SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), a biotech firm focusing on cell and exosome-based therapies for rare diseases, announced plans to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for its lead product candidate, deramiocel, to treat cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The company expects to begin the rolling submission process in October 2024 and complete it by the end of the year.
The decision to proceed with the BLA filing follows recent discussions with the FDA and is supported by cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, as well as natural history data from Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center. Deramiocel has been granted FDA Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation, which may expedite its regulatory review process.
Capricor's CEO, Linda Marbán, Ph.D., emphasized the robust cardiac data presented to the FDA and the lack of approved therapies for DMD cardiomyopathy, which is the leading cause of death in patients with Duchenne. She expressed gratitude to the patients, their families, and the FDA for their efforts to advance treatments for this rare disease.
Deramiocel, also known as CAP-1002, consists of allogeneic cardiosphere-derived cells that have shown promise in preclinical and clinical studies for their immunomodulatory, antifibrotic, and regenerative effects in dystrophinopathies and heart failure. Over 200 human subjects have received CDCs in various trials, with over 100 peer-reviewed scientific publications supporting their use.
DMD is a genetic disorder that results in progressive muscle weakness and chronic inflammation, leading to mortality at a median age of around 30 years. It is estimated to affect one in every 3,500 male births in the U.S., with a patient population of approximately 15,000-20,000.
Capricor has established internal GMP manufacturing capabilities to support the anticipated BLA submission and potential commercialization of deramiocel. Furthermore, the company is planning a post-approval study for label expansion to treat DMD skeletal muscle myopathy, combining Cohorts A and B of the Phase 3 HOPE-3 clinical trial without unblinding Cohort A as previously expected.
The company's updates were discussed in a webcast held today at 8:30 a.m. ET. This article is based on a press release statement from Capricor Therapeutics.
In other recent news, Capricor Therapeutics has been making significant strides in the biopharmaceutical sector. The company recently 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. The firm's primary focus is the development of deramiocel, a potential treatment for Duchenne muscular dystrophy. Capricor has secured a significant financial agreement with Nippon Shinyaku, totaling up to $35 million, which includes a $15 million equity investment and an upfront payment of $20 million. This funding is anticipated to support the development and global distribution of deramiocel.
The company is also preparing for a potential commercial launch of deramiocel and is in advanced partnership discussions for distribution in Europe. In addition, Capricor Therapeutics has maintained its Buy rating from H.C. Wainwright and has seen its price target upgraded to $15 by Oppenheimer. The firm's positive stance comes ahead of Capricor's anticipated regulatory update on deramiocel. This extended partnership with Nippon Shinyaku is expected to be finalized in the fourth quarter of 2024 and is seen as a stepping stone for potential future global outreach.
These recent developments highlight Capricor's ongoing progress in the biopharmaceutical sector and the potential impact of its product candidate, deramiocel. The optimism is further bolstered by Capricor's expanded partnership with Nippon Shinyaku, now encompassing the U.S., EU, and Japan.
InvestingPro Insights
As Capricor Therapeutics (NASDAQ: CAPR) forges ahead with plans to file a Biologics License Application for deramiocel, investors are keenly observing the company's financial health and stock performance. According to InvestingPro data, Capricor boasts a market capitalization of approximately $210.96 million. Despite the company's significant revenue growth of 187.15% over the last twelve months as of Q2 2024, it faces challenges with a negative gross profit margin of -37.73% and an operating income margin of -110.5% in the same period. This indicates substantial costs relative to revenue, which could impact the company's financial sustainability in the long term.
InvestingPro Tips suggest that while Capricor holds more cash than debt, which is a positive sign of liquidity, the company is quickly burning through cash. This rapid cash burn could be a concern for investors as the company prepares for potential commercialization of its lead product. Furthermore, Capricor's stock has experienced significant volatility, with a price/book multiple of 18.34, indicating that the stock may be trading at a premium compared to its book value. This is further supported by the fact that analysts do not anticipate the company will be profitable this year, and net income is expected to drop.
For those interested in the company's stock performance, Capricor has seen a strong return over the last week, month, and three months, with price total returns of 17.52%, 30.92%, and 24.63%, respectively. However, the stock's performance over the last six months shows a decline of 6.43%, reflecting the stock's volatility. These insights are particularly relevant for investors considering Capricor's stock as the company navigates the critical phase of seeking FDA approval for deramiocel.
For a deeper dive into Capricor Therapeutics' financials and stock analysis, investors can access additional InvestingPro Tips at https://www.investing.com/pro/CAPR, which currently lists a total of 15 tips to help inform investment decisions.
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