Capricor Therapeutics (CAPR) Stock News

Capricor Therapeutics (NASDAQ: CAPR) has announced a scheduled Type A meeting with the FDA to discuss the regulatory pathway for their Biologics License Application (BLA) for Deramiocel, their lead cell therapy candidate for treating cardiomyopathy in Duchenne muscular dystrophy (DMD).

Due to this meeting, the company has rescheduled its Q2 2025 financial results release to August 11, 2025, after market close, with a conference call at 4:30 p.m. ET. Deramiocel, currently in late-stage development, has shown promising immunomodulatory and anti-fibrotic properties in preserving cardiac and skeletal muscle function in DMD patients. The company has an exclusive commercialization agreement with Nippon Shinyaku Co., Ltd. for Deramiocel in the US and Japan.

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on cell and exosome-based therapeutics for rare diseases, will release its Q2 2025 financial results on August 13, 2025, after market close. The company will host a conference call at 4:30 p.m. ET the same day.

Capricor's lead product candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage development for Duchenne Muscular Dystrophy (DMD). The company has partnered with Nippon Shinyaku for exclusive commercialization rights in the US and Japan. Additionally, Capricor is developing its proprietary StealthX™ platform for targeted delivery of therapeutics.

Capricor Therapeutics (NASDAQ: CAPR) announced a webinar in collaboration with Parent Project Muscular Dystrophy (PPMD) scheduled for July 29, 2025, at 1:00 p.m. ET. The webinar will discuss the status of Capricor's Biologics License Application (BLA) for Deramiocel, their lead product candidate for treating Duchenne Muscular Dystrophy (DMD).

Deramiocel is an allogeneic cardiac-derived cell therapy in late-stage development, demonstrating immunomodulatory and anti-fibrotic properties for preserving cardiac and skeletal muscle function in muscular dystrophies. The company has secured an exclusive commercialization agreement with Nippon Shinyaku Co., Ltd. for Deramiocel distribution in the United States and Japan, pending regulatory approval.

Capricor Therapeutics (NASDAQ: CAPR) received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, its cell therapy candidate for treating cardiomyopathy in Duchenne muscular dystrophy (DMD).

The FDA stated it cannot approve the BLA in its current form, citing insufficient evidence of effectiveness and the need for additional clinical data. Capricor plans to resubmit the BLA with data from its ongoing Phase 3 HOPE-3 trial, which includes 104 patients, with topline results expected in Q3 2025.

The company will request a Type A meeting with the FDA to discuss next steps. Deramiocel has received multiple designations including Orphan Drug, RMAT, and Rare Pediatric Disease Designation, potentially qualifying for a Priority Review Voucher upon approval.

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.