San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) ended the second quarter with about $237.9 million in cash, cash equivalents and marketable securities, down from about $318.1 million at Dec. 31, 2025. The decline came as an FDA advisory committee voted 9 against and 3 in favor on whether evidence shows Deramiocel is effective for cardiomyopathy in Duchenne muscular dystrophy (DMD) patients.
Advisory panel vote is non-binding and limited to cardiomyopathy
The Cellular, Tissue and Gene Therapies Advisory Committee voted on July 29, 2026. Capricor says the panel was not asked to vote on the HOPE-3 primary endpoint or on overall benefit-risk. In a separate discussion of upper limb function, the committee's feedback was directionally supportive of the HOPE-3 evidence, according to the company. The recommendation is advisory and non-binding, and the Biologics License Application (BLA) remains under active FDA review.
CEO Linda Marbán said the indication Capricor requested in 2024 was cardiomyopathy in DMD, and that "we continue to believe there is a path to approval." Capricor planned to give a regulatory update on its earnings call.
Corrected LVEF analysis weakens a secondary cardiac result
The full HOPE-3 Phase 3 dataset was published in The Lancet in July. Deramiocel met the primary endpoint of upper limb function, slowing disease progression as measured by PUL 2.0 at p=0.029. Supportive functional and cardiac results are characterized as nominally significant under the hierarchical testing procedures.
During peer review, Capricor found an issue with the statistical model in the clinical study report and reverted to the statistical analysis plan in place before unblinding. Only left ventricular ejection fraction (LVEF) was affected. It now shows p=0.09 and a 1.8 percentage point treatment difference, against p=0.04 and 2.4 points in the topline data. The pre-specified cardiomyopathy subgroup is unchanged at p=0.02.
FDA inspection yields a single Form 483 observation
The FDA conducted a Bioresearch Monitoring inspection in July 2026 and issued a Form 483 citing one observation. Capricor has submitted its responses and is awaiting feedback.
Operating expenses rise to $42.9M as net loss widens to $40.7M
Capricor recognized no revenue in the first half of 2026 or 2025. Second-quarter operating expenses were about $42.9 million, against $27.7 million a year earlier. First-half operating expenses reached about $79.7 million, up from $52.7 million.
The second-quarter net loss was about $40.7 million, or $0.70 per share, versus $25.9 million, or $0.57 per share, in the prior-year quarter. The first-half net loss was about $74.7 million, or $1.29 per share, compared with $50.3 million, or $1.10 per share.
Capricor says current cash is sufficient to fund operations for at least the next twelve months. That outlook excludes product sales revenue and any monetization of a Priority Review Voucher, if received. Longer-term guidance will follow greater regulatory clarity.
Launch preparations slow while non-Deramiocel programs go on hold
Capricor's San Diego GMP facility is operational and can support an initial commercial launch if Deramiocel is approved. Validation and FDA inspection of the second-floor expansion are targeted for 2027. Chief Commercial Officer Michael Maurer is building out the launch organization, but Capricor is advancing commercial readiness at a slower pace pending regulatory clarity.
Programs unrelated to Deramiocel, including the exosome-based platform, are on hold. Trials in younger DMD patients and Becker muscular dystrophy are stage-gated to the U.S. regulatory pathway. Capricor has opened regulatory engagement in Europe and Japan.
Capricor withdraws injunction motion against NS Pharma and heads to arbitration
A state court was due to hear Capricor's motion for a preliminary injunction against NS Pharma on Aug. 10, 2026, ahead of the PDUFA action date. Capricor withdrew the motion without prejudice, preferring to resolve the contractual dispute in arbitration after the FDA decision. It estimates arbitration will begin this fall. Capricor still contends the pricing structure in the U.S. Distribution Agreement is fundamentally flawed and would impede patient access, and it continues to seek rescission.
Safety database spans 1,300 infusions in more than 200 patients
Capricor has administered about 1,300 intravenous Deramiocel infusions to more than 200 DMD patients across three clinical trials. More than 80 patients are enrolled in open-label extension studies, some receiving continuous infusions for over five years. The company describes the long-term safety profile as consistent and well characterized.
Capricor will present at the Wells Fargo Healthcare Conference (Sept. 8-10, Boston), the Cantor Global Healthcare Conference (Sept. 9-11, New York) and the H.C. Wainwright Annual Global Investment Conference (Sept. 14-16, New York).