Capricor Therapeutics Presents at 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Presentation Highlights Positive 24-Month Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy

SAN DIEGO, March 06, 2024 (GLOBE NEWSWIRE) -- 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 cell therapy 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.”