Capricor Therapeutics, Inc. today announced 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 targeting full approval based on existing cardiac data and natural history data from Phase 2 studies.
The BLA submission process began in October 2024, with the company expecting to complete it by the end of the year. Data for the application includes results from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, along with natural history data from Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center.
In preparation for potential future expansion of the indication to include the treatment of skeletal muscle myopathy in DMD, Capricor plans to combine Cohorts A and B of the ongoing Phase 3 HOPE-3 study for a post-approval study, delaying unveiling of Cohort A initially expected in the fourth quarter of 2024.
Additionally, Capricor’s exosome-based multivalent StealthX™ vaccine for the prevention of SARS-CoV-2 was selected for Project NextGen, a U.S. Department of Health and Human Services initiative to advance innovative vaccines. The company is currently manufacturing the vaccine candidate and plans to deliver it to the National Institute of Allergy and Infectious Diseases (NIAID) later this year.
Capricor also completed a private placement of common shares to Nippon Shinyaku on September 16, 2024, raising approximately €15.0 million at a premium price. Additionally, the company sold all available shares under its at-the-market program, generating gross proceeds of approximately €53.9 million.
The company estimates it has approximately €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 in accordance with U.S. Accounting Principles (GAAP) and should not be relied upon excessively.
In other recent news, Capricor Therapeutics reported significant progress in its operations. The company reaffirmed its Outperform rating from Oppenheimer, buoyed by optimism about its product, deramiocel, a treatment for Duchenne muscular dystrophy (DMD). Analysts at HC Wainwright and Maxim Group also maintained their Buy ratings for Capricor.
In financial updates, Capricor reported a net loss of approximately €11 million for the second quarter of 2024, generating revenue of approximately €4 million. Despite the loss, the company maintains a solid cash position of €29.5 million, supported by a financing 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. Finally, Capricor is preparing for potential indication expansion to address muscle myopathy skeletal in DMD and is in advanced stages of 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 biotech sector. This optimism is further supported by the stock’s impressive performance, with a total price return of 659.23% over the past year and a return of 389.8% in the past month alone.
However, investors should note that Capricor is not yet profitable, with a negative gross profit margin of -37.73% over the trailing twelve months. This aligns with InvestingPro’s suggestion that the company “suffers from weak gross profit margins.” Despite this, Capricor’s revenue growth is substantial, showing an increase of 187.15% over the past twelve months, which could be attributed to its advancing pipeline and recent financial activities.
The company’s strategic moves, including its planned BLA filing and participation in Project NextGen, are crucial as analysts expect sales to decline this year, according to another InvestingPro tip. These initiatives could help Capricor maintain its growth trajectory and potentially improve its financial position.
For investors looking for a more comprehensive analysis, InvestingPro offers 17 additional recommendations for Capricor Therapeutics, providing a deeper understanding of the company’s financial health and market position.
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