Capricor Therapeutics Announces Upcoming Type-B Meeting with the FDA to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy
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Insights
The scheduled Type-B meeting between Capricor Therapeutics and the FDA represents a pivotal step for the company, specifically regarding their cell and exosome-based therapeutic, CAP-1002, aimed at treating Duchenne muscular dystrophy (DMD). The focus on chemistry, manufacturing and controls (CMC) is a critical aspect of the drug development process, as it pertains to the quality and consistency of the product. Successful discussions could significantly streamline the path to a Biologics License Application (BLA), which is the FDA's authorization for a biologic product to be marketed in the U.S.
From a business perspective, the RMAT and orphan drug designations previously granted to CAP-1002 could offer Capricor advantages such as tax credits, grant funding and potentially seven years of market exclusivity upon approval. These incentives are designed to encourage the development of treatments for rare diseases, which often have a limited patient population and thus a smaller market. The HOPE-3 trial reaching full enrollment and the anticipation of top-line data release are significant milestones that can impact the company's valuation and investor sentiment, especially if the results are positive and suggest a clear path forward for approval and commercialization.
Investors will be closely monitoring the outcome of Capricor's FDA meeting, as it may have a substantial impact on the company's financial trajectory. The ability to expedite the pathway to a BLA submission could lead to earlier-than-expected revenue generation from CAP-1002. Moreover, the market for DMD treatments is relatively untapped, with high unmet medical needs, which could translate into a significant market opportunity for Capricor if CAP-1002 is approved.
However, investors should also consider the risks associated with the drug development process. Regulatory setbacks or negative clinical trial results could adversely affect the stock price. In addition, the biotech sector is known for its volatility and even the anticipation of news can lead to speculative trading. Hence, it is crucial for investors to analyze the company's fundamentals, including its cash runway, pipeline diversity and the competitive landscape of DMD treatments.
For stakeholders in the medical community, the progress of Capricor's CAP-1002 through the regulatory process is of high interest. DMD is a genetic disorder characterized by progressive muscle degeneration and current treatment options are limited. The Regenerative Medicine Advanced Therapy (RMAT) designation indicates that the FDA recognizes the potential of CAP-1002 to address unmet medical needs. A successful meeting with the FDA regarding CMC plans can be indicative of the company's readiness for commercial-scale production, which is often a challenge for cell and exosome-based therapies due to their complexity.
The potential expedited approval process, facilitated by the RMAT and orphan drug designations, could mean that patients with DMD have access to new treatments sooner than typically expected. The medical community will be particularly interested in the quality of the manufacturing process and the reproducibility of the therapy's efficacy and safety profile across a larger population, as this has direct implications for patient care.
SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- – Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March. In the upcoming meeting with the FDA, the Company intends to discuss its proposed chemistry, manufacturing and controls (CMC) plans for commercial launch, if approved which may enable opportunities to expedite the pathway to a Biologics License Application (BLA) submission. The Company has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for the use of CAP-1002 in Duchenne muscular dystrophy (DMD) and these designations enable sponsors to work closely with the FDA and receive its guidance on potential expedited pathways for approval.
“At this time, with our pivotal Phase 3, HOPE-3 trial fully enrolled in Cohort A and top-line data expected in the fourth quarter of this year, we believe that discussing our CMC plans for potential commercialization with the FDA will allow us the opportunity to bring CAP-1002 to patients in the most expeditious manner possible for patients in need,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “At Capricor, we remain focused on getting to approval as quickly as possible and we look forward to providing more details after this meeting.”
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, CAP-1002 — an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 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 which is 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 Facebook, Instagram 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; 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 royalty streams and revenue 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, 2022, as filed with the Securities and Exchange Commission on March 17, 2023 and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, as filed with the Securities and Exchange Commission on November 14, 2023. 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 a partnership for the exclusive commercialization and distribution of 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. 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
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