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SRPT Plummets on Second Patient Death After DMD Gene Therapy Infusion
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Key Takeaways
SRPT reported a second fatality from acute liver failure after Elevidys gene therapy infusion.
Sarepta suspends Elevidys dosing commercially and pauses the global ENVISION trial.
Elevidys generated over half of the company's Q1 2025 revenue.
Shares of Sarepta Therapeutics (SRPT - Free Report) nosedived 42% in pre-market today after the company reported a second patient death following treatment with Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD).
Based on these events, Sarepta has suspended Elevidys dosing for non-ambulatory patients in the commercial setting and is considering an enhanced immunosuppression regimen to make the therapy safer for these patients. It has also voluntarily paused dosing in the ongoing late-stage ENVISION study designed to satisfy the regulatory requirements for Elevidys outside the United States. This study is evaluating the therapy in non-ambulatory and ambulatory DMD patients.
These recent developments do not impact Elevidys’ label for ambulatory patients. Sarepta claimed that the therapy’s benefit-risk profile in ambulatory DMD remains positive.
Sarepta has informed the FDA and other global health authorities about these developments.
SRPT Stock Under Pressure
The timing of the second case could not have come at a worse time for Sarepta, which has already seen a substantial downfall in its share price since the onset of this year. While some stakeholders could have dismissed the first case as an isolated incident or one-off, the second death raises more serious concerns about Elevidys’ safety profile and may further slow market adoption, as doctors are likely to be more cautious in prescribing the therapy.
Year to date, Sarepta’s shares have plunged 70% compared with the industry’s 1% decline.
Image Source: Zacks Investment Research
Elevidys is a major revenue driver for Sarepta. During the first quarter of 2025, sales from the therapy accounted for over half of its top line. After Sarepta reported the first case, the EMA placed a clinical hold on all Elevidys-related studies, affecting launch plans for the therapy in Europe. Though Sarepta had attributed the first fatality to a CMV infection at the time, uptake for the gene therapy was negatively impacted, forcing it to lower its net product revenue guidance for full-year 2025 to $2.3-$2.6 billion from $2.9-$3.1 billion.
Such continued setbacks to Elevidys’ adoption could be detrimental to SRPT’s growth trajectory and stock performance. Since its commercial launch in June 2023, the therapy has shown blockbuster potential. Sarepta generated around $821 million from Elevidys sales in 2024, up from $200 million in the year-ago period.
Elevidys remains the first and only one-time gene therapy for DMD in the United States. It is currently approved for use in individuals aged four years and older, regardless of ambulation status. While the therapy has been granted full approval to treat ambulatory DMD patients, it has only received accelerated approval for use in non-ambulatory patients. The ENVISION study serves as the confirmatory trial for converting this accelerated approval to a full one.
Sarepta developed Elevidys in partnership with pharma giant Roche (RHHBY - Free Report) . In 2019, the company and Roche entered into a licensing agreement to develop Elevidys. Per the agreement, Roche has exclusive rights to launch and market the therapy in non-U.S. markets.
SRPT’s Zacks Rank
Sarepta currently carries a Zacks Rank #5 (Strong Sell).
In the past 60 days, loss per share estimates for Immunocore’s 2025 have improved from $1.50 to 86 cents. Loss per share estimates for 2026 have narrowed from $1.68 to $1.33 during the same period. IMCR stock has gained around 18% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 76.18%.
In the past 60 days, Agenus’ bottom-line estimates for 2025 have significantly improved from a loss of $4.66 per share to earnings of $1.56. During the same timeframe, estimates for 2026 loss per share have narrowed from $5.02 to $1.99. AGEN stock has soared nearly 72% so far this year.
Agenus’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%.
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SRPT Plummets on Second Patient Death After DMD Gene Therapy Infusion
Key Takeaways
Shares of Sarepta Therapeutics (SRPT - Free Report) nosedived 42% in pre-market today after the company reported a second patient death following treatment with Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD).
This latest fatality was caused by acute liver failure (ALF) — also the reason behind the first patient's death reported three months back. Both deaths occurred in DMD patients who are non-ambulatory (unable to walk).
Based on these events, Sarepta has suspended Elevidys dosing for non-ambulatory patients in the commercial setting and is considering an enhanced immunosuppression regimen to make the therapy safer for these patients. It has also voluntarily paused dosing in the ongoing late-stage ENVISION study designed to satisfy the regulatory requirements for Elevidys outside the United States. This study is evaluating the therapy in non-ambulatory and ambulatory DMD patients.
These recent developments do not impact Elevidys’ label for ambulatory patients. Sarepta claimed that the therapy’s benefit-risk profile in ambulatory DMD remains positive.
Sarepta has informed the FDA and other global health authorities about these developments.
SRPT Stock Under Pressure
The timing of the second case could not have come at a worse time for Sarepta, which has already seen a substantial downfall in its share price since the onset of this year. While some stakeholders could have dismissed the first case as an isolated incident or one-off, the second death raises more serious concerns about Elevidys’ safety profile and may further slow market adoption, as doctors are likely to be more cautious in prescribing the therapy.
Year to date, Sarepta’s shares have plunged 70% compared with the industry’s 1% decline.
Image Source: Zacks Investment Research
Elevidys is a major revenue driver for Sarepta. During the first quarter of 2025, sales from the therapy accounted for over half of its top line. After Sarepta reported the first case, the EMA placed a clinical hold on all Elevidys-related studies, affecting launch plans for the therapy in Europe. Though Sarepta had attributed the first fatality to a CMV infection at the time, uptake for the gene therapy was negatively impacted, forcing it to lower its net product revenue guidance for full-year 2025 to $2.3-$2.6 billion from $2.9-$3.1 billion.
Such continued setbacks to Elevidys’ adoption could be detrimental to SRPT’s growth trajectory and stock performance. Since its commercial launch in June 2023, the therapy has shown blockbuster potential. Sarepta generated around $821 million from Elevidys sales in 2024, up from $200 million in the year-ago period.
Elevidys remains the first and only one-time gene therapy for DMD in the United States. It is currently approved for use in individuals aged four years and older, regardless of ambulation status. While the therapy has been granted full approval to treat ambulatory DMD patients, it has only received accelerated approval for use in non-ambulatory patients. The ENVISION study serves as the confirmatory trial for converting this accelerated approval to a full one.
Sarepta developed Elevidys in partnership with pharma giant Roche (RHHBY - Free Report) . In 2019, the company and Roche entered into a licensing agreement to develop Elevidys. Per the agreement, Roche has exclusive rights to launch and market the therapy in non-U.S. markets.
SRPT’s Zacks Rank
Sarepta currently carries a Zacks Rank #5 (Strong Sell).
Sarepta Therapeutics, Inc. Price
Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote
Key Picks Among Biotech Stocks
Some better-ranked stocks from the sector are Immunocore (IMCR - Free Report) and Agenus (AGEN - Free Report) . While IMCR sports a Zacks Rank #1 (Strong Buy) at present, AGEN carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, loss per share estimates for Immunocore’s 2025 have improved from $1.50 to 86 cents. Loss per share estimates for 2026 have narrowed from $1.68 to $1.33 during the same period. IMCR stock has gained around 18% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 76.18%.
In the past 60 days, Agenus’ bottom-line estimates for 2025 have significantly improved from a loss of $4.66 per share to earnings of $1.56. During the same timeframe, estimates for 2026 loss per share have narrowed from $5.02 to $1.99. AGEN stock has soared nearly 72% so far this year.
Agenus’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%.