TLDR
- Capricor Therapeutics (CAPR) stock jumped over 440% after announcing positive Phase 3 trial results for Deramiocel
- The HOPE-3 study met both primary and key secondary endpoints with statistical significance
- Trial showed 54% slowing of skeletal muscle disease progression and 91% slowing of cardiac function decline
- Study involved 106 participants across 20 U.S. centers over 12 months
- Company plans to submit response to FDA’s Complete Response Letter incorporating new data
Capricor Therapeutics watched its stock price explode Wednesday morning after the company announced that its experimental cell therapy Deramiocel hit both primary and key secondary goals in a pivotal Phase 3 trial. The trial focused on treating Duchenne muscular dystrophy, a rare genetic disorder.
Capricor Therapeutics, Inc., CAPR
The HOPE-3 study delivered results that exceeded expectations. Deramiocel showed a 54% slowing of skeletal muscle disease progression compared to placebo. The treatment also demonstrated a 91% slowing of cardiac function decline.
Trial Details and Patient Population
The study enrolled 106 boys and young men across 20 U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for 12 months. The average age was approximately 15 years.
All participants continued their standard corticosteroid therapy during the trial. About 90% were already taking cardiac medications at baseline. Over 75% had been diagnosed with cardiomyopathy before entering the study.
The trial was randomized, double-blind, and placebo-controlled. This design helps ensure the results reflect the treatment’s true effects. Deramiocel maintained a favorable safety profile consistent with earlier clinical experience.
The primary endpoint measured Performance of Upper Limb function using the PUL v2.0 scoring system. The treatment group showed statistically better results with a p-value of 0.029. This metric directly relates to a patient’s ability to maintain independence in daily activities.
The key secondary endpoint tracked Left Ventricular Ejection Fraction through cardiac MRI scans. Results reached statistical significance with a p-value of 0.041. Cardiomyopathy represents the leading cause of death in Duchenne patients.
Company Response and Next Steps
CEO Linda Marbán called the results “strong and definitive evidence” that Deramiocel can improve the course of the disease. The findings reinforce benefits seen in the earlier HOPE-2 study and its open-label extension. That extension has now continued for over 48 months.
The company previously received a Complete Response Letter from the FDA earlier this year. Capricor plans to submit its response incorporating the HOPE-3 data. The FDA had previously indicated that HOPE-3 results should be sufficient to support regulatory approval.
Craig McDonald, the national principal investigator for HOPE-3, described the nearly 54% slowing of skeletal muscle disease progression as extraordinary. He noted this preservation of function translates into real benefits for patients. The effect on cardiomyopathy could potentially improve long-term survival.
Jonathan Soslow from Vanderbilt University Medical Center emphasized the cardiac findings. Stabilizing cardiac function has remained a major unmet need in Duchenne treatment. The preservation of left ventricular ejection fraction addresses one of the disease’s most critical aspects.
The trial marks the first Phase 3 study in a largely non-ambulatory Duchenne population to successfully meet its primary endpoint. Statistical significance was achieved in all type 1 error controlled secondary endpoints. The company plans to submit detailed results for presentation at a future scientific meeting and publication in a peer-reviewed journal.
Get 3 Free Stock Ebooks
Discover top-performing stocks in AI, Crypto, and Technology with expert analysis.
- Top 10 AI Stocks - Leading AI companies
- Top 10 Crypto Stocks - Blockchain leaders
- Top 10 Tech Stocks - Tech giants
