SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR) has announced positive three-year results from its HOPE-2 open label extension study for deramiocel, a treatment for Duchenne muscular dystrophy (DMD). The data, presented at the World Muscle Society Congress, indicates sustained efficacy and safety in treating DMD, particularly in stabilizing cardiac and skeletal muscle function.
The study's findings are significant, as there are currently no approved therapies specifically for DMD-related cardiomyopathy, a leading cause of death in patients with DMD. According to Dr. Craig McDonald of the University of California, Davis, the improvements in cardiac function and skeletal muscle function over the three-year period are promising and unprecedented compared to other DMD therapeutics.
Capricor's CEO, Linda Marbán, Ph.D., emphasized the potential for deramiocel to become a long-term treatment option with quarterly infusions, particularly for preserving cardiac function as new therapies emerge for skeletal muscle loss in DMD.
The HOPE-2 study demonstrated improvements in left ventricular ejection fraction and indexed volumes, which are critical for predicting long-term cardiac outcomes. Patients treated with deramiocel also showed statistically significant benefits in skeletal muscle function compared to an external comparator dataset.
Capricor is on track to submit a Biologics License Application to the FDA by the end of 2024, with the hope of fast-tracking approval for deramiocel. The therapy has received Orphan Drug Designation and is supported by the Regenerative Medicine Advanced Therapy Designation. If approved, Capricor would be eligible for a Priority Review Voucher due to its rare pediatric disease designation.
DMD is a severe genetic disorder that leads to progressive muscle weakness and chronic inflammation, affecting approximately 15,000-20,000 patients in the United States. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells, which have shown immunomodulatory, antifibrotic, and regenerative actions in clinical studies.
The company continues to explore the potential of its exosome technology platform, StealthX™, in various therapeutic areas while progressing with its clinical development for DMD. This announcement is based on a press release statement from Capricor Therapeutics.
In other recent news, Capricor Therapeutics has made significant strides in the development of deramiocel, a treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. The company has initiated a rolling Biologics License Application (BLA) submission to the FDA, with completion expected by the end of 2024. Analysts from H.C. Wainwright and Oppenheimer have maintained their Buy and Outperform ratings for Capricor, respectively, while Maxim Group has also sustained a Buy rating.
Capricor reported a net loss of approximately $11 million for Q2 2024, while generating revenues of around $4 million. Despite this, the company maintains a strong cash position of $29.5 million, supported by a financial agreement with Nippon Shinyaku, totaling up to $35 million.
In addition to these financial updates, the company is preparing for potential label expansion to address DMD skeletal muscle myopathy and is in advanced partnership discussions for distribution in Europe. These are some of the recent developments at Capricor Therapeutics.
InvestingPro Insights
Capricor Therapeutics' recent announcement of positive three-year results for its DMD treatment aligns with several key financial metrics and insights from InvestingPro. The company's stock has shown remarkable performance, with a 505.65% price total return over the past year, reflecting investor optimism about its pipeline developments, particularly deramiocel.
InvestingPro data reveals that Capricor's revenue growth stands at an impressive 187.15% for the last twelve months as of Q2 2024, indicating strong momentum in its business operations. This growth could be attributed to the company's progress in its clinical programs and the potential market for its DMD treatment.
However, it's important to note that Capricor is currently operating at a loss, with a negative gross profit margin of -37.73%. This is not uncommon for biotech companies in the development stage, as they often incur significant research and development expenses before bringing products to market.
An InvestingPro Tip highlights that analysts anticipate a sales decline in the current year, which may seem at odds with the positive clinical results. This could be due to the company's focus on research and development rather than immediate commercialization.
Another relevant InvestingPro Tip indicates that the stock price movements are quite volatile. This volatility is typical for biotech stocks, especially as they approach critical milestones like the planned Biologics License Application submission for deramiocel by the end of 2024.
For investors seeking a more comprehensive analysis, InvestingPro offers 16 additional tips for Capricor Therapeutics, providing a deeper understanding of 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.