Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of limb-girdle muscular dystrophy (LGMD), a group of rare genetic disorders marked by progressive muscle weakness.

Following positive FDA feedback, the company is advancing a fourth LGMD program to clinical development. Sarepta will soon start screening and dosing patients in the first-in-human clinical SRP-9005-101 (COMPASS) study, to evaluate its investigational gene therapy SRP-9005 for LGMD type 2C/R5 (LGMD2C/R5, or gamma-sarcoglycanopathy).

SRPT also announced completion of enrollment and dosing in the phase I proof-of-concept SRP-9004-102 (DISCOVERY) study, which is evaluating SRP-9004 in patients with LGMD type 2D/R3 (LGMD2D/R3, or alpha-sarcoglycanopathy).

The company also reiterated its plans to report data from the late-stage EMERGENE study, evaluating SRP-9003 in patients with LGMD type 2E/R4 (LGMD2E/R4, or beta-sarcoglycanopathy), by first-half 2025. This study’s primary endpoint is the biomarker expression of the beta-sarcoglycan protein, whose absence is the sole cause of the disease. Following a pre-BLA meeting, the FDA has confirmed eligibility for the accelerated approval pathway, and Sarepta plans to submit a regulatory filing before 2025-end, contingent on positive results.

LGMD encompasses more than 30 genetically distinct subtypes, all of which currently lack approved disease-modifying treatments. Sarepta’s pipeline, which targets subtypes accounting for over 70% of known LGMD cases, represents meaningful progress toward addressing this unmet need.

SRPT Stock’s Performance

Year to date, Sarepta’s shares have plunged 55% compared with the industry’s 7% decline.

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Recent Developments in SRPT Stock

Though Sarepta remains a dominant player in the Duchenne muscular dystrophy (DMD) space, there is currently a negative sentiment around the stock. This is due to the safety concerns around Elevidys, its one-shot gene therapy for DMD.

Last month, Sarepta reported the death of a young patient after treatment with Elevidys. Though this patient died of acute liver failure — a known risk of Elevidys — the company believes that the patient’s recent CMV infection might have contributed to the outcome. Based on this event, the EMA recently placed a clinical hold on all Elevidys-related studies. An investigation into the patient’s death is currently ongoing.

Elevidys is currently the first and only one-time gene therapy for DMD approved in the United States. 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.

Despite this strong commercial performance, the recent adverse event has raised concerns among investors and clinicians. There is growing apprehension that physicians may become more cautious in prescribing Elevidys, potentially limiting market adoption in the United States. The clinical hold further delays the launch plans for the gene therapy in Europe.

Elevidys is a major revenue driver for Sarepta. During the fourth quarter of 2024, the therapy accounted for nearly 60% of total company revenues. For full-year 2025, Sarepta projects net product revenues of $2.9-$3.1 billion, with two-thirds expected to come from Elevidys. Any additional safety concern or regulatory hurdle could pose significant risks to SRPT’s revenue growth and stock performance. Such concerns are the primary reasons behind the sharp decline in Sarepta’s share price.

Sarepta developed Elevidys in partnership with pharmaceutical giant Roche RHHBY. In 2019, SRPT and RHHBY 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 ANI Pharmaceuticals ANIP and CytomX Therapeutics CTMX. While ANIP sports a Zacks Rank #1 (Strong Buy) at present, CTMX 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, estimates for ANI Pharmaceuticals’ 2025 earnings per share (EPS) have risen from $5.54 to $6.34. EPS estimates for 2026 have increased from $6.75 to $7.13 during the same period.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 17.32%.

In the past 60 days, estimates for CytomX Therapeutics’ 2025 EPS have improved from a loss of 31 cents to a profit of 44 cents. During the same timeframe, estimates for 2026 loss per share have narrowed from 65 cents to 25 cents.

CytomX Therapeutics’ earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 180.70%.

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