SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), a biotechnology firm, has reached a significant milestone in the development of its lead drug candidate, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD).
Following recent discussions with the U.S. Food and Drug Administration (FDA), the company has established non-clinical comparability for its drug product, enabling the use of its San Diego facility for manufacturing upon potential approval.
The FDA has advised Capricor to prepare for a pre-Biologics License Application (BLA) meeting and a rolling BLA submission. A subsequent Type-B meeting with the FDA is scheduled for the second quarter of 2024 to discuss these steps, which could lead to an accelerated filing process.
Capricor plans to present its HOPE-2 open label extension 3-year safety and efficacy data in the upcoming quarter as part of its strategy to support the BLA submission. The FDA has agreed that additional data from Cohort B of the HOPE-3 clinical trial will not be required for approval, suggesting confidence in the existing data presented by Capricor.
Linda Marbán, Ph.D., CEO of Capricor, expressed satisfaction with the FDA's feedback, highlighting the successful demonstration of product comparability between the company's Los Angeles and San Diego facilities. This alignment with the FDA is expected to conserve resources and streamline preparations for the potential launch of CAP-1002.
CAP-1002 has received Orphan Drug Designation and is supported by the Regenerative Medicine Advanced Therapy Designation. If approved by the FDA for DMD treatment, Capricor could be eligible for a Priority Review Voucher due to its rare pediatric disease designation.
Capricor is also preparing for a potential launch in partnership with Nippon Shinyaku's U.S. subsidiary, NS Pharma, Inc. The company will host a virtual investor webcast on April 29, 2024, to discuss these updates.
This news is based on a press release statement from Capricor Therapeutics.
InvestingPro Insights
As Capricor Therapeutics (NASDAQ: CAPR) continues to make strides in the development of its lead drug candidate, CAP-1002, for Duchenne muscular dystrophy (DMD), investors and stakeholders are keeping a close eye on the company's financial health and market performance. With a market capitalization of $151.84 million and significant revenue growth over the last twelve months, Capricor shows a promising trajectory. However, the company's gross profit margin stands at a concerning -44.76%, reflecting challenges in profitability.
InvestingPro Tips for Capricor indicate a mixed picture: the company holds more cash than debt, which is a positive sign for financial stability. On the flip side, Capricor is quickly burning through cash and has not been profitable over the last twelve months. Analysts also do not anticipate the company will be profitable this year, which is a critical consideration for potential investors.
Stock price volatility is another factor to consider, as Capricor's stock has experienced significant fluctuations recently. Despite a large price uptick over the last six months, with a 75.59% return, the stock has taken a considerable hit over the last week and month, with total returns of -13.15% and -24.45%, respectively. This volatility could present both risks and opportunities for investors.
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