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SAN DIEGO - Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company with a market capitalization of $546 million and strong liquidity metrics according to InvestingPro data, reported four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension study of Deramiocel for Duchenne Muscular Dystrophy (DMD), showing preservation of cardiac function in treated patients.
The data, presented at the Parent Project Muscular Dystrophy 2025 Annual Conference in Las Vegas, demonstrated that patients receiving continuous treatment with Deramiocel for four years showed a median change of -0.5 points in left ventricular ejection fraction compared to baseline. The company’s strong clinical progress has contributed to its impressive 150% return over the past year, with analysts maintaining a bullish outlook and forecasting significant revenue growth of 242% for fiscal year 2025.
Patients with baseline LVEF greater than 45% showed even greater clinical benefit, according to the company. The treatment also continued to slow skeletal muscle disease progression, with patients experiencing a smaller average decline in Performance of the Upper Limb (PUL v2.0) scores in the fourth year (0.6 points) compared to the first year (1.8 points).
"Cardiomyopathy remains one of the leading causes of mortality in Duchenne and addressing this aspect of the disease is critical to improving outcomes," said Pat Furlong, Founding President and CEO of PPMD.
Deramiocel consists of allogeneic cardiosphere-derived cells that have immunomodulatory and anti-fibrotic properties. The therapy has received Orphan Drug Designation for DMD treatment from both the U.S. FDA and European Medicines Agency, along with Regenerative Medicine Advanced Therapy designation in the U.S.
The company’s Biologics License Application for Deramiocel is currently under priority review by the FDA.
DMD is a severe genetic disorder affecting approximately 15,000 individuals in the United States, primarily boys. It causes progressive muscle degeneration and leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD patients. For detailed financial analysis and additional insights about Capricor’s market position and growth potential, InvestingPro subscribers can access comprehensive research reports and 12 more exclusive ProTips about the company’s financial health and market performance.
The information in this article is based on a press release statement from Capricor Therapeutics.
In other recent news, Capricor Therapeutics announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its cell therapy candidate Deramiocel for the treatment of Becker Muscular Dystrophy. This expands the potential use of Deramiocel, which is also under priority review for Duchenne Muscular Dystrophy with a target action date of August 31, 2025. Capricor also reported the successful completion of its Pre-License Inspection for Deramiocel, noting that the FDA issued a Form 483 with several observations related to quality systems and documentation practices. The company has addressed these observations and stated that no material changes to the manufacturing process are required.
In another development, H.C. Wainwright reaffirmed its Buy rating and $77.00 price target for Capricor Therapeutics, despite the FDA withdrawing its notice for an Advisory Committee meeting on Deramiocel. The research firm maintained a positive outlook on the approval prospects of Deramiocel, attributing the FDA’s action to administrative reasons. Additionally, Capricor disclosed that its stockholders approved the adoption of the Capricor Therapeutics 2025 Equity Incentive Plan, which reserves 3.5 million shares for equity-based awards. The plan will see an annual increase in shares starting in 2026.
These recent developments reflect Capricor’s ongoing efforts in advancing its therapeutic candidates and managing corporate governance. The company’s Biologics License Application for Deramiocel remains under priority review, with a mid-cycle review completed without significant issues. Capricor’s actions and regulatory progress continue to be closely monitored by investors and analysts alike.
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