On Wednesday, H.C. Wainwright reaffirmed its Buy rating and $40.00 price target for Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), following the company's announcement on September 24 regarding its regulatory strategy for deramiocel, which is aimed at treating all patients diagnosed with Duchenne muscular dystrophy (DMD)-cardiomyopathy. The firm is poised to submit a Biologics License Application (BLA) for full approval based on existing cardiac and natural history outcomes data.
Capricor plans to initiate a rolling BLA submission in October 2024, with the goal of securing full approval and a broader label for deramiocel in the treatment of DMD-cardiomyopathy. The final submission is projected to be completed by the end of 2024. The BLA is supported by cardiac data from the Phase 2 HOPE-2 and HOPE-2 OLE studies, which are benchmarked against natural history data from Vanderbilt University Medical Center and Cincinnati Children's Hospital Medical Center.
The treatment has shown to improve various measures of cardiovascular function in DMD patients compared to established natural history comparators. This progress is significant as it could pave the way for deramiocel to reach the market more swiftly, leveraging the Regenerative Medicine Advanced Therapy (RMAT) designation and the potential revenue from a Priority Review Voucher (PRV) sale.
Additionally, Capricor is preparing for potential label expansion to address DMD skeletal muscle myopathy. The company intends to combine Cohorts A and B from the Phase 3 HOPE-3 clinical trial to act as a post-approval study. It has also decided not to unblind Cohort A as previously expected in the fourth quarter of 2024, which is viewed as a strategic move to de-risk and expedite the BLA submission process.
H.C. Wainwright expressed a positive outlook on Capricor's strategic shift, emphasizing the potential for deramiocel to reach the market sooner and the company's continued efforts to explore treatments for skeletal muscle myopathy. The firm's reiterated Buy rating and price target reflect confidence in Capricor's regulatory strategy and its implications for the treatment of DMD-cardiomyopathy.
In other recent news, Capricor Therapeutics, a biotech firm, recently announced plans to submit a Biologics License Application (BLA) for deramiocel, a treatment for Duchenne muscular dystrophy (DMD). The firm has also reported a net loss of approximately $11 million for Q2 2024, while generating revenue of around $4 million. Despite this, Capricor maintains a strong cash position of $29.5 million, supported by a financial agreement with Nippon Shinyaku, totaling up to $35 million.
In addition, Capricor has maintained its Buy rating from H.C. Wainwright and had its price target upgraded to $15 by Oppenheimer. These recent developments highlight the company's ongoing progress in the biopharmaceutical sector and the potential impact of its product candidate, deramiocel. The BLA submission process for deramiocel, a potential treatment for DMD, is expected to begin in October 2024 and complete by the end of the year.
The company is also preparing for a potential commercial launch of deramiocel and is in advanced partnership discussions for distribution in Europe.
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