Capricor gives update on Deramiocel BLA after adcomm setback, reports Q2 2026 results

Capricor Therapeutics reported on August 13, 2026 that its Biologics License Application for Deramiocel remains under FDA review, and the company is continuing to work with the agency, planning to give a regulatory update on its conference call and further updates as they become available.¹

On July 29, 2026, an FDA advisory panel reviewed whether evidence supports Deramiocel's effectiveness for cardiomyopathy in Duchenne muscular dystrophy patients. The panel's vote was 3 in favor and 9 against on that question.¹ Capricor noted that the committee was not asked to vote on the HOPE-3 primary endpoint or on overall benefit-risk, and in a separate discussion of upper limb function its feedback was directionally supportive of the HOPE-3 evidence, since the indication requested in 2024 was for cardiomyopathy specifically.¹ The committee's recommendation is advisory and non-binding.¹

The full HOPE-3 Phase 3 dataset was published in The Lancet in July following independent peer review, showing a statistically significant slowing of upper limb disease progression measured by PUL 2.0 (p=0.029), with additional functional and cardiac measures characterized as nominally significant under the trial's hierarchical testing procedures.¹ During that review, Capricor found a problem in the statistical model used in its clinical study report and returned to the original pre-unblinding analysis plan; the change affected only left ventricular ejection fraction, moving it to p=0.09 with a 1.8 percentage point treatment difference from the previously reported p=0.04 and 2.4 point difference, while the cardiomyopathy subgroup result and the primary endpoint were unchanged.¹

Separately, the FDA conducted a bioresearch monitoring inspection in July 2026 and issued a Form 483 citing one observation, to which Capricor has responded and is awaiting feedback.¹ In its NS Pharma dispute, Capricor withdrew its motion for a preliminary injunction ahead of an August 10 hearing, without prejudice, opting to pursue arbitration after the FDA's decision, with arbitration expected to begin in the fall.¹

Cash, cash equivalents and marketable securities were approximately $237.9 million as of June 30, 2026, down from about $318.1 million at year-end 2025.¹ The company said based on its current operating plan, its available cash is sufficient to fund operations for at least the next twelve months.¹