SAN DIEGO, Calif. — Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company 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 this year’s 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 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.
“We are pleased to be presenting our positive long-term open label extension data at this year’s MDA Conference,” said Linda Marbán, Ph.D., chief executive officer of Capricor. “DMD is a progressive disease associated with loss of function over time and there is a critical need for treatment options to slow the rate of decline and preserve upper limb function to enable key activities of daily living. These two-year results suggest that CAP-1002 has the potential to slow the decline of DMD progression and speaks to the potential long-term benefit for patients. At this time, as these patients enter their fourth year of CAP-1002 continuous treatment, we plan to discuss options for potential expedited approval pathways with the FDA and in connection with these efforts, look forward to sharing the three-year results in the second quarter of this year.”
Podium Presentation in Clinical Trial Updates Session
| Title: | Long term safety and efficacy of CAP-1002 in late-stage patients with DMD: A new treatment approach to target skeletal and cardiac muscle pathogenesis |
| Date: | Wednesday, March 6, 2024, 11:45 a.m. – 12:00 p.m. ET |
About the 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. 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. These 24-month results were presented previously at the 2023 Parent Project Muscular Dystrophy (PPMD) Annual Conference.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, CAP-1002 — an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our 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, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need.
Capricor Company Contact
AJ Bergmann, Chief Financial Officer
858.727.1755