en.Wedoany.com Reported - Capricor Therapeutics Inc. (CAPR:NASDAQ) reported its first-quarter 2026 financial results and disclosed the latest regulatory and clinical progress of its investigational cell therapy Deramiocel (CAP-1002) for Duchenne muscular dystrophy (DMD). The Biologics License Application (BLA) for Deramiocel is under review by the U.S. Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026.
Duchenne muscular dystrophy is a severe X-linked genetic disorder caused by the absence of functional dystrophin protein, leading to progressive degeneration of skeletal, respiratory, and cardiac muscles. The disease affects approximately 15,000 people in the United States, primarily boys. Cardiomyopathy and heart failure resulting from cardiac muscle degeneration are the leading causes of death in DMD patients, with no cure currently available and limited treatment options.
Deramiocel is composed of allogeneic cardiosphere-derived cells (CDCs). This population of cardiac cells has demonstrated immunomodulatory and anti-fibrotic effects in preclinical and clinical studies. Exosomes secreted by CDCs can target macrophages and alter their expression profile, inducing a healing phenotype. The drug has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA), as well as Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, Advanced Therapy Medicinal Product (ATMP) designation from the EMA, and Rare Pediatric Disease Designation from the FDA.
Financially, as of March 31, 2026, the company held approximately $278.6 million in cash, cash equivalents, and marketable securities, compared to approximately $318.1 million as of December 31, 2025. Total operating expenses for the first quarter of 2026 were approximately $36.8 million, compared to approximately $25.0 million in the same period last year; net loss was approximately $33.9 million, or $0.59 per share, compared to a net loss of approximately $24.4 million, or $0.53 per share, in the same period last year. The company expects its existing funds to cover expenses and capital requirements through the fourth quarter of 2027.
In research and operations, cardiac MRI analysis showed that Deramiocel significantly reduced myocardial fibrosis as assessed by late gadolinium enhancement compared to placebo. A home-based Duchenne video assessment of the "Eat 10 Bites" metric demonstrated statistically significant improvements in upper limb function and activities of daily living. The company's manufacturing facility in San Diego has passed FDA pre-approval inspection and is prepared to support initial commercial launch. Upon completion of the second-floor expansion, full production capacity is expected to cover approximately 2,000 to 2,500 patients per year, corresponding to approximately 10,000 doses annually. The company is awaiting comprehensive facility validation, with FDA approval targeted for the first half of 2027.
Approximately 90 patients are currently enrolled in the open-label extension study, with multiple patients having received continuous intravenous infusions of Deramiocel for up to five years, involving over 800 infusions, and safety data continues to be favorable. Capricor has hired Michael Maurer as Chief Commercial Officer, who brings direct DMD commercial experience. Following potential approval, the company expects to be eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), which is transferable and saleable.
Dr. Linda Marbán, Chief Executive Officer of the company, stated that with the FDA's review of the BLA ongoing, the team's top priority is execution, working closely with the agency to prepare for a potential launch and continuing to build the capabilities of a commercial-stage company. Multiple analysts issued or reiterated ratings following the earnings report. Oppenheimer gave an "Outperform" rating with a 12- to 18-month price target of $54.00; H.C. Wainwright & Co. gave a "Buy" rating with a price target of $60; Roth analysts conducted a discounted cash flow analysis of the company's valuation. Capricor is also advancing the StealthX™ platform for muscle-targeted delivery of siRNA, proteins, and small molecules, and preparing to expand Deramiocel to Becker muscular dystrophy.
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