San Diego — Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, announced that the Company presented the 18-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 this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which took place virtually and in-person in Dallas, Texas from March 19-22, 2023. Dr. Craig McDonald, national Principal Investigator and University of California, Davis, Professor and Department of Physical Medicine and Rehabilitation Chair, presented the findings during a late-breaking session.
Key results from the study, include:
- 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).
- Both groups experienced reduced disease progression once all patients began treatment in the OLE study.
- CAP-1002 treatment during the OLE portion of the study continues to yield a consistent 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 18-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.
“There are thousands of boys and young men across the world who continue to face the devastating and immobilizing effects of DMD, and for whom few therapeutic options currently exist,” said Dr. Linda Marbán, CEO of Capricor. “Our 18-month results suggest that CAP-1002 has the potential to slow the decline of DMD progression and speaks to the long-term benefit for patients. We were pleased to present these results alongside other industry leaders who share Capricor’s mission of developing novel treatments for patients suffering from rare neuromuscular conditions.”
Capricor is currently conducting the HOPE-3, Phase 3 trial, designed as a randomized, double-blind, placebo-controlled study with enrollment criteria similar to HOPE-2. The Phase 3 study is currently enrolling subjects (NCT05126758).
For more information on the MDA conference, please visit www.mdaconference.org.
About HOPE-2 Open Label Extension (OLE) Study
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 and are non-ambulant, the later stage of the disease process. The study was conducted at nine sites across the United States. 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 18 months. Patients continued through the gap phase (off treatment for both groups) and the OLE Phase (on treatment for both groups).
Patients in the study were evaluated using the Performance of the Upper Limb (PUL 2.0), a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist and hand) function, with a conceptual framework reflecting the progression of weakness in upper limb function.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy 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 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.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Capricor’s focus is on developing exosomes capable of delivering nucleic acids, including mRNA, as well as proteins to treat or prevent a variety of diseases. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.