SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR) has begun the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its product candidate deramiocel, aimed at treating cardiomyopathy in Duchenne muscular dystrophy (DMD) patients. The company anticipates completing the submission process by the end of 2024 and is hopeful that the application will qualify for priority review, which could accelerate the approval timeline.
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) which have demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies. These cells secrete exosomes that target macrophages, encouraging a healing response rather than promoting inflammation. The therapy has been administered to over 200 human subjects across various trials and has been the subject of more than 100 peer-reviewed scientific publications.
DMD is a severe genetic disorder that leads to progressive muscle weakening and chronic inflammation, affecting approximately one in every 3,500 male births in the United States. The condition results from a deficiency in dystrophin, a protein that helps keep muscle cells intact, leading to cell damage and fibrosis. With no approved treatment options currently available for DMD cardiomyopathy, the potential approval of deramiocel represents a significant advancement.
Capricor's CEO, Linda Marbán, Ph.D., expressed the company's commitment to working with the FDA throughout the approval process and highlighted the importance of bringing this first-in-class treatment to patients in need. If approved, Capricor could also receive a Priority Review Voucher based on deramiocel's orphan drug and rare pediatric disease designations.
Capricor Therapeutics focuses on cell and exosome-based therapeutics for the treatment of rare diseases. Its lead product candidate, deramiocel, is in Phase 3 clinical development for DMD treatment. The company is also exploring exosome technology for potential applications in vaccinology and targeted delivery of therapeutics.
The information in this article is based on a press release statement and does not constitute an endorsement of Capricor's claims. It is intended to provide a factual report on the company's recent regulatory submission and the potential implications for DMD treatment.
In other recent news, Capricor Therapeutics has been the subject of several important updates. The company has sustained an Outperform rating from Oppenheimer ahead of an anticipated update on the HOPE-2 trial for deramiocel, a treatment for Duchenne muscular dystrophy. Maxim Group has increased Capricor's stock price target to $25.00 and maintained a Buy rating, following the company's plan to submit a Biologics License Application for deramiocel. A similar sentiment was echoed by H.C. Wainwright and Oppenheimer, who maintained their Buy ratings and set price targets at $40 and $15, respectively.
In financial news, Capricor reported a net loss of approximately $11 million for Q2 2024, while generating revenues of around $4 million. However, the company maintains a strong cash position of $29.5 million, supported by a financial agreement with Nippon Shinyaku, totaling up to $35 million.
On a regulatory front, Capricor plans to initiate a rolling Biologics License Application submission in October 2024, with the goal of securing full approval and a broader label for deramiocel in the treatment of DMD-associated cardiomyopathy.
Lastly, Capricor is preparing for potential label expansion to address DMD skeletal muscle myopathy and is in advanced partnership discussions for distribution in Europe, marking significant developments in the company's operations.
InvestingPro Insights
Capricor Therapeutics' recent initiation of the rolling submission for its Biologics License Application (BLA) for deramiocel comes amid a period of significant market performance for the company. According to InvestingPro data, CAPR has seen a remarkable 595.32% price total return over the past year, with a particularly strong 360.24% return in the last month alone. This surge in stock price may reflect investor optimism about the potential approval of deramiocel and its impact on the company's future.
Despite the positive market sentiment, InvestingPro Tips highlight some financial challenges. The company is not currently profitable, with a negative gross profit margin of -37.73% over the last twelve months as of Q2 2024. This aligns with the developmental stage of Capricor's lead product candidate and the significant investments required in the biotechnology sector.
An InvestingPro Tip notes that analysts anticipate a sales decline in the current year, which could be attributed to the company's focus on regulatory submissions rather than immediate revenue generation. However, with a market capitalization of $683.96 million, investors appear to be valuing Capricor based on its future potential rather than current financials.
For readers interested in a deeper analysis, InvestingPro offers 17 additional tips for Capricor Therapeutics, providing a more comprehensive view of the company's financial health and market position.
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