Capricor Therapeutics Advances in DMD Treatment with FDA BLA

Capricor Therapeutics Takes Significant Step with FDA Submission

-If approved, deramiocel would be the first therapy for Duchenne muscular dystrophy cardiomyopathy-

-BLA submission triggers a $10 million milestone payment to Capricor from Nippon Shinyaku-

Capricor Therapeutics (NASDAQ: CAPR), a pioneering biotechnology company originating from an innovative background in cell and exosome-based therapeutics, has recently completed a major milestone by submitting a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). This submission seeks full approval for deramiocel, an investigational therapy designed to treat patients suffering from Duchenne muscular dystrophy (DMD) cardiomyopathy.

Impact of the BLA Submission

Linda Marbán, Ph.D., the CEO of Capricor, emphasized the importance of this application: "The submission of the BLA marks a pivotal moment for Capricor and the families impacted by DMD. This is the result of extensive research and dedication to providing a transformative therapy for those in need." With multiple clinical trials demonstrating deramiocel's ability to potentially mitigate cardiac concerns related to DMD, the company looks forward to collaborating closely with the FDA during the review process.

The Regulatory Pathway and Milestone Payments

The completion of the rolling BLA was anticipated and confirmed as per the company's previous statements made in late December. Capricor has backed this application with data from the Phase 2 HOPE-2 trial and its Open Label Extension (OLE) trials. This data is benchmarked against historical datasets funded and published by the FDA that outline the cardiac implications of DMD and its disease progression biomarkers.

In addition to the milestone of submitting the BLA, Capricor will also receive a payment of $10 million from Nippon Shinyaku Co., Ltd. as part of their collaboration for commercialization in the United States.

Designations Holding Hope for Patients

Deramiocel comes with significant designations, having obtained the Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA). The regulatory journey is further supported by the Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., alongside the Advanced Therapy Medicinal Product (ATMP) designation in Europe. Should deramiocel secure FDA approval, Capricor may be eligible for a Priority Review Voucher due to its previous rare pediatric disease designation.

Understanding Deramiocel and Its Mechanism

Deramiocel, scientifically referenced as CAP-1002, utilizes allogeneic cardiosphere-derived cells (CDCs). These specialized stromal cells have garnered attention due to their immunomodulatory and regenerative properties observed in rigorous clinical and preclinical studies. The mode of action revolves around the secretion of exosomes by CDCs, which target macrophages and encourage a switch to a healing state rather than a pro-inflammatory one.

This therapy has significant implications as CDCs have already been part of over 100 peer-reviewed publications and have been administered to more than 200 human subjects across numerous clinical trials, illustrating its potential and the wide array of studies backing its efficacy.

Addressing Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe genetic ailment linked to progressive skeletal muscle weakness and severe inflammation affecting the skeletal, cardiac, and respiratory functions. Individuals diagnosed with DMD have a challenging prognosis and often face mortality by their thirties. It is a rare condition, affecting approximately one in 3,500 male births, accumulating around 15,000-20,000 cases in the U.S.

Capricor Therapeutics: A Focus on Innovation

Capricor Therapeutics, Inc. (NASDAQ: CAPR), stands at the forefront of biotechnology with a mission focused on advancing innovative cell and exosome-based therapeutics, redefining the treatment landscape for rare diseases. The firm’s flagship product candidate, deramiocel, exemplifies their commitment to addressing urgent medical needs, particularly in the area of DMD. Beyond deramiocel, Capricor is exploring its proprietary StealthX™ platform, targeting various therapeutic applications including vaccinology and targeted drug delivery, widening their scope for innovation.

Staying Connected

For any inquiries or additional information, individuals can connect with Capricor through social media platforms like Facebook, Instagram, and Twitter, or visit the official Capricor website. Additionally, key contacts for media relations and company inquiries are also accessible for more insights.

Frequently Asked Questions

What is the significance of the BLA submission for Capricor?

The BLA submission is a major milestone in seeking approval for deramiocel, an important therapeutic option for patients with DMD.

How does deramiocel work?

Deramiocel utilizes cardiosphere-derived cells that release exosomes facilitating healing by modulating macrophage activity.

What are the designations received by deramiocel?

Deramiocel has received Orphan Drug Designation and RMAT designation, which expedite its review and approval process.

What other areas is Capricor exploring?

Capricor is also working on its StealthX™ platform for targeted delivery and diverse therapeutic applications.

How can I stay updated with Capricor Therapeutics?

Stay connected with Capricor via their website and social media channels for the latest updates and information.

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