- Sarepta Therapeutics Inc. (SRPT) shares dropped nearly 40% to $21.74 in premarket trading after suspending Elevidys shipments for non-ambulatory Duchenne muscular dystrophy patients due to a second patient death from acute liver failure.
- The company paused dosing in a clinical trial and is enhancing immunosuppressive regimens to mitigate the risk of acute liver failure, as stated by Chief Scientific Officer Louise Rodino-Klapac.
- The safety concerns surrounding Elevidys, a key gene therapy for DMD, have led to analyst downgrades and heightened scrutiny, impacting investor confidence in Sarepta’s growth prospects.
Sarepta Therapeutics Inc. (SRPT) faced a sharp decline in its stock price, plummeting nearly 40% to $21.74 in premarket trading on Monday, following the company’s decision to suspend shipments of its gene therapy Elevidys for non-ambulatory patients with Duchenne muscular dystrophy (DMD). The suspension was prompted by the death of a second non-ambulatory DMD patient due to acute liver failure, raising serious safety concerns about the therapy. Sarepta’s chief scientific officer, Louise Rodino-Klapac, announced that the company has voluntarily paused dosing in a related clinical trial and is taking immediate steps to investigate and mitigate the risk of acute liver failure, including strengthening the immunosuppressive regimen.
Elevidys, approved for DMD patients aged 4 and older, targets a rare genetic disorder that causes progressive muscle degeneration, primarily in boys, and has been a cornerstone of Sarepta’s portfolio. The reported fatalities, both in non-ambulatory patients, have intensified scrutiny on the therapy’s safety profile, particularly for those with advanced disease stages. The decision to halt shipments reflects Sarepta’s proactive response to these adverse events but has triggered significant market backlash, with analysts from Piper Sandler and BMO Capital downgrading the stock to ‘Neutral’ and ‘Market Perform,’ respectively, and slashing price targets to $36 and $70. The company’s efforts to enhance immunosuppression protocols aim to address the liver failure risk, but the path forward remains uncertain as regulatory bodies and investors await further clarity. Sarepta’s stock reaction underscores the high-stakes nature of gene therapy development, where safety setbacks can severely impact market confidence and future revenue potential, particularly for a treatment projected to be a key growth driver.
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