Capricor Faces Activist Pressure and Cash Burn Concerns Amid Deramiocel's Narrowed Regulatory Pivot
Capricor Therapeutics is navigating an intense regulatory and corporate crisis as its lead cell therapy candidate for Duchenne muscular dystrophy (DMD), deramiocel (CAP-1002), faces a major delay. The FDA's planned August 22, 2026, Prescription Drug User Fee Act (PDUFA) action date has officially been postponed. Following a devastating 9-3 non-binding rejection by the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) on July 29, 2026, regarding the drug's efficacy in treating Duchenne-related cardiomyopathy, Capricor has pivoted its regulatory strategy.
During its second-quarter earnings call, Capricor announced plans to submit a major amendment to its Biologics License Application (BLA). This amendment shifts the target indication from heart function (cardiomyopathy) to skeletal muscle function, specifically upper-limb function (which was the primary endpoint of its HOPE-3 trial).1 The amendment will also include 24-month open-label extension data. The FDA has signaled its willingness to review this amendment and has postponed the August 22 deadline, extending the review timeline once the amendment is formally received.
This regulatory delay has intensified pressure from activist investor Kaos Capital. In a letter to Capricor shareholders, Kaos Capital called for an immediate special meeting, board changes, and a capital-preservation plan. Kaos raised concerns over Capricor's cash burn, noting that the company has paused all other pipeline activities to focus entirely on deramiocel, leaving it highly vulnerable to a potential regulatory failure.
What This Means
By shifting its target indication from cardiomyopathy (the heart) to upper-limb function (the arm), Capricor is attempting to salvage its BLA by aligning it with the HOPE-3 trial's primary endpoint. While the FDA's willingness to review the amendment avoids an immediate Complete Response Letter (CRL), it leaves investors facing a protracted delay. The corporate pressure from Kaos Capital highlights growing shareholder anxiety over the company’s high cash burn and singular reliance on deramiocel's regulatory survival.
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An instance of Narrowing a drug's clinical claims is the ultimate escape hatch for applications doomed by trial design disputes. — Capricor amended its BLA to target a narrower upper-limb skeletal muscle claim rather than risk an outright rejection of its heart-focused data. ↩︎