Sarepta Therapeutics SRPT announced that it has completed enrolment and dosing in the phase III EMERGENE study evaluating its investigational gene therapy, SRP-9003 (bidridistrogene xeboparvovec) for treating limb-girdle muscular dystrophy (LGMD) Type 2E/R4 (LGMD2E/R4, or beta-sarcoglycanopathy).

Data from the EMERGENE study is expected in the first half of 2025. If the data is positive, Sarepta intends to submit a regulatory filing seeking accelerated approval for SRP-9003 in the given indication before the end of next year.

The primary endpoint of the study is the biomarker expression of the beta-sarcoglycan protein, whose absence is the sole cause of the disease.

LGMD2E/R4 is an ultra-rare form of LGMD for which there is no treatment beyond symptom management. Patients suffering from this genetic disease exhibit neuromuscular symptoms, which include difficulty in running, jumping and climbing stairs, before the age of 10. Because of the lack of treatments, the disease often progresses to loss of mobility during the teenage years and is associated with early mortality.

Other Announcements by SRPT

Alongside the above news, Sarepta also announced that it has started the clinical study on gene therapy candidate SRP-9004 in LGMD type 2D. Management is on track to start a clinical study on another gene therapy SRP-9005 for LGMD type 2C in first-quarter 2025.

Both studies have been designed to support the FDA’s approval under the accelerated pathway.

SRPT Stock Performance

Year to date, Sarepta Therapeutics’ shares have risen 24.7% against the industry’s 10.5% decline.

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Sarepta’s Strong Commercial DMD Portfolio

Sarepta’s commercial portfolio includes three approved RNA-based PMO therapies — Exondys 51, Vyondys 53 and Amondys 45 — and the one-shot gene therapy Elevidys, all targeting DMD indications.

Currently, Elevidys is the first and only one-shot gene therapy for DMD in the United States. Since its commercial launch last year in June, the gene therapy has demonstrated blockbuster potential. Sarepta has added around $437 million in product sales in the first nine months of 2024. In comparison, the therapy generated more than $200 million in sales last year.

Elevidys has been developed by Sarepta in collaboration with pharma giant Roche RHHBY. Sarepta and Roche entered into a licensing agreement in 2019 to develop Elevidys. Per the agreement, RHHBY has exclusive rights to launch and commercialize Elevidys in ex-U.S. markets.

Apart from Elevidys, Sarepta has three other therapies in its commercial portfolio targeting the DMD patient population. Exondys 51 is the first approved disease-modifying therapy for DMD in the United States as well as the company’s first product to receive marketing approval. Exondys 51 recorded impressive sales growth in the past few quarters despite the COVID-19 pandemic. Vyondys 53 and Amondys 45 have also seen strong demand since their launch. Per management, these three drugs have the potential to address nearly a third of all patients with DMD in the United States.

Sarepta Therapeutics, Inc. Price

Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote

SRPT’s Zacks Rank

Sarepta Therapeutics currently carries a Zacks Rank #2 (Buy).

Other Key Picks Among Biotech Stocks

Some other top-ranked stocks from the sector are Castle Biosciences CSTL and CytomX Therapeutics CTMX, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Bottom-line estimates for Castle Biosciences have improved from a loss of 59 cents per share to earnings of 34 cents for 2024 in the past 60 days. During the same timeframe, loss per share estimates for 2025 have narrowed from $2.15 to $1.84. Year to date, shares of Castle Biosciences have surged 30.4%.

CSTL’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 172.72%.

In the past 60 days, estimates for CytomX Therapeutics’ 2024 loss per share have narrowed from 29 cents to 5 cents. Estimates for 2025 loss per share have narrowed from 56 cents to 35 cents during the same timeframe. Year to date, CTMX’s shares have lost 27.1%.

CytomX’s earnings beat estimates in two of the trailing four quarters and missed the mark in the other two, delivering an average surprise of 115.70%.

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