Capricor Stock Plummets 62% After FDA Rejects Duchenne Treatment

Capricor Therapeutics, a biotechnology company headquartered in San Diego, has experienced a significant drop in its stock price following the rejection of its application for a treatment targeting a fatal muscle disease by the U.S. Food and Drug Administration (FDA). The company has been focused on developing a cell therapy aimed at addressing heart-related complications associated with Duchenne muscular dystrophy, a genetic disorder that predominantly affects boys and is characterized by muscle wasting and weakness.

The FDA's decision to reject the application was based on the company's inability to provide "substantial evidence of effectiveness." In its response, Capricor Therapeutics acknowledged that the FDA's complete response letter identified unresolved issues related to the drug's chemistry, manufacturing, and quality control. However, the company asserted that it has addressed the majority of these concerns.

In light of the FDA's rejection, Capricor Therapeutics has announced plans to submit data from a late-stage trial to address the regulatory body's concerns. The results of this trial are anticipated to be released in the third quarter of this year. The company's proactive approach in responding to the FDA's feedback demonstrates its commitment to advancing the development of its cell therapy for Duchenne muscular dystrophy.

The impact of the FDA's decision on investor sentiment was evident, as the company's stock price initially plummeted by as much as 62% in pre-market trading. This significant drop reflects the market's reaction to the regulatory setback and the potential implications for the company's future prospects. Despite the challenges, Capricor Therapeutics remains focused on its mission to develop innovative treatments for rare and life-threatening diseases, and its ongoing efforts to address the FDA's concerns are a testament to its dedication to this goal.