Capricor Therapeutics (NASDAQ: CAPR) reported that its pivotal Phase 3 HOPE‑3 study met both its primary and key secondary endpoints, demonstrating statistically significant preservation of left ventricular ejection fraction (LVEF) and a 54 % slowing of skeletal muscle disease progression as measured by the Performance of Upper Limb test, version 2 (PUL v2.0). The trial enrolled 106 boys with Duchenne muscular dystrophy who received 150 million Deramiocel cells or placebo every three months for 12 months, and 91 % of treated patients experienced measurable improvement in cardiac function.
The study’s design and results provide the most robust evidence to date that Deramiocel can modify the disease course in both cardiac and skeletal muscle systems. The primary endpoint—PUL v2.0—was achieved with a 54 % improvement, while the key secondary endpoint—LVEF—showed a 91 % reduction in the rate of decline, confirming the therapy’s dual‑organ benefit. These findings directly address the concerns raised in the FDA’s July 2025 Complete Response Letter, which requested additional data to demonstrate efficacy in both organ systems.
Capricor plans to resubmit a Biologics License Application to the FDA that incorporates the HOPE‑3 data, positioning the company to resolve the regulatory hurdles that delayed approval. The company also maintains a commercialization and distribution agreement with Nippon Shinyaku for the United States and Japan, which could accelerate market entry once regulatory clearance is obtained. The successful trial results therefore represent a critical step toward a first‑in‑class therapy that could fill a substantial unmet need in the DMD patient population.
Financially, Capricor reported a net loss of $24.57 million for Q3 2025, up from $12.56 million in Q3 2024, reflecting continued investment in research and development. Despite the losses, the company’s cash position is sufficient to fund operations through the end of Q4 2026, and a potential $80 million milestone payment from its Japanese partner could extend the runway further. The trial success, combined with the company’s strong cash base, supports confidence in its ability to pursue regulatory approval and subsequent commercialization.
Chief Executive Officer Linda Marbán emphasized that the HOPE‑3 data “deliver strong and definitive evidence that Deramiocel can meaningfully improve the course of Duchenne muscular dystrophy, demonstrating statistically significant improvements in both skeletal and cardiac function.” She added that the results reinforce the durable benefits seen in earlier studies and position Capricor to address the FDA’s remaining questions. The company’s leadership views the data as a pivotal milestone that could transform the therapeutic landscape for DMD patients worldwide.
Investors reacted strongly to the announcement, citing the unambiguous efficacy across both cardiac and skeletal muscle endpoints, the favorable safety profile, and the potential for Deramiocel to become the first disease‑modifying therapy for DMD cardiomyopathy. The trial’s success is expected to accelerate the company’s regulatory strategy and could unlock significant commercial opportunities once approval is achieved.
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