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SAN DIEGO - Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company with a market capitalization of $566 million and strong liquidity metrics, announced Tuesday 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 (BMD). According to InvestingPro data, the company maintains a healthy current ratio of 6.55, indicating robust short-term financial stability.
The designation expands Capricor’s focus beyond Duchenne Muscular Dystrophy (DMD), for which Deramiocel is currently under priority review with a PDUFA target action date of August 31, 2025.
BMD, like DMD, is a progressive X-linked neuromuscular disorder causing skeletal and cardiac muscle deterioration. It affects approximately 5,000 individuals in the United States, presenting later in life with slower progression than DMD.
"The granting of Orphan Drug Designation for Becker Muscular Dystrophy is a significant milestone that expands the potential reach of Deramiocel," said Linda Marbán, CEO of Capricor Therapeutics. The company’s growth prospects look promising, with InvestingPro analysts forecasting revenue growth of 242% for fiscal year 2025, while the stock has delivered an impressive 149.6% return over the past year.
Deramiocel consists of allogeneic cardiosphere-derived cells that have shown immunomodulatory and anti-fibrotic properties in preserving cardiac and skeletal muscle function. The therapy has been administered to over 250 human subjects across multiple clinical trials.
The company also reported successful completion of its Pre-License Inspection, a regulatory requirement for the approval of its Biologics License Application for DMD.
Deramiocel has previously received Orphan Drug Designation for DMD from both the FDA and European Medicines Agency, along with Regenerative Medicine Advanced Therapy designation in the U.S. and Advanced Therapy Medicinal Product designation in Europe.
The information in this article is based on a press release statement from Capricor Therapeutics. For comprehensive analysis and additional insights, including 13 more exclusive ProTips and detailed financial metrics, visit InvestingPro, where you can access the full Pro Research Report on Capricor Therapeutics, one of over 1,400 detailed company analyses available to subscribers.
In other recent news, Capricor Therapeutics has been in the spotlight following several key developments. The company announced that the FDA completed its Pre-License Inspection of its San Diego facility for Deramiocel, its lead therapy candidate for Duchenne Muscular Dystrophy. The inspection resulted in a Form 483 with observations primarily related to quality systems and documentation, but Capricor has stated that no material changes to the manufacturing process are required. In addition, Capricor’s Biologics License Application for Deramiocel remains under priority review with an action date set for August 31, 2025.
H.C. Wainwright has reaffirmed its Buy rating on Capricor, maintaining a price target of $77, despite the FDA’s withdrawal of a notice for an Advisory Committee meeting for Deramiocel. The firm views the market’s reaction to this regulatory uncertainty as an overreaction. Meanwhile, Roth/MKM initiated coverage of Capricor with a Buy rating and a $31 price target, citing the potential of Deramiocel to significantly impact the market for DMD treatments.
Furthermore, Capricor disclosed the adoption of its 2025 Equity Incentive Plan, which reserves 3.5 million shares for equity-based awards. This plan was approved by stockholders during the company’s Annual Meeting. These developments indicate Capricor’s strategic movements as it progresses toward the potential commercialization of Deramiocel.
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