Capricor Therapeutics Surges 18% on Positive FDA Drug Update
Regulator provides a clear path forward for the company's Duchenne muscular dystrophy therapy, Deramiocel, boosting investor confidence.
Shares of Capricor Therapeutics (NASDAQ: CAPR) surged more than 18% in trading on Tuesday after the biotechnology firm announced a significant breakthrough in its discussions with the U.S. Food and Drug Administration. Trading volume spiked to more than five times the daily average as investors reacted positively to news that provides a clearer regulatory path for its lead drug candidate, Deramiocel.
The rally was ignited after the company disclosed regarding the Biologics License Application (BLA) for Deramiocel, a cell therapy designed to treat Duchenne muscular dystrophy (DMD), a rare and fatal genetic disorder.
This crucial meeting followed a Complete Response Letter issued by the agency earlier this year. In a significant win for Capricor, the FDA has agreed that data from the company's completed late-stage HOPE-3 clinical trial can serve as the confirmatory evidence required for the BLA. This decision potentially saves the company from undertaking a costly and lengthy new study, accelerating the timeline for resubmission and review.
"The outcome of our Type A meeting is extremely encouraging and provides us with a clear strategy for our BLA resubmission for Deramiocel," said Linda Marbán, Ph.D., Capricor's chief executive officer. The FDA also aligned with Capricor on using the Performance of Upper Limb version 2.0 (PUL v2.0) as the primary efficacy endpoint, reinforcing the trial's design. The agency is by agreeing to review the existing data set.
For patients and investors, the update is a pivotal development. DMD is a progressive muscle-wasting disease with limited treatment options, and Deramiocel aims to address both cardiac and skeletal muscle function. The positive regulatory feedback significantly de-risks the path to market for what could be a transformative therapy.
Capricor plans to resubmit the BLA with the HOPE-3 data, with topline results expected in the fourth quarter of 2025. The company has also stated it has a of Deramiocel through the final stages of regulatory review and towards a potential commercial launch.