Sarepta Sees ELEVIDYS Demand Stabilizing as FSHD, DM1 Readouts Near

Key Points

• ELEVIDYS demand is stabilizing after 2025 safety-related disruption, aided by expanded commercial and medical support, though second-half 2026 demand is expected to be modestly below the first half.
• Sarepta expects multiple-ascending-dose readouts for its FSHD and DM1 programs in the second half of 2026, with early data showing target engagement and no dose-limiting toxicities.
• The company is enrolling non-ambulatory patients in a study testing whether sirolimus can reduce ELEVIDYS-related acute liver injury, with enrollment expected to finish in 2026 and data anticipated in early 2027.

Sarepta Therapeutics NASDAQ: SRPT outlined plans to stabilize demand for its ELEVIDYS gene therapy while advancing RNA-based programs for facioscapulohumeral muscular dystrophy, or FSHD, and myotonic dystrophy type 1, or DM1, during a Morgan Stanley Global Healthcare Conference fireside chat.

Chief Executive Officer Michael Severino, who said he had been with the company for nearly two months, described Sarepta as a company with marketed products that generate revenue and support a pipeline intended to expand its therapeutic footprint. He said the company’s near-term strategy centers on supporting its commercial products and progressing its siRNA pipeline through upcoming clinical readouts.

ELEVIDYS Demand Shows Stabilization, CEO Says

Severino said ELEVIDYS experienced disruption in 2025 following disclosed safety events, shifting attention among physicians and families toward product risks. He said Sarepta is working to return the discussion to a balanced assessment of benefit and risk, citing data from registrational trials, longer-term studies and three-year data released earlier this year.

“ELEVIDYS clearly demonstrates a strong benefit for patients,” Severino said, while noting that the disease it treats is progressive, debilitating and life-shortening.

The company has expanded its commercial field force and medical support for ELEVIDYS, according to Severino. He said Sarepta has increased the number of physician discussions and has seen demand stabilize, with early signs of positive momentum.

However, Severino said second-half demand is expected to be modestly below first-half levels. First-half performance benefited from patients already in the treatment queue from 2025, he said. In addition, the average journey from enrollment form to treatment is roughly six months, meaning improvements in new enrollment activity are more likely to affect revenue in 2027 than in the latter half of 2026.

Severino characterized the outlook for the currently indicated population as a gradual build rather than a sharp inflection in demand. He also said the company is not providing long-term guidance.

Non-Ambulatory Study Enrollment Underway

Sarepta is conducting Cohort 8 of its ENDEAVOR study in non-ambulatory patients to evaluate whether sirolimus can reduce acute liver injury associated with ELEVIDYS. The company’s stated hypothesis is that sirolimus could reduce the incidence of acute liver injury by 50% in that population.

Severino said the study is enrolling and is expected to complete enrollment in 2026, with 12-week data anticipated in the first quarter of 2027. Sarepta plans to discuss those data with the Food and Drug Administration during the first quarter of next year as it considers a potential return to the non-ambulatory population.

FSHD and DM1 Programs Advance Toward Readouts

The company expects separate readouts for the multiple-ascending-dose portions of its SRP-1001 FSHD study and SRP-1003 DM1 study during the second half of 2026. Severino said the DM1 release is likely to come first, while the FSHD update may follow later in the period because of assay-validation work involving circulating biomarkers.

For SRP-1001, Sarepta previously reported data from the single-ascending-dose portion of its Phase 1 study. Severino said the company observed systemic pharmacokinetics, muscle delivery that increased proportionally with dose, no dose-limiting toxicities and early evidence of DUX4 knockdown in muscle biopsy samples.

Chief Medical Officer James Richardson said Sarepta observed more than 90% correction in DUX4 gene-regulated panels in pooled single-ascending-dose data. He compared that with approximately 40% to 50% reported in the Avidity program, while noting that the company evaluated the same gene panels used by Avidity and Fulcrum.

Upcoming FSHD data will include six-month results through an 8 milligram-per-kilogram dose, Richardson said. Sarepta also plans to report circulating biomarker data related to DUX4 correction.

For SRP-1003 in DM1, Sarepta previously reported favorable pharmacokinetics, dose-proportional muscle exposure, no dose-limiting toxicities and early target-engagement evidence, Severino said. The multiple-ascending-dose data expected later this year are intended to further evaluate DMPK knockdown and its relationship to splicing correction.

Severino said Sarepta is seeking differentiated levels of DMPK knockdown, arguing that greater knockdown could support a larger clinical benefit. Discussing a Novartis Phase 3 DM1 study that missed its primary endpoint, he said Sarepta remains confident in the biological target but will assess additional data as they become available, including questions around endpoint selection and study design.

PMO Franchise Positioned for Future Competition

Severino described Sarepta’s phosphorodiamidate morpholino oligomer, or PMO, franchise as durable, citing long-term patient benefit, published real-world evidence and support systems including reimbursement assistance and home infusion. He said those services have contributed to adherence rates above 90% in real-world use.

While acknowledging potential competition, including a 2027 PDUFA date for Dyne Therapeutics, Severino said Sarepta believes its established reimbursement pathways, patient support infrastructure and long-term clinical experience position the franchise to compete.

About Sarepta Therapeutics (NASDAQ:SRPT)

Sarepta Therapeutics, Inc is a biotechnology company focused on developing and commercializing genetic medicines for rare diseases. Its research platforms include RNA-based technologies, gene therapy and gene-editing approaches designed to address the underlying causes of genetic disorders.

The company's primary commercial focus has been Duchenne muscular dystrophy (DMD). Sarepta markets Exondys 51, Vyondys 53 and Amondys 45, exon-skipping therapies for eligible patients with specific genetic mutations, as well as Elevidys, a gene therapy developed to treat certain patients with DMD.

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