Duyuru • 3h
Capricor Therapeutics Publishes Phase 3 Hope-3 Trial Results for Deramiocel in Duchenne Muscular Dystrophy Capricor 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 27
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 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). Duyuru • Jun 26
Capricor Therapeutics Presents Positive Five-Year HOPE-2 Ole Data and HOPE-3 Phase 3 Results for Deramiocel in Duchenne Muscular Dystrophy Capricor 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. Duyuru • 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 Duyuru • 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. 
