Capricor Therapeutics（4LN2）株式概要臨床段階のバイオテクノロジー企業であるカプリコル・セラピューティクス社は、デュシェンヌ型筋ジストロフィー（DMD）およびその他のアンメット・メディカル・ニーズのある疾患を治療するための、変革的な細胞およびエクソソームに基づく治療薬の開発に米国で取り組んでいる。 詳細4LN2 ファンダメンタル分析スノーフレーク・スコア評価4/6将来の成長5/6過去の実績0/6財務の健全性5/6配当金0/6報酬当社が推定した公正価値より94.2%で取引されている 収益は年間63.61%増加すると予測されています リスク分析収益が 100 万ドル未満 ( $0 )German市場と比較して、過去 3 か月間の株価の変動が非常に大きい過去1年間で株主の希薄化が進んだ すべてのリスクチェックを見る4LN2 Community Fair Values Create NarrativeSee what others think this stock is worth. Follow their fair value or set your own to get alerts.NEW492,148 membersJoin community and earn perksGain real feedbackFrom our editorial team, personally. Not silence.Grow your followingReal investors. The kind who actually invest, not scroll past.Unlock free accessFree premium subscription for consistent and quality authors.Learn moreCreate NarrativeBLINRODA492,148 investors already sharing narrativesYour Fair Value€Current Price€5.74該当なし内在価値ディスカウントEst. Revenue$PastFuture-115m27m2016201920222025202620282031Revenue US$6.8Earnings US$1.2AdvancedSet Fair ValueView all narrativesCapricor Therapeutics, Inc. 競合他社Biotest GmbH KGaASymbol: HMSE:BIO0Market cap: €1.3bBiotestSymbol: XTRA:BIO3Market cap: €1.4bFormyconSymbol: XTRA:FYBMarket cap: €334.0mHamlet BioPharmaSymbol: DB:7ZTMarket cap: €1.4b価格と性能株価の高値、安値、推移の概要Capricor Therapeutics過去の株価現在の株価US$5.7452週高値US$30.1052週安値US$3.73ベータ0.421ヶ月の変化-75.55%3ヶ月変化-78.73%1年変化-13.19%3年間の変化39.95%5年間の変化53.42%IPOからの変化-42.62%最新ニュースお知らせ • 5hCapricor Therapeutics Publishes Phase 3 Hope-3 Trial Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced that The Lancet has published results from the Company’s pivotal Phase 3 HOPE-3 clinical trial evaluating Deramiocel, its investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The manuscript underwent independent expert peer review, providing external validation of the trial’s design, statistical methodology and findings. The results are based on the Company’s prespecified Statistical Analysis Plan version 3.0 (SAP 3.0). The paper, titled “Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial” can be accessed. The randomized, double-blind, placebo-controlled HOPE-3 Phase 3 trial (n=106) met primary endpoint, with Deramiocel slowing upper limb function decline by 54% versus placebo (PUL 2.0, p=0.03) and showing clinically meaningful cardiac benefit. Deramiocel BLA remains under active FDA review, with PDUFA target action date of August 22, 2026. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.New Risk • Jul 27New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 21% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (21% average weekly change). Revenue is less than US$1m. Minor Risk Shareholders have been diluted in the past year (27% increase in shares outstanding).お知らせ • Jun 29+ 4 more updatesCapricor Therapeutics Announces FDA Advisory Committee Meeting To Review BLA For Deramiocel For The Treatment Of Duchenne Muscular DystrophyCapricor Therapeutics announced that the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) of the U.S. Food and Drug Administration (FDA) is planning to convene an advisory committee meeting to discuss the Company's Biologics License Application (BLA) seeking approval of Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The BLA is supported by the Company's Phase 2 HOPE-2 trial and long-term outcomes from the HOPE-2-OLE trial, as well as positive results from the Phase 3 HOPE-3 trial, which achieved statistical significance on its primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints. The date for the Advisory Committee meeting is July 29, 2026, and the meeting will be available for live streaming. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.お知らせ • Jun 26Capricor Therapeutics Presents Positive Five-Year HOPE-2 Ole Data and HOPE-3 Phase 3 Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, taking place June 25-27, 2026, in Orlando, Florida, alongside previously reported results from the Company’s HOPE-3 Phase 3 trial. Among the patients who remain enrolled in the HOPE-2 OLE study (n=9), treatment with Deramiocel continued to attenuate disease progression over five years, as measured by Performance of the Upper Limb (PUL 2.0). Patients experienced a mean total-score decline of less than 5 points over five years. By comparison, a cohort-matched external comparator of standard-of-care DMD patients showed a modeled decline of roughly 2.4 points per year, which if continued would project to approximately 12 points over five years. A separately published natural-history analysis (Coratti, et al., J Neuromuscular Dis, 2025) reported a decline in non-ambulant patients of approximately 8.1 points over three years, consistent with a meaningful treatment benefit. Cardiac function, as measured by left ventricular ejection fraction (LVEF) on cardiac MRI, remained stable over the full five-year period, in contrast to the modeled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator. Deramiocel continued to demonstrate a favorable safety profile throughout the study, consistent with over 800 intravenous infusions administered to date across the Deramiocel clinical development program. The multicenter, randomized, double-blind, placebo-controlled trial enrolled 106 patients with DMD and met its primary endpoint (PUL 2.0, p=0.03) and key secondary cardiac endpoint (LVEF, p=0.04), along with all other Type I error-controlled secondary endpoints – consistent with the durable skeletal and cardiac muscle benefit observed in the HOPE-2 OLE study. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.お知らせ • May 05Capricor Therapeutics, Inc. to Report Q1, 2026 Results on May 12, 2026Capricor Therapeutics, Inc. announced that they will report Q1, 2026 results After-Market on May 12, 2026お知らせ • Apr 22Capricor Therapeutics Announces Late-Breaking Presentation of Hope-3 Phase 3 ResultsCapricor Therapeutics had announced the presentation of data from its Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago, Illinois. The data were presented by Dr. Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children's Hospital, during the Late-Breaking Science 2 session on Tuesday, April 21, 2026. These findings were further supported by an alternative measure of upper limb function, specifically video recordings of tasks performed at home (the Duchenne Video Assessment, or DVA), which showed meaningful slowing of disease progression in the ability to self-feed, a function central to patient independence. These data reinforce the potential of Deramiocel to make a meaningful difference in the lives of those living with Duchenne. The HOPE-3 data tell a compelling story of preserved function, slowed decline, and real-world impact on patients' daily lives, evidence we believe positions Deramiocel as a potentially transformative therapy for Duchenne. With our BLA currently under FDA review and a PDUFA target action date of August 22, 2026, we remain on track and focused on bringing this therapy to the patients who need it as quickly as possible. Data from this presentation were released under AAN embargo on April 21, 2026 at 4:45 p.m. ET. Additional information about the 2026 AAN Annual Meeting is available at www.aan.com. The Company's presentation will be made available in the Investors section of Capricor's website shortly. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.最新情報をもっと見るRecent updatesお知らせ • 5hCapricor Therapeutics Publishes Phase 3 Hope-3 Trial Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced that The Lancet has published results from the Company’s pivotal Phase 3 HOPE-3 clinical trial evaluating Deramiocel, its investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The manuscript underwent independent expert peer review, providing external validation of the trial’s design, statistical methodology and findings. The results are based on the Company’s prespecified Statistical Analysis Plan version 3.0 (SAP 3.0). The paper, titled “Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial” can be accessed. The randomized, double-blind, placebo-controlled HOPE-3 Phase 3 trial (n=106) met primary endpoint, with Deramiocel slowing upper limb function decline by 54% versus placebo (PUL 2.0, p=0.03) and showing clinically meaningful cardiac benefit. Deramiocel BLA remains under active FDA review, with PDUFA target action date of August 22, 2026. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.New Risk • Jul 27New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 21% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (21% average weekly change). Revenue is less than US$1m. Minor Risk Shareholders have been diluted in the past year (27% increase in shares outstanding).お知らせ • Jun 29+ 4 more updatesCapricor Therapeutics Announces FDA Advisory Committee Meeting To Review BLA For Deramiocel For The Treatment Of Duchenne Muscular DystrophyCapricor Therapeutics announced that the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) of the U.S. Food and Drug Administration (FDA) is planning to convene an advisory committee meeting to discuss the Company's Biologics License Application (BLA) seeking approval of Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The BLA is supported by the Company's Phase 2 HOPE-2 trial and long-term outcomes from the HOPE-2-OLE trial, as well as positive results from the Phase 3 HOPE-3 trial, which achieved statistical significance on its primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints. The date for the Advisory Committee meeting is July 29, 2026, and the meeting will be available for live streaming. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.お知らせ • Jun 26Capricor Therapeutics Presents Positive Five-Year HOPE-2 Ole Data and HOPE-3 Phase 3 Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, taking place June 25-27, 2026, in Orlando, Florida, alongside previously reported results from the Company’s HOPE-3 Phase 3 trial. Among the patients who remain enrolled in the HOPE-2 OLE study (n=9), treatment with Deramiocel continued to attenuate disease progression over five years, as measured by Performance of the Upper Limb (PUL 2.0). Patients experienced a mean total-score decline of less than 5 points over five years. By comparison, a cohort-matched external comparator of standard-of-care DMD patients showed a modeled decline of roughly 2.4 points per year, which if continued would project to approximately 12 points over five years. A separately published natural-history analysis (Coratti, et al., J Neuromuscular Dis, 2025) reported a decline in non-ambulant patients of approximately 8.1 points over three years, consistent with a meaningful treatment benefit. Cardiac function, as measured by left ventricular ejection fraction (LVEF) on cardiac MRI, remained stable over the full five-year period, in contrast to the modeled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator. Deramiocel continued to demonstrate a favorable safety profile throughout the study, consistent with over 800 intravenous infusions administered to date across the Deramiocel clinical development program. The multicenter, randomized, double-blind, placebo-controlled trial enrolled 106 patients with DMD and met its primary endpoint (PUL 2.0, p=0.03) and key secondary cardiac endpoint (LVEF, p=0.04), along with all other Type I error-controlled secondary endpoints – consistent with the durable skeletal and cardiac muscle benefit observed in the HOPE-2 OLE study. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.お知らせ • May 05Capricor Therapeutics, Inc. to Report Q1, 2026 Results on May 12, 2026Capricor Therapeutics, Inc. announced that they will report Q1, 2026 results After-Market on May 12, 2026お知らせ • Apr 22Capricor Therapeutics Announces Late-Breaking Presentation of Hope-3 Phase 3 ResultsCapricor Therapeutics had announced the presentation of data from its Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago, Illinois. The data were presented by Dr. Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children's Hospital, during the Late-Breaking Science 2 session on Tuesday, April 21, 2026. These findings were further supported by an alternative measure of upper limb function, specifically video recordings of tasks performed at home (the Duchenne Video Assessment, or DVA), which showed meaningful slowing of disease progression in the ability to self-feed, a function central to patient independence. These data reinforce the potential of Deramiocel to make a meaningful difference in the lives of those living with Duchenne. The HOPE-3 data tell a compelling story of preserved function, slowed decline, and real-world impact on patients' daily lives, evidence we believe positions Deramiocel as a potentially transformative therapy for Duchenne. With our BLA currently under FDA review and a PDUFA target action date of August 22, 2026, we remain on track and focused on bringing this therapy to the patients who need it as quickly as possible. Data from this presentation were released under AAN embargo on April 21, 2026 at 4:45 p.m. ET. Additional information about the 2026 AAN Annual Meeting is available at www.aan.com. The Company's presentation will be made available in the Investors section of Capricor's website shortly. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.お知らせ • Apr 02Capricor Therapeutics, Inc., Annual General Meeting, Jun 04, 2026Capricor Therapeutics, Inc., Annual General Meeting, Jun 04, 2026. Location: 10865 road to the cure, suite 150, 92121., san diego, Canadaお知らせ • Mar 13Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular DystrophyCapricor Therapeutics announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD), which were presented at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference in Orlando, Florida. Cardiac MRI analyses demonstrated Deramiocel’s impact on cardiac structure in patients with DMD. Evaluation of late gadolinium enhancement (LGE), a marker of myocardial fibrosis, showed a significant reduction in fibrotic segments in patients treated with Deramiocel versus placebo, corresponding to a three-segment treatment difference at 12 months (p=0.022). The presence of LGE reflects replacement of viable myocardium with fibrotic tissue and is associated with progressive cardiac dysfunction and heart failure risk in DMD cardiomyopathy. In patients with baseline cardiomyopathy, Deramiocel demonstrated an even greater treatment effect on cardiac function. In this subgroup, treatment resulted in a 3.3 percentage-point improvement in LVEF versus placebo, corresponding to greater than 100% attenuation of expected cardiac decline (p=0.017). A Global Statistical Test (GST), a patient-level composite including Performance of Upper Limb (PUL v2.0), left ventricular ejection fraction (LVEF), and Patient Global Impression of Severity (PGI-S), demonstrated a statistically significant overall treatment benefit favoring Deramiocel (p=0.017). This composite integrates multiple clinically meaningful domains of disease, reflecting how patients feel and function. Additional functional outcomes evaluating hand-to-mouth activity, an important measure of patient independence, were also presented. Data from the Duchenne Video Assessment (DVA), a measure of activities of daily living in individuals with Duchenne, showed that the “eat 10 bites” task resulted in approximately 83% slowing of disease progression compared with placebo (p=0.018). These findings align with results from the mid-level (elbow) PUL v2.0 assessment (p=0.008), providing concordant evidence across both validated clinical and real-world functional measures. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.お知らせ • Mar 10Capricor Therapeutics, Inc. Establishes New PDUFA Date For Deramiocel BLACapricor Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has lifted the previously issued Complete Response Letter and resumed review of its Biologics License Application (BLA) seeking full approval of Deramiocel, an investigational cell therapy, for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. The submission has been classified as a Class 2 resubmission, with a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026. The Company received a Complete Response Letter (CRL) from the FDA in July 2025. Following submission of data and supporting documentation from the HOPE-3 clinical trial, the FDA resumed review of the application and assigned a PDUFA target action date of August 22, 2026. At this time, the FDA has not identified any potential review issues in its response to the Company. Capricor also expects to be eligible to receive a Priority Review Voucher (PRV) upon potential approval of Deramiocel. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co. Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX vaccine are investigational candidates and have not been approved for commercial use in any indication.お知らせ • Mar 09Capricor Therapeutics, Inc. to Report Q4, 2025 Results on Mar 12, 2026Capricor Therapeutics, Inc. announced that they will report Q4, 2025 results After-Market on Mar 12, 2026お知らせ • Jan 20Capricor Therapeutics Provides Regulatory Updates Regarding Its Biologics License Application for DeramiocelCapricor Therapeutics provided a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, the Company’s investigational first-in-class cell therapy for the treatment of Duchenne muscular dystrophy (DMD). As previously disclosed, the Company provided topline results from its Phase 3 HOPE-3 clinical study to the U.S. Food and Drug Administration (FDA) in late 2025. Following its review of these data, the FDA has formally requested the full HOPE-3 clinical study report (CSR) and supporting data to address the Complete Response Letter (CRL). The FDA did not request any additional clinical studies or new patient data as part of this request. Preparation of the HOPE-3 CSR is well underway, and the Company plans to submit the requested materials to the FDA in February 2026. The Company expects that this submission will address the items outlined in the CRL and support continued review of the BLA, including the assignment of a new Prescription Drug User Fee Act (PDUFA) target action date.お知らせ • Dec 17Capricor Therapeutics Announces Positive Topline Results From Phase 3 HOPE-3 Trial Of Deramiocel For Duchenne Muscular DystrophyCapricor Therapeutics announced that Parent Project Muscular Dystrophy (PPMD) will host a community webinar to share and discuss positive topline results from Capricor’s Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The webinar will provide patients and families with an overview of the HOPE-3 results and how the findings inform ongoing regulatory discussions, including next steps with the U.S. Food and Drug Administration (FDA). Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co. Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX™ vaccine are investigational candidates and have not been approved for commercial use in any indication.お知らせ • Dec 05+ 1 more updateCapricor Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $150 million.Capricor Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $150 million. Security Name: Common Stock Security Type: Common Stock Securities Offered: 6,000,000 Price\Range: $25お知らせ • Dec 03Capricor Therapeutics, Inc. Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics, Inc. announced positive topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with Duchenne muscular dystrophy. The study randomized 106 participants across 20 leading U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period. The average age of participants was approximately 15 years, and all were on a stable corticosteroid regimen throughout the study. Baseline demographics were well balanced between treatment arms, approximately 90 percent were receiving cardiac medications at baseline, and over 75 percent had a clinical diagnosis of cardiomyopathy. Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience. They expect that detailed HOPE-3 results will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.お知らせ • Nov 25Capricor Therapeutics, Inc. Presents New Data Demonstrating a Scalable Framework for Loading Therapeutic Oligonucleotides into ExosomesCapricor Therapeutics, Inc. announced new data describing a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into exosomes. The data were presented at the 2025 American Association for Extracellular Ventures (AAEV) Annual Meeting in Salt Lake City, Utah, held from November 20-23, 2025. The poster titled, "A Systematic Framework for the Scalable Loading of Therapeutic siRNA and PMO into Exosomes," highlighted proprietary data showcasing Capricor's exosome-based technology. The data provided an overview of both scale-up and scale-out electroporation strategies that can be integrated to achieve substantially larger yields of exogenously loaded engineered exosomes, which is a critical requirement for further clinical development. Key findings include: Engineered exosomes derived from 293F cells were successfully loaded with therapeutic siRNA and PMO cargo using optimized electroporation conditions. Both scale-up and scale- out strategies produced comparable loading efficiencies to standard small-volume electroporation. Integrating the two approaches enables manufacturing of significantly larger batches of therapeutic exosomes. At the forefront of innovation is the lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for the treatment of Duchenne muscular dystrophy (DMD).お知らせ • Oct 31Capricor Therapeutics, Inc. to Report Q3, 2025 Results on Nov 10, 2025Capricor Therapeutics, Inc. announced that they will report Q3, 2025 results After-Market on Nov 10, 2025お知らせ • Sep 25Capricor Therapeutics Provides Regulatory Update on Deramiocel Program for Duchenne Muscular DystrophyCapricor Therapeutics announced a regulatory update for its Biologics License Application (BLA) for Deramiocel, the Company's investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). This update follows a recent Type A meeting with the U.S. Food and Drug Administration (FDA) after the receipt of a Complete Response Letter (CRL) in July 2025. The goal of the Type A meeting was to establish a path toward potential approval of Deramiocel for the treatment of DMD. Key outcomes included: The HOPE-3 clinical trial should serve as the "add additional study" requested in the CRL. The HOPE-3 data can be submitted within the current BLA, maintaining PUL v2.0 as the primary efficacy endpoint and suggesting left ventricular ejection fraction (LVEF) as a key secondary endpoint, which Capricor intends to request for labeling consideration. Capricor plans to submit HOPE-3 data with its complete response to the CRL, with the goal of securing a label encompassing both cardiac and skeletal muscle function in DMD. In its meeting minutes, the FDA further emphasized its commitment, stating: "The FDA remains committed to collaborating with the applicant and will exercise further regulatory flexibility by reviewing data from the HOPE-3 trial". Importantly, prior to issuance of the CRL, the majority of the BLA had undergone rigorous review with no significant deficiencies identified by the FDA during the mid-cycle review or pre-licensing inspections. All CMC items identified in the CRL have been addressed and communicated to the FDA. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of Duchenane Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial consisting of two supports evaluating the safety and efficacy of Deramiocel in participants with DMD.お知らせ • Sep 09Capricor Therapeutics Responds to FDA Posting of Complete Response Letter for DeramiocelCapricor Therapeutics issued a statement regarding the U.S. Food and Drug Administration’s (FDA) public posting of its Complete Response Letter (CRL) for the Biologics License Application (BLA) for Deramiocel, the Company’s investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The Company was not notified in advance that the CRL would be posted, but acknowledges the FDA’s decision to publish the letter, originally received in July 2025. However, the FDA did not release the comprehensive preliminary response that Capricor submitted shortly after receipt of the CRL. This written response provided clarifications to the Agency’s feedback and outlined the Company’s proposed plan to address the outstanding issues. To ensure transparency, Capricor will make its preliminary response available on the investor section of its website for patients, families, and other stakeholders to review. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD afflicts approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials.Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.お知らせ • Aug 19Capricor Therapeutics Announces First Subjects Dosed in Phase 1 Clinical Trial of Novel Exosome-Based VaccineCapricor Therapeutics announced that the first subjects have been dosed in a Phase 1 clinical trial evaluating its StealthX exosome-based vaccine. The study, funded by the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) under the U.S. Department of Health and Human Services' Project NextGen, follows review and clearance of the Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). The trial is being conducted by an NIAID-funded network of clinical trial sites. Project NextGen is a federal initiative aimed at accelerating the development of next-generation vaccine platforms that provide broader and more durable protection against respiratory viruses and other infectious threats. The Phase 1 trial includes four dosing arms and is initially focused on the spike (S) protein of SARS-CoV-2. An additional arm of Capricor's StealthX multivalent vaccine incorporating the nucleocapsid (N) protein is planned, pending separate FDA clearance. The StealthX vaccine is a proprietary exosome-based platform developed internally by Capricor, using exosomes engineered to display either the spike or nucleocapsid proteins on their surface. Preclinical results published in Microbiology Spectrum demonstrated that StealthX elicited robust antibody production, potent neutralizing activity, a strong T-cell response and a favorable safety profile.お知らせ • Aug 06Capricor Therapeutics, Inc. to Report Q2, 2025 Results on Aug 11, 2025Capricor Therapeutics, Inc. announced that they will report Q2, 2025 results After-Market on Aug 11, 2025お知らせ • Aug 01Levi & Korsinsky, LLP Notifies Investors in Capricor Therapeutics, Inc. of A Class Action Securities LawLevi & Korsinsky, LLP notified investors in Capricor Therapeutics, Inc. of a class action securities lawsuit. The lawsuit seeks to recover losses on behalf of Capricor investors who were adversely affected by alleged securities fraud between October 9, 2024 and July 10, 2025. According to the complaint, defendants provided investors with material information concerning Capricor's lead cell therapy candidate drug deramiocel for the treatment of cardiomyopathy associated withuchenne muscular dystrophy (DMD). Participants' statements included, among other things, Capricor's ability to obtain a Biologics License Application (BLA) for deramiocel from the U.S. Food and Drug Administration (FDA). Defendants provided these overwhelmingly positive statements to investors while, at the same time, disseminating false and misleading statements and/or concealing material adverse facts concerning its four-year safety and efficacy data from its Phase 2 HOPE-2 trial study of deramiocel. On July 11, 2025, Capricor issued a press release announcing it received a Complete Response Letter (CRL) from the FDA denying the BLA specifically citing it did not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data. Further, the CRL referenced outstanding items in the Chemistry, Manufacturing, and Controls section of the application.お知らせ • Jul 11Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular DystrophyCapricor Therapeutics announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). In the CRL, the FDA stated that it had completed its review of the application but is unable to approve the BLA in its current form, specifically citing that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data. The CRL also referenced certain outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section of the application, most of which Capricor believes it has addressed in prior communications to the FDA. However, these materials were not reviewed by the FDA due to the timing of the CRL issuance. The FDA confirmed that it will restart the review clock upon resubmission. In addition, the agency offered the company the opportunity to request a Type A meeting to discuss the path forward. Capricor plans to engage further with the FDA to determine the appropriate next steps. Capricor's BLA for Deramiocel was granted Priority Review in March 2025 and was supported by data from the HOPE-2 trial, its open-label extension (OLE), and natural history comparisons from FDA-funded datasets.お知らせ • Jun 30+ 5 more updatesCapricor Therapeutics, Inc.(NasdaqCM:CAPR) dropped from Russell 3000E Value IndexCapricor Therapeutics, Inc.(NasdaqCM:CAPR) dropped from Russell 3000E Value Indexお知らせ • Jun 24Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular DystrophyCapricor Therapeutics provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)--associated cardiomyopathy. As part of the FDA's ongoing review, the Company has been informed that an Advisory Committee meeting is not indicated at this time. The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running treatment datasets in DMD, including measures of both cardiac and skeletal muscle function. The data demonstrated continued preservation of cardiac function, along with sustained clinical benefit from long-term Deramiocel treatment. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of Duchenane Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. For Becker Muscular Dystrophy (BMD), Deramiocel has also received Orphan Drug Designation from the FDA.お知らせ • Jun 20Capricor Therapeutics Announces Positive 4-Year Data from Hope-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced positive four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company's lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be featured in the session titled "Therapies that slow Progression" at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference, taking place June 21, 2025, in Las Vegas, Nevada. After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline. Further, a subgroup analysis of patients with baseline LVEF >45% showed an even greater clinical benefit, supporting early intervention with Deramiocel to potentially preserve cardiac function. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™? platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, company stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need.お知らせ • Jun 13Capricor Therapeutics Announces the Successful Completion of the U.S. Food and Drug Administration's License Inspection of Its San Diego Manufacturing FacilityCapricor Therapeutics announced the successful completion of the U.S. Food and Drug Administration's (FDA) Pre-License Inspection (PLI) of its San Diego manufacturing facility for Deramiocel, the company's lead cell therapy candidate with a Biologics License Application (BLA) under FDA review for potential approval in the treatment of Duchenne Muscular Dystrophy (DMD). The inspection concluded with a Form 483 containing several observations. The Company has submitted its responses to the FDA, none of which required material changes to the cGMP process or facility. The observations were primarily related to routine quality systems and documentation practices. The company is confident that the facility will meet the necessary requirements to support product licensure and, pending approval, commercial launch.お知らせ • Jun 12Capricor Therapeutics Announces Key Regulatory Updates for Its Duchenne Muscular Dystrophy ProgramCapricor Therapeutics announced the successful completion of the U.S. Food and Drug Administration’s (FDA) Pre-License Inspection (PLI) of its San Diego manufacturing facility for Deramiocel, the Company’s lead cell therapy candidate with a Biologics License Application (BLA) under FDA review for potential approval in the treatment of Duchenne Muscular Dystrophy (DMD). The inspection concluded with a Form 483 containing several observations. The Company has submitted its responses to the FDA, none of which required material changes to the cGMP process or facility. The observations were primarily related to routine quality systems and documentation practices. The Company is confident that the facility will meet the necessary requirements to support product licensure and, pending approval, commercial launch. The FDA informed Capricor of its intent to hold the Advisory Committee meeting on July 30, 2025, although that date is pending confirmation by the FDA. At the time of the mid-cycle review, no significant issues or major deficiencies were noted. A late-cycle meeting is planned for mid-July 2025. The BLA for Deramiocel remains under priority review with a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025.お知らせ • May 06+ 1 more updateCapricor Therapeutics, Inc. to Report Q1, 2025 Results on May 13, 2025Capricor Therapeutics, Inc. announced that they will report Q1, 2025 results After-Market on May 13, 2025お知らせ • Apr 10Capricor Therapeutics, Inc., Annual General Meeting, May 22, 2025Capricor Therapeutics, Inc., Annual General Meeting, May 22, 2025. Location: 10865 road to the cure, suite 150, san diego, california 92121, United Statesお知らせ • Apr 09Earl Collier Not to Stand for Re-Election as Member of the Board of Capricor Therapeutics, IncCapricor Therapeutics, Inc. announced that on April 8, 2025, Mr. Earl Collier, a member of the Board of Directors of the company informed the Board he will not stand for re-election to the Board following the Annual Meeting of Shareholders which is scheduled to occur on May 22, 2025. Mr. Collier's decision to resign is not due to any disagreement with the Company with respect to any of the Company's operations, policies or practices. In connection with Mr. Collier's resignation, the Company plans to enter into a consulting agreement with Mr. Collier pursuant to which, from time to time, Mr. Collier will perform certain strategic advisory relations.お知らせ • Mar 18Capricor Therapeutics, Inc. Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics, Inc. announced positive long-term data from its ongoing HOPE-2 open label extension (“OLE”) clinical trial, demonstrating the potential of the Company’s lead asset, deramiocel, to slow disease progression and preserve upper limb function in patients with Duchenne muscular dystrophy (“DMD”). The data is presented as a late breaking poster at this year’s Muscular Dystrophy Association Clinical and Scientific Conference, which began on March 16 and runs through March 19 in Dallas, Texas. In a cohort-matched external comparator analysis, the study showed that patients treated with deramiocel over three years experienced an average decline in Performance of the Upper Limb (PUL 2.0) total score of 3.46 points, compared to a 7.19-point decline in the external comparator group (p=0.019). This equates to a 52% slowing of disease progression, reinforcing deramiocel’s potential long-term therapeutic durability. Additional findings include: Treatment effect increases year over year – Patients on deramiocel showed a reduction in disease progression, with a mean annual PUL 2.0 decline of 1.8 points in Year 1, 1.2 points in Year 2 and 1.1 points in Year 3. Potential disease-modifying effects – During a 1-year gap of treatment, those originally randomized to deramiocel showed a slower rate of decline (2.8 points per year) compared to untreated patients (3.7 points per year). Favorable safety profile – Deramiocel was well tolerated with no new safety signals identified and continues to maintain a favorable long-term benefit-risk profile. Capricor recently announced the acceptance by the U.S. Food and Drug Administration (“FDA”) of its Biologics License Application (“BLA”) for the cardiomyopathy associated with DMD with a Prescription Drug User Fee Act (“PDUFA”) target action date set for August 31, 2025, seeking full approval of its application.お知らせ • Mar 11Capricor Therapeutics, Inc. to Report Q4, 2024 Results on Mar 19, 2025Capricor Therapeutics, Inc. announced that they will report Q4, 2024 results After-Market on Mar 19, 2025お知らせ • Mar 04Capricor Therapeutics Announces FDA Acceptance and Priority Review of Its Biologics License Application for Deramiocel to Treat Duchenne Muscular DystrophyCapricor Therapeutics announced the U.S. Food and Drug Administration ("FDA") has accepted for review its Biologics License Application ("BLA") seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy ("DMD") cardiomyopathy. Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act ("PDUFA") target action date of August 31, 2025 and at this time, the FDA has not identified any potential review issues. The BLA submission is supported by Capricor's existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Lab Extension ("OLE") trials compared to natural history data from an FDA-funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. The FDA also informed the Company they have not yet decided whether an Advisory Committee meeting is needed in relation to this application. The FDA grants Priority Review to applications for medicines that, if approved, provide significant improvements in the safety or effectiveness of the treatment of a serious condition. Deramiocel for the treatment of DMD has received Orphan Drug Designation from the FDA and European Medicines Agency ("EMA"). In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD by September 30, 2026, Capricor would be eligible to receive a Priority Review Voucher ("PRV") based on its previous receipt of a rare pediatric disease designation.お知らせ • Jan 02Capricor Therapeutics Completes Submission of Biologics License Application to U.S. FDA for Deramiocel for Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. The full submission of the rolling BLA was completed as the Company had previously guided in late December 2024 and is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data from an FDA funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. Capricor has requested a priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA. In conjunction with this achievement, Capricor will receive a milestone payment of $10 million from its distribution partner, Nippon Shinyaku Co. Ltd., under the terms of its U.S. Commercialization and Distribution Agreement. Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.Reported Earnings • Nov 14Third quarter 2024 earnings released: US$0.38 loss per share (vs US$0.25 loss in 3Q 2023)Third quarter 2024 results: US$0.38 loss per share (further deteriorated from US$0.25 loss in 3Q 2023). Revenue: US$2.26m (down 63% from 3Q 2023). Net loss: US$12.6m (loss widened 97% from 3Q 2023). Revenue is forecast to grow 52% p.a. on average during the next 3 years, compared to a 22% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has fallen by 2% per year but the company’s share price has increased by 74% per year, which means it is well ahead of earnings.お知らせ • Nov 05Capricor Therapeutics, Inc. to Report Q3, 2024 Results on Nov 13, 2024Capricor Therapeutics, Inc. announced that they will report Q3, 2024 results After-Market on Nov 13, 2024New Risk • Oct 20New major risk - Shareholder dilutionThe company's shareholders have been substantially diluted in the past year. Increase in shares outstanding: 76% This is considered a major risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (27% average weekly change). Shareholders have been substantially diluted in the past year (76% increase in shares outstanding).お知らせ • Oct 18+ 1 more updateCapricor Therapeutics, Inc. Intends to File A Biologics License ApplicationFollowing recent meetings with the U.S. Food and Drug Administration Capricor Therapeutics, Inc. announced its intent to file a Biologics License Application based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (“DMD”) cardiomyopathy. Capricor commenced the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024. The BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (“OLE”) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center. In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024. StealthX™Exosome Platform The Company’s proprietary StealthX™ exosome-based multivalent vaccine (StealthX™ vaccine) for the prevention of SARS-CoV-2 was selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines. Under the terms of the collaboration, Capricor will supply the investigational product and NIAID's Division of Microbiology and Infectious Diseases will conduct the trial. Currently, its vaccine candidate is in the manufacturing phase with plans to deliver it to NIAID by the end of 2024.お知らせ • Oct 17Capricor Therapeutics, Inc. has filed a Follow-on Equity Offering.Capricor Therapeutics, Inc. has filed a Follow-on Equity Offering. Security Name: Common Stock Security Type: Common Stockお知らせ • Oct 12Capricor Therapeutics Announces Positive Long-Term Data from Hope-2 OLE Study in Duchenne Muscular Dystrophy at 2024 World Muscle Society CongressCapricor Therapeutics announced positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study for its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD). The data was highlighted in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society (WMS), October 8-12, 2024 in Prague, Czechia. The 3-year data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures of cardiac function, including left ventricular ejection fraction (LVEF%), as well as indexed volumes, which are considered highly relevant in terms of predicting long-term cardiac outcomes. In addition, there was clear bifurcation in the treatment effect seen in those that had ejection fractions greater than 45% at the end of HOPE-2 which suggests that early and sustained intervention will be key in attenuating the impacts of DMD cardiomyopathy. In order to evaluate the relevance of the data to disease progression as well as the chronic and progressive nature of DMD where cardiac function can decline year over year, a natural history data set was used to compare the trajectory of those treated with deramiocel to standard of care. In addition to the cardiac data, patients demonstrated a statistically and clinically relevant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel. In conclusion, the results of this study showed sustained cardiac and skeletal benefits after 3 years of continuous treatment with deramiocel. This data was previously highlighted at the PPMD Annual Meeting in June 2024.お知らせ • Oct 09Capricor Therapeutics Announces Initiation of Rolling Submission of Biologics License Application with U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced that it has initiated its rolling submission process with the U.S. Food and Drug Administration (FDA) for a Biologics License Application (BLA), seeking full approval for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. Capricor plans to complete its rolling BLA submission by the end of 2024. The application may be eligible for priority review as deramiocel could potentially provide significant improvements in the safety and/or effectiveness of the treatment for the serious condition of DMD cardiomyopathy, where there are currently no approved treatment options available. Once the rolling BLA submission is completed, the FDA will notify the Company when it is formally accepted for review.Breakeven Date Change • Sep 24The 5 analysts covering Capricor Therapeutics previously expected the company to break even in . New consensus forecast suggests the company will make a profit of US$0 in . Earnings growth of 53% is required to achieve expected profit on schedule.New Risk • Sep 24New minor risk - ProfitabilityThe company is currently unprofitable and not forecast to become profitable over the next 3 years. Trailing 12-month net loss: US$28m Forecast net loss in 3 years: US$13m This is considered a minor risk. Companies that are not profitable are more likely to be burning through cash and less likely to be well established. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. Without profits, the company is under pressure to grow significantly while potentially having to reduce costs and possibly needing to take on debt or raise capital to remain afloat. Currently, the following risks have been identified for the company: Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$13m net loss in 3 years). Share price has been volatile over the past 3 months (10% average weekly change). Shareholders have been diluted in the past year (37% increase in shares outstanding).お知らせ • Sep 18Capricor Therapeutics, Inc. announced that it has received $14.999998 million in funding from Nippon Shinyaku Co., Ltd.Capricor Therapeutics, Inc. announced that it has entered into a subscription agreement to issue $5.36 per share for the gross proceeds of $14,999,998 on September 16, 2024. The transaction included participation from new investor, Nippon Shinyaku Co., Ltd. The Subscription Agreement also includes lock-up provisions restricting Nippon Shinyaku from selling or otherwise disposing of shares of Common Stock until the six month anniversary of the Closing Date.Reported Earnings • Aug 08Second quarter 2024 earnings released: US$0.34 loss per share (vs US$0.29 loss in 2Q 2023)Second quarter 2024 results: US$0.34 loss per share (further deteriorated from US$0.29 loss in 2Q 2023). Revenue: US$3.97m (up 1.4% from 2Q 2023). Net loss: US$11.0m (loss widened 49% from 2Q 2023). Revenue is forecast to grow 40% p.a. on average during the next 3 years, compared to a 20% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has fallen by 3% per year whereas the company’s share price has fallen by 1% per year.お知らせ • Jul 31Capricor Therapeutics, Inc. to Report Q2, 2024 Results on Aug 07, 2024Capricor Therapeutics, Inc. announced that they will report Q2, 2024 results After-Market on Aug 07, 2024New Risk • Jul 01New major risk - Financial positionThe company has less than a year of cash runway based on its current free cash flow trend. Free cash flow: -US$33m This is considered a major risk. With less than a year's worth of cash, the company will need to raise capital or take on debt unless its cash flows improve. This would dilute existing shareholders or increase balance sheet risk. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$33m free cash flow). Share price has been highly volatile over the past 3 months (11% average weekly change). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$6.2m net loss in 3 years). Shareholders have been diluted in the past year (27% increase in shares outstanding).お知らせ • Jun 29Capricor Therapeutics, Inc. Announces Long-Term Benefit of Deramiocel (Cap-1002) in Both Skeletal Muscle and Cardiac Function in the Hope-2 Ole Study in Duchenne Muscular DystrophyCapricor Therapeutics, Inc. announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, deramiocel (CAP-1002), for the treatment of Duchenne muscular dystrophy. Data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures, including left ventricular ejection fraction (LVEF), as well as indexed volumes (left ventricular end systolic volume (LVESV) and left ventricular end diastolic volume (LVEDV)). These are measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes. In addition, greater improvements in cardiac function were observed in those patients that had higher ejection fractions (> 45%) at the beginning of the HOPE-2 randomized trial. Published data supports the need for early intervention in order to maintain function and potentially slow the progression of the cardiomyopathy, one of the leading causes of death in patients with DMD. Currently, there is no approved treatment specifically for DMD cardiomyopathy, which underscores the need for additional therapies to treat DMD. Additionally, as previously reported, patients showed a statistically significant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel. HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, deramiocel, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either deramiocel (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment entered the HOPE-2-OLE study where they receive deramiocel (150 million cells per infusion) every three months. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 (p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety, cardiac and functional performance. Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.お知らせ • Jun 25Capricor Therapeutics, Inc. Announces for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD). Capricor’s goal for this meeting will be to finalize its BLA filing plans based on all currently available data as well as to work with the FDA to outline the rolling BLA submission timeline. Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000 to 20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.New Risk • Jun 07New minor risk - ProfitabilityThe company is currently unprofitable and not forecast to become profitable over the next 3 years. Trailing 12-month net loss: US$24m Forecast net loss in 3 years: US$11m This is considered a minor risk. Companies that are not profitable are more likely to be burning through cash and less likely to be well established. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. Without profits, the company is under pressure to grow significantly while potentially having to reduce costs and possibly needing to take on debt or raise capital to remain afloat. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (12% average weekly change). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$11m net loss in 3 years). Shareholders have been diluted in the past year (26% increase in shares outstanding).お知らせ • Jun 05Capricor Therapeutics Announces Positive 3-Year Efficacy Results from Hope-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular DystrophyCapricor Therapeutics announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study with CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Patients treated with CAP-1002 continue to show positive benefits after 3 years of treatment in both the PUL 2.0 and LVEF measures when compared to an external comparator dataset of similar DMD patients (dataset provided by Cincinnati Children's Hospital Medical Center (CCHMC). Further, CAP-1002 treatment during the HOPE-2-OLE study continues to have a consistently well-tolerated safety profile throughout the study. The Company plans to present these results in more detail at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024. HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment entered the HOPE-2-OLE study where they receive CAP-1002 (150 million cells per infusion) every three months. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL 2.0 (p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety and functional performance.Breakeven Date Change • May 17Forecast breakeven date pushed back to 2026The 5 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$15.1m in 2026. Average annual earnings growth of 66% is required to achieve expected profit on schedule.Reported Earnings • May 14First quarter 2024 earnings released: US$0.31 loss per share (vs US$0.31 loss in 1Q 2023)First quarter 2024 results: US$0.31 loss per share (further deteriorated from US$0.31 loss in 1Q 2023). Revenue: US$4.91m (up 64% from 1Q 2023). Net loss: US$9.79m (loss widened 26% from 1Q 2023). Revenue is forecast to grow 39% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has fallen by 6% per year but the company’s share price has increased by 16% per year, which means it is well ahead of earnings.お知らせ • May 10Capricor Therapeutics, Inc. to Present Exosome Platform Updates At the American Society of Gene and Cell Therapy 27Th Annual MeetingCapricor Therapeutics, Inc. announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™? exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore, Maryland from May 7-11, 2024. The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.お知らせ • May 08Capricor Therapeutics, Inc. to Report Q1, 2024 Results on May 13, 2024Capricor Therapeutics, Inc. announced that they will report Q1, 2024 results After-Market on May 13, 2024お知らせ • Apr 24Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular DystrophyCapricor Therapeutics announced an update from the Company’s recent Type-B Chemistry, Manufacturing and Controls meeting with the U.S. Food and Drug Administration on next steps for the Biologics License Application submission with its lead asset CAP-1002 in treating Duchenne muscular dystrophy. The FDA has affirmed alignment with Capricor on the following topics: Pre-BLA Meeting and Rolling BLA Submission The FDA advised Capricor to include discussion for a pre-BLA meeting and rolling BLA schedule in the upcoming Type-B meeting. Based on this feedback, Capricor has already been granted a subsequent Type-B meeting to be held in the second quarter of 2024 to discuss these topics, with the results of those discussions to potentially lead to an accelerated BLA filing. Capricor plans to share with FDA its HOPE-2 open label extension 3-year safety and efficacy data which is expected to be available in the second quarter of 2024 as part of Capricor’s ISS and ISE strategy. Establishment of Non-Clinical Comparability The FDA agreed that comparability between drug product manufactured at its two different facilities (Los Angeles and San Diego) has been demonstrated using the provided analytical comparability data. This allows for the use of CAP-1002 drug product manufactured at its San Diego manufacturing facility upon potential product approval. Data from Cohort B of the HOPE-3 clinical trial will not be necessary for FDA approval of the product.お知らせ • Apr 02Capricor Therapeutics, Inc., Annual General Meeting, May 14, 2024Capricor Therapeutics, Inc., Annual General Meeting, May 14, 2024, at 10:00 Pacific Daylight. Location: 10865 Road to the Cure, Suite 150 San Diego California United States Agenda: To elect the nine nominees named in this proxy statement to the Company’s board of directors to serve for a one-year term expiring at 2025 Annual Meeting of Stockholders; to ratify the appointment of Rose, Snyder & Jacobs LLP as the Company’s independent registered public accounting firm for the fiscal year ending December 31, 2024; to approve, by non-binding advisory vote, the resolution approving named executive officer compensation; to approve an amendment in the form set forth on Annex A to this proxy statement to the Company’s Certificate of Incorporation, as amended from time to time (the “ Certificate of Incorporation ”) to increase the number of authorized shares of common stock from 50,000,000 to 100,000,000; and to transact such other business as may properly come before the Annual Meeting or any adjournment or postponement thereof.New Risk • Mar 29New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 10% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (10% average weekly change). Minor Risk Shareholders have been diluted in the past year (24% increase in shares outstanding).お知らせ • Mar 07Capricor Therapeutics, Inc. to Present Positive 24-Month Results from its HOPE-2 Open-Label Extension Study at 2024 Muscular Dystrophy Association Clinical & Scientific ConferenceCapricor Therapeutics, Inc. will present the positive 24-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at MDA Clinical and Scientific Conference which is taking place in Orlando, Florida from March 3-6, 2024. Key results from the study, include: CAP-1002 is an investigational cell therapy which aims to slow disease progression through immunomodulatory, anti-inflammatory and anti-fibrotic actions with the goal of potentially improving the rate of decline in skeletal and cardiac muscle function in patients with DMD. Skeletal muscle function as measured by the Performance of the Upper Limb (PUL v2.0) showed a mean PUL v2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7 point decline on average observed over 24-months in the placebo patient group. Delta change=4.9 points; p=0.021. The average rate of decline in CAP-1002 treated patients showed an attenuation of disease progression by approximately 64%. CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function. Cardiac function as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in 67% of patients, compared to a steady decline in a comparable natural history population. CAP-1002 treatment during the OLE portion of the study continues to yield a consistent favorable safety profile and has been well-tolerated throughout the study. The HOPE-2 OLE study previously met the primary endpoint at the one-year time-point and these 24-month results suggest that patients accumulate benefit over time with preservation of skeletal muscle function, which underscore the potential long-term benefit of CAP-1002. HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 24-months. These 24-month results were presented previously at the 2023 Parent Project Muscular Dystrophy (PPMD) Annual Conference.Breakeven Date Change • Mar 05Forecast breakeven date pushed back to 2026The 3 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests losses will reduce by 22% per year to 2025. The company is expected to make a profit of US$956.7k in 2026. Average annual earnings growth of 30% is required to achieve expected profit on schedule.Reported Earnings • Mar 03Full year 2023 earnings released: US$0.83 loss per share (vs US$1.18 loss in FY 2022)Full year 2023 results: US$0.83 loss per share (improved from US$1.18 loss in FY 2022). Revenue: US$25.2m (up US$22.6m from FY 2022). Net loss: US$22.3m (loss narrowed 23% from FY 2022). Revenue is forecast to grow 21% p.a. on average during the next 3 years, compared to a 13% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has fallen by 10% per year but the company’s share price has only fallen by 1% per year, which means it has not declined as severely as earnings.お知らせ • Feb 23Capricor Therapeutics, Inc. to Report Q4, 2023 Results on Feb 29, 2024Capricor Therapeutics, Inc. announced that they will report Q4, 2023 results After-Market on Feb 29, 2024お知らせ • Jan 24Capricor Therapeutics Announces Collaboration with the National Institutes of Health for Clinical Trial of Novel Exosome-Based Multivalent Vaccine for SARS-CoV-2Capricor Therapeutics announced that Capricor's proprietary StealthX exosome-based multivalent vaccine (StealthX™? vaccine) for the prevention of SARS-CoV-2 has been selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines providing broader and more durable protection for COVID-19. As part of Project NextGen, the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, will conduct a Phase 1 clinical study with Capricor's StealthX™? vaccine, subject to regulatory approval. NIAID's Division of Microbiology and Infectious Diseases (DMID) would oversee the study. The StealthX™? vaccine is a proprietary vaccine developed internally by Capricor utilizing exosomes that were engineered to express either spike or nucleocapsid proteins on the surface. Preclinical results from murine and Rabbit models published in Microbiology Spectrum, showed the StealthX™? vaccine, resulted in robust antibody production, potent neutralizing antibodies, a strong T-cell response and a favorable safety profile. These effects were obtained with administration of only nanogram amounts of protein and without adjuvant or synthetic lipid nanoparticles (LNPs). Exosomes offer a new antigen delivery system that potentially could be utilized to rapidly generate multivalent protein-based vaccines. Exosomes, first identified as extracellular vesicles, are small vesicles enriched in specific subsets of proteins, RNAs and lipids and responsible for cell-to-cell communication.お知らせ • Dec 12Capricor Therapeutics Announces Continuation of Phase 3 Hope-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility AnalysisCapricor Therapeutics announced a positive outcome of the interim futility analysis for HOPE-3, the pivotal Phase 3 trial evaluating CAP-1002 in patients with Duchenne muscular dystrophy (DMD). The results of the interim futility analysis, reviewed by the Data Safety Monitoring Board (DSMB), resulted in a favorable recommendation to continue the HOPE-3 trial as planned. The company believe CAP-1002 may address the high unmet medical need for these patients and remain committed to its expeditious advancement towards approval. Based on this important milestone, the company will be requesting a meeting with the U.S. Food and Drug Administration (FDA) to further discuss options for expedited review and approval. In addition, the company believe CAP-1002 will be requesting a Meeting with the U.S. food and Drug Administration (FDA). In addition, the company believe the company are well positioned to execute on additional value-driving clinical and regulatory milestones, including reporting topline data from HOPE-3 (Cohort A) in the fourth quarter of 2024. HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial comprised of two cohorts evaluating the safety and efficacy of CAP-1002 in participants with DMD and impaired skeletal muscle function. Non-ambulatory and ambulatory boys who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. Approximately 102 eligible study subjects will participate in this dual-cohort study. Enrollment has been completed for Cohort A which is designed to enroll approximately 58 subjects randomized to either CAP-1002 or trial in a 1:1 ratio and is intended to support a Biologics License Application submission. Enrollment has commenced for Cohort B which is designed to enroll approximately 44 participants randomized to either CAP-100 2 or placebo in a 1:1 ratio. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials. CAP-1002 for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for CAP-1002 is supported RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for CAP-1002 for thetreatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.New Risk • Nov 19New major risk - Financial positionThe company has less than a year of cash runway based on its current free cash flow trend. Free cash flow: -US$23m This is considered a major risk. With less than a year's worth of cash, the company will need to raise capital or take on debt unless its cash flows improve. This would dilute existing shareholders or increase balance sheet risk. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$23m free cash flow). Share price has been highly volatile over the past 3 months (25% average weekly change). Negative equity (-US$1.8m). Minor Risks Shareholders have been diluted in the past year (25% increase in shares outstanding). Market cap is less than US$100m (€85.8m market cap, or US$93.6m).お知らせ • Nov 08Capricor Therapeutics, Inc. to Report Q3, 2023 Results on Nov 14, 2023Capricor Therapeutics, Inc. announced that they will report Q3, 2023 results After-Market on Nov 14, 2023New Risk • Oct 03New minor risk - Market cap sizeThe company's market capitalization is less than US$100m. Market cap: €87.0m (US$91.2m) This is considered a minor risk. Companies with a small market capitalization are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (25% average weekly change). Minor Risks Shareholders have been diluted in the past year (26% increase in shares outstanding). Market cap is less than US$100m (€87.0m market cap, or US$91.2m).お知らせ • Oct 01Capricor Therapeutics Announces Positive Type-B Meeting with the FDA to Discuss Pathway to BLA for CAP-1002 in Duchenne Muscular DystrophyCapricor Therapeutics announced an update on the Company’s positive Type-B clinical meeting with the U.S. Food and Drug Administration (“FDA”) on the design and execution of HOPE-3, Capricor’s pivotal Phase 3 trial with lead asset CAP-1002 in treating Duchenne muscular dystrophy (“DMD”). Feedback from the FDA on trial design and timeline confirms CAP-1002’s path towards a future Biologics License Application (“BLA”) submission. The FDA has affirmed alignment on the Phase 3 clinical trial’s design and timeline. Key details for HOPE-3 are as follows: Primary endpoints remain unchanged. HOPE-3 will aim to enroll approximately 58 patients and enrollment is estimated to be completed in the fourth quarter of 2023. Capricor has treated 52 patients. Topline data for HOPE-3 is expected in the fourth quarter of 2024. Capricor plans to submit a BLA for CAP-1002 in 2025, which will be supported by results using product manufactured at the Los Angeles site. Capricor and the FDA discussed the potential for alternative approval pathways and Capricor plans to further discuss these options with the FDA following the completion of enrollment. CAP-1002 for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for CAP-1002 is supported RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for CAP-1002 for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (“PRV”) based on its previous receipt of a rare pediatric disease designation. Capricor retains full rights to the PRV, if received. Duchenne muscular dystrophy (“DMD”) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in approximately one in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited and there is no cure. CAP-1002 consists of allogeneic cardiosphere-derived cells (“CDCs”), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.お知らせ • Sep 07Capricor Therapeutics, Inc. Appoints Michael Kelliher to Its Board of DirectorsCapricor Therapeutics announced that the Company has appointed Michael Kelliher to its Board of Directors, effective immediately. Mr. Kelliher, Group Vice President of M&A and Business Development at Horizon Therapeutics, brings to Capricor extensive expertise in business development, strategy, finance and operational leadership across the life sciences industry. Mr. Kelliher is an experienced business development and finance professional with expertise in corporate strategy, mergers and acquisitions, strategic partnerships and licensing, with a career spanning more than 20 years with leading biotechnology and global pharmaceutical companies. He currently serves as Group Vice President, M&A and Business Development, at Horizon Therapeutics, a publicly traded global biotechnology company focused on researching, developing and commercializing medicines for rare, autoimmune and severe inflammatory diseases. Mr. Kelliher joined Horizon in 2014 and led an aggressive growth and expansion agenda through acquisitions, development collaborations and other transactions. He was instrumental in transforming Horizon into a $28 billion innovation-driven biotech company. Prior to his time at Horizon, from 2009 to 2014, Mr. Kelliher held progressive financial roles at Elan Corporation (now Perrigo Company), a leading global pharmaceutical company where he oversaw strategic partnerships and collaborations and advised its Board of Directors and senior leadership on investments, business development, product commercialization and asset monetization. Mr. Kelliher began his career in banking, public accounting and corporate finance and holds a Bachelor of Commerce degree from the University College Cork (Ireland). He is also an Associated Chartered Accountant.New Risk • Aug 25New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 5.0% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (5.0% average weekly change). Minor Risk Shareholders have been diluted in the past year (5.9% increase in shares outstanding).Board Change • Aug 25Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 7 experienced directors. 5 highly experienced directors. Independent Director George Dunbar was the last director to join the board, commencing their role in 2013. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.Breakeven Date Change • Aug 08Forecast breakeven date moved forward to 2024The 2 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$3.12m in 2024. Average annual earnings growth of 61% is required to achieve expected profit on schedule.お知らせ • Aug 03Capricor Therapeutics, Inc. to Report Q2, 2023 Results on Aug 07, 2023Capricor Therapeutics, Inc. announced that they will report Q2, 2023 results After-Market on Aug 07, 2023Board Change • Aug 01Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 7 experienced directors. 5 highly experienced directors. Independent Director George Dunbar was the last director to join the board, commencing their role in 2013. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.お知らせ • Jul 25+ 1 more updateCapricor Therapeutics Announces the Appointment of Philip J. Gotwals to Its Board of DirectorsCapricor Therapeutics announced the appointment of Philip J. Gotwals, Ph.D. to its Board of Directors, effective immediately. Dr. Gotwals, most recently the Global Head, Vice President of Business Development and Licensing at Novartis Institutes for Biomedical Research, bring his strategic vision for research, innovation for drug development and valuable experience to the Company. Dr. Gotwals has experience in drug development, research, corporate strategy and business development with a career spanning nearly 30 years in the biotechnology industry. Dr. Gotwals most recently served as the Global Head, Vice President of Business Development and Licensing at Novartis Institutes for Biomedical Research (NIBR) where he oversaw business development efforts for all disease areas and technology platforms. Under Dr. Gotwals’ leadership, NIBR business development and licensing executed over 50 major strategic transactions which included licensing deals, collaborations, acquisitions and new company creations. These transactions led to significant corporate evolution and growth. During his 13 years at Novartis, Dr. Gotwals was instrumental in building the company’s immuno-oncology strategic research area and spearheading the collaboration with the University of Pennsylvania to develop chimeric antigen receptor (CAR) T-cell therapies. Prior to Novartis, he was Vice President of Program Management at Altus Pharmaceuticals, where he was responsible for all product development project management activities. Prior to Altus, he was Senior Director of Program and Alliance Management at Biogen, where he oversaw leadership of internal and allied early product development teams in the autoimmune, neurology and oncology therapeutic areas. Dr. Gotwals has a B.A. in Biology from Amherst College, holds a Ph.D. in Genetics from the University of California at Berkeley, completed postdoctoral research at the Massachusetts Institute of Technology, business training at Harvard Business School and has published extensively in the area of integrin biology.お知らせ • Jul 14Capricor Therapeutics, Inc Announces Board ChangesOn July 11, 2023, Mr. Louis Manzo, a member of the Board of Directors of Capricor Therapeutics, Inc. resigned from the Board effective immediately. Mr. Manzo’s decision to resign is not due to any disagreement with the Company on any of the Company’s operations, policies or practices. On July 11, 2023, the Board elected Paul Auwaerter, M.D. as a member of the Board, effective immediately. Dr. Auwaerter will receive an initial grant of a nonstatutory stock option to purchase 115,000 shares of the Company’s common stock (the “Initial Grant”) as standard non-employee director compensation under the Company’s policies. The Initial Grant will vest with respect to 25% of such shares of common stock on the first anniversary of the first day of the month following the date of grant of the Initial Grant, and the Initial Grant will vest with respect to 1/36 of such shares of common stock each month thereafter. Dr. Auwaerter will also enter into the Company’s standard form of indemnification agreement for directors. Dr. Auwaerter is the Sherrilyn and Ken Fisher Professor of Medicine at the Johns Hopkins University School of Medicine, serving as the Clinical Director for the Division of Infectious Diseases and Director of the Sherrilyn and Ken Fisher Center for Environmental Infectious Diseases. Dr. Auwaerter serves as the Executive Director of the Johns Hopkins Point of Care-Information Technology (POC-IT) Center producing the Johns Hopkins ABX (Antibiotic), JH HIV, JH Osler, JH Psychiatry and JH Diabetes Guides. Dr. Auwaerter also serves as Editor-in-Chief of the ABX Guide. Dr. Auwaerter’s research and clinical interests include improving the diagnosis and care for patients with infectious diseases, specifically Lyme disease, respiratory infections, surgical infections, prosthetic joint infections, Epstein-Barr virus, and fever of unknown origin. He is a Past President of the Infectious Diseases Society of America (IDSA), the largest professional society worldwide related to infectious diseases. He currently serves as Chairman of the Infectious Diseases Society of America Foundation and is also on the Board of Directors of the American Lyme Disease Foundation. Dr. Auwaerter has been a member of the Board of Directors of the Baltimore Area Council of the Boy Scouts of America and has been awarded the national Distinguished Eagle Scout award. Dr. Auwaerter holds undergraduate and medical degrees from Columbia University and subsequent training in medicine and infectious diseases at Johns Hopkins, where he has been employed since 1988; he also trained in the virology and immunology laboratory of Dr. Diane Griffin.お知らせ • Jul 01Capricor Therapeutics Announces Positive 24-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular DystrophyCapricor Therapeutics announced positive 24-month safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). Data from the OLE study demonstrated that the majority of patients had an improvement in left ventricular ejection fraction (LVEF), after two years of CAP-1002 treatment, which suggests preservation of cardiac function. Additionally, patients continue to show statistically significant benefit (p=0.021) after two years of treatment in the Performance of the Upper Limb (PUL v2.0) scale when compared to the original rate of decline of the placebo group from HOPE-2 after one year. Furthermore, the OLE study continues to show a favorable safety profile for long-term treatment of CAP-1002. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale (p=0.02). At the 24-month timepoint, data showed statistically significant differences in the PUL v2.0 in the OLE treatment group when compared to the original rate of decline of the placebo group from HOPE-2 after one-year (p=0.021). LVEF was measured using cardiac magnetic resonance imaging (cMRI) and six of nine patients showed improvements in heart function with CAP-1002 treatment compared to their final assessment at the end of the HOPE-2 study. Over time, there was an increasing correlation with PUL v2.0 and ejection fraction results (24-month OLE results r=0.75, p=0.02). HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 24 months. Patients continued through the gap phase (off treatment for both groups) and the OLE phase (on treatment for both groups).お知らせ • Jun 08Capricor Therapeutics Announces Follow-up Type-B Clinical Meeting with the FDA for CAP-1002 for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced an upcoming Type-B clinical meeting with the U.S. Food and Drug Administration (FDA), planned in early Third Quarter 2023. During the planned meeting with the FDA, Capricor will outline its proposed path towards submission of a potential Biologics License Application (BLA) and further discuss its ongoing HOPE-3 clinical trial with the agency. The regulatory pathway for CAP-1002 is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) as well as Orphan Drug Designation. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher based on its designation as a rare pediatric disease. HOPE-3, the Company's Phase 3 clinical trial of CAP-1002 is a multi-center, randomized, double-blind, placebo-controlled study (NCT05126758) which is designed to treat up to 68 subjects in the United States. Duchenne muscular dystrophy is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. CAP-1002 consists of allogeneic cardiosphere-derived cells (CDCs), a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity and is being investigated for its potential to modify the immune system's activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.Breakeven Date Change • May 14Forecast breakeven date moved forward to 2024The 2 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$11.8m in 2024. Average annual earnings growth of 58% is required to achieve expected profit on schedule.お知らせ • May 05Capricor Therapeutics, Inc. to Report Q1, 2023 Results on May 11, 2023Capricor Therapeutics, Inc. announced that they will report Q1, 2023 results After-Market on May 11, 2023Reported Earnings • Mar 18Full year 2022 earnings released: US$1.18 loss per share (vs US$0.87 loss in FY 2021)Full year 2022 results: US$1.18 loss per share (further deteriorated from US$0.87 loss in FY 2021). Net loss: US$29.0m (loss widened 45% from FY 2021). Revenue is forecast to grow 40% p.a. on average during the next 3 years, compared to a 21% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has increased by 16% per year but the company’s share price has increased by 57% per year, which means it is tracking significantly ahead of earnings growth.Breakeven Date Change • Mar 18Forecast breakeven date moved forward to 2024The 2 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$12.9m in 2024. Average annual earnings growth of 54% is required to achieve expected profit on schedule.お知らせ • Jan 26Capricor Therapeutics, Inc. Announces Positive 18-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy PatientsCapricor Therapeutics, Inc. announced positive 18-month results from its ongoing HOPE-2 open label extension (OLE) study in patients with later-stage Duchenne muscular dystrophy (DMD). Data from the OLE study continues to show evidence for disease modification with statistically significant differences in the Performance of the Upper Limb (PUL version 2.0) scale in the CAP-1002 original treatment group when compared to the original placebo group from HOPE-2 (p=0.02). In addition, disease progression is attenuated equally in both groups once patients begin treatment in the OLE. The study met its primary endpoint at the one-year timepoint and continues to show statistically significant improvements (p=0.02) on the Performance of the Upper Limb scale for patients treated with CAP-1002 at 18-months. In the study, CAP-1002 was made available to the original 20 patients enrolled in the HOPE-2 study. Of those, 13 entered and 12 completed 18-months of study follow-up. The breakdown of patients included seven from the original placebo group and six from the original CAP-1002 treatment group. All patients were off CAP-1002 or placebo for a mean of approximately one-year before resumption or initiation of therapy under the HOPE-2 OLE program. As in HOPE-2, CAP-1002 was administered quarterly and current results are from the 18-month analysis. CAP-1002 treatment during the open-label portion of the study continues to yield a consistent safety profile and has been well-tolerated throughout the study. The HOPE-2 OLE study is ongoing, and all participants continue to be monitored for safety and functional performance. The company is currently conducting a pivotal Phase 3 trial, HOPE-3, designed as a randomized, double-blind, placebo-controlled study with approximately 70 patients and enrollment criteria similar to HOPE-2. The Phase 3 study is currently enrolling subjects (NCT05126758).Breakeven Date Change • Dec 31Forecast to breakeven in 2025The 3 analysts covering Capricor Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$89.3m in 2025. Average annual earnings growth of 59% is required to achieve expected profit on schedule.Board Change • Nov 17Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 6 experienced directors. 6 highly experienced directors. Independent Director George Dunbar was the last director to join the board, commencing their role in 2013. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.Reported Earnings • Nov 12Third quarter 2022 earnings released: US$0.26 loss per share (vs US$0.17 loss in 3Q 2021)Third quarter 2022 results: US$0.26 loss per share (further deteriorated from US$0.17 loss in 3Q 2021). Net loss: US$6.37m (loss widened 62% from 3Q 2021). Revenue is forecast to grow 64% p.a. on average during the next 3 years, compared to a 24% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has increased by 33% per year but the company’s share price has increased by 44% per year, which means it is tracking significantly ahead of earnings growth.株主還元4LN2DE BiotechsDE 市場7D-66.3%0.8%1.5%1Y-13.2%4.6%0.8%株主還元を見る業界別リターン: 4LN2過去 1 年間で4.6 % の収益を上げたGerman Biotechs業界を下回りました。リターン対市場: 4LN2は、過去 1 年間で0.8 % のリターンを上げたGerman市場を下回りました。価格変動Is 4LN2's price volatile compared to industry and market?4LN2 volatility4LN2 Average Weekly Movement20.9%Biotechs Industry Average Movement7.1%Market Average Movement5.3%10% most volatile stocks in DE Market12.8%10% least volatile stocks in DE Market2.8%安定した株価: 4LN2の株価は、 German市場と比較して過去 3 か月間で変動しています。時間の経過による変動: 4LN2の 週次ボラティリティ は過去 1 年間で54%から21%に減少しましたが、依然としてGerman株の 75% よりも高くなっています。会社概要設立従業員CEO（最高経営責任者ウェブサイト2005231Linda Marbánwww.capricor.comCapricor Therapeutics, Inc.は臨床段階のバイオテクノロジー企業で、米国においてデュシェンヌ型筋ジストロフィー（DMD）やその他のアンメット・メディカル・ニーズのある疾患を治療するための、形質転換細胞およびエクソソームに基づく治療薬の開発に従事している。同社の主要製品候補は、同種心筋球由来の細胞であるデラミオセルで、DMDの治療薬として第3相臨床試験中である。また、SARS-CoV-2治療薬として前臨床試験中のエクソソーム・タンパク質ベース・ワクチン、StealthXエクソソーム・プラットフォーム、ワクチンや治療薬開発のための人工エクソソームからなるエクソソーム・プラットフォーム・プログラムを開発し、前臨床試験中である。さらに、さまざまな疾患の治療や予防のための標的RNA、タンパク質、低分子治療薬など、さまざまな治療用途のために第1相臨床試験中のエクソソームベースの人工ワクチン候補であるStealthXや、DMDの治療のために前臨床試験中のCAP-2003、同種心筋由来細胞エクソソームの開発に取り組んでいる。また、ジョンズ・ホプキンス大学、ローマ大学ライセンス校、シダーズ・サイナイ・メディカル・センターとライセンス契約を結び、全分野の特許権およびノウハウの非独占的権利のもと、ライセンス製品およびライセンスサービスを開発・商業化する。同社は2005年に設立され、カリフォルニア州サンディエゴに本社を置いている。もっと見るCapricor Therapeutics, Inc. 基礎のまとめCapricor Therapeutics の収益と売上を時価総額と比較するとどうか。4LN2 基礎統計学時価総額€332.27m収益（TTM）-€100.07m売上高(TTM)n/a0.0xP/Sレシオ-3.3xPER（株価収益率4LN2 は割高か？公正価値と評価分析を参照収益と収入最新の決算報告書（TTM）に基づく主な収益性統計4LN2 損益計算書（TTM）収益US$0売上原価US$92.92m売上総利益-US$92.92mその他の費用US$21.68m収益-US$114.59m直近の収益報告Mar 31, 2026次回決算日該当なし一株当たり利益（EPS）-1.98グロス・マージン0.00%純利益率0.00%有利子負債／自己資本比率0%4LN2 の長期的なパフォーマンスは？過去の実績と比較を見るView Valuation企業分析と財務データの現状データ最終更新日（UTC時間）企業分析2026/07/29 23:12終値2026/07/29 00:00収益2026/03/31年間収益2025/12/31データソース企業分析に使用したデータはS&P Global Market Intelligence LLC のものです。本レポートを作成するための分析モデルでは、以下のデータを使用しています。データは正規化されているため、ソースが利用可能になるまでに時間がかかる場合があります。パッケージデータタイムフレーム米国ソース例会社財務10年損益計算書キャッシュ・フロー計算書貸借対照表SECフォーム10-KSECフォーム10-Qアナリストのコンセンサス予想+プラス3年予想財務アナリストの目標株価アナリストリサーチレポートBlue Matrix市場価格30年株価配当、分割、措置ICEマーケットデータSECフォームS-1所有権10年トップ株主インサイダー取引SECフォーム4SECフォーム13Dマネジメント10年リーダーシップ・チーム取締役会SECフォーム10-KSECフォームDEF 14A主な進展10年会社からのお知らせSECフォーム8-K* 米国証券を対象とした例であり、非米国証券については、同等の規制書式および情報源を使用。特に断りのない限り、すべての財務データは1年ごとの期間に基づいていますが、四半期ごとに更新されます。これは、TTM（Trailing Twelve Month）またはLTM（Last Twelve Month）データとして知られています。詳細はこちら。分析モデルとスノーフレークこのレポートを生成するために使用した分析モデルの詳細は、当社のGitHubページでご覧いただけます。また、レポートの活用方法に関するガイドやYouTubeのチュートリアルも用意しています。シンプリー・ウォールストリート分析モデルを設計・構築した世界トップクラスのチームについてご紹介します。業界およびセクターの指標私たちの業界とセクションの指標は、Simply Wall Stによって6時間ごとに計算されます。アナリスト筋Capricor Therapeutics, Inc. 10 これらのアナリストのうち、弊社レポートのインプットとして使用した売上高または利益の予想を提出したのは、 。アナリストの投稿は一日中更新されます。10 アナリスト機関Matthew VeneziaAlliance Global PartnersMadison Wynne El-SaadiB. Riley Securities, Inc.Kristen KluskaCantor Fitzgerald & Co.7 その他のアナリストを表示
お知らせ • 5hCapricor Therapeutics Publishes Phase 3 Hope-3 Trial Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced that The Lancet has published results from the Company’s pivotal Phase 3 HOPE-3 clinical trial evaluating Deramiocel, its investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The manuscript underwent independent expert peer review, providing external validation of the trial’s design, statistical methodology and findings. The results are based on the Company’s prespecified Statistical Analysis Plan version 3.0 (SAP 3.0). The paper, titled “Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial” can be accessed. The randomized, double-blind, placebo-controlled HOPE-3 Phase 3 trial (n=106) met primary endpoint, with Deramiocel slowing upper limb function decline by 54% versus placebo (PUL 2.0, p=0.03) and showing clinically meaningful cardiac benefit. Deramiocel BLA remains under active FDA review, with PDUFA target action date of August 22, 2026. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
New Risk • Jul 27New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 21% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (21% average weekly change). Revenue is less than US$1m. Minor Risk Shareholders have been diluted in the past year (27% increase in shares outstanding).
お知らせ • Jun 29+ 4 more updatesCapricor Therapeutics Announces FDA Advisory Committee Meeting To Review BLA For Deramiocel For The Treatment Of Duchenne Muscular DystrophyCapricor Therapeutics announced that the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) of the U.S. Food and Drug Administration (FDA) is planning to convene an advisory committee meeting to discuss the Company's Biologics License Application (BLA) seeking approval of Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The BLA is supported by the Company's Phase 2 HOPE-2 trial and long-term outcomes from the HOPE-2-OLE trial, as well as positive results from the Phase 3 HOPE-3 trial, which achieved statistical significance on its primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints. The date for the Advisory Committee meeting is July 29, 2026, and the meeting will be available for live streaming. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • Jun 26Capricor Therapeutics Presents Positive Five-Year HOPE-2 Ole Data and HOPE-3 Phase 3 Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, taking place June 25-27, 2026, in Orlando, Florida, alongside previously reported results from the Company’s HOPE-3 Phase 3 trial. Among the patients who remain enrolled in the HOPE-2 OLE study (n=9), treatment with Deramiocel continued to attenuate disease progression over five years, as measured by Performance of the Upper Limb (PUL 2.0). Patients experienced a mean total-score decline of less than 5 points over five years. By comparison, a cohort-matched external comparator of standard-of-care DMD patients showed a modeled decline of roughly 2.4 points per year, which if continued would project to approximately 12 points over five years. A separately published natural-history analysis (Coratti, et al., J Neuromuscular Dis, 2025) reported a decline in non-ambulant patients of approximately 8.1 points over three years, consistent with a meaningful treatment benefit. Cardiac function, as measured by left ventricular ejection fraction (LVEF) on cardiac MRI, remained stable over the full five-year period, in contrast to the modeled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator. Deramiocel continued to demonstrate a favorable safety profile throughout the study, consistent with over 800 intravenous infusions administered to date across the Deramiocel clinical development program. The multicenter, randomized, double-blind, placebo-controlled trial enrolled 106 patients with DMD and met its primary endpoint (PUL 2.0, p=0.03) and key secondary cardiac endpoint (LVEF, p=0.04), along with all other Type I error-controlled secondary endpoints – consistent with the durable skeletal and cardiac muscle benefit observed in the HOPE-2 OLE study. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • May 05Capricor Therapeutics, Inc. to Report Q1, 2026 Results on May 12, 2026Capricor Therapeutics, Inc. announced that they will report Q1, 2026 results After-Market on May 12, 2026
お知らせ • Apr 22Capricor Therapeutics Announces Late-Breaking Presentation of Hope-3 Phase 3 ResultsCapricor Therapeutics had announced the presentation of data from its Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago, Illinois. The data were presented by Dr. Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children's Hospital, during the Late-Breaking Science 2 session on Tuesday, April 21, 2026. These findings were further supported by an alternative measure of upper limb function, specifically video recordings of tasks performed at home (the Duchenne Video Assessment, or DVA), which showed meaningful slowing of disease progression in the ability to self-feed, a function central to patient independence. These data reinforce the potential of Deramiocel to make a meaningful difference in the lives of those living with Duchenne. The HOPE-3 data tell a compelling story of preserved function, slowed decline, and real-world impact on patients' daily lives, evidence we believe positions Deramiocel as a potentially transformative therapy for Duchenne. With our BLA currently under FDA review and a PDUFA target action date of August 22, 2026, we remain on track and focused on bringing this therapy to the patients who need it as quickly as possible. Data from this presentation were released under AAN embargo on April 21, 2026 at 4:45 p.m. ET. Additional information about the 2026 AAN Annual Meeting is available at www.aan.com. The Company's presentation will be made available in the Investors section of Capricor's website shortly. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • 5hCapricor Therapeutics Publishes Phase 3 Hope-3 Trial Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced that The Lancet has published results from the Company’s pivotal Phase 3 HOPE-3 clinical trial evaluating Deramiocel, its investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The manuscript underwent independent expert peer review, providing external validation of the trial’s design, statistical methodology and findings. The results are based on the Company’s prespecified Statistical Analysis Plan version 3.0 (SAP 3.0). The paper, titled “Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial” can be accessed. The randomized, double-blind, placebo-controlled HOPE-3 Phase 3 trial (n=106) met primary endpoint, with Deramiocel slowing upper limb function decline by 54% versus placebo (PUL 2.0, p=0.03) and showing clinically meaningful cardiac benefit. Deramiocel BLA remains under active FDA review, with PDUFA target action date of August 22, 2026. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
New Risk • Jul 27New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 21% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (21% average weekly change). Revenue is less than US$1m. Minor Risk Shareholders have been diluted in the past year (27% increase in shares outstanding).
お知らせ • Jun 29+ 4 more updatesCapricor Therapeutics Announces FDA Advisory Committee Meeting To Review BLA For Deramiocel For The Treatment Of Duchenne Muscular DystrophyCapricor Therapeutics announced that the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) of the U.S. Food and Drug Administration (FDA) is planning to convene an advisory committee meeting to discuss the Company's Biologics License Application (BLA) seeking approval of Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The BLA is supported by the Company's Phase 2 HOPE-2 trial and long-term outcomes from the HOPE-2-OLE trial, as well as positive results from the Phase 3 HOPE-3 trial, which achieved statistical significance on its primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints. The date for the Advisory Committee meeting is July 29, 2026, and the meeting will be available for live streaming. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • Jun 26Capricor Therapeutics Presents Positive Five-Year HOPE-2 Ole Data and HOPE-3 Phase 3 Results for Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, taking place June 25-27, 2026, in Orlando, Florida, alongside previously reported results from the Company’s HOPE-3 Phase 3 trial. Among the patients who remain enrolled in the HOPE-2 OLE study (n=9), treatment with Deramiocel continued to attenuate disease progression over five years, as measured by Performance of the Upper Limb (PUL 2.0). Patients experienced a mean total-score decline of less than 5 points over five years. By comparison, a cohort-matched external comparator of standard-of-care DMD patients showed a modeled decline of roughly 2.4 points per year, which if continued would project to approximately 12 points over five years. A separately published natural-history analysis (Coratti, et al., J Neuromuscular Dis, 2025) reported a decline in non-ambulant patients of approximately 8.1 points over three years, consistent with a meaningful treatment benefit. Cardiac function, as measured by left ventricular ejection fraction (LVEF) on cardiac MRI, remained stable over the full five-year period, in contrast to the modeled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator. Deramiocel continued to demonstrate a favorable safety profile throughout the study, consistent with over 800 intravenous infusions administered to date across the Deramiocel clinical development program. The multicenter, randomized, double-blind, placebo-controlled trial enrolled 106 patients with DMD and met its primary endpoint (PUL 2.0, p=0.03) and key secondary cardiac endpoint (LVEF, p=0.04), along with all other Type I error-controlled secondary endpoints – consistent with the durable skeletal and cardiac muscle benefit observed in the HOPE-2 OLE study. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • May 05Capricor Therapeutics, Inc. to Report Q1, 2026 Results on May 12, 2026Capricor Therapeutics, Inc. announced that they will report Q1, 2026 results After-Market on May 12, 2026
お知らせ • Apr 22Capricor Therapeutics Announces Late-Breaking Presentation of Hope-3 Phase 3 ResultsCapricor Therapeutics had announced the presentation of data from its Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago, Illinois. The data were presented by Dr. Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children's Hospital, during the Late-Breaking Science 2 session on Tuesday, April 21, 2026. These findings were further supported by an alternative measure of upper limb function, specifically video recordings of tasks performed at home (the Duchenne Video Assessment, or DVA), which showed meaningful slowing of disease progression in the ability to self-feed, a function central to patient independence. These data reinforce the potential of Deramiocel to make a meaningful difference in the lives of those living with Duchenne. The HOPE-3 data tell a compelling story of preserved function, slowed decline, and real-world impact on patients' daily lives, evidence we believe positions Deramiocel as a potentially transformative therapy for Duchenne. With our BLA currently under FDA review and a PDUFA target action date of August 22, 2026, we remain on track and focused on bringing this therapy to the patients who need it as quickly as possible. Data from this presentation were released under AAN embargo on April 21, 2026 at 4:45 p.m. ET. Additional information about the 2026 AAN Annual Meeting is available at www.aan.com. The Company's presentation will be made available in the Investors section of Capricor's website shortly. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • Apr 02Capricor Therapeutics, Inc., Annual General Meeting, Jun 04, 2026Capricor Therapeutics, Inc., Annual General Meeting, Jun 04, 2026. Location: 10865 road to the cure, suite 150, 92121., san diego, Canada
お知らせ • Mar 13Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular DystrophyCapricor Therapeutics announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD), which were presented at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference in Orlando, Florida. Cardiac MRI analyses demonstrated Deramiocel’s impact on cardiac structure in patients with DMD. Evaluation of late gadolinium enhancement (LGE), a marker of myocardial fibrosis, showed a significant reduction in fibrotic segments in patients treated with Deramiocel versus placebo, corresponding to a three-segment treatment difference at 12 months (p=0.022). The presence of LGE reflects replacement of viable myocardium with fibrotic tissue and is associated with progressive cardiac dysfunction and heart failure risk in DMD cardiomyopathy. In patients with baseline cardiomyopathy, Deramiocel demonstrated an even greater treatment effect on cardiac function. In this subgroup, treatment resulted in a 3.3 percentage-point improvement in LVEF versus placebo, corresponding to greater than 100% attenuation of expected cardiac decline (p=0.017). A Global Statistical Test (GST), a patient-level composite including Performance of Upper Limb (PUL v2.0), left ventricular ejection fraction (LVEF), and Patient Global Impression of Severity (PGI-S), demonstrated a statistically significant overall treatment benefit favoring Deramiocel (p=0.017). This composite integrates multiple clinically meaningful domains of disease, reflecting how patients feel and function. Additional functional outcomes evaluating hand-to-mouth activity, an important measure of patient independence, were also presented. Data from the Duchenne Video Assessment (DVA), a measure of activities of daily living in individuals with Duchenne, showed that the “eat 10 bites” task resulted in approximately 83% slowing of disease progression compared with placebo (p=0.018). These findings align with results from the mid-level (elbow) PUL v2.0 assessment (p=0.008), providing concordant evidence across both validated clinical and real-world functional measures. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • Mar 10Capricor Therapeutics, Inc. Establishes New PDUFA Date For Deramiocel BLACapricor Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has lifted the previously issued Complete Response Letter and resumed review of its Biologics License Application (BLA) seeking full approval of Deramiocel, an investigational cell therapy, for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. The submission has been classified as a Class 2 resubmission, with a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026. The Company received a Complete Response Letter (CRL) from the FDA in July 2025. Following submission of data and supporting documentation from the HOPE-3 clinical trial, the FDA resumed review of the application and assigned a PDUFA target action date of August 22, 2026. At this time, the FDA has not identified any potential review issues in its response to the Company. Capricor also expects to be eligible to receive a Priority Review Voucher (PRV) upon potential approval of Deramiocel. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co. Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX vaccine are investigational candidates and have not been approved for commercial use in any indication.
お知らせ • Mar 09Capricor Therapeutics, Inc. to Report Q4, 2025 Results on Mar 12, 2026Capricor Therapeutics, Inc. announced that they will report Q4, 2025 results After-Market on Mar 12, 2026
お知らせ • Jan 20Capricor Therapeutics Provides Regulatory Updates Regarding Its Biologics License Application for DeramiocelCapricor Therapeutics provided a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, the Company’s investigational first-in-class cell therapy for the treatment of Duchenne muscular dystrophy (DMD). As previously disclosed, the Company provided topline results from its Phase 3 HOPE-3 clinical study to the U.S. Food and Drug Administration (FDA) in late 2025. Following its review of these data, the FDA has formally requested the full HOPE-3 clinical study report (CSR) and supporting data to address the Complete Response Letter (CRL). The FDA did not request any additional clinical studies or new patient data as part of this request. Preparation of the HOPE-3 CSR is well underway, and the Company plans to submit the requested materials to the FDA in February 2026. The Company expects that this submission will address the items outlined in the CRL and support continued review of the BLA, including the assignment of a new Prescription Drug User Fee Act (PDUFA) target action date.
お知らせ • Dec 17Capricor Therapeutics Announces Positive Topline Results From Phase 3 HOPE-3 Trial Of Deramiocel For Duchenne Muscular DystrophyCapricor Therapeutics announced that Parent Project Muscular Dystrophy (PPMD) will host a community webinar to share and discuss positive topline results from Capricor’s Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The webinar will provide patients and families with an overview of the HOPE-3 results and how the findings inform ongoing regulatory discussions, including next steps with the U.S. Food and Drug Administration (FDA). Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co. Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX™ vaccine are investigational candidates and have not been approved for commercial use in any indication.
お知らせ • Dec 05+ 1 more updateCapricor Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $150 million.Capricor Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $150 million. Security Name: Common Stock Security Type: Common Stock Securities Offered: 6,000,000 Price\Range: $25
お知らせ • Dec 03Capricor Therapeutics, Inc. Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics, Inc. announced positive topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with Duchenne muscular dystrophy. The study randomized 106 participants across 20 leading U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period. The average age of participants was approximately 15 years, and all were on a stable corticosteroid regimen throughout the study. Baseline demographics were well balanced between treatment arms, approximately 90 percent were receiving cardiac medications at baseline, and over 75 percent had a clinical diagnosis of cardiomyopathy. Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience. They expect that detailed HOPE-3 results will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.
お知らせ • Nov 25Capricor Therapeutics, Inc. Presents New Data Demonstrating a Scalable Framework for Loading Therapeutic Oligonucleotides into ExosomesCapricor Therapeutics, Inc. announced new data describing a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into exosomes. The data were presented at the 2025 American Association for Extracellular Ventures (AAEV) Annual Meeting in Salt Lake City, Utah, held from November 20-23, 2025. The poster titled, "A Systematic Framework for the Scalable Loading of Therapeutic siRNA and PMO into Exosomes," highlighted proprietary data showcasing Capricor's exosome-based technology. The data provided an overview of both scale-up and scale-out electroporation strategies that can be integrated to achieve substantially larger yields of exogenously loaded engineered exosomes, which is a critical requirement for further clinical development. Key findings include: Engineered exosomes derived from 293F cells were successfully loaded with therapeutic siRNA and PMO cargo using optimized electroporation conditions. Both scale-up and scale- out strategies produced comparable loading efficiencies to standard small-volume electroporation. Integrating the two approaches enables manufacturing of significantly larger batches of therapeutic exosomes. At the forefront of innovation is the lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for the treatment of Duchenne muscular dystrophy (DMD).
お知らせ • Oct 31Capricor Therapeutics, Inc. to Report Q3, 2025 Results on Nov 10, 2025Capricor Therapeutics, Inc. announced that they will report Q3, 2025 results After-Market on Nov 10, 2025
お知らせ • Sep 25Capricor Therapeutics Provides Regulatory Update on Deramiocel Program for Duchenne Muscular DystrophyCapricor Therapeutics announced a regulatory update for its Biologics License Application (BLA) for Deramiocel, the Company's investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). This update follows a recent Type A meeting with the U.S. Food and Drug Administration (FDA) after the receipt of a Complete Response Letter (CRL) in July 2025. The goal of the Type A meeting was to establish a path toward potential approval of Deramiocel for the treatment of DMD. Key outcomes included: The HOPE-3 clinical trial should serve as the "add additional study" requested in the CRL. The HOPE-3 data can be submitted within the current BLA, maintaining PUL v2.0 as the primary efficacy endpoint and suggesting left ventricular ejection fraction (LVEF) as a key secondary endpoint, which Capricor intends to request for labeling consideration. Capricor plans to submit HOPE-3 data with its complete response to the CRL, with the goal of securing a label encompassing both cardiac and skeletal muscle function in DMD. In its meeting minutes, the FDA further emphasized its commitment, stating: "The FDA remains committed to collaborating with the applicant and will exercise further regulatory flexibility by reviewing data from the HOPE-3 trial". Importantly, prior to issuance of the CRL, the majority of the BLA had undergone rigorous review with no significant deficiencies identified by the FDA during the mid-cycle review or pre-licensing inspections. All CMC items identified in the CRL have been addressed and communicated to the FDA. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of Duchenane Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial consisting of two supports evaluating the safety and efficacy of Deramiocel in participants with DMD.
お知らせ • Sep 09Capricor Therapeutics Responds to FDA Posting of Complete Response Letter for DeramiocelCapricor Therapeutics issued a statement regarding the U.S. Food and Drug Administration’s (FDA) public posting of its Complete Response Letter (CRL) for the Biologics License Application (BLA) for Deramiocel, the Company’s investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The Company was not notified in advance that the CRL would be posted, but acknowledges the FDA’s decision to publish the letter, originally received in July 2025. However, the FDA did not release the comprehensive preliminary response that Capricor submitted shortly after receipt of the CRL. This written response provided clarifications to the Agency’s feedback and outlined the Company’s proposed plan to address the outstanding issues. To ensure transparency, Capricor will make its preliminary response available on the investor section of its website for patients, families, and other stakeholders to review. Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD afflicts approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials.Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
お知らせ • Aug 19Capricor Therapeutics Announces First Subjects Dosed in Phase 1 Clinical Trial of Novel Exosome-Based VaccineCapricor Therapeutics announced that the first subjects have been dosed in a Phase 1 clinical trial evaluating its StealthX exosome-based vaccine. The study, funded by the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) under the U.S. Department of Health and Human Services' Project NextGen, follows review and clearance of the Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). The trial is being conducted by an NIAID-funded network of clinical trial sites. Project NextGen is a federal initiative aimed at accelerating the development of next-generation vaccine platforms that provide broader and more durable protection against respiratory viruses and other infectious threats. The Phase 1 trial includes four dosing arms and is initially focused on the spike (S) protein of SARS-CoV-2. An additional arm of Capricor's StealthX multivalent vaccine incorporating the nucleocapsid (N) protein is planned, pending separate FDA clearance. The StealthX vaccine is a proprietary exosome-based platform developed internally by Capricor, using exosomes engineered to display either the spike or nucleocapsid proteins on their surface. Preclinical results published in Microbiology Spectrum demonstrated that StealthX elicited robust antibody production, potent neutralizing activity, a strong T-cell response and a favorable safety profile.
お知らせ • Aug 06Capricor Therapeutics, Inc. to Report Q2, 2025 Results on Aug 11, 2025Capricor Therapeutics, Inc. announced that they will report Q2, 2025 results After-Market on Aug 11, 2025
お知らせ • Aug 01Levi & Korsinsky, LLP Notifies Investors in Capricor Therapeutics, Inc. of A Class Action Securities LawLevi & Korsinsky, LLP notified investors in Capricor Therapeutics, Inc. of a class action securities lawsuit. The lawsuit seeks to recover losses on behalf of Capricor investors who were adversely affected by alleged securities fraud between October 9, 2024 and July 10, 2025. According to the complaint, defendants provided investors with material information concerning Capricor's lead cell therapy candidate drug deramiocel for the treatment of cardiomyopathy associated withuchenne muscular dystrophy (DMD). Participants' statements included, among other things, Capricor's ability to obtain a Biologics License Application (BLA) for deramiocel from the U.S. Food and Drug Administration (FDA). Defendants provided these overwhelmingly positive statements to investors while, at the same time, disseminating false and misleading statements and/or concealing material adverse facts concerning its four-year safety and efficacy data from its Phase 2 HOPE-2 trial study of deramiocel. On July 11, 2025, Capricor issued a press release announcing it received a Complete Response Letter (CRL) from the FDA denying the BLA specifically citing it did not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data. Further, the CRL referenced outstanding items in the Chemistry, Manufacturing, and Controls section of the application.
お知らせ • Jul 11Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular DystrophyCapricor Therapeutics announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). In the CRL, the FDA stated that it had completed its review of the application but is unable to approve the BLA in its current form, specifically citing that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data. The CRL also referenced certain outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section of the application, most of which Capricor believes it has addressed in prior communications to the FDA. However, these materials were not reviewed by the FDA due to the timing of the CRL issuance. The FDA confirmed that it will restart the review clock upon resubmission. In addition, the agency offered the company the opportunity to request a Type A meeting to discuss the path forward. Capricor plans to engage further with the FDA to determine the appropriate next steps. Capricor's BLA for Deramiocel was granted Priority Review in March 2025 and was supported by data from the HOPE-2 trial, its open-label extension (OLE), and natural history comparisons from FDA-funded datasets.
お知らせ • Jun 30+ 5 more updatesCapricor Therapeutics, Inc.(NasdaqCM:CAPR) dropped from Russell 3000E Value IndexCapricor Therapeutics, Inc.(NasdaqCM:CAPR) dropped from Russell 3000E Value Index
お知らせ • Jun 24Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular DystrophyCapricor Therapeutics provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)--associated cardiomyopathy. As part of the FDA's ongoing review, the Company has been informed that an Advisory Committee meeting is not indicated at this time. The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running treatment datasets in DMD, including measures of both cardiac and skeletal muscle function. The data demonstrated continued preservation of cardiac function, along with sustained clinical benefit from long-term Deramiocel treatment. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of Duchenane Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. For Becker Muscular Dystrophy (BMD), Deramiocel has also received Orphan Drug Designation from the FDA.
お知らせ • Jun 20Capricor Therapeutics Announces Positive 4-Year Data from Hope-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular DystrophyCapricor Therapeutics announced positive four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company's lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be featured in the session titled "Therapies that slow Progression" at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference, taking place June 21, 2025, in Las Vegas, Nevada. After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline. Further, a subgroup analysis of patients with baseline LVEF >45% showed an even greater clinical benefit, supporting early intervention with Deramiocel to potentially preserve cardiac function. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™? platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, company stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need.
お知らせ • Jun 13Capricor Therapeutics Announces the Successful Completion of the U.S. Food and Drug Administration's License Inspection of Its San Diego Manufacturing FacilityCapricor Therapeutics announced the successful completion of the U.S. Food and Drug Administration's (FDA) Pre-License Inspection (PLI) of its San Diego manufacturing facility for Deramiocel, the company's lead cell therapy candidate with a Biologics License Application (BLA) under FDA review for potential approval in the treatment of Duchenne Muscular Dystrophy (DMD). The inspection concluded with a Form 483 containing several observations. The Company has submitted its responses to the FDA, none of which required material changes to the cGMP process or facility. The observations were primarily related to routine quality systems and documentation practices. The company is confident that the facility will meet the necessary requirements to support product licensure and, pending approval, commercial launch.
お知らせ • Jun 12Capricor Therapeutics Announces Key Regulatory Updates for Its Duchenne Muscular Dystrophy ProgramCapricor Therapeutics announced the successful completion of the U.S. Food and Drug Administration’s (FDA) Pre-License Inspection (PLI) of its San Diego manufacturing facility for Deramiocel, the Company’s lead cell therapy candidate with a Biologics License Application (BLA) under FDA review for potential approval in the treatment of Duchenne Muscular Dystrophy (DMD). The inspection concluded with a Form 483 containing several observations. The Company has submitted its responses to the FDA, none of which required material changes to the cGMP process or facility. The observations were primarily related to routine quality systems and documentation practices. The Company is confident that the facility will meet the necessary requirements to support product licensure and, pending approval, commercial launch. The FDA informed Capricor of its intent to hold the Advisory Committee meeting on July 30, 2025, although that date is pending confirmation by the FDA. At the time of the mid-cycle review, no significant issues or major deficiencies were noted. A late-cycle meeting is planned for mid-July 2025. The BLA for Deramiocel remains under priority review with a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025.
お知らせ • May 06+ 1 more updateCapricor Therapeutics, Inc. to Report Q1, 2025 Results on May 13, 2025Capricor Therapeutics, Inc. announced that they will report Q1, 2025 results After-Market on May 13, 2025
お知らせ • Apr 10Capricor Therapeutics, Inc., Annual General Meeting, May 22, 2025Capricor Therapeutics, Inc., Annual General Meeting, May 22, 2025. Location: 10865 road to the cure, suite 150, san diego, california 92121, United States
お知らせ • Apr 09Earl Collier Not to Stand for Re-Election as Member of the Board of Capricor Therapeutics, IncCapricor Therapeutics, Inc. announced that on April 8, 2025, Mr. Earl Collier, a member of the Board of Directors of the company informed the Board he will not stand for re-election to the Board following the Annual Meeting of Shareholders which is scheduled to occur on May 22, 2025. Mr. Collier's decision to resign is not due to any disagreement with the Company with respect to any of the Company's operations, policies or practices. In connection with Mr. Collier's resignation, the Company plans to enter into a consulting agreement with Mr. Collier pursuant to which, from time to time, Mr. Collier will perform certain strategic advisory relations.
お知らせ • Mar 18Capricor Therapeutics, Inc. Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics, Inc. announced positive long-term data from its ongoing HOPE-2 open label extension (“OLE”) clinical trial, demonstrating the potential of the Company’s lead asset, deramiocel, to slow disease progression and preserve upper limb function in patients with Duchenne muscular dystrophy (“DMD”). The data is presented as a late breaking poster at this year’s Muscular Dystrophy Association Clinical and Scientific Conference, which began on March 16 and runs through March 19 in Dallas, Texas. In a cohort-matched external comparator analysis, the study showed that patients treated with deramiocel over three years experienced an average decline in Performance of the Upper Limb (PUL 2.0) total score of 3.46 points, compared to a 7.19-point decline in the external comparator group (p=0.019). This equates to a 52% slowing of disease progression, reinforcing deramiocel’s potential long-term therapeutic durability. Additional findings include: Treatment effect increases year over year – Patients on deramiocel showed a reduction in disease progression, with a mean annual PUL 2.0 decline of 1.8 points in Year 1, 1.2 points in Year 2 and 1.1 points in Year 3. Potential disease-modifying effects – During a 1-year gap of treatment, those originally randomized to deramiocel showed a slower rate of decline (2.8 points per year) compared to untreated patients (3.7 points per year). Favorable safety profile – Deramiocel was well tolerated with no new safety signals identified and continues to maintain a favorable long-term benefit-risk profile. Capricor recently announced the acceptance by the U.S. Food and Drug Administration (“FDA”) of its Biologics License Application (“BLA”) for the cardiomyopathy associated with DMD with a Prescription Drug User Fee Act (“PDUFA”) target action date set for August 31, 2025, seeking full approval of its application.
お知らせ • Mar 11Capricor Therapeutics, Inc. to Report Q4, 2024 Results on Mar 19, 2025Capricor Therapeutics, Inc. announced that they will report Q4, 2024 results After-Market on Mar 19, 2025
お知らせ • Mar 04Capricor Therapeutics Announces FDA Acceptance and Priority Review of Its Biologics License Application for Deramiocel to Treat Duchenne Muscular DystrophyCapricor Therapeutics announced the U.S. Food and Drug Administration ("FDA") has accepted for review its Biologics License Application ("BLA") seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy ("DMD") cardiomyopathy. Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act ("PDUFA") target action date of August 31, 2025 and at this time, the FDA has not identified any potential review issues. The BLA submission is supported by Capricor's existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Lab Extension ("OLE") trials compared to natural history data from an FDA-funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. The FDA also informed the Company they have not yet decided whether an Advisory Committee meeting is needed in relation to this application. The FDA grants Priority Review to applications for medicines that, if approved, provide significant improvements in the safety or effectiveness of the treatment of a serious condition. Deramiocel for the treatment of DMD has received Orphan Drug Designation from the FDA and European Medicines Agency ("EMA"). In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD by September 30, 2026, Capricor would be eligible to receive a Priority Review Voucher ("PRV") based on its previous receipt of a rare pediatric disease designation.
お知らせ • Jan 02Capricor Therapeutics Completes Submission of Biologics License Application to U.S. FDA for Deramiocel for Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. The full submission of the rolling BLA was completed as the Company had previously guided in late December 2024 and is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data from an FDA funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. Capricor has requested a priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA. In conjunction with this achievement, Capricor will receive a milestone payment of $10 million from its distribution partner, Nippon Shinyaku Co. Ltd., under the terms of its U.S. Commercialization and Distribution Agreement. Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.
Reported Earnings • Nov 14Third quarter 2024 earnings released: US$0.38 loss per share (vs US$0.25 loss in 3Q 2023)Third quarter 2024 results: US$0.38 loss per share (further deteriorated from US$0.25 loss in 3Q 2023). Revenue: US$2.26m (down 63% from 3Q 2023). Net loss: US$12.6m (loss widened 97% from 3Q 2023). Revenue is forecast to grow 52% p.a. on average during the next 3 years, compared to a 22% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has fallen by 2% per year but the company’s share price has increased by 74% per year, which means it is well ahead of earnings.
お知らせ • Nov 05Capricor Therapeutics, Inc. to Report Q3, 2024 Results on Nov 13, 2024Capricor Therapeutics, Inc. announced that they will report Q3, 2024 results After-Market on Nov 13, 2024
New Risk • Oct 20New major risk - Shareholder dilutionThe company's shareholders have been substantially diluted in the past year. Increase in shares outstanding: 76% This is considered a major risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (27% average weekly change). Shareholders have been substantially diluted in the past year (76% increase in shares outstanding).
お知らせ • Oct 18+ 1 more updateCapricor Therapeutics, Inc. Intends to File A Biologics License ApplicationFollowing recent meetings with the U.S. Food and Drug Administration Capricor Therapeutics, Inc. announced its intent to file a Biologics License Application based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (“DMD”) cardiomyopathy. Capricor commenced the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024. The BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (“OLE”) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center. In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024. StealthX™Exosome Platform The Company’s proprietary StealthX™ exosome-based multivalent vaccine (StealthX™ vaccine) for the prevention of SARS-CoV-2 was selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines. Under the terms of the collaboration, Capricor will supply the investigational product and NIAID's Division of Microbiology and Infectious Diseases will conduct the trial. Currently, its vaccine candidate is in the manufacturing phase with plans to deliver it to NIAID by the end of 2024.
お知らせ • Oct 17Capricor Therapeutics, Inc. has filed a Follow-on Equity Offering.Capricor Therapeutics, Inc. has filed a Follow-on Equity Offering. Security Name: Common Stock Security Type: Common Stock
お知らせ • Oct 12Capricor Therapeutics Announces Positive Long-Term Data from Hope-2 OLE Study in Duchenne Muscular Dystrophy at 2024 World Muscle Society CongressCapricor Therapeutics announced positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study for its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD). The data was highlighted in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society (WMS), October 8-12, 2024 in Prague, Czechia. The 3-year data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures of cardiac function, including left ventricular ejection fraction (LVEF%), as well as indexed volumes, which are considered highly relevant in terms of predicting long-term cardiac outcomes. In addition, there was clear bifurcation in the treatment effect seen in those that had ejection fractions greater than 45% at the end of HOPE-2 which suggests that early and sustained intervention will be key in attenuating the impacts of DMD cardiomyopathy. In order to evaluate the relevance of the data to disease progression as well as the chronic and progressive nature of DMD where cardiac function can decline year over year, a natural history data set was used to compare the trajectory of those treated with deramiocel to standard of care. In addition to the cardiac data, patients demonstrated a statistically and clinically relevant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel. In conclusion, the results of this study showed sustained cardiac and skeletal benefits after 3 years of continuous treatment with deramiocel. This data was previously highlighted at the PPMD Annual Meeting in June 2024.
お知らせ • Oct 09Capricor Therapeutics Announces Initiation of Rolling Submission of Biologics License Application with U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced that it has initiated its rolling submission process with the U.S. Food and Drug Administration (FDA) for a Biologics License Application (BLA), seeking full approval for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. Capricor plans to complete its rolling BLA submission by the end of 2024. The application may be eligible for priority review as deramiocel could potentially provide significant improvements in the safety and/or effectiveness of the treatment for the serious condition of DMD cardiomyopathy, where there are currently no approved treatment options available. Once the rolling BLA submission is completed, the FDA will notify the Company when it is formally accepted for review.
Breakeven Date Change • Sep 24The 5 analysts covering Capricor Therapeutics previously expected the company to break even in . New consensus forecast suggests the company will make a profit of US$0 in . Earnings growth of 53% is required to achieve expected profit on schedule.
New Risk • Sep 24New minor risk - ProfitabilityThe company is currently unprofitable and not forecast to become profitable over the next 3 years. Trailing 12-month net loss: US$28m Forecast net loss in 3 years: US$13m This is considered a minor risk. Companies that are not profitable are more likely to be burning through cash and less likely to be well established. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. Without profits, the company is under pressure to grow significantly while potentially having to reduce costs and possibly needing to take on debt or raise capital to remain afloat. Currently, the following risks have been identified for the company: Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$13m net loss in 3 years). Share price has been volatile over the past 3 months (10% average weekly change). Shareholders have been diluted in the past year (37% increase in shares outstanding).
お知らせ • Sep 18Capricor Therapeutics, Inc. announced that it has received $14.999998 million in funding from Nippon Shinyaku Co., Ltd.Capricor Therapeutics, Inc. announced that it has entered into a subscription agreement to issue $5.36 per share for the gross proceeds of $14,999,998 on September 16, 2024. The transaction included participation from new investor, Nippon Shinyaku Co., Ltd. The Subscription Agreement also includes lock-up provisions restricting Nippon Shinyaku from selling or otherwise disposing of shares of Common Stock until the six month anniversary of the Closing Date.
Reported Earnings • Aug 08Second quarter 2024 earnings released: US$0.34 loss per share (vs US$0.29 loss in 2Q 2023)Second quarter 2024 results: US$0.34 loss per share (further deteriorated from US$0.29 loss in 2Q 2023). Revenue: US$3.97m (up 1.4% from 2Q 2023). Net loss: US$11.0m (loss widened 49% from 2Q 2023). Revenue is forecast to grow 40% p.a. on average during the next 3 years, compared to a 20% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has fallen by 3% per year whereas the company’s share price has fallen by 1% per year.
お知らせ • Jul 31Capricor Therapeutics, Inc. to Report Q2, 2024 Results on Aug 07, 2024Capricor Therapeutics, Inc. announced that they will report Q2, 2024 results After-Market on Aug 07, 2024
New Risk • Jul 01New major risk - Financial positionThe company has less than a year of cash runway based on its current free cash flow trend. Free cash flow: -US$33m This is considered a major risk. With less than a year's worth of cash, the company will need to raise capital or take on debt unless its cash flows improve. This would dilute existing shareholders or increase balance sheet risk. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$33m free cash flow). Share price has been highly volatile over the past 3 months (11% average weekly change). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$6.2m net loss in 3 years). Shareholders have been diluted in the past year (27% increase in shares outstanding).
お知らせ • Jun 29Capricor Therapeutics, Inc. Announces Long-Term Benefit of Deramiocel (Cap-1002) in Both Skeletal Muscle and Cardiac Function in the Hope-2 Ole Study in Duchenne Muscular DystrophyCapricor Therapeutics, Inc. announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, deramiocel (CAP-1002), for the treatment of Duchenne muscular dystrophy. Data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures, including left ventricular ejection fraction (LVEF), as well as indexed volumes (left ventricular end systolic volume (LVESV) and left ventricular end diastolic volume (LVEDV)). These are measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes. In addition, greater improvements in cardiac function were observed in those patients that had higher ejection fractions (> 45%) at the beginning of the HOPE-2 randomized trial. Published data supports the need for early intervention in order to maintain function and potentially slow the progression of the cardiomyopathy, one of the leading causes of death in patients with DMD. Currently, there is no approved treatment specifically for DMD cardiomyopathy, which underscores the need for additional therapies to treat DMD. Additionally, as previously reported, patients showed a statistically significant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel. HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, deramiocel, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either deramiocel (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment entered the HOPE-2-OLE study where they receive deramiocel (150 million cells per infusion) every three months. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 (p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety, cardiac and functional performance. Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.
お知らせ • Jun 25Capricor Therapeutics, Inc. Announces for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD). Capricor’s goal for this meeting will be to finalize its BLA filing plans based on all currently available data as well as to work with the FDA to outline the rolling BLA submission timeline. Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000 to 20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.
New Risk • Jun 07New minor risk - ProfitabilityThe company is currently unprofitable and not forecast to become profitable over the next 3 years. Trailing 12-month net loss: US$24m Forecast net loss in 3 years: US$11m This is considered a minor risk. Companies that are not profitable are more likely to be burning through cash and less likely to be well established. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. Without profits, the company is under pressure to grow significantly while potentially having to reduce costs and possibly needing to take on debt or raise capital to remain afloat. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (12% average weekly change). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$11m net loss in 3 years). Shareholders have been diluted in the past year (26% increase in shares outstanding).
お知らせ • Jun 05Capricor Therapeutics Announces Positive 3-Year Efficacy Results from Hope-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular DystrophyCapricor Therapeutics announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study with CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Patients treated with CAP-1002 continue to show positive benefits after 3 years of treatment in both the PUL 2.0 and LVEF measures when compared to an external comparator dataset of similar DMD patients (dataset provided by Cincinnati Children's Hospital Medical Center (CCHMC). Further, CAP-1002 treatment during the HOPE-2-OLE study continues to have a consistently well-tolerated safety profile throughout the study. The Company plans to present these results in more detail at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024. HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment entered the HOPE-2-OLE study where they receive CAP-1002 (150 million cells per infusion) every three months. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL 2.0 (p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety and functional performance.
Breakeven Date Change • May 17Forecast breakeven date pushed back to 2026The 5 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$15.1m in 2026. Average annual earnings growth of 66% is required to achieve expected profit on schedule.
Reported Earnings • May 14First quarter 2024 earnings released: US$0.31 loss per share (vs US$0.31 loss in 1Q 2023)First quarter 2024 results: US$0.31 loss per share (further deteriorated from US$0.31 loss in 1Q 2023). Revenue: US$4.91m (up 64% from 1Q 2023). Net loss: US$9.79m (loss widened 26% from 1Q 2023). Revenue is forecast to grow 39% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has fallen by 6% per year but the company’s share price has increased by 16% per year, which means it is well ahead of earnings.
お知らせ • May 10Capricor Therapeutics, Inc. to Present Exosome Platform Updates At the American Society of Gene and Cell Therapy 27Th Annual MeetingCapricor Therapeutics, Inc. announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™? exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore, Maryland from May 7-11, 2024. The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.
お知らせ • May 08Capricor Therapeutics, Inc. to Report Q1, 2024 Results on May 13, 2024Capricor Therapeutics, Inc. announced that they will report Q1, 2024 results After-Market on May 13, 2024
お知らせ • Apr 24Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular DystrophyCapricor Therapeutics announced an update from the Company’s recent Type-B Chemistry, Manufacturing and Controls meeting with the U.S. Food and Drug Administration on next steps for the Biologics License Application submission with its lead asset CAP-1002 in treating Duchenne muscular dystrophy. The FDA has affirmed alignment with Capricor on the following topics: Pre-BLA Meeting and Rolling BLA Submission The FDA advised Capricor to include discussion for a pre-BLA meeting and rolling BLA schedule in the upcoming Type-B meeting. Based on this feedback, Capricor has already been granted a subsequent Type-B meeting to be held in the second quarter of 2024 to discuss these topics, with the results of those discussions to potentially lead to an accelerated BLA filing. Capricor plans to share with FDA its HOPE-2 open label extension 3-year safety and efficacy data which is expected to be available in the second quarter of 2024 as part of Capricor’s ISS and ISE strategy. Establishment of Non-Clinical Comparability The FDA agreed that comparability between drug product manufactured at its two different facilities (Los Angeles and San Diego) has been demonstrated using the provided analytical comparability data. This allows for the use of CAP-1002 drug product manufactured at its San Diego manufacturing facility upon potential product approval. Data from Cohort B of the HOPE-3 clinical trial will not be necessary for FDA approval of the product.
お知らせ • Apr 02Capricor Therapeutics, Inc., Annual General Meeting, May 14, 2024Capricor Therapeutics, Inc., Annual General Meeting, May 14, 2024, at 10:00 Pacific Daylight. Location: 10865 Road to the Cure, Suite 150 San Diego California United States Agenda: To elect the nine nominees named in this proxy statement to the Company’s board of directors to serve for a one-year term expiring at 2025 Annual Meeting of Stockholders; to ratify the appointment of Rose, Snyder & Jacobs LLP as the Company’s independent registered public accounting firm for the fiscal year ending December 31, 2024; to approve, by non-binding advisory vote, the resolution approving named executive officer compensation; to approve an amendment in the form set forth on Annex A to this proxy statement to the Company’s Certificate of Incorporation, as amended from time to time (the “ Certificate of Incorporation ”) to increase the number of authorized shares of common stock from 50,000,000 to 100,000,000; and to transact such other business as may properly come before the Annual Meeting or any adjournment or postponement thereof.
New Risk • Mar 29New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 10% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (10% average weekly change). Minor Risk Shareholders have been diluted in the past year (24% increase in shares outstanding).
お知らせ • Mar 07Capricor Therapeutics, Inc. to Present Positive 24-Month Results from its HOPE-2 Open-Label Extension Study at 2024 Muscular Dystrophy Association Clinical & Scientific ConferenceCapricor Therapeutics, Inc. will present the positive 24-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at MDA Clinical and Scientific Conference which is taking place in Orlando, Florida from March 3-6, 2024. Key results from the study, include: CAP-1002 is an investigational cell therapy which aims to slow disease progression through immunomodulatory, anti-inflammatory and anti-fibrotic actions with the goal of potentially improving the rate of decline in skeletal and cardiac muscle function in patients with DMD. Skeletal muscle function as measured by the Performance of the Upper Limb (PUL v2.0) showed a mean PUL v2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7 point decline on average observed over 24-months in the placebo patient group. Delta change=4.9 points; p=0.021. The average rate of decline in CAP-1002 treated patients showed an attenuation of disease progression by approximately 64%. CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function. Cardiac function as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in 67% of patients, compared to a steady decline in a comparable natural history population. CAP-1002 treatment during the OLE portion of the study continues to yield a consistent favorable safety profile and has been well-tolerated throughout the study. The HOPE-2 OLE study previously met the primary endpoint at the one-year time-point and these 24-month results suggest that patients accumulate benefit over time with preservation of skeletal muscle function, which underscore the potential long-term benefit of CAP-1002. HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 24-months. These 24-month results were presented previously at the 2023 Parent Project Muscular Dystrophy (PPMD) Annual Conference.
Breakeven Date Change • Mar 05Forecast breakeven date pushed back to 2026The 3 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests losses will reduce by 22% per year to 2025. The company is expected to make a profit of US$956.7k in 2026. Average annual earnings growth of 30% is required to achieve expected profit on schedule.
Reported Earnings • Mar 03Full year 2023 earnings released: US$0.83 loss per share (vs US$1.18 loss in FY 2022)Full year 2023 results: US$0.83 loss per share (improved from US$1.18 loss in FY 2022). Revenue: US$25.2m (up US$22.6m from FY 2022). Net loss: US$22.3m (loss narrowed 23% from FY 2022). Revenue is forecast to grow 21% p.a. on average during the next 3 years, compared to a 13% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has fallen by 10% per year but the company’s share price has only fallen by 1% per year, which means it has not declined as severely as earnings.
お知らせ • Feb 23Capricor Therapeutics, Inc. to Report Q4, 2023 Results on Feb 29, 2024Capricor Therapeutics, Inc. announced that they will report Q4, 2023 results After-Market on Feb 29, 2024
お知らせ • Jan 24Capricor Therapeutics Announces Collaboration with the National Institutes of Health for Clinical Trial of Novel Exosome-Based Multivalent Vaccine for SARS-CoV-2Capricor Therapeutics announced that Capricor's proprietary StealthX exosome-based multivalent vaccine (StealthX™? vaccine) for the prevention of SARS-CoV-2 has been selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines providing broader and more durable protection for COVID-19. As part of Project NextGen, the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, will conduct a Phase 1 clinical study with Capricor's StealthX™? vaccine, subject to regulatory approval. NIAID's Division of Microbiology and Infectious Diseases (DMID) would oversee the study. The StealthX™? vaccine is a proprietary vaccine developed internally by Capricor utilizing exosomes that were engineered to express either spike or nucleocapsid proteins on the surface. Preclinical results from murine and Rabbit models published in Microbiology Spectrum, showed the StealthX™? vaccine, resulted in robust antibody production, potent neutralizing antibodies, a strong T-cell response and a favorable safety profile. These effects were obtained with administration of only nanogram amounts of protein and without adjuvant or synthetic lipid nanoparticles (LNPs). Exosomes offer a new antigen delivery system that potentially could be utilized to rapidly generate multivalent protein-based vaccines. Exosomes, first identified as extracellular vesicles, are small vesicles enriched in specific subsets of proteins, RNAs and lipids and responsible for cell-to-cell communication.
お知らせ • Dec 12Capricor Therapeutics Announces Continuation of Phase 3 Hope-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility AnalysisCapricor Therapeutics announced a positive outcome of the interim futility analysis for HOPE-3, the pivotal Phase 3 trial evaluating CAP-1002 in patients with Duchenne muscular dystrophy (DMD). The results of the interim futility analysis, reviewed by the Data Safety Monitoring Board (DSMB), resulted in a favorable recommendation to continue the HOPE-3 trial as planned. The company believe CAP-1002 may address the high unmet medical need for these patients and remain committed to its expeditious advancement towards approval. Based on this important milestone, the company will be requesting a meeting with the U.S. Food and Drug Administration (FDA) to further discuss options for expedited review and approval. In addition, the company believe CAP-1002 will be requesting a Meeting with the U.S. food and Drug Administration (FDA). In addition, the company believe the company are well positioned to execute on additional value-driving clinical and regulatory milestones, including reporting topline data from HOPE-3 (Cohort A) in the fourth quarter of 2024. HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial comprised of two cohorts evaluating the safety and efficacy of CAP-1002 in participants with DMD and impaired skeletal muscle function. Non-ambulatory and ambulatory boys who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. Approximately 102 eligible study subjects will participate in this dual-cohort study. Enrollment has been completed for Cohort A which is designed to enroll approximately 58 subjects randomized to either CAP-1002 or trial in a 1:1 ratio and is intended to support a Biologics License Application submission. Enrollment has commenced for Cohort B which is designed to enroll approximately 44 participants randomized to either CAP-100 2 or placebo in a 1:1 ratio. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials. CAP-1002 for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for CAP-1002 is supported RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for CAP-1002 for thetreatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.
New Risk • Nov 19New major risk - Financial positionThe company has less than a year of cash runway based on its current free cash flow trend. Free cash flow: -US$23m This is considered a major risk. With less than a year's worth of cash, the company will need to raise capital or take on debt unless its cash flows improve. This would dilute existing shareholders or increase balance sheet risk. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$23m free cash flow). Share price has been highly volatile over the past 3 months (25% average weekly change). Negative equity (-US$1.8m). Minor Risks Shareholders have been diluted in the past year (25% increase in shares outstanding). Market cap is less than US$100m (€85.8m market cap, or US$93.6m).
お知らせ • Nov 08Capricor Therapeutics, Inc. to Report Q3, 2023 Results on Nov 14, 2023Capricor Therapeutics, Inc. announced that they will report Q3, 2023 results After-Market on Nov 14, 2023
New Risk • Oct 03New minor risk - Market cap sizeThe company's market capitalization is less than US$100m. Market cap: €87.0m (US$91.2m) This is considered a minor risk. Companies with a small market capitalization are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (25% average weekly change). Minor Risks Shareholders have been diluted in the past year (26% increase in shares outstanding). Market cap is less than US$100m (€87.0m market cap, or US$91.2m).
お知らせ • Oct 01Capricor Therapeutics Announces Positive Type-B Meeting with the FDA to Discuss Pathway to BLA for CAP-1002 in Duchenne Muscular DystrophyCapricor Therapeutics announced an update on the Company’s positive Type-B clinical meeting with the U.S. Food and Drug Administration (“FDA”) on the design and execution of HOPE-3, Capricor’s pivotal Phase 3 trial with lead asset CAP-1002 in treating Duchenne muscular dystrophy (“DMD”). Feedback from the FDA on trial design and timeline confirms CAP-1002’s path towards a future Biologics License Application (“BLA”) submission. The FDA has affirmed alignment on the Phase 3 clinical trial’s design and timeline. Key details for HOPE-3 are as follows: Primary endpoints remain unchanged. HOPE-3 will aim to enroll approximately 58 patients and enrollment is estimated to be completed in the fourth quarter of 2023. Capricor has treated 52 patients. Topline data for HOPE-3 is expected in the fourth quarter of 2024. Capricor plans to submit a BLA for CAP-1002 in 2025, which will be supported by results using product manufactured at the Los Angeles site. Capricor and the FDA discussed the potential for alternative approval pathways and Capricor plans to further discuss these options with the FDA following the completion of enrollment. CAP-1002 for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for CAP-1002 is supported RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for CAP-1002 for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (“PRV”) based on its previous receipt of a rare pediatric disease designation. Capricor retains full rights to the PRV, if received. Duchenne muscular dystrophy (“DMD”) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in approximately one in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited and there is no cure. CAP-1002 consists of allogeneic cardiosphere-derived cells (“CDCs”), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.
お知らせ • Sep 07Capricor Therapeutics, Inc. Appoints Michael Kelliher to Its Board of DirectorsCapricor Therapeutics announced that the Company has appointed Michael Kelliher to its Board of Directors, effective immediately. Mr. Kelliher, Group Vice President of M&A and Business Development at Horizon Therapeutics, brings to Capricor extensive expertise in business development, strategy, finance and operational leadership across the life sciences industry. Mr. Kelliher is an experienced business development and finance professional with expertise in corporate strategy, mergers and acquisitions, strategic partnerships and licensing, with a career spanning more than 20 years with leading biotechnology and global pharmaceutical companies. He currently serves as Group Vice President, M&A and Business Development, at Horizon Therapeutics, a publicly traded global biotechnology company focused on researching, developing and commercializing medicines for rare, autoimmune and severe inflammatory diseases. Mr. Kelliher joined Horizon in 2014 and led an aggressive growth and expansion agenda through acquisitions, development collaborations and other transactions. He was instrumental in transforming Horizon into a $28 billion innovation-driven biotech company. Prior to his time at Horizon, from 2009 to 2014, Mr. Kelliher held progressive financial roles at Elan Corporation (now Perrigo Company), a leading global pharmaceutical company where he oversaw strategic partnerships and collaborations and advised its Board of Directors and senior leadership on investments, business development, product commercialization and asset monetization. Mr. Kelliher began his career in banking, public accounting and corporate finance and holds a Bachelor of Commerce degree from the University College Cork (Ireland). He is also an Associated Chartered Accountant.
New Risk • Aug 25New major risk - Share price stabilityThe company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 5.0% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (5.0% average weekly change). Minor Risk Shareholders have been diluted in the past year (5.9% increase in shares outstanding).
Board Change • Aug 25Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 7 experienced directors. 5 highly experienced directors. Independent Director George Dunbar was the last director to join the board, commencing their role in 2013. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.
Breakeven Date Change • Aug 08Forecast breakeven date moved forward to 2024The 2 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$3.12m in 2024. Average annual earnings growth of 61% is required to achieve expected profit on schedule.
お知らせ • Aug 03Capricor Therapeutics, Inc. to Report Q2, 2023 Results on Aug 07, 2023Capricor Therapeutics, Inc. announced that they will report Q2, 2023 results After-Market on Aug 07, 2023
Board Change • Aug 01Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 7 experienced directors. 5 highly experienced directors. Independent Director George Dunbar was the last director to join the board, commencing their role in 2013. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.
お知らせ • Jul 25+ 1 more updateCapricor Therapeutics Announces the Appointment of Philip J. Gotwals to Its Board of DirectorsCapricor Therapeutics announced the appointment of Philip J. Gotwals, Ph.D. to its Board of Directors, effective immediately. Dr. Gotwals, most recently the Global Head, Vice President of Business Development and Licensing at Novartis Institutes for Biomedical Research, bring his strategic vision for research, innovation for drug development and valuable experience to the Company. Dr. Gotwals has experience in drug development, research, corporate strategy and business development with a career spanning nearly 30 years in the biotechnology industry. Dr. Gotwals most recently served as the Global Head, Vice President of Business Development and Licensing at Novartis Institutes for Biomedical Research (NIBR) where he oversaw business development efforts for all disease areas and technology platforms. Under Dr. Gotwals’ leadership, NIBR business development and licensing executed over 50 major strategic transactions which included licensing deals, collaborations, acquisitions and new company creations. These transactions led to significant corporate evolution and growth. During his 13 years at Novartis, Dr. Gotwals was instrumental in building the company’s immuno-oncology strategic research area and spearheading the collaboration with the University of Pennsylvania to develop chimeric antigen receptor (CAR) T-cell therapies. Prior to Novartis, he was Vice President of Program Management at Altus Pharmaceuticals, where he was responsible for all product development project management activities. Prior to Altus, he was Senior Director of Program and Alliance Management at Biogen, where he oversaw leadership of internal and allied early product development teams in the autoimmune, neurology and oncology therapeutic areas. Dr. Gotwals has a B.A. in Biology from Amherst College, holds a Ph.D. in Genetics from the University of California at Berkeley, completed postdoctoral research at the Massachusetts Institute of Technology, business training at Harvard Business School and has published extensively in the area of integrin biology.
お知らせ • Jul 14Capricor Therapeutics, Inc Announces Board ChangesOn July 11, 2023, Mr. Louis Manzo, a member of the Board of Directors of Capricor Therapeutics, Inc. resigned from the Board effective immediately. Mr. Manzo’s decision to resign is not due to any disagreement with the Company on any of the Company’s operations, policies or practices. On July 11, 2023, the Board elected Paul Auwaerter, M.D. as a member of the Board, effective immediately. Dr. Auwaerter will receive an initial grant of a nonstatutory stock option to purchase 115,000 shares of the Company’s common stock (the “Initial Grant”) as standard non-employee director compensation under the Company’s policies. The Initial Grant will vest with respect to 25% of such shares of common stock on the first anniversary of the first day of the month following the date of grant of the Initial Grant, and the Initial Grant will vest with respect to 1/36 of such shares of common stock each month thereafter. Dr. Auwaerter will also enter into the Company’s standard form of indemnification agreement for directors. Dr. Auwaerter is the Sherrilyn and Ken Fisher Professor of Medicine at the Johns Hopkins University School of Medicine, serving as the Clinical Director for the Division of Infectious Diseases and Director of the Sherrilyn and Ken Fisher Center for Environmental Infectious Diseases. Dr. Auwaerter serves as the Executive Director of the Johns Hopkins Point of Care-Information Technology (POC-IT) Center producing the Johns Hopkins ABX (Antibiotic), JH HIV, JH Osler, JH Psychiatry and JH Diabetes Guides. Dr. Auwaerter also serves as Editor-in-Chief of the ABX Guide. Dr. Auwaerter’s research and clinical interests include improving the diagnosis and care for patients with infectious diseases, specifically Lyme disease, respiratory infections, surgical infections, prosthetic joint infections, Epstein-Barr virus, and fever of unknown origin. He is a Past President of the Infectious Diseases Society of America (IDSA), the largest professional society worldwide related to infectious diseases. He currently serves as Chairman of the Infectious Diseases Society of America Foundation and is also on the Board of Directors of the American Lyme Disease Foundation. Dr. Auwaerter has been a member of the Board of Directors of the Baltimore Area Council of the Boy Scouts of America and has been awarded the national Distinguished Eagle Scout award. Dr. Auwaerter holds undergraduate and medical degrees from Columbia University and subsequent training in medicine and infectious diseases at Johns Hopkins, where he has been employed since 1988; he also trained in the virology and immunology laboratory of Dr. Diane Griffin.
お知らせ • Jul 01Capricor Therapeutics Announces Positive 24-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular DystrophyCapricor Therapeutics announced positive 24-month safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). Data from the OLE study demonstrated that the majority of patients had an improvement in left ventricular ejection fraction (LVEF), after two years of CAP-1002 treatment, which suggests preservation of cardiac function. Additionally, patients continue to show statistically significant benefit (p=0.021) after two years of treatment in the Performance of the Upper Limb (PUL v2.0) scale when compared to the original rate of decline of the placebo group from HOPE-2 after one year. Furthermore, the OLE study continues to show a favorable safety profile for long-term treatment of CAP-1002. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale (p=0.02). At the 24-month timepoint, data showed statistically significant differences in the PUL v2.0 in the OLE treatment group when compared to the original rate of decline of the placebo group from HOPE-2 after one-year (p=0.021). LVEF was measured using cardiac magnetic resonance imaging (cMRI) and six of nine patients showed improvements in heart function with CAP-1002 treatment compared to their final assessment at the end of the HOPE-2 study. Over time, there was an increasing correlation with PUL v2.0 and ejection fraction results (24-month OLE results r=0.75, p=0.02). HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 24 months. Patients continued through the gap phase (off treatment for both groups) and the OLE phase (on treatment for both groups).
お知らせ • Jun 08Capricor Therapeutics Announces Follow-up Type-B Clinical Meeting with the FDA for CAP-1002 for the Treatment of Duchenne Muscular DystrophyCapricor Therapeutics announced an upcoming Type-B clinical meeting with the U.S. Food and Drug Administration (FDA), planned in early Third Quarter 2023. During the planned meeting with the FDA, Capricor will outline its proposed path towards submission of a potential Biologics License Application (BLA) and further discuss its ongoing HOPE-3 clinical trial with the agency. The regulatory pathway for CAP-1002 is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) as well as Orphan Drug Designation. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher based on its designation as a rare pediatric disease. HOPE-3, the Company's Phase 3 clinical trial of CAP-1002 is a multi-center, randomized, double-blind, placebo-controlled study (NCT05126758) which is designed to treat up to 68 subjects in the United States. Duchenne muscular dystrophy is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. CAP-1002 consists of allogeneic cardiosphere-derived cells (CDCs), a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity and is being investigated for its potential to modify the immune system's activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.
Breakeven Date Change • May 14Forecast breakeven date moved forward to 2024The 2 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$11.8m in 2024. Average annual earnings growth of 58% is required to achieve expected profit on schedule.
お知らせ • May 05Capricor Therapeutics, Inc. to Report Q1, 2023 Results on May 11, 2023Capricor Therapeutics, Inc. announced that they will report Q1, 2023 results After-Market on May 11, 2023
Reported Earnings • Mar 18Full year 2022 earnings released: US$1.18 loss per share (vs US$0.87 loss in FY 2021)Full year 2022 results: US$1.18 loss per share (further deteriorated from US$0.87 loss in FY 2021). Net loss: US$29.0m (loss widened 45% from FY 2021). Revenue is forecast to grow 40% p.a. on average during the next 3 years, compared to a 21% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has increased by 16% per year but the company’s share price has increased by 57% per year, which means it is tracking significantly ahead of earnings growth.
Breakeven Date Change • Mar 18Forecast breakeven date moved forward to 2024The 2 analysts covering Capricor Therapeutics previously expected the company to break even in 2025. New consensus forecast suggests the company will make a profit of US$12.9m in 2024. Average annual earnings growth of 54% is required to achieve expected profit on schedule.
お知らせ • Jan 26Capricor Therapeutics, Inc. Announces Positive 18-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy PatientsCapricor Therapeutics, Inc. announced positive 18-month results from its ongoing HOPE-2 open label extension (OLE) study in patients with later-stage Duchenne muscular dystrophy (DMD). Data from the OLE study continues to show evidence for disease modification with statistically significant differences in the Performance of the Upper Limb (PUL version 2.0) scale in the CAP-1002 original treatment group when compared to the original placebo group from HOPE-2 (p=0.02). In addition, disease progression is attenuated equally in both groups once patients begin treatment in the OLE. The study met its primary endpoint at the one-year timepoint and continues to show statistically significant improvements (p=0.02) on the Performance of the Upper Limb scale for patients treated with CAP-1002 at 18-months. In the study, CAP-1002 was made available to the original 20 patients enrolled in the HOPE-2 study. Of those, 13 entered and 12 completed 18-months of study follow-up. The breakdown of patients included seven from the original placebo group and six from the original CAP-1002 treatment group. All patients were off CAP-1002 or placebo for a mean of approximately one-year before resumption or initiation of therapy under the HOPE-2 OLE program. As in HOPE-2, CAP-1002 was administered quarterly and current results are from the 18-month analysis. CAP-1002 treatment during the open-label portion of the study continues to yield a consistent safety profile and has been well-tolerated throughout the study. The HOPE-2 OLE study is ongoing, and all participants continue to be monitored for safety and functional performance. The company is currently conducting a pivotal Phase 3 trial, HOPE-3, designed as a randomized, double-blind, placebo-controlled study with approximately 70 patients and enrollment criteria similar to HOPE-2. The Phase 3 study is currently enrolling subjects (NCT05126758).
Breakeven Date Change • Dec 31Forecast to breakeven in 2025The 3 analysts covering Capricor Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$89.3m in 2025. Average annual earnings growth of 59% is required to achieve expected profit on schedule.
Board Change • Nov 17Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 6 experienced directors. 6 highly experienced directors. Independent Director George Dunbar was the last director to join the board, commencing their role in 2013. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.
Reported Earnings • Nov 12Third quarter 2022 earnings released: US$0.26 loss per share (vs US$0.17 loss in 3Q 2021)Third quarter 2022 results: US$0.26 loss per share (further deteriorated from US$0.17 loss in 3Q 2021). Net loss: US$6.37m (loss widened 62% from 3Q 2021). Revenue is forecast to grow 64% p.a. on average during the next 3 years, compared to a 24% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has increased by 33% per year but the company’s share price has increased by 44% per year, which means it is tracking significantly ahead of earnings growth.
