Sarepta Therapeutics Q3 Earnings Call Highlights

Key Points

• Sarepta raised the low end of its 2026 product revenue forecast by $50 million, to $1.25 billion–$1.3 billion. Third-quarter product revenue fell 13% year over year to $324 million, including $83 million from ELEVIDYS and $240 million from its exon-skipping franchise.
• ELEVIDYS demand indicators were stable, and Sarepta expects fourth-quarter revenue to be roughly in line with the third quarter, but management said it is too early to predict when sales growth may resume. The company plans to seek FDA approval for use in children under age 4 around mid-next year.
• Sarepta will not restart the ENVISION trial; its strategy for potentially treating non-ambulatory patients centers on a safety study testing sirolimus with ELEVIDYS. The company also expects near-term data from its DM1 and FSHD candidates, while anticipating a competing exon 51 treatment could be approved in January 2027.

Sarepta Therapeutics NASDAQ: SRPT raised the low end of its 2026 product revenue forecast after reporting stable demand indicators for Duchenne muscular dystrophy gene therapy ELEVIDYS and continued strength in its exon-skipping drug franchise.

The company now expects full-year net product revenue of $1.25 billion to $1.3 billion, an increase of $50 million at the bottom of its previous range. Third-quarter product revenue fell 13% from a year earlier to $324 million, as lower ELEVIDYS sales offset the contribution from its other Duchenne treatments.

CEO Mike Severino said ELEVIDYS enrollment form activity in the third quarter was in line with the second quarter, providing a baseline for assessing the company’s commercial initiatives. Sarepta expects fourth-quarter ELEVIDYS revenue to be in line with the third quarter, while noting that sales of the one-time gene therapy can vary from quarter to quarter.

ELEVIDYS Sales Stabilize as Sarepta Expands Evidence

ELEVIDYS generated $83 million in net revenue during the quarter, while Sarepta’s phosphorodiamidate morpholino oligomer, or PMO, exon-skipping franchise brought in $240 million. Chief Commercial Officer Patrick Moss said new patient enrollments in the exon-skipping business increased and adherence remained supportive of a stable patient base.

Moss said physicians and families were responding to longer-term ELEVIDYS evidence, including recently published three-year results from the EMBARK study. The company also presented two-year data at the World Muscle Society meeting showing functional benefits in patients treated between ages 8 and 12 compared with an external control group, according to Louise Rodino-Klapac, president of research and development and technical operations.

Severino said it was too early to specify when ELEVIDYS sales growth might return. The company wants to see longer-term trends, given the variability associated with a one-time therapy, he said.

Sarepta is pursuing a broader treatment population. Rodino-Klapac said the company expects to submit a supplemental biologics license application for children under age 4 around the middle of next year, following discussions with the Food and Drug Administration.

Non-Ambulatory Strategy Centers on Safety Study

Sarepta said it would not resume enrollment in its ENVISION study, which had been on hold for more than a year and had recruited about half its intended participants. Rodino-Klapac cited operational and technical barriers to restarting the trial.

Severino said ENVISION was not part of the company’s plan for a potential return to treating non-ambulatory patients. That plan instead centers on ENDEAVOR Cohort 8, which is evaluating whether sirolimus given before and after ELEVIDYS infusion can help mitigate acute liver injury, a known risk associated with AAV gene therapy.

Sarepta expects to complete enrollment in the cohort by year-end and plans to approach the FDA toward the end of the first quarter of 2027 with 12-week safety data. Chief Medical Officer James Richardson said the study design and primary outcome were agreed with the FDA in the latter half of 2025.

In a separate observational study presented at a scientific meeting, no acute liver injury events were observed among the subset of participants who received prophylactic sirolimus, Rodino-Klapac said. She described those findings as preliminary real-world data.

Pipeline Readouts Expected in Coming Weeks

Sarepta expects to share multiple-ascending-dose data in the coming weeks for SRP-1003, its candidate for myotonic dystrophy type 1, or DM1, and SRP-1001, its candidate for facioscapulohumeral muscular dystrophy, or FSHD. The company is preparing for registrational studies in both programs, including manufacturing for planned phase 3 trials.

Rodino-Klapac said the DM1 readout will examine the relationship between dose, muscle exposure, DMPK RNA knockdown and splicing correction, as well as safety and exploratory functional measures. For FSHD, Sarepta is looking for evidence of muscle delivery, suppression of the DUX4 pathway and consistent downstream biomarker effects.

She cautioned against placing too much weight on early functional outcomes in the FSHD study because it is small, participants were not selected for functional similarity at baseline, and follow-up is limited. Severino similarly said the DM1 cohorts are small for assessing functional endpoints.

Management said accelerated approval remains a possible pathway for both programs, but the strategy will depend on the data and discussions with regulators. Higher-dose cohorts are also underway, with full readouts expected early next year, Severino said.

Profitability and Competitive Outlook

Sarepta reported a third-quarter GAAP operating loss of $9 million and a non-GAAP operating profit of $23 million. Total revenue, including collaboration and other revenue, declined 5% to $381 million. Cost of sales included $52 million in take-or-pay charges related to non-cancelable manufacturing obligations for ELEVIDYS inventory the company elected not to produce.

Chief Financial Officer Ryan Wong said cash and investments increased by $54 million during the quarter to nearly $1 billion. Sarepta also narrowed its full-year guidance for combined non-GAAP research and development and selling, general and administrative expenses to $800 million to $825 million.

On competition, Severino said Sarepta’s baseline assumption is that Dyne will receive approval for a competing exon 51 treatment in January 2027. He said he did not expect wholesale switching of stable patients early on and anticipated any competitive impact would be more likely toward the latter part of 2027, while noting that Sarepta would need to see the competitor’s label before offering a more specific assessment.

About Sarepta Therapeutics (NASDAQ:SRPT)

Sarepta Therapeutics, Inc NASDAQ: SRPT is a biotechnology company focused on developing and commercializing precision genetic medicines for rare diseases. Its research programs use RNA-based technologies, gene therapy and other genetic approaches to address the underlying causes of serious neuromuscular disorders.

The company's primary focus is Duchenne muscular dystrophy (DMD), a progressive genetic disease that causes muscle weakness and loss of function. Sarepta markets Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen), RNA-based therapies designed to help certain patients produce a shortened but functional form of dystrophin.

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