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2026-08-21 12:54 UTC → 2026-08-28 19:18 UTC ·
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Deramiocel DMD trial, FDA panel rejection, pivot, and ongoing lawsuit
The FDA’s Cellular, Tissue, Capricor Therapeutics is navigating a shifting regulatory and Gene Therapies Advisory Committee voted 9-to-3 strategic landscape following an FDA advisory committee vote against recommending approval of Capricor Therapeutics’ experimental stem-cell therapy deramiocel the original effectiveness case for Duchenne-related deramiocel in treating Duchenne cardiomyopathy. The panel reiterated concerns that efficacy evidence was insufficient and that key statistical analyses had been altered after the Phase III HOPE-3 study was completed. While In response, the trial met company has narrowed its primary upper-limb endpoint—showing a 54% slower decline in arm function—the overall cardiac endpoint did not reach significance. Safety concerns included allergic-type reactions in 42% of treated patients versus 15% in the placebo group. Following the advisory vote, Capricor announced it is preparing an amendment to the FDA Biologics License Application to refine the drug’s indication, shifting focus toward on upper limb skeletal muscle function rather than cardiomyopathy. function, supported by the HOPE-3 trial meeting its primary endpoint for that specific metric. This regulatory pivot follows HOPE-3 data showing a 4.55 percent mean difference has resulted in the slowing of muscle function decline. Due to this regulatory shift, the FDA is expected to extend extending the current review decision deadline to November 22, 2026. Financially, Capricor reported $237.9 million in cash, cash equivalents, and marketable securities as of 22 August. In parallel, June 30, 2026, while recording a net loss of $74.7 million for the first half of the year. Despite recent stock pullbacks, Oppenheimer recently upgraded the company faces a to ‘Outperform,’ suggesting potential upside. Simultaneously, the company continues to face legal challenges via the federal securities class-action lawsuit, Nkamga v. Capricor Therapeutics, Inc., filed in the U.S. District Court for the Southern District of California. Inc. The suit, representing investors who purchased stock securities between December 2025 and July 2026, alleges the company misled shareholders about the likelihood of FDA approval and failed to disclose material changes to that Capricor made misleading statements or omissions regarding the HOPE-3 trial’s statistical analysis plan. The regulatory plan and legal turmoil contributed to a massive market sell-off, with shares erasing approximately 80% of the company’s market capitalization during its worst week likelihood of trading. FDA approval.