SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), a biotechnology company focused on developing therapies for rare diseases, announced positive three-year results from its HOPE-2 open-label extension study of CAP-1002, a treatment for Duchenne muscular dystrophy (DMD). The study showed sustained benefits in skeletal muscle function and stabilization of cardiac function in patients with DMD.
Patients treated with CAP-1002 demonstrated a statistically significant reduction in the decline of Performance of Upper Limb (PUL 2.0) scores and stabilization in Left Ventricular Ejection Fraction (LVEF) compared to an external comparator group from Cincinnati Children’s Hospital Medical Center. The treatment was also consistently well-tolerated over the course of the study.
Capricor's CEO, Linda Marbán, Ph.D., expressed encouragement over the results, which suggest long-term impact of CAP-1002 in treating DMD, a genetic disorder characterized by progressive muscle degeneration. The recent findings were shared with the FDA during a Type-B meeting in May 2024, as the company prepares for a potential Biologics License Application (BLA) and commercialization.
The HOPE-2 study was initially a randomized, double-blind, placebo-controlled trial where patients were treated with CAP-1002 or a placebo every three months. Following the trial, a gap phase occurred before patients entered the ongoing HOPE-2 open-label extension study, receiving CAP-1002 every three months. The study met its primary endpoint at the one-year mark and continues into its fourth year.
Capricor plans to present detailed results at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference from June 27-29, 2024, and expects to announce topline results from the Phase 3 HOPE-3 pivotal trial in the fourth quarter of 2024.
DMD affects approximately one in every 3,500 male births in the United States, with current treatment options limited and no cure available. CAP-1002, Capricor's lead product candidate, is an allogeneic cardiac-derived cell therapy that has shown promise in preclinical and clinical studies for its potential immunomodulatory, antifibrotic, and regenerative actions.
The information in this article is based on a press release statement from Capricor Therapeutics.
In other recent news, Capricor Therapeutics has been making significant strides in the development of its Duchenne muscular dystrophy (DMD) therapy, CAP-1002. The company announced in its first quarter earnings call the completion of enrollment for Cohort A of its HOPE-3 Phase 3 pivotal trial and plans to submit a biologics license application based on the trial's outcomes.
Moreover, Capricor's financial position appears stable, with a cash runway until Q1 2025, bolstered by a milestone payment from Nippon Shinyaku and funds raised through their at-the-market program.
In terms of analyst outlook, Oppenheimer initiated coverage on Capricor, assigning an Outperform rating and expressing optimism about the company's lead candidate, CAP-1002. The firm forecasts that the cell therapy could achieve approximately $1 billion in net sales in the U.S. by 2030, in partnership with NS Pharma, resulting in around $310 million in revenue for Capricor.
Ladenburg Thalmann also updated its outlook on Capricor, increasing the stock's price target to $25 from $24, while reiterating a Buy rating.
Furthermore, Capricor is preparing for potential commercialization and discussing label expansion with the FDA, while its San Diego manufacturing facility is ready to meet demand. The company is also making strides in its regulatory discussions with the FDA and is planning for global expansion. These developments underline the recent progress made by the company and its promising future prospects.
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