Notizie in diretta • Jun 13
Capricor Therapeutics Advances Toward FDA Decision on Duchenne Therapy With Market Launch in Sight Capricor Therapeutics is preparing for an FDA review of its lead cell therapy Deramiocel for Duchenne muscular dystrophy, with a PDUFA target date set for August 22, 2026.
The HOPE-3 trial met its primary and key secondary endpoints, with reported skeletal and cardiac benefits in treated patients.
The company has completed an FDA Pre-License Inspection at its San Diego GMP facility, plans to independently launch Deramiocel, and expects manufacturing expansion in the first half of 2027, with funding stated to extend operations through at least Q4 2027.
The combination of positive HOPE-3 data, a defined PDUFA date, and an inspected manufacturing facility places Capricor at a pivotal point, where regulatory, clinical and operational elements are aligning around a potential Deramiocel launch.
Investors may wish to monitor how the company executes on its independent commercialization plans, including pricing, market access and scaling capacity, as well as any updates around demand expectations and reimbursement for a high-touch cell therapy in a rare disease setting. Notizie in diretta • Jun 05
Capricor Therapeutics Eyes FDA Decision With Positive Phase 3 Data for Duchenne Therapy Capricor Therapeutics reported positive Phase 3 HOPE-3 data for Deramiocel in Duchenne muscular dystrophy, with results indicating a meaningful slowing of disease progression and improved upper limb function.
The FDA is actively reviewing the Biologics License Application for Deramiocel, with a PDUFA target action date set for August 22, 2026, following acceptance of the resubmission as a Class II application.
The company is preparing for a potential independent launch of Deramiocel amid a legal dispute over a distribution agreement with NS Pharma, while holding about US$279 million in cash as of March 31, 2026, which it expects to support operations into late 2027.
The combination of positive Phase 3 data, an ongoing FDA review and preparations for commercialization places regulatory decisions and launch execution at the center of the story over the next few years.
Investors may wish to monitor the outcome of the NS Pharma legal process, the timing and terms of any partnership deals, and how rising R&D and operating costs are managed in the absence of current commercial revenues. Recent Insider Transactions Derivative • May 20
Independent Director notifies of intention to sell stock David Musket intends to sell 89k shares in the next 90 days after lodging an Intent To Sell Form on the 19th of May. If the sale is conducted around the recent share price of US$27.00, it would amount to US$2.4m. David currently holds less than 1% of total shares outstanding. Company insiders have collectively sold US$5.9m more than they bought, via options and on-market transactions in the last 12 months. Major Estimate Revision • May 20
Consensus EPS estimates fall by 105% The consensus outlook for earnings per share (EPS) in fiscal year 2026 has deteriorated. 2026 revenue forecast decreased from US$115.3m to US$104.3m. Losses expected to increase from US$0.39 per share to US$0.80. Biotechs industry in the US expected to see average net income decline 9.3% next year. Consensus price target broadly unchanged at US$54.11. Share price fell 4.4% to US$29.01 over the past week. New Risk • May 12
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of American stocks, typically moving 11% a week. This is considered a minor risk. Share price volatility indicates 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. It also 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. Currently, the following risks have been identified for the company: Major Risk Revenue is less than US$1m. Minor Risks Share price has been volatile over the past 3 months (11% average weekly change). Shareholders have been diluted in the past year (27% increase in shares outstanding). Recent Insider Transactions Derivative • May 05
Executive VP exercised options and sold US$713k worth of stock On the 1st of May, Karen Krasney exercised 25k options at a strike price of around US$3.18 and sold these shares for an average price of US$31.70 per share. This trade did not impact their existing holding. Since December 2025, Karen has owned 30.55k shares directly. Company insiders have collectively sold US$5.9m more than they bought, via options and on-market transactions in the last 12 months. Annuncio • May 05
Capricor Therapeutics, Inc. to Report Q1, 2026 Results on May 12, 2026 Capricor Therapeutics, Inc. announced that they will report Q1, 2026 results After-Market on May 12, 2026 Price Target Changed • Apr 22
Price target increased by 7.4% to US$54.67 Up from US$50.89, the current price target is an average from 9 analysts. New target price is 56% above last closing price of US$35.00. Stock is up 166% over the past year. The company is forecast to post a net loss per share of US$0.39 next year compared to a net loss per share of US$2.26 last year. Breakeven Date Change • Apr 22
Forecast breakeven date pushed back to 2028 The 9 analysts covering Capricor Therapeutics previously expected the company to break even in 2026. New consensus forecast suggests losses will reduce by 44% per year to 2027. The company is expected to make a profit of US$109.6m in 2028. Average annual earnings growth of 62% is required to achieve expected profit on schedule. Annuncio • Apr 22
Capricor Therapeutics Announces Late-Breaking Presentation of Hope-3 Phase 3 Results Capricor 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. Annuncio • Apr 02
Capricor Therapeutics, Inc., Annual General Meeting, Jun 04, 2026 Capricor Therapeutics, Inc., Annual General Meeting, Jun 04, 2026. Location: 10865 road to the cure, suite 150, 92121., san diego, Canada Breakeven Date Change • Mar 16
Forecast breakeven date moved forward to 2026 The 10 analysts covering Capricor Therapeutics previously expected the company to break even in 2028. New consensus forecast suggests the company will make a profit of US$1.69m in 2026. Earnings growth of 62% is required to achieve expected profit on schedule. New Risk • Mar 15
New major risk - Revenue size The company makes less than US$1m in revenue. This is considered a major risk. Companies with a small amount of revenue 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 Revenue is less than US$1m. Minor Risks Share price has been volatile over the past 3 months (11% average weekly change). Shareholders have been diluted in the past year (26% increase in shares outstanding). Price Target Changed • Mar 13
Price target increased by 12% to US$53.70 Up from US$48.11, the current price target is an average from 10 analysts. New target price is 76% above last closing price of US$30.50. Stock is up 131% over the past year. The company is forecast to post a net loss per share of US$0.44 next year compared to a net loss per share of US$1.81 last year. New Risk • Mar 13
New minor risk - Shareholder dilution The company's shareholders have been diluted in the past year. Increase in shares outstanding: 26% This is considered a minor 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. This is currently the only risk that has been identified for the company. Annuncio • Mar 13
Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular Dystrophy Capricor 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. Annuncio • Mar 10
Capricor Therapeutics, Inc. Establishes New PDUFA Date For Deramiocel BLA Capricor 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. Annuncio • Mar 09
Capricor Therapeutics, Inc. to Report Q4, 2025 Results on Mar 12, 2026 Capricor Therapeutics, Inc. announced that they will report Q4, 2025 results After-Market on Mar 12, 2026 Annuncio • Jan 20
Capricor Therapeutics Provides Regulatory Updates Regarding Its Biologics License Application for Deramiocel Capricor 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. Annuncio • Dec 17
Capricor Therapeutics Announces Positive Topline Results From Phase 3 HOPE-3 Trial Of Deramiocel For Duchenne Muscular Dystrophy Capricor 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. New Risk • Dec 08
New minor risk - Shareholder dilution The company's shareholders have been diluted in the past year. Increase in shares outstanding: 27% This is considered a minor 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 Risk Share price has been highly volatile over the past 3 months (47% average daily change). Minor Risk Shareholders have been diluted in the past year (27% increase in shares outstanding). Breakeven Date Change • Dec 04
Forecast to breakeven in 2027 The 10 analysts covering Capricor Therapeutics expect the company to break even for the first time. New consensus forecast suggests losses will reduce by 64% per year to 2026. The company is expected to make a profit of US$28.2m in 2027. Average annual earnings growth of 77% is required to achieve expected profit on schedule. New Risk • Dec 04
New major risk - Share price stability The company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of American stocks, typically moving 47% a day. 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. This is currently the only risk that has been identified for the company. Annuncio • Dec 03
Capricor Therapeutics, Inc. Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy Capricor 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. Annuncio • Nov 25
Capricor Therapeutics, Inc. Presents New Data Demonstrating a Scalable Framework for Loading Therapeutic Oligonucleotides into Exosomes Capricor 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). Major Estimate Revision • Nov 17
Consensus revenue estimates decrease by 50% The consensus outlook for fiscal year 2025 has been updated. 2025 revenue forecast fell from US$1.19m to US$600.0k. EPS estimate unchanged at -US$2.17 per share. Biotechs industry in the US expected to see average net income decline 10% next year. Consensus price target broadly unchanged at US$20.70. Share price was steady at US$5.60 over the past week. Reported Earnings • Nov 11
Third quarter 2025 earnings released: US$0.54 loss per share (vs US$0.38 loss in 3Q 2024) Third quarter 2025 results: US$0.54 loss per share (further deteriorated from US$0.38 loss in 3Q 2024). Net loss: US$24.6m (loss widened 96% from 3Q 2024). Revenue is forecast to grow 53% p.a. on average during the next 3 years, compared to a 21% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has fallen by 13% per year but the company’s share price has increased by 15% per year, which means it is well ahead of earnings. Annuncio • Oct 31
Capricor Therapeutics, Inc. to Report Q3, 2025 Results on Nov 10, 2025 Capricor Therapeutics, Inc. announced that they will report Q3, 2025 results After-Market on Nov 10, 2025 Annuncio • Sep 25
Capricor Therapeutics Provides Regulatory Update on Deramiocel Program for Duchenne Muscular Dystrophy Capricor 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. Annuncio • Sep 09
Capricor Therapeutics Responds to FDA Posting of Complete Response Letter for Deramiocel Capricor 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. Annuncio • Aug 19
Capricor Therapeutics Announces First Subjects Dosed in Phase 1 Clinical Trial of Novel Exosome-Based Vaccine Capricor 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. Major Estimate Revision • Aug 18
Consensus revenue estimates fall by 45% The consensus outlook for revenues in fiscal year 2025 has deteriorated. 2025 revenue forecast decreased from US$2.18m to US$1.19m. Forecast losses increased from -US$1.90 to -US$2.18 per share. Biotechs industry in the US expected to see average net income decline 12% next year. Consensus price target of US$20.60 unchanged from last update. Share price was steady at US$7.96 over the past week. Reported Earnings • Aug 12
First half 2025 earnings released: US$1.10 loss per share (vs US$0.66 loss in 1H 2024) First half 2025 results: US$1.10 loss per share (further deteriorated from US$0.66 loss in 1H 2024). Net loss: US$50.3m (loss widened 142% from 1H 2024). Revenue is forecast to grow 50% p.a. on average during the next 3 years, compared to a 19% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has fallen by 8% per year but the company’s share price has increased by 16% per year, which means it is well ahead of earnings. Annuncio • Aug 06
Capricor Therapeutics, Inc. to Report Q2, 2025 Results on Aug 11, 2025 Capricor Therapeutics, Inc. announced that they will report Q2, 2025 results After-Market on Aug 11, 2025 Annuncio • Aug 01
Levi & Korsinsky, LLP Notifies Investors in Capricor Therapeutics, Inc. of A Class Action Securities Law Levi & 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. Major Estimate Revision • Jul 13
Consensus revenue estimates fall by 51% The consensus outlook for revenues in fiscal year 2025 has deteriorated. 2025 revenue forecast decreased from US$100.2m to US$48.6m. Forecast losses increased from -US$0.377 to -US$1.35 per share. Biotechs industry in the US expected to see average net income decline 12% next year. Consensus price target down from US$34.60 to US$22.90. Share price fell 25% to US$7.64 over the past week. Annuncio • Jul 11
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy Capricor 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. Breakeven Date Change • Jun 25
Forecast breakeven date moved forward to 2025 The 8 analysts covering Capricor Therapeutics previously expected the company to break even in 2027. New consensus forecast suggests the company will make a profit of US$3.46m in 2025. Earnings growth of 63% is required to achieve expected profit on schedule. Annuncio • Jun 24
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy Capricor 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. Annuncio • Jun 20
Capricor Therapeutics Announces Positive 4-Year Data from Hope-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular Dystrophy Capricor 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. Annuncio • Jun 13
Capricor Therapeutics Announces the Successful Completion of the U.S. Food and Drug Administration's License Inspection of Its San Diego Manufacturing Facility Capricor 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. Annuncio • Jun 12
Capricor Therapeutics Announces Key Regulatory Updates for Its Duchenne Muscular Dystrophy Program Capricor 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. Major Estimate Revision • May 21
Consensus revenue estimates increase by 64% The consensus outlook for revenues in fiscal year 2025 has improved. 2025 revenue forecast increased from US$63.3m to US$103.8m. Now expected to report a profit of US$0.02 instead of losses of -US$0.567 per share. Biotechs industry in the US expected to see average net income decline 11% next year. Consensus price target of US$42.13 unchanged from last update. Share price rose 45% to US$11.12 over the past week. Breakeven Date Change • May 14
Forecast breakeven date pushed back to 2027 The 7 analysts covering Capricor Therapeutics previously expected the company to break even in 2026. New consensus forecast suggests losses will reduce by 45% per year to 2026. The company is expected to make a profit of US$60.1m in 2027. Average annual earnings growth of 65% is required to achieve expected profit on schedule. New Risk • May 06
New major risk - Share price stability The company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of American stocks, typically moving 17% 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 (17% average weekly change). Shareholders have been substantially diluted in the past year (45% increase in shares outstanding). Annuncio • Apr 10
Capricor Therapeutics, Inc., Annual General Meeting, May 22, 2025 Capricor Therapeutics, Inc., Annual General Meeting, May 22, 2025. Location: 10865 road to the cure, suite 150, san diego, california 92121, United States Annuncio • Apr 09
Earl Collier Not to Stand for Re-Election as Member of the Board of Capricor Therapeutics, Inc Capricor 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. Reported Earnings • Mar 20
Full year 2024 earnings: EPS and revenues exceed analyst expectations Full year 2024 results: US$1.15 loss per share (further deteriorated from US$0.83 loss in FY 2023). Revenue: US$22.3m (down 12% from FY 2023). Net loss: US$40.5m (loss widened 82% from FY 2023). Revenue exceeded analyst estimates by 6.2%. Earnings per share (EPS) also surpassed analyst estimates by 1.2%. Revenue is forecast to grow 43% p.a. on average during the next 3 years, compared to a 20% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has fallen by 1% per year but the company’s share price has increased by 39% per year, which means it is well ahead of earnings. Annuncio • Mar 18
Capricor Therapeutics, Inc. Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy Capricor 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. Annuncio • Mar 11
Capricor Therapeutics, Inc. to Report Q4, 2024 Results on Mar 19, 2025 Capricor Therapeutics, Inc. announced that they will report Q4, 2024 results After-Market on Mar 19, 2025 Annuncio • Mar 04
Capricor Therapeutics Announces FDA Acceptance and Priority Review of Its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy Capricor 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. New Risk • Jan 16
New major risk - Shareholder dilution The company's shareholders have been substantially diluted in the past year. Increase in shares outstanding: 48% 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 Risk Shareholders have been substantially diluted in the past year (48% increase in shares outstanding). Minor Risk Share price has been volatile over the past 3 months (13% average weekly change). Annuncio • Jan 02
Capricor Therapeutics Completes Submission of Biologics License Application to U.S. FDA for Deramiocel for Treatment of Duchenne Muscular Dystrophy Capricor 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 14
Third quarter 2024 earnings: EPS and revenues miss analyst expectations 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 missed analyst estimates by 37%. Earnings per share (EPS) also missed analyst estimates by 6.0%. 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 the US. 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 79% per year, which means it is well ahead of earnings. Annuncio • Nov 05
Capricor Therapeutics, Inc. to Report Q3, 2024 Results on Nov 13, 2024 Capricor Therapeutics, Inc. announced that they will report Q3, 2024 results After-Market on Nov 13, 2024 New Risk • Oct 20
New major risk - Shareholder dilution The 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 (25% average weekly change). Shareholders have been substantially diluted in the past year (76% increase in shares outstanding). Annuncio • Oct 17
Capricor 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 Annuncio • Oct 12
Capricor Therapeutics Announces Positive Long-Term Data from Hope-2 OLE Study in Duchenne Muscular Dystrophy at 2024 World Muscle Society Congress Capricor 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. Annuncio • Oct 09
Capricor Therapeutics Announces Initiation of Rolling Submission of Biologics License Application with U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy Capricor 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. Price Target Changed • Sep 24
Price target increased by 28% to US$25.40 Up from US$19.80, the current price target is an average from 5 analysts. New target price is 179% above last closing price of US$9.10. Stock is up 59% over the past year. The company is forecast to post a net loss per share of US$1.20 next year compared to a net loss per share of US$0.83 last year. Breakeven Date Change • Sep 24
Forecast to breakeven in 2026 The 5 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$5.61m in 2026. Average annual earnings growth of 68% is required to achieve expected profit on schedule. New Risk • Sep 24
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of American stocks, typically moving 10% a week. This is considered a minor risk. Share price volatility indicates 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. It also 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. Currently, the following risks have been identified for the company: Minor Risks 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). 
