Capricor Therapeutics (NASDAQ:CAPR), Inc. announced today its plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration for deramiocel, a treatment for cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The company is aiming for full approval based on existing cardiac data and natural history data from Phase 2 trials.
The BLA submission process began in October 2024, with the company expecting to complete it by the end of the year. The data for the application includes results from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, alongside natural history data from Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center.
In preparation for potential future label expansion to include DMD skeletal muscle myopathy treatment, Capricor plans to merge Cohorts A and B from the ongoing Phase 3 HOPE-3 trial for a post-approval study, delaying the unblinding of Cohort A which was initially scheduled for the fourth quarter of 2024.
Additionally, Capricor’s StealthX™ exosome-based multivalent vaccine for SARS-CoV-2 prevention has been selected for Project NextGen, a U.S. Department of Health and Human Services initiative to foster innovative vaccines. The company is currently manufacturing the vaccine candidate and intends to deliver it to the National Institute of Allergy and Infectious Diseases (NIAID) by year-end.
Capricor has also completed a private placement of common stock to Nippon Shinyaku on September 16, 2024, raising approximately $15.0 million at a premium price. Furthermore, the company has sold all available shares under its at-the-market program, generating gross proceeds of approximately $53.9 million.
The company estimates it had around $85.0 million in cash, cash equivalents, and short-term investments as of September 30, 2024. This figure is a preliminary estimate and may differ from the final financial position. The estimate is not a substitute for financial statements prepared according to U.S. GAAP and should not be unduly relied upon.
In other recent news, Capricor Therapeutics has reported significant progress in its operations. The company has reaffirmed its Outperform rating by Oppenheimer, buoyed by optimism surrounding its product, deramiocel, a treatment for Duchenne muscular dystrophy (DMD). Analysts from H.C. Wainwright and Maxim (NASDAQ:MXIM) Group have also maintained their Buy ratings for Capricor.
In financial updates, Capricor reported a net loss of approximately $11 million for Q2 2024, while generating revenues of around $4 million. Despite the loss, the company maintains a strong cash position of $29.5 million, supported by a financial agreement with Nippon Shinyaku, totaling up to $35 million. The company has initiated a rolling Biologics License Application (BLA) submission to the FDA for deramiocel, with completion expected by the end of 2024. Lastly, Capricor is preparing for potential label expansion to address DMD skeletal muscle myopathy and is in advanced partnership discussions for distribution in Europe.
InvestingPro Insights
Capricor Therapeutics' recent announcements align with several key financial metrics and trends highlighted by InvestingPro. The company's market capitalization stands at $720.18 million, reflecting investor optimism about its potential in the biotechnology sector. This optimism is further supported by the stock's impressive performance, with a 659.23% price total return over the past year and a 389.8% return in the last month alone.
However, investors should note that Capricor is not yet profitable, with a negative gross profit margin of -37.73% in the last twelve months. This aligns with the InvestingPro Tip that the company "suffers from weak gross profit margins." Despite this, Capricor's revenue growth is substantial, showing a 187.15% increase in the last twelve months, which could be attributed to its advancing pipeline and recent financial activities.
The company's strategic moves, including the planned BLA submission and participation in Project NextGen, are crucial given that analysts anticipate a sales decline in the current year, according to another InvestingPro Tip. These initiatives may help Capricor maintain its growth trajectory and potentially improve its financial position.
For investors seeking a more comprehensive analysis, InvestingPro offers 17 additional tips for Capricor Therapeutics, providing a deeper understanding of the company's financial health and market position.
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