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13 May, 2025
Capricor Therapeutics, a biotechnology company focused on developing transformative cell and exosome-based therapies for rare diseases, has announced its financial results for the first quarter ending March 31, 2025, along with an update on its corporate developments.
In a statement, Linda Marbán, Ph.D., Capricor’s Chief Executive Officer, highlighted the company's progress in 2025. She emphasized their commitment to delivering the first approved therapy for Duchenne cardiomyopathy, a condition that currently has no approved treatments. Marbán shared that Capricor remains in active discussions with the FDA as it reviews the company's Biologics License Application (BLA) and is on track for the Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. Preparations for an FDA advisory committee meeting, pre-approval inspection, and potential commercial launch are progressing. Additionally, the StealthX™ vaccine program is advancing towards a Phase 1 clinical trial initiation in the third quarter of 2025, led by the National Institute of Allergy and Infectious Diseases (NIAID), pending regulatory clearance. Capricor ended the first quarter with a strong cash position of approximately $145 million, which will support its continued strategic execution as it approaches critical milestones.
First Quarter 2025 and Recent Developments
In March 2025, the FDA accepted Capricor’s BLA for full approval of deramiocel for the treatment of individuals with DMD-cardiomyopathy. The application has been granted priority review, with a PDUFA action date of August 31, 2025. In May 2025, Capricor completed a mid-cycle review meeting with the FDA, where no significant deficiencies were identified, and the FDA confirmed plans to convene an advisory committee meeting. The BLA is supported by data from Capricor’s Phase 2 HOPE-2 trial and its open-label extension (OLE) trial, which demonstrated significant improvements in cardiac function for up to three years post-treatment and a consistent safety profile. Notably, the ongoing HOPE-3 Phase 3 trial, which assesses skeletal muscle function, is not part of the BLA for full approval of deramiocel.
Capricor expanded its internal manufacturing capacity in the first quarter of 2025 by amending its lease to secure additional GMP manufacturing space at its San Diego, California headquarters, to support increased commercial manufacturing needs.
The company also announced that the NIAID intends to initiate a Phase 1 clinical trial of its StealthX™ exosome-based vaccine in Q3 2025, pending regulatory approval. The vaccine was selected for participation in Project NextGen, a U.S. Department of Health and Human Services initiative focused on advancing innovative vaccines for future pandemics.
Furthermore, Michael Binks, M.D., was appointed as Capricor’s Chief Medical Officer. Dr. Binks brings over 25 years of global clinical development and translational research experience, having held senior leadership roles at Pfizer and GlaxoSmithKline in various therapeutic areas, including immunology, neurology, cardiology, nephrology, and hematology.
First Quarter 2025 Financial Results
Cash Position: As of March 31, 2025, Capricor had approximately $144.8 million in cash, cash equivalents, and marketable securities, down from $151.5 million as of December 31, 2024. In January 2025, the company received $10 million from the second development milestone payment under its U.S. Commercialization and Distribution Agreement with Nippon Shinyaku.
Revenues: Capricor reported no revenues for the first quarter of 2025, compared to $4.9 million for the same period in 2024. The revenue in the prior year was mainly from the ratable recognition of the $40 million upfront and first development milestone payments, as well as the $10 million second milestone payment, all of which were fully recognized by December 31, 2024.
Costs and Expenses: The company incurred total operating expenses of approximately $25.0 million in the first quarter of 2025, up from $15.2 million in Q1 2024.
Net Loss: Capricor reported a net loss of approximately $24.4 million, or $0.53 per share, for the first quarter of 2025, compared to a net loss of $9.8 million, or $0.31 per share, for the same period in 2024.
Financial Outlook: Capricor believes that its available cash, cash equivalents, and marketable securities will be sufficient to cover anticipated expenses and capital requirements through 2027, excluding any additional potential milestone payments or strategic capital uses not currently factored into the company’s base-case planning assumptions.
Upcoming Events
Capricor is scheduled to participate in the following upcoming events:
H.C. Wainwright 3rd Annual BioConnect Investor Conference, May 20, 2025, New York, NY
BIO International Convention, June 16-19, 2025, Boston, MA
Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference, June 19-21, 2025, Las Vegas, NV
Conference Call and Webcast
To participate in the conference call, interested parties can dial 1-800-717-1738 (Domestic) or 1-646-307-1865 (International) and reference the conference ID: 73741. A webcast will also be available on the company’s website, and a replay of the webcast will be accessible following the live broadcast.
About Duchenne Muscular Dystrophy (DMD)
DMD is a severe genetic disorder characterized by progressive muscle weakness and chronic inflammation, leading to heart and respiratory muscle failure, with mortality typically around 30 years of age. It is estimated to affect approximately 1 in 3,500 male births, with a patient population of about 15,000-20,000 in the United States. DMD is caused by the absence of functional dystrophin, which leads to muscle cell damage and eventually muscle cell death, particularly in the heart, resulting in cardiomyopathy, the leading cause of death in DMD patients.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), which have demonstrated potent immunomodulatory and anti-fibrotic effects in preclinical and clinical studies, particularly in the context of dystrophiopathies such as DMD. CDCs work by secreting exosomes that target macrophages, prompting them to adopt a healing rather than a pro-inflammatory phenotype. CDCs have been studied in over 250 peer-reviewed scientific publications and have been administered to more than 250 human subjects across multiple clinical trials.
Deramiocel has received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA). It is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and ATMP (Advanced Therapy Medicinal Product) Designation in the European Union. If approved by the FDA for the treatment of DMD, Capricor could be eligible to receive a Priority Review Voucher based on its prior receipt of rare pediatric disease designation.