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Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular Dystrophy with an Aim to Expedite BLA Pathway

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Capricor Therapeutics (NASDAQ: CAPR) announced a successful Type-B meeting with the FDA for their lead asset, CAP-1002, aimed at treating Duchenne muscular dystrophy (DMD). The FDA approved a rolling Biologics License Application (BLA) submission, set to begin in Q3 2024. The rolling BLA process could streamline regulatory review and reduce risks. The company also received positive clinical results from their HOPE-2 and HOPE-2 OLE studies, with additional data pending from the HOPE-3 trial. The WHO selected 'Deramiocel' as the International Nonproprietary Name for CAP-1002. Capricor could also receive a Priority Review Voucher following FDA approval due to its rare pediatric disease designation. This regulatory progress signifies pivotal steps toward the potential commercialization of CAP-1002.

Positive
  • Successful Type-B meeting with FDA for CAP-1002.
  • FDA approved rolling BLA submission starting Q3 2024.
  • Positive outcomes from HOPE-2 and HOPE-2 OLE studies.
  • WHO selected 'Deramiocel' as INN for CAP-1002.
  • Potential Priority Review Voucher (PRV) upon FDA approval.
  • Orphan Drug Designation and RMAT support for CAP-1002.
  • Plans to present additional data at PPMD conference.
Negative
  • Final HOPE-3 trial data awaited until Q4 2024, causing potential delays in decision-making.
  • Pending commercialization steps may require additional financial resources.
  • Ongoing regulatory processes create uncertainty until final approval.

The recent announcement from Capricor Therapeutics regarding their meeting with the FDA and the progression of their CAP-1002 program for Duchenne Muscular Dystrophy (DMD) is highly significant. The agreement to commence a rolling BLA submission is a positive development as it can expedite the review process. This step is particularly important for rare diseases like DMD, where timely access to new therapies can significantly impact patient outcomes. The HOPE-2 and HOPE-2 OLE studies have already demonstrated encouraging results and the upcoming data from HOPE-3 will likely further solidify the case for CAP-1002's efficacy and safety. Importantly, the designation of Deramiocel as the INN and the potential to receive a Priority Review Voucher add additional layers of validation and possible financial incentives for the company.

Rating: 1

From a financial perspective, the news of Capricor Therapeutics moving towards a rolling BLA submission for CAP-1002 is a notable event. This pathway can reduce the time to market, potentially leading to earlier revenue generation. The Orphan Drug and RMAT designations can provide market exclusivity and additional regulatory support, which are critical for a biotech company focusing on rare diseases. Furthermore, the potential acquisition of a Priority Review Voucher can be a substantial financial asset, as these vouchers can either be used to expedite future drug reviews or sold to other companies, often fetching a significant price. These elements combined could lead to a positive reevaluation of the company's valuation, driving investor interest and potentially increasing the stock price.

Rating: 1

The progression of Capricor Therapeutics towards potential approval of CAP-1002 for DMD represents a strategic move in a niche, high-impact market. Duchenne Muscular Dystrophy is a rare and severe disease with limited treatment options, making any advancements in this area highly impactful. The company's ability to secure significant regulatory designations such as Orphan Drug and RMAT further positions it competitively within the biotech sector. The upcoming data presentations and manufacturing preparations indicate a well-structured pathway towards commercialization. Investors should note the strategic importance of these developments in capturing market share in the rare disease therapeutic space and the potential long-term benefits this could bring to Capricor's market position.

Rating: 1

--FDA Grants Pre-BLA Meeting Request and Rolling BLA Submission after Review of HOPE-2 and HOPE-2 OLE 3-Year Results--

SAN DIEGO, June 11, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the successful completion of a Type-B meeting with the U.S. Food and Drug Administration (FDA) on next steps for the Biologics License Application (BLA) submission with its lead asset, CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Additionally, Deramiocel has been selected as the proposed International Nonproprietary Name (INN) for CAP-1002 by the World Health Organization.

“The last year has been transformative for Capricor as we have moved rapidly towards potential approval for CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “The most recent meeting had several very important outcomes; the first of which was that the FDA has agreed to a pre-BLA meeting based on their review of our clinical data from HOPE-2, HOPE-2 open label extension (OLE) and available data from HOPE-3. This key agreement will allow us to begin our BLA filing with a rolling submission planned to initiate in the third quarter of 2024.”

The rolling submission process allows for the submission of pre-agreed components of the BLA to be submitted as they are completed, which can streamline the regulatory review process and de-risk the BLA. Capricor plans to further outline the submission schedule with the FDA in the coming months.

Dr. Marbán continued, “We continue to make significant progress on this program as we are awaiting final data in the fourth quarter of 2024 from our already enrolled HOPE-3 pivotal trial and prepare for potential commercialization, including completion of our commercial manufacturing facility and actively preparing for our pre-BLA meeting and rolling BLA submission. Furthermore, we are looking forward to our presentation of additional 3-year data from our HOPE-2 OLE study at the upcoming PPMD conference which we believe will add further evidence of the efficacy and safety of CAP-1002.”

Deramiocel (CAP-1002) for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for this drug is supported by RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for Deramiocel for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation. Capricor retains full rights to the PRV, if received.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, Deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown Deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on FacebookInstagram and Twitter.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as filed with the Securities and Exchange Commission on May 14, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel (CAP-1002) is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755


FAQ

What recent decision did the FDA make regarding CAPR's CAP-1002?

The FDA has agreed to a rolling BLA submission for CAP-1002 starting in Q3 2024.

What are the key clinical trials associated with CAP-1002?

HOPE-2, HOPE-2 OLE, and HOPE-3 are the key clinical trials associated with CAP-1002.

What is the significance of the rolling BLA submission for CAP-1002?

The rolling BLA submission allows for parts of the application to be submitted as completed, potentially streamlining the review process.

What is the new INN for CAP-1002?

The WHO has selected 'Deramiocel' as the International Nonproprietary Name for CAP-1002.

When will Capricor submit the rolling BLA for CAP-1002?

Capricor plans to initiate the rolling BLA submission in the third quarter of 2024.

What designations have been granted to CAP-1002?

CAP-1002 has received Orphan Drug Designation and RMAT designation.

What could Capricor receive if the FDA approves CAP-1002?

Capricor could receive a Priority Review Voucher (PRV) upon FDA approval of CAP-1002.

Capricor Therapeutics, Inc.

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About CAPR

capricor therapeutics, inc. (nasdaq: capr) is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. our innovative technology builds upon a large body of scientific research and enables us to approach the treatment of diseases in novel ways. capricor’s lead candidate, cap-1002, is an “off-the-shelf” cardiac cell therapy that is currently in clinical development for the treatment of duchenne muscular dystrophy (dmd). cap-1002 consists of allogeneic cardiosphere-derived cells, or cdcs, a unique population of cells which include progenitor cells that have been shown to exert potent immunomodulatory activity, which alters the immune system’s activity to stimulate cellular regeneration. cdcs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials. capricor has