SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), a biotech firm focused on cell and exosome-based treatments for rare diseases, today announced that the European Medicines Agency (EMA) has awarded Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to deramiocel, its primary product candidate for Duchenne muscular dystrophy (DMD). These designations could accelerate the development and potential market exclusivity in Europe for deramiocel.
The Orphan Drug designation offers Capricor benefits including a decade of market exclusivity upon approval, and significantly reduced regulatory fees. Similarly, the ATMP designation is expected to facilitate the development process for cell-based therapies, potentially hastening the time to market and providing access to crucial resources.
Capricor's CEO, Linda Marbán, Ph.D., emphasized the importance of these milestones in the global effort to provide deramiocel to DMD patients. These European designations complement the Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations previously granted by the U.S. Food and Drug Administration (FDA).
Capricor has initiated a rolling Biologics License Application (BLA) with the FDA, targeting full approval of deramiocel for DMD-cardiomyopathy, with expectations to complete the submission by year-end.
Deramiocel (CAP-1002) comprises allogeneic cardiosphere-derived cells, which have demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies. These cells release exosomes that influence macrophages to promote healing. Over 200 patients have received CDCs in various trials, and deramiocel is currently in Phase 3 development for DMD treatment.
DMD is a severe genetic disorder leading to progressive muscle weakness and chronic inflammation, with an average life expectancy of about 30 years. It affects roughly one in 3,500 male births, with an estimated 15,000-20,000 patients in the U.S. The disease is caused by a lack of functional dystrophin, a protein essential for muscle integrity, leading to cell damage and fibrosis.
This news is based on a press release from Capricor Therapeutics. The company continues to investigate its exosome technology, including the StealthX™ platform, for potential applications in vaccinology and targeted delivery of therapeutics. Capricor's partnership with Nippon Shinyaku Co., Ltd. for the commercialization of deramiocel in the U.S. and Japan is contingent on regulatory approvals. Deramiocel is not yet approved for any indications, and Capricor's exosome-based candidates have not received clinical investigation approval.
In other recent news, Capricor Therapeutics has made significant strides in the development of deramiocel, a promising treatment for cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The company has received a Buy rating from Jones Trading, highlighting the potential of this innovative therapy. Capricor Therapeutics has also successfully raised funds, amassing a cash balance of approximately $165 million to support the upcoming commercial launch and manufacturing expansion.
In addition, the company has established a partnership with Nippon Shinyaku for the commercial distribution of deramiocel in the United States, European Union, and Japan. However, the company reported a net loss of approximately $12.6 million for the third quarter of 2024. Despite this, Capricor's San Diego facility is ready for production, and plans for a new manufacturing site are in progress to meet anticipated demand.
Capricor Therapeutics is also exploring opportunities to expand the treatment's use to Becker muscular dystrophy and advancing its StealthX exosome technology for future therapeutics. The company plans to submit a Biologics License Application (BLA) to the FDA by the end of 2024. These are among the recent developments at Capricor Therapeutics.
InvestingPro Insights
Capricor Therapeutics' recent regulatory milestones in Europe align with its strong financial performance and market position. According to InvestingPro data, the company's market capitalization stands at $839.37 million, reflecting investor confidence in its potential. This valuation is particularly noteworthy given the company's revenue of $23.23 million in the last twelve months as of Q3 2024, indicating a high revenue multiple that suggests market optimism about Capricor's future prospects.
InvestingPro Tips highlight that Capricor has seen a significant return over the last week, with a 9.17% price increase. This short-term gain is part of a broader trend, as the stock has shown a remarkable 521.55% return over the past year. These figures underscore the market's positive reaction to Capricor's progress with deramiocel and its potential in the DMD treatment space.
Despite these positive indicators, it's important to note that Capricor is not yet profitable, with a negative gross profit margin of -95.25% in the last twelve months. This is typical for biotech companies in the development stage, where substantial investments in research and clinical trials often precede revenue generation. The company's strong cash position, with more cash than debt on its balance sheet, provides a financial buffer as it pursues regulatory approvals and potential commercialization of deramiocel.
For investors seeking a more comprehensive analysis, InvestingPro offers 15 additional tips for Capricor Therapeutics, providing deeper insights into the company's financial health and market position.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.