Capricor Therapeutics Inc. (NASDAQ: CAPR) stock fell 13% on Friday following the announcement that the U.S. Food and Drug Administration (FDA) will convene an advisory committee meeting to review its Biologics License Application (BLA) for Deramiocel. The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) is scheduled to meet on July 29, 2026. This regulatory step precedes the PDUFA target action date of August 22, 2026. The BLA seeks approval of Deramiocel, an investigational cell therapy for the treatment of Duchenne Muscular Dystrophy (DMD), supported by data from the HOPE-2 and HOPE-3 clinical trials.

The market decline reflects investor reaction to the additional regulatory hurdle. This occurs as the FDA reconsiders multiple cell and gene therapy applications, a trend noted following the tenure of Dr. Vinay Prasad. Recent industry activity includes Replimune Group Inc. aligning with the FDA on a path forward for its melanoma therapy BLA, and Regenxbio Inc. gaining clarity on potential accelerated approval for Navsunli.

Clinical Data Supporting the BLA

The application is backed by positive five-year data from the HOPE-2 Open-Label Extension (OLE) study, demonstrating durable skeletal and cardiac muscle benefits. These findings were presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference. Additionally, the BLA includes results from the HOPE-3 Phase 3 trial, a multicenter, randomized, double-blind, placebo-controlled study enrolling 106 patients with DMD.

Five-Year HOPE-2 OLE Results

In the HOPE-2 OLE study, patients receiving Deramiocel experienced a mean total-score decline of less than 5 points on the Performance of the Upper Limb (PUL 2.0) scale over five years. By comparison, a cohort-matched external comparator of standard-of-care DMD patients showed a modeled decline of roughly 2.4 points per year, projecting to approximately 12 points over five years. A separate natural-history analysis reported a decline of approximately 8.1 points over three years in non-ambulant patients.

Cardiac function, measured by left ventricular ejection fraction (LVEF) on cardiac MRI, remained stable over the five-year period. This contrasts with a modeled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator. Deramiocel maintained a favorable safety profile, consistent with over 800 intravenous infusions administered to date across the clinical development program.

HOPE-3 Phase 3 Results

The HOPE-3 Phase 3 trial met its primary endpoint (PUL 2.0, p=0.03) and key secondary cardiac endpoint (LVEF, p=0.04), along with all other Type I error-controlled secondary endpoints. These results align with the durable skeletal and cardiac muscle benefits observed in the HOPE-2 OLE study.

Linda Marbán, Ph.D., CEO of Capricor, stated that the combined data represent the totality of clinical evidence for Deramiocel, showing durable, statistically significant benefit to both skeletal and cardiac muscle function. The July 29, 2026, advisory committee meeting will be available for live streaming.

Capricor Therapeutics, a biotechnology company developing cell and exosome-based therapeutics for rare diseases, announced that senior management will participate in two upcoming investor conferences on Wednesday, June 10. The company will engage with investors to discuss its lead product candidate, Deramiocel, and its proprietary StealthX exosome platform.

The company is scheduled to attend the Goldman Sachs 47th Annual Global Healthcare Conference in Miami, FL. Management will conduct a fireside chat and investor meetings at 10:00 a.m. ET. A live webcast of the session will be accessible, with a replay available in the Investors section of the Capricor Therapeutics website following the event.

Additionally, Capricor Therapeutics will participate in the Oppenheimer CNS and Neuro-Muscular Summit, also taking place in Miami, FL. Senior management will take part in an industry panel and hold investor meetings throughout the day.

Conference Schedule

Capricor Therapeutics focuses on advancing therapies for rare diseases, with Deramiocel in late-stage development for Duchenne muscular dystrophy (DMD). The company is also developing its StealthX platform for targeted delivery of therapeutics across various diseases.