downgrades this morning:
HC Wainwright Downgrades Sarepta Therapeutics to Sell From Neutral, Adjusts PT to $10 From $40
Piper Sandler Downgrades Sarepta Therapeutics to Neutral From Overweight, Adjusts PT to $36 From $70
BMO Capital Downgrades Sarepta Therapeutics to Market Perform From Outperform, Adjusts PT to $70 From $120
Sarepta Therapeutics, Inc. (SRPT) stock experienced a steep decline of approximately 40% in premarket trading on Monday following a sobering safety update for its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys.
What Happened On Sunday, Sarepta provided a safety update regarding Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the company is taking to strengthen the safety profile in non-ambulatory patients.
These steps follow a second reported case of acute liver failure (ALF), resulting in death.
In March, Sarepta Therapeutics (SRPT) reported a patient death following treatment with Elevidys, having suffered acute liver failure.
The cases of ALF to date have both occurred in non-ambulatory (patients who cannot walk independently) individuals with Duchenne.
Sarepta and Roche Holdings AG (OTC:RHHBY) temporarily halted several clinical studies in April.
During its first-quarter earnings release, Sarepta revised its 2025 total net product revenues guidance to $2.3-$2.6 billion from $2.9-$3.1 billion.
William Blair writes that the revised guidance primarily concerns delayed Elevidys turnaround times.
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Key Safety Initiatives As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients.
A panel will evaluate data and assess the company’s proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events.
Sarepta will share the panel’s recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance.
In the meantime, Sarepta is temporarily suspending shipments of Elevidys for non-ambulatory patients.
For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after Elevidys infusion and post-treatment monitoring remains the same.
Sarepta has voluntarily paused dosing in the ENVISION clinical study (also known as Study SRP-9001-303).
ENVISION is a global, randomized, double-blind, placebo-controlled trial evaluating Elevidys in older ambulatory and non-ambulatory Duchenne muscular dystrophy patients.
In the U.S., it serves as the confirmatory trial required under the FDA’s accelerated approval pathway for non-ambulatory patients. |
