Sarepta Therapeutics Q1 Earnings Call Highlights

Key Points

• Sarepta reaffirmed full-year revenue guidance of $1.2 billion to $1.4 billion after reporting Q1 total revenue of $731 million (product revenue $331 million), a GAAP operating profit of $358 million, and ending the quarter with $748 million in cash and investments, with $400 million of collaboration/other revenue (including $325 million non-cash related to Roche).
• ELEVIDYS commercial recovery remains a focus: Sarepta has more than doubled its salesforce, added a contract team and expanded physician education to close an “information gap,” but management cautioned that converting interest to infusions is typically a ~6-month process and therefore kept guidance unchanged despite “green shoots.”
• Sarepta submitted sNDAs to convert VYONDYS 53 and AMONDYS 45 from accelerated to traditional approval (expecting a standard review and a potential PDUFA in February), is enrolling ENDEAVOR Cohort 8 to test prophylactic sirolimus to reduce acute liver injury with early supportive real‑world signals, and reported encouraging early siRNA data in FSHD and DM1 plus progress toward first Huntington’s program dosing.

Sarepta Therapeutics NASDAQ: SRPT reported first-quarter 2026 results and reiterated its full-year revenue guidance of $1.2 billion to $1.4 billion, with executives emphasizing efforts to “stabilize the business” following disruption in 2025 and rebuild momentum for its Duchenne gene therapy ELEVIDYS.

First-quarter revenue mix and product updates

Chief Executive Officer Douglas Ingram said the company entered 2026 with priorities focused on restoring growth, maintaining financial strength, and advancing its pipeline. Total net product revenue in the quarter was $331 million, including $229 million from the company’s PMO exon-skipping therapies (EXONDYS, VYONDYS, and AMONDYS) and $102 million from ELEVIDYS.

Ingram highlighted progress toward converting VYONDYS 53 and AMONDYS 45 from accelerated approval to traditional approval. He said Sarepta submitted supplemental NDAs (sNDAs) seeking conversion, following FDA feedback that the company could submit clinical data and real-world evidence. President of Research and Development and Technical Operations Louise Rodino-Klapac said the company submitted the sNDAs at the end of April, supported by data from the ESSENCE confirmatory study and published real-world evidence.

On timing, Ingram told analysts Sarepta did not request priority review, and the company expects a standard review cycle with a potential PDUFA date “sometime in February of next year.”

ELEVIDYS: physician education, “green shoots,” and measured expectations

Management spent much of the call discussing efforts to address what executives described as an “information gap” around ELEVIDYS in the ambulatory population. Chief Commercial Officer Patrick Moss said first-quarter ELEVIDYS results reflected “measured demand” influenced by ongoing gaps in understanding of benefit-risk, rather than deterioration in underlying demand fundamentals.

Moss outlined commercial initiatives intended to improve reach and education across the “multiple touch points” required for treatment, including referrals, evaluations, and payer review. He said Sarepta increased field resources, deployed a contract sales force focused on referring physicians and exon-skipping prescribers, and expects that contract team to be fully operational entering the second half of the year. Moss also said the time from enrollment form to infusion is typically “a 6-month process,” which he said will delay when efforts show up in sales.

Executives pointed to early signs of recovery. Moss said enrollment form activity has become more geographically diverse, and some sites that paused activity in 2025 have resumed participation. Ingram said Sarepta has “more than doubled the size of our sales force,” and the company is expanding promotional and educational materials incorporating longer-term data.

When asked what aspects of the three-year EMBARK data are resonating with physicians, Moss said efficacy data and muscle MRI evidence are moving the needle, particularly the “divergence over time from natural history.” Ingram added that EMBARK showed increasing separation between treated and untreated patients over years one, two, and three and argued the MRI findings reinforce the urgency of intervening before irreversible muscle damage occurs.

Despite citing “green shoots,” Ingram reiterated guidance and urged caution. “We would counsel prudence in raising estimates prematurely,” he said, noting the long cycle time to convert interest into infusions. In response to a question about whether the company is more comfortable with the midpoint or high end of guidance, Ingram said Sarepta is making progress but is not changing its outlook.

Non-ambulatory cohort 8 and sirolimus: trial design and real-world observations

Rodino-Klapac said screening and enrollment are underway in ENDEAVOR Cohort 8 (SRP-9001-103), which is evaluating prophylactic sirolimus in non-ambulatory Duchenne patients receiving ELEVIDYS. The cohort is enrolling about 25 U.S. participants and dosing is underway. She said the regimen includes 14 days of sirolimus before infusion and continues for 12 weeks after, with primary endpoints including incidence of acute liver injury (ALI) and ELEVIDYS dystrophin expression at 12 weeks.

Discussing trial expectations, Rodino-Klapac said the study is targeting a 50% reduction in ALI incidence. Chief Medical Officer James Richardson added that ALI rates differ between clinical trials and the real-world setting, partly due to use of GLDH as a biomarker in trials. He said the primary analysis will compare against historical clinical trial rates that include GLDH, while also evaluating rates without GLDH. As an open-label study, Richardson said Sarepta can adjust sample size if observations deviate from assumptions.

Ingram said Sarepta has seen early supportive evidence where sirolimus pretreatment was associated with no liver enzyme increases in interim analyses of real-world experience, while stressing it is “early days” and more patients must be dosed to confirm outcomes.

Richardson also described an additional phase IV effort evaluating commercially dosed patients receiving the same sirolimus regimen, calling it a hypothesis-driven study to evaluate mitigation of ALI in a broader Duchenne population. He said it is “not part of a broader regulatory strategy” for non-ambulatory patients but could provide supportive context.

On next steps after Cohort 8 data are available, Rodino-Klapac said Sarepta will meet with the FDA to determine the regulatory path forward and has committed to share the data with the agency as soon as it is available. Ingram said the company hopes the FDA will work with Sarepta to restore the non-ambulatory indication as quickly as possible if the data significantly improves the risk-benefit profile.

Pipeline: early siRNA signals in FSHD and DM1, and progress in Huntington’s

Rodino-Klapac highlighted preliminary data from Sarepta’s integrin-targeting siRNA platform in facioscapulohumeral muscular dystrophy (FSHD) and myotonic dystrophy type 1 (DM1). She said the company’s alpha-v beta-6 integrin-targeting ligand is intended to enhance skeletal muscle uptake and, based on data to date, has shown superior muscle concentration compared to transferrin-based approaches “without dose-limiting toxicity.”

For SRP-1001 in FSHD1, Rodino-Klapac cited preliminary phase I/II single-ascending dose findings including dose-dependent plasma exposure, no apparent saturation of uptake, suppression of DUX4-related genes, and reductions in creatine kinase (CK), along with what she described as favorable safety and tolerability and “no indication of anemia.” She said Sarepta expects to share multiple-ascending dose data later in the year, including at least six months of follow-up for the 6 mg/kg and 12 mg/kg cohorts (equivalent to 4 mg/kg and 8 mg/kg siRNA dosing), including safety, PK/PD, biomarkers, gene expression, and early functional data.

For SRP-1003 in DM1, she said early data supported dose-dependent delivery to muscle and “impressive” DMPK knockdown. Sarepta expects additional readouts later in the year, including safety, serum and muscle PK, DMPK knockdown, CSI-22 splicing indices, and vHOT analyses, with at least six months of follow-up in the 6 mg/kg cohort (equivalent to 4 mg/kg siRNA). Richardson said PD biomarkers are expected to guide dose selection for phase III and that functional measures are early, adding he would not expect “significant movement” in FSHD functional measures at six months, while DM1 may show a signal in vHOT at that timeframe.

Rodino-Klapac also said Sarepta is on track for first patient in its Huntington’s disease program and that its SCA2 trial is fully enrolled. Discussing Huntington’s differentiation, she pointed to preclinical data supporting delivery to deep brain regions such as the striatum using a TfR ligand and subcutaneous dosing, and said initial “proof of biology” data are expected earlier next year.

Financial results: collaboration revenue, profitability, and cash position

Chief Financial Officer Ryan Wong reported first-quarter total revenues of $731 million, down 2% year over year. He said Sarepta recorded $400 million in collaboration and other revenue, including $325 million of non-cash collaboration revenue related to Roche declining a program option and $40 million in milestone revenue tied to the first commercial sale of ELEVIDYS in Japan.

Total cost of sales was $109 million, down 21% year over year, and Wong said gross margins were 82% on a unit sales basis. Combined R&D and SG&A expenses were $263 million on a GAAP basis and $224 million on a non-GAAP basis, both including a $50 million annual Arrowhead collaboration license fee recorded to R&D. Wong said expenses declined compared with the prior year due to cost restructuring and because the prior-year quarter included upfront transaction costs from the Arrowhead collaboration.

Sarepta reported a GAAP operating profit of $358 million and a non-GAAP operating profit of $398 million. The company ended the quarter with $748 million in cash and investments, down sequentially by $206 million. Wong attributed the decrease primarily to $250 million in payments to Arrowhead, including a DM1 milestone and the annual collaboration payment.

Looking ahead, Wong said Sarepta reaffirmed its revenue and operating expense guidance and expects to remain profitable while growing its cash balance from current levels. Ingram said the company believes its financial position enables it to fund its pipeline “without relying on the equity markets.”

About Sarepta Therapeutics NASDAQ: SRPT

Sarepta Therapeutics, Inc is a biopharmaceutical company focused on the discovery and development of precision genetic medicines for rare neuromuscular diseases. Headquartered in Cambridge, Massachusetts, Sarepta's core expertise lies in designing RNA-targeted therapies and gene therapies that address underlying genetic mutations. The company's mission is to transform the treatment paradigm for patients with Duchenne muscular dystrophy (DMD) and related disorders through innovative modalities.

Sarepta's commercial products include several exon-skipping therapies approved by the U.S.

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