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SAN DIEGO - Capricor Therapeutics (NASDAQ:CAPR), currently trading at $11.40 with a market capitalization of $521 million, announced Friday it received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration regarding its Biologics License Application for Deramiocel, its cell therapy candidate for treating cardiomyopathy associated with Duchenne muscular dystrophy. According to InvestingPro analysis, the company appears undervalued based on its Fair Value estimates.
In the CRL, the FDA stated it could not approve the application in its current form, citing insufficient evidence of effectiveness and the need for additional clinical data. The agency also noted outstanding items in the Chemistry, Manufacturing, and Controls section, which Capricor believes it had addressed in previous communications. Despite this setback, InvestingPro data shows the company maintains strong liquidity with a current ratio of 6.55, and analysts expect both revenue and net income growth in the current year.
Capricor plans to resubmit its application in the third quarter of 2025, incorporating data from its ongoing Phase 3 HOPE-3 clinical trial. This randomized, double-blind, placebo-controlled study involves 104 patients, with topline results expected in the same quarter.
"We are surprised by this decision by the FDA. We have followed their guidance throughout the process," said Linda Marbán, CEO of Capricor, in the press release. The company noted that prior to the CRL, the review had progressed without major issues, including a successful pre-licensure inspection.
The FDA has offered Capricor the opportunity to request a Type A meeting to discuss next steps toward potential approval and confirmed it will restart the review clock upon resubmission.
Deramiocel has received Orphan Drug Designation for DMD treatment from both the FDA and European Medicines Agency, along with Regenerative Medicine Advanced Therapy designation in the U.S.
Duchenne muscular dystrophy is a severe genetic disorder affecting approximately 15,000 individuals in the United States, primarily boys. The condition causes progressive muscle degeneration, with cardiomyopathy and heart failure being the leading causes of death. With the next earnings report due on August 7, 2025, investors can access comprehensive analysis and 12+ additional ProTips through InvestingPro’s detailed research reports.
In other recent news, Capricor Therapeutics is navigating significant developments concerning its Duchenne muscular dystrophy (DMD) therapy, Deramiocel. The company’s Biologics License Application for Deramiocel is under Priority Review by the FDA, with a target action date set for August 31, 2025. Notably, the FDA will not require an Advisory Committee meeting for this therapy candidate, which aims to treat DMD-associated cardiomyopathy. B.Riley has initiated coverage on Capricor Therapeutics with a buy rating, highlighting the potential of Deramiocel to become a leading therapy for DMD-cardiomyopathy, projecting favorable commercial prospects post-approval. Meanwhile, H.C. Wainwright has maintained its Buy rating, advising investors to avoid overreacting to recent FDA personnel changes, which they believe reflect broader regulatory shifts. Conversely, Oppenheimer has lowered its price target for Capricor from $43.00 to $22.00 due to concerns about the current FDA review cycle, though it maintains an Outperform rating. The recent administrative leave of key FDA officials, including those overseeing Capricor’s application, has raised concerns about potential regulatory unpredictability. Despite these challenges, Capricor continues to report positive clinical data, with four-year results from the HOPE-2 Open-Label Extension study showing sustained cardiac function in patients treated with Deramiocel.
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