Capricor Therapeutics Inc (CAPR)
Muscular Dystrophies · FDA target action date (PDUFA) 2026-11-22 (per 8-K filed 2026-08-24) · NCT05126758
Published 2026-09-02Market cap $558M11 sourced statements
Deramiocel (CAP-1002) — BLA for Duchenne muscular dystrophy cardiomyopathy · Class 2 resubmission under review
What this sheet is scored on
callCash of $100M against operating outflow of $33M per quarter gives roughly 3.0 quarters of runway, clearing the FDA target action date (PDUFA) 0.9 quarters out but leaving under a year. On these figures the company raises capital within four quarters.
Regulatory
factThe FDA extended the PDUFA target action date for the deramiocel BLA from August 22, 2026 to November 22, 2026, classifying the company's submission as a major amendment.
“the U.S. Food and Drug Administration ("FDA") has extended the Prescription Drug User Fee Act ("PDUFA") target action date for its Biologics License Application ("BLA") for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy ("DMD"), from August 22, 2026 to November 22, 2026. The FDA has classified the submission as a major amendment…”
factThe FDA advisory committee voted 3 for and 9 against on whether the available evidence supports the effectiveness of deramiocel for DMD cardiomyopathy. The vote is non-binding.
“voted that available evidence did not support the effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy ("DMD"), a rare and life-threatening disease for which no approved cardiomyopathy treatment currently exists (3 for, 9 against, 0 abstain). The Advisory Committee's recommendation is non-binding…”
factThe BLA previously received a Complete Response Letter in July 2025 stating the application did not meet the statutory requirement for substantial evidence of effectiveness and requesting additional clinical data. The resubmission added data from the Phase 3 HOPE-3 trial.
“In July 2025, we received a Complete Response Letter ("CRL") from the FDA stating that the application did not meet the statutory requirement for substantial evidence of effectiveness and requesting additional clinical data. Following a Type A meeting with the FDA in August 2025, we aligned with the Agency on a regulatory path forward to address the CRL, including the submission of additional clinical data from the P…”
factDesignations disclosed: RMAT, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review Voucher. These accelerate review and, for Orphan, confer exclusivity if approved; none is evidence of efficacy.
Balance sheet
factA facility lease signed in July 2026 gives either party a termination right if deramiocel is not approved by December 31, 2026.
“if Capricor does not receive FDA approval of Deramiocel for the treatment of Duchenne muscular dystrophy by December 31, 2026, then either Capricor or Landlord may terminate the Lease by delivering written notice within five business days after such date…”
Share count and dilution
fact58,138,989 shares outstanding against 75,887,396 if every outstanding option, warrant and convertible were exercised. In that case 23.4% of a gain in enterprise value would accrue to those holders rather than to current shareholders.
factShare count up 27% year over year to 58,138,989 as of 2026-08-13.
Market
factNo approved product carries Muscular Dystrophies on label.
Trial registry
factRegistered design for NCT05126758: randomized, quadruple-masked, 106 participants (actual). Primary outcome: Change in the upper limb function.
Pipeline
fact1 distinct molecule in 2 active company-sponsored trials; CAP-1002 accounts for 2 of them at Phase 2/Phase 3.
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