Capricor’s DMD therapy clears FDA pre-license inspection

Published 11/06/2025, 14:30
Capricor’s DMD therapy clears FDA pre-license inspection

SAN DIEGO - Capricor Therapeutics (NASDAQ:CAPR) announced Wednesday that the U.S. Food and Drug Administration has completed its Pre-License Inspection of the company’s San Diego manufacturing facility for Deramiocel, its cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The company, currently valued at $635 million, has seen its stock surge 150% over the past year, reflecting strong investor confidence in its development pipeline. According to InvestingPro analysis, the stock appears fairly valued based on current market conditions.

The inspection concluded with a Form 483 containing several observations primarily related to quality systems and documentation practices. Capricor has submitted responses to the FDA and stated that none of the observations required material changes to the manufacturing process or facility. The company’s strong financial position, with a current ratio of 6.55 and minimal debt, provides ample resources to address any regulatory requirements. InvestingPro subscribers can access detailed financial health metrics and 13 additional ProTips about Capricor’s outlook.

The FDA has scheduled an Advisory Committee meeting for July 30, 2025, to review Deramiocel’s Biologics License Application (BLA), which remains under priority review with a target action date of August 31, 2025.

According to the company, a mid-cycle review was recently completed with no significant issues identified, and a late-cycle meeting is planned for mid-July 2025.

With analyst price targets ranging from $25 to $77, and projected revenue growth of 242% for 2025, market expectations remain high for Capricor’s potential. "This inspection outcome is a major regulatory milestone, particularly in a field where standards are exceptionally high," said Linda Marbán, Capricor’s Chief Executive Officer, in the press release.

Deramiocel consists of allogeneic cardiosphere-derived cells that have been granted Orphan Drug Designation by both the FDA and European Medicines Agency. The therapy has also received Regenerative Medicine Advanced Therapy Designation in the U.S.

DMD is a genetic disorder affecting approximately 15,000-20,000 patients in the United States. It causes progressive muscle weakness and has a median mortality age of about 30 years.

If approved, Deramiocel would be eligible to receive a Priority Review Voucher based on its previous rare pediatric disease designation.

In other recent news, Capricor Therapeutics reported a wider-than-expected loss for the first quarter of 2025, with earnings per share at -$0.53, missing the forecasted -$0.32. The company also reported no revenue for the quarter, a decrease from $4.9 million in the same period last year. Despite these financial setbacks, Capricor maintains a strong cash position with $144.8 million on hand. Additionally, Roth/MKM initiated coverage of Capricor Therapeutics with a Buy rating and a 12-month price target of $31.00, citing the potential of its product, deramiocel™, for treating Duchenne muscular dystrophy-related cardiomyopathy. In a strategic move, Capricor’s stockholders recently approved the 2025 Equity Incentive Plan, which reserves 3.5 million shares for equity-based awards. The plan includes an automatic annual increase in shares authorized for issuance starting in 2026. Furthermore, Capricor is preparing for potential FDA approval of its DMD therapy in late 2025, with plans to expand its manufacturing capabilities by mid-to-late 2026.

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