Capricor’s DMD therapy progresses without FDA advisory committee

SAN DIEGO - Capricor Therapeutics (NASDAQ:CAPR), currently trading at $7.68 and identified as undervalued by InvestingPro analysis, announced Tuesday that the U.S. Food and Drug Administration will not require an Advisory Committee meeting for its Duchenne muscular dystrophy (DMD) therapy candidate Deramiocel.

The company’s Biologics License Application (BLA) for Deramiocel remains under Priority Review with a target action date of August 31, 2025. An in-person late-cycle review meeting with the FDA is scheduled for mid-July. According to InvestingPro data, Capricor maintains a strong financial position with more cash than debt and a healthy current ratio of 6.55x, providing adequate resources to support its regulatory initiatives.

Deramiocel is being developed to treat DMD-associated cardiomyopathy, a condition that leads to heart failure and is the leading cause of death in DMD patients. The therapy consists of allogeneic cardiosphere-derived cells that have shown immunomodulatory and anti-fibrotic properties in clinical studies.

"To date, all regulatory milestones have proceeded as expected, including a successful pre-license inspection and a mid-cycle review with no major issues," said Linda Marbán, Chief Executive Officer of Capricor, according to the press release.

The company also reported that four-year data from its HOPE-2 Open-Label Extension study, presented at the 2025 Parent Project Muscular Dystrophy Conference, demonstrated sustained cardiac function in patients receiving long-term Deramiocel treatment.

DMD is a rare genetic disorder affecting approximately 15,000 individuals in the United States, primarily boys. The disease causes progressive muscle degeneration due to the absence of dystrophin, a key structural protein in muscle cells.

Deramiocel has received Orphan Drug Designation for DMD treatment from both U.S. and European regulators, along with Regenerative Medicine Advanced Therapy designation in the U.S.

The information in this article is based on a press release statement from Capricor Therapeutics. Looking ahead, analysts expect both sales and net income growth for the company in the current year, with revenue projected to grow by 142%. For deeper insights into Capricor’s financial health and growth prospects, including 13 additional ProTips and comprehensive analysis, visit InvestingPro.

In other recent news, Capricor Therapeutics reported significant developments regarding its cell therapy candidate, Deramiocel. The company announced four-year safety and efficacy results from its HOPE-2 Open-Label Extension study, showing preservation of cardiac function in patients with Duchenne Muscular Dystrophy (DMD). This data was presented at the Parent Project Muscular Dystrophy 2025 Annual Conference, highlighting the treatment’s ability to slow skeletal muscle disease progression. Additionally, the U.S. FDA granted Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, expanding Capricor’s focus beyond DMD.

In related regulatory news, the FDA’s recent personnel changes have impacted Capricor, with key officials overseeing cell and gene therapies placed on administrative leave. This development has raised concerns about the continuity of regulatory service. Despite these changes, H.C. Wainwright reaffirmed its Buy rating on Capricor, emphasizing that the company’s drug candidate remains promising. In contrast, Oppenheimer lowered its price target for the company due to uncertainties surrounding the FDA review process for Deramiocel. The investment firm does not expect the drug to receive approval in its current review cycle, suggesting potential regulatory hurdles ahead.

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