FMP
Jun 16, 2025 1:24 PM - Parth Sanghvi
Image credit: Financial Modeling Prep (FMP)
Sarepta Therapeutics (NASDAQ:SRPT) faced intense market pressure on Monday after reporting a second death linked to its Duchenne muscular dystrophy gene therapy, Elevidys, in a non-ambulatory patient. Shares plunged nearly 35% in premarket trading, extending losses that began after the first fatality in March.
Two major brokerages—BMO Capital Markets and Piper Sandler—downgraded the stock, citing rising uncertainty:
BMO cut its rating to Market Perform, reducing its price target to $70, and warning of potential hits to Sarepta's 2025 revenue guidance and broader regulatory challenges.
Piper Sandler lowered its rating to Neutral, slashing the price target to $36 from $55. The firm also reduced peak Elevidys sales expectations from $2.4 billion to $1.7 billion, citing growing hesitation among physicians and regulators.
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Sarepta confirmed that it has:
Suspended shipments of Elevidys for non-ambulatory use.
Paused dosing in its ENVISION trial.
Proposed an enhanced immunosuppressive regimen (including sirolimus) and plans to seek FDA approval to resume treatment.
Despite the setback, Elevidys remains available for ambulatory patients, although analysts warn that confidence in its safety profile is now under review.
The latest fatality, like the previous one in March, was attributed to acute liver failure—a known risk associated with AAV-based gene therapies. While not unexpected from a scientific standpoint, the timing adds pressure on Sarepta's near-term outlook, especially given the stock's volatile performance year-to-date.
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Sarepta's future hinges on regulatory decisions and how quickly confidence in Elevidys can be rebuilt—particularly in the non-ambulatory setting. Until clarity emerges, the stock is likely to remain under scrutiny.
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