Capricor Therapeutics Inc Stock Price, News & Analysis

Capricor Therapeutics (NASDAQ: CAPR) has received positive regulatory updates for Deramiocel, its cell therapy candidate for treating Duchenne Muscular Dystrophy (DMD)-associated cardiomyopathy. The FDA has indicated that an Advisory Committee meeting is not required, and the Biologics License Application (BLA) continues under Priority Review with a PDUFA target date of August 31, 2025.

Key developments include a successful pre-license inspection, mid-cycle review with no major issues, and an upcoming late-cycle review meeting in mid-July. Additionally, four-year data from the HOPE-2 Open-Label Extension study presented at PPMD 2025 Conference showed sustained cardiac function and clinical benefits from long-term Deramiocel treatment.

Capricor Therapeutics (CAPR) announced positive 4-year data from the HOPE-2 Open-Label Extension study of Deramiocel for Duchenne Muscular Dystrophy (DMD). The results show preservation of cardiac function with a median change of -0.5 points from baseline, and even better outcomes in patients with baseline LVEF >45%. The treatment demonstrated continued slowing of skeletal muscle disease progression, with patients showing a smaller decline in the fourth year (0.6 points) compared to the first year (1.8 points) on the PUL v2.0 scale. Deramiocel maintains a favorable safety profile throughout the study. The company's Biologics License Application (BLA) is under priority review, with regulatory discussions progressing as planned. The cell therapy candidate has received multiple regulatory designations including Orphan Drug, RMAT, and Rare Pediatric Disease Designation from the FDA.

Capricor Therapeutics (CAPR) announced two significant regulatory developments for its lead cell therapy candidate Deramiocel. First, the FDA granted Orphan Drug Designation for treating Becker Muscular Dystrophy (BMD), expanding the therapy's potential beyond Duchenne Muscular Dystrophy (DMD). Second, following a successful FDA Pre-License Inspection, the company remains on track for its August 31, 2025 PDUFA date for Deramiocel in DMD treatment. Deramiocel consists of allogeneic cardiosphere-derived cells that have shown promise in preserving cardiac and skeletal muscle function in dystrophiopathies. The therapy has been tested in over 250 human subjects and documented in numerous peer-reviewed publications. BMD affects approximately 5,000 individuals in the US, while DMD affects around 15,000, primarily boys.

Capricor Therapeutics (CAPR) announced significant regulatory progress for Deramiocel, its cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The FDA successfully completed a Pre-License Inspection of Capricor's San Diego facility, with minor Form 483 observations related to quality systems and documentation. The company's BLA remains under priority review with a PDUFA date of August 31, 2025. An FDA Advisory Committee meeting is scheduled for July 30, 2025, and a mid-cycle review was completed with no major issues. Deramiocel, which has received Orphan Drug, RMAT, and ATMP designations, consists of allogeneic cardiosphere-derived cells showing promising immunomodulatory and anti-fibrotic properties in DMD treatment. Upon potential FDA approval, Capricor would be eligible for a Priority Review Voucher.

Capricor Therapeutics (CAPR) reported Q1 2025 financial results and provided updates on its lead candidate deramiocel. The company's BLA for deramiocel in treating Duchenne muscular dystrophy (DMD) remains under FDA priority review with a PDUFA date of August 31, 2025. A mid-cycle review meeting revealed no significant deficiencies, and an advisory committee meeting is planned. The company reported a cash position of $144.8M, expected to fund operations into 2027. Q1 financial results showed zero revenue compared to $4.9M in Q1 2024, with a net loss of $24.4M ($0.53 per share) versus $9.8M ($0.31 per share) in Q1 2024. Notable developments include appointing Dr. Michael Binks as CMO and NIAID's plans to initiate a Phase 1 trial of Capricor's StealthX™ exosome vaccine in Q3 2025.

Capricor Therapeutics (NASDAQ: CAPR) has appointed Dr. Michael Binks as Chief Medical Officer, effective immediately. Dr. Binks, a board-certified rheumatologist with 25 years of experience, joins Capricor ahead of the potential approval of deramiocel in Q3 2025. He previously served as Vice President and Head of Rare Disease Clinical and Translational Research at Pfizer, where he led global clinical efforts across multiple studies, including several in Duchenne muscular dystrophy (DMD). His expertise spans immunology, neurology, cardiology, nephrology, and hematology, with a track record of advancing first-in-class therapies including Benlysta, Hympavzi, Paxlovid, and Beqvez. The appointment strengthens Capricor's medical leadership as the company advances deramiocel toward potential commercialization for DMD treatment and explores pipeline expansion opportunities.

Capricor Therapeutics (NASDAQ: CAPR) has announced it will release its Q1 2025 financial results on Tuesday, May 13, 2025, after market close, followed by a conference call at 4:30 p.m. ET. The company, focused on developing cell and exosome-based therapeutics for rare diseases, is advancing its lead candidate deramiocel, an allogeneic cardiac-derived cell therapy. Deramiocel is in late-stage development for treating Duchenne muscular dystrophy (DMD), showing promising immunomodulatory and anti-fibrotic properties. Capricor has secured an exclusive commercialization agreement with Nippon Shinyaku for deramiocel in the US and Japan. The company is also developing its proprietary StealthX™ platform for various therapeutic applications including vaccinology and targeted delivery of therapeutics.

Capricor Therapeutics (NASDAQ: CAPR) has completed its mid-cycle review meeting with the FDA regarding deramiocel, a cell therapy treatment for Duchenne Muscular Dystrophy (DMD) cardiomyopathy. The FDA reported no significant deficiencies and confirmed the PDUFA target action date of August 31, 2025. An advisory committee meeting will be held before the target date. The BLA submission is supported by cardiac data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials. Deramiocel, which has received Orphan Drug Designation from both FDA and EMA, consists of allogeneic cardiosphere-derived cells that help preserve cardiac and skeletal muscle function through immunomodulatory and anti-fibrotic actions. DMD affects approximately 15,000-20,000 patients in the United States, occurring in about one in 3,500 male births, with heart failure being the leading cause of death.

OmniAb (NASDAQ: OABI) has announced two strategic appointments to its Board of Directors: Philip J. Gotwals, Ph.D., and Steve Crouse, while Director Sarah Boyce steps down after nearly six years of service. These changes increase the board size to seven members.

Dr. Gotwals brings 30 years of biopharmaceutical experience, notably as Global Head of Business Development and Licensing at Novartis Institutes for BioMedical Research, where his team executed over 50 major strategic transactions. He will serve on the Human Capital Management & Compensation Committee and Science & Technology Committee.

Steve Crouse, currently Senior Vice President and General Manager of Bio-Techne's Analytical Solutions Division, contributes over 20 years of life sciences expertise. He will serve on the Audit Committee and Science & Technology Committee.