U.S.-Japan trade pact; Alphabet, Tesla to report - what’s moving markets
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), currently valued at $1.38 billion, announced Monday that the U.S. Food and Drug Administration has placed a clinical hold on its investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD). The news comes as the stock trades near its 52-week low, having declined over 90% in the past year according to InvestingPro data. The hold applies to Sarepta’s clinical programs for product candidates SRP-9003 (LGMD2E/R4/bidridistrogene xeboparvovec), SRP-9004 (LGMD2D/patidistrogene bexoparvovec), SRP-6004 (LGMD2B/R2), and SRP-9005 (LGMD2C/R5 γ-sarcoglycan).
The company previously disclosed on July 16 that it had paused each of these LGMD programs as part of a strategic restructuring, except for SRP-9003. Sarepta reported on December 18, 2024, that it had completed enrollment and dosing in the Phase 3 clinical trial of SRP-9003. Following the clinical hold, Sarepta stated it intends to discuss with the FDA the potential pathway to submit a Biologics License Application seeking accelerated approval for SRP-9003 after the hold is lifted. Despite recent challenges, InvestingPro data shows the company maintains a healthy liquidity position with a current ratio of 4.02, indicating strong ability to meet short-term obligations.
Also on Monday, Sarepta announced that the FDA has revoked the platform technology designation for its AAVrh74 platform technology, which was previously granted on June 2, 2025.
These announcements were made in a press release and disclosed in a filing with the Securities and Exchange Commission.
In other recent news, Sarepta Therapeutics is navigating significant regulatory and financial challenges. The company reported a request from the FDA to halt shipments of its gene therapy Elevidys following several patient deaths. Despite this, Sarepta plans to continue supplying the treatment to ambulatory patients, citing a favorable risk/benefit profile. Guggenheim has maintained its Buy rating, while TD Cowen reiterated a Hold rating, both expressing concerns over the company’s disclosure practices and credibility with patient groups. H.C. Wainwright took a more drastic step by slashing its price target to $0, maintaining a Sell rating due to financial pressures and regulatory issues. BofA Securities continues to hold a Neutral rating, noting that Elevidys will likely remain available but with limited uptake. Wells Fargo (NYSE:WFC) remains optimistic, maintaining an Overweight rating, citing no deaths among ambulatory patients and a resilient benefit-risk profile. Sarepta faces ongoing scrutiny as it addresses these challenges and works to maintain its market presence.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.