Capricor Therapeutics, Inc.

What they do

Capricor develops cell and exosome-based therapeutics, led by Deramiocel, a cardiosphere-derived cell (CDC) therapy intended to slow DMD progression through immunomodulatory, anti-inflammatory, pro-angiogenic and anti-fibrotic activity. The company has administered approximately 1,300 intravenous infusions to over 200 DMD patients across three clinical trials and describes its clinical program as focused on adolescents and young adults, many non-ambulatory. Its earlier-stage StealthX exosome platform is pursued through collaborations including NIH, NIAID, Johns Hopkins University, the Department of Defense, the U.S. Army Institute of Surgical Research and Cedars-Sinai. It operates a GMP manufacturing facility in San Diego.

Revenue drivers

• Deramiocel (cell therapy) — No product revenue generated; the company states it currently has no products approved for commercial sale, and future revenue depends on FDA approval and commercialization in the U.S., with possible partners in select international markets.
• StealthX exosome platform — Preclinical/early-stage program exploring exosome-based approaches for infectious diseases, monogenic diseases and other indications, advanced through collaborations and partnerships rather than internal revenue.
• Reported historical revenue — Annual revenue was $25.2M in 2023, $22.3M in 2024 and $0.00 in 2025; recent quarterly revenue ended in 2024 ($11.1M in the quarter ended 2024-12-31), and no product sales are reported.

Recent performance

Revenue was $0.00 in 2025, following $22.3M in 2024 and $25.2M in 2023, and net loss widened to $105.0M in 2025 from $40.5M in 2024 (diluted EPS of -$2.26 versus -$1.15). Operating cash flow was -$69.8M in 2025 versus -$40.0M in 2024. The company reported cash, cash equivalents and marketable securities of approximately $238 million as of June 30, 2026. On July 29, 2026, the FDA Cellular, Tissue and Gene Therapies Advisory Committee voted 3 in favor and 9 against on whether available evidence provides substantial evidence of effectiveness of Deramiocel for cardiomyopathy in DMD. In July 2026 the FDA issued a Form 483 with one observation following a BIMO inspection, and the company has submitted responses.

Strategy

The priority is advancing the Deramiocel BLA through FDA review and preparing for a potential U.S. commercial launch. HOPE-3 Phase 3 results were published in The Lancet, reporting a statistically significant slowing of upper limb disease progression on PUL 2.0 (p=0.029), with supportive functional and cardiac results described as nominally significant. The San Diego GMP facility is operational for initial launch, with second-floor expansion targeted for full validation and FDA inspection in 2027. Michael Maurer joined as Chief Commercial Officer, and the company says it is building out the launch organization at a slower pace pending regulatory clarity. The exosome platform is to be advanced mainly through collaborations and partnerships.

Risks

• No approved product or revenue — Capricor has no products approved for commercial sale and reported $0.00 revenue in 2025, so any product revenue depends on Deramiocel approval.
• FDA advisory committee vote against — The FDA advisory committee voted 3 in favor and 9 against on substantial evidence of effectiveness for cardiomyopathy in DMD, and the recommendation, while non-binding, creates regulatory uncertainty.
• Statistical model issue in HOPE-3 — The company identified an issue with the statistical model in the clinical study report and reverted to the prior statistical analysis plan, revising the left ventricular ejection fraction result to p=0.09 and a 1.8 percentage point treatment difference from p=0.04 and 2.4 points reported as topline.
• Form 483 and funding needs — The FDA issued a Form 483 with one observation after a July 2026 BIMO inspection, and the 10-K risk factors state that substantial additional funding may be required to complete development and potential commercialization.

Outlook

Management states the BLA remains under active FDA review with a PDUFA target action date of August 22, 2026, and that it continues to work with the Agency on a path forward. The company plans to provide further regulatory updates as they become available. Commercial readiness is proceeding at a slower pace pending regulatory clarity, with the second-floor manufacturing expansion targeted for full validation and FDA inspection in 2027. Capricor also reports that the NS Pharma dispute remains active; it withdrew its motion for preliminary injunction without prejudice.