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SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program
Read MoreHide Full Article
Key Takeaways
SRPT shares fell 35.9% after a third death in its muscular dystrophy gene therapy program from liver failure.
FDA placed LGMD studies on hold and urged a halt to Elevidys shipments; SRPT refused, citing safety data.
Sarepta will cut 36% of staff and focus on siRNA assets to save $400M annually and stabilize operations.
Shares of Sarepta Therapeutics (SRPT - Free Report) nosedived 35.9% on Friday following the death of a patient dosed with one of its experimental gene therapies.
The company confirmed that a 51-year-old, non-ambulant (unable to walk) limb-girdle muscular dystrophy (LGMD) patient died due to acute liver failure (ALF). The unfortunate event occurred in a phase I study of the investigational gene therapy candidate, SRP-9004, which is being developed for LGMD Type 2D.
This marks the third patient death confirmed by Sarepta from the use of its gene therapies. Earlier this year, SRPT reported two cases of patient death following treatment with Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). Like the latest setback, these two deaths were also due to ALF and occurred in non-ambulatory patients.
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. However, the therapy has been mandated by the FDA to carry a black box warning for ALF and acute liver injury.
Sarepta is heavily dependent on Elevidys for revenue generation. During the first quarter of 2025, sales from the therapy accounted for over half of its top line. The safety concerns are bound to severely impact sales of the drug in the second half of the year, which will cause the company’s top line to suffer. The first two deaths have already negatively impacted the uptake of the drug, forcing Sarepta to lower its net product revenue guidance for full-year 2025 to $2.3-$2.6 billion from $2.9-$3.1 billion.
SRPT Defies FDA Request to Halt All Elevidys Shipments
Following the third death, the FDA placed all Sarepta’s clinical studies for LGMD on hold. The regulatory body cited the deaths, potentially tied to ALF following treatment with gene therapies using the AAVrh74 serotype found in Elevidys, as the reason for its action. The agency determined that participants in these studies may face an unreasonable and significant risk of serious illness or injury.
Considering the emerging safety concerns, the FDA requested Sarepta to voluntarily stop all shipments of Elevidys in the United States, effective immediately. Sarepta, via a press release, declined the FDA's request, citing its scientific analysis, which found no new or changed safety signals in the ambulatory LGMD patient population. The company stated that it will continue supplying Elevidys to the ambulatory group.
In its press release, Sarepta clarified that the most recent patient death was not linked to Elevidys, but to SRP-9004 — an investigational therapy developed for a different form of LGMD (LGMD Type 2D). The company emphasized that SRP-9004 is administered at a different dose and produced through a manufacturing process distinct from that of Elevidys. Following the death, dosing in the phase I study for SRP-9004 has already been concluded. SRPT also expressed a willingness to maintain open dialogue with the FDA, aiming to support their shared goal of ensuring informed access to care while safeguarding patient well-being.
Year to date, Sarepta shares have plunged 88.4% compared with the industry’s 2.2% decline.
Image Source: Zacks Investment Research
Furthermore, the FDA has also revoked Sarepta’s AAVrh74 platform technology designation. The agency stated that current evidence no longer sufficiently demonstrates the platform’s potential to support multiple drugs without compromising safety. The FDA continues to assess the risk of severe liver complications, including hospitalization and death, from gene therapies based on this platform and is considering additional regulatory measures.
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 study for converting this accelerated approval to a full one. Per Sarepta, the recent developments do not impact Elevidys’ label for ambulatory patients. It claimed that the therapy’s benefit-risk profile in ambulatory DMD remains positive.
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 to Cut Costs & Focus on siRNA Pipeline
Last week, Sarepta announced a major restructuring plan aimed at improving long-term financial sustainability. The company will cut 36% of its workforce, about 500 employees, generating annual savings of $120 million, and reprioritize its pipeline to save an additional $300 million per year, totaling $400 million in expected yearly savings by 2026.
As part of this shift, Sarepta is now focusing on its siRNA pipeline acquired through its recent deal with Arrowhead Pharmaceuticals, which includes several clinical and preclinical programs targeting conditions like facioscapulohumeral muscular dystrophy, idiopathic pulmonary fibrosis, myotonic dystrophy type 1, spinocerebellar ataxia 2 and Huntington’s disease. Initial clinical data from the studies of some of the acquired candidates are expected by year-end. Despite incurring up to $37 million in one-time charges, Sarepta anticipates more than $100 million in cost savings this year and projects 2026 adjusted R&D and SG&A expenses to be between $800 million and $900 million.
In the past 90 days, Verona Pharma’s bottom-line estimates for 2025 have significantly improved from a loss of 7 cents per share to earnings of 22 cents. During the same timeframe, estimates for 2026 earnings per share have improved from $2.21 to $2.88. VRNA stock has soared 125.9% so far this year.
Verona Pharma’s earnings beat estimates in one of the trailing four quarters and missed the mark on the other three occasions, delivering an average negative surprise of 6.76%.
BAYRY’s 2025 earnings per share estimate has increased from $1.19 to $1.30 for 2025 over the past 90 days, while that for 2026 has gone up from $1.28 to $1.35 over the same timeframe. Year to date, shares of Bayer have surged 62.9%.
BAYRY’s earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%.
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SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program
Key Takeaways
Shares of Sarepta Therapeutics (SRPT - Free Report) nosedived 35.9% on Friday following the death of a patient dosed with one of its experimental gene therapies.
The company confirmed that a 51-year-old, non-ambulant (unable to walk) limb-girdle muscular dystrophy (LGMD) patient died due to acute liver failure (ALF). The unfortunate event occurred in a phase I study of the investigational gene therapy candidate, SRP-9004, which is being developed for LGMD Type 2D.
This marks the third patient death confirmed by Sarepta from the use of its gene therapies. Earlier this year, SRPT reported two cases of patient death following treatment with Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). Like the latest setback, these two deaths were also due to ALF and occurred in non-ambulatory patients.
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. However, the therapy has been mandated by the FDA to carry a black box warning for ALF and acute liver injury.
Sarepta is heavily dependent on Elevidys for revenue generation. During the first quarter of 2025, sales from the therapy accounted for over half of its top line. The safety concerns are bound to severely impact sales of the drug in the second half of the year, which will cause the company’s top line to suffer. The first two deaths have already negatively impacted the uptake of the drug, forcing Sarepta to lower its net product revenue guidance for full-year 2025 to $2.3-$2.6 billion from $2.9-$3.1 billion.
SRPT Defies FDA Request to Halt All Elevidys Shipments
Following the third death, the FDA placed all Sarepta’s clinical studies for LGMD on hold. The regulatory body cited the deaths, potentially tied to ALF following treatment with gene therapies using the AAVrh74 serotype found in Elevidys, as the reason for its action. The agency determined that participants in these studies may face an unreasonable and significant risk of serious illness or injury.
Considering the emerging safety concerns, the FDA requested Sarepta to voluntarily stop all shipments of Elevidys in the United States, effective immediately. Sarepta, via a press release, declined the FDA's request, citing its scientific analysis, which found no new or changed safety signals in the ambulatory LGMD patient population. The company stated that it will continue supplying Elevidys to the ambulatory group.
In its press release, Sarepta clarified that the most recent patient death was not linked to Elevidys, but to SRP-9004 — an investigational therapy developed for a different form of LGMD (LGMD Type 2D). The company emphasized that SRP-9004 is administered at a different dose and produced through a manufacturing process distinct from that of Elevidys. Following the death, dosing in the phase I study for SRP-9004 has already been concluded. SRPT also expressed a willingness to maintain open dialogue with the FDA, aiming to support their shared goal of ensuring informed access to care while safeguarding patient well-being.
Year to date, Sarepta shares have plunged 88.4% compared with the industry’s 2.2% decline.
Image Source: Zacks Investment Research
Furthermore, the FDA has also revoked Sarepta’s AAVrh74 platform technology designation. The agency stated that current evidence no longer sufficiently demonstrates the platform’s potential to support multiple drugs without compromising safety. The FDA continues to assess the risk of severe liver complications, including hospitalization and death, from gene therapies based on this platform and is considering additional regulatory measures.
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 study for converting this accelerated approval to a full one. Per Sarepta, the recent developments do not impact Elevidys’ label for ambulatory patients. It claimed that the therapy’s benefit-risk profile in ambulatory DMD remains positive.
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 to Cut Costs & Focus on siRNA Pipeline
Last week, Sarepta announced a major restructuring plan aimed at improving long-term financial sustainability. The company will cut 36% of its workforce, about 500 employees, generating annual savings of $120 million, and reprioritize its pipeline to save an additional $300 million per year, totaling $400 million in expected yearly savings by 2026.
As part of this shift, Sarepta is now focusing on its siRNA pipeline acquired through its recent deal with Arrowhead Pharmaceuticals, which includes several clinical and preclinical programs targeting conditions like facioscapulohumeral muscular dystrophy, idiopathic pulmonary fibrosis, myotonic dystrophy type 1, spinocerebellar ataxia 2 and Huntington’s disease. Initial clinical data from the studies of some of the acquired candidates are expected by year-end. Despite incurring up to $37 million in one-time charges, Sarepta anticipates more than $100 million in cost savings this year and projects 2026 adjusted R&D and SG&A expenses to be between $800 million and $900 million.
Sarepta Therapeutics, Inc. Price and Consensus
Sarepta Therapeutics, Inc. price-consensus-chart | Sarepta Therapeutics, Inc. Quote
SRPT’s Zacks Rank & Stocks to Consider
Sarepta currently carries a Zacks Rank #5 (Strong Sell).
Some better-ranked stocks in the biotech sector are Verona Pharma (VRNA - Free Report) and Bayer (BAYRY - Free Report) . While VRNA sports a Zacks Rank #1 (Strong Buy), BAYRY carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 90 days, Verona Pharma’s bottom-line estimates for 2025 have significantly improved from a loss of 7 cents per share to earnings of 22 cents. During the same timeframe, estimates for 2026 earnings per share have improved from $2.21 to $2.88. VRNA stock has soared 125.9% so far this year.
Verona Pharma’s earnings beat estimates in one of the trailing four quarters and missed the mark on the other three occasions, delivering an average negative surprise of 6.76%.
BAYRY’s 2025 earnings per share estimate has increased from $1.19 to $1.30 for 2025 over the past 90 days, while that for 2026 has gone up from $1.28 to $1.35 over the same timeframe. Year to date, shares of Bayer have surged 62.9%.
BAYRY’s earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%.