Sarepta Therapeutics Sees Transformational 2027 as Rare Disease Programs Advance

Key Points

• Sarepta expects its DM1 and FSHD programs to enter registrational studies within 12 months and aims to show biological activity in Huntington’s disease around mid-2027; CEO Michael Severino called 2027 potentially transformational.
• The company plans to use cash flow from its Duchenne business to fund pipeline development without raising equity, while navigating competition and seeking clearer evidence of ELEVIDYS’s growth trajectory.
• Upcoming DM1 and FSHD data will focus on biological markers and functional results; Sarepta says accelerated approval could be possible for both programs, subject to results and regulatory discussions.

Sarepta Therapeutics NASDAQ: SRPT expects updates from its experimental neuromuscular disease programs to help shape its next phase of growth, while relying on its Duchenne muscular dystrophy business to fund development, CEO Michael Severino said at Guggenheim’s Third Healthcare Innovation conference.

Severino said he expects the company’s myotonic dystrophy type 1, or DM1, and facioscapulohumeral muscular dystrophy, or FSHD, programs to be in registrational studies within 12 months. He also expects proof of biological activity from Sarepta’s Huntington’s disease program around the middle of next year.

“I think 2027 is going to be a very transformational year for the company,” Severino said. He described the three programs as high-value assets that he believes are not reflected in Sarepta’s valuation.

Duchenne Business Faces Competition

Severino said Sarepta can use cash flow from its Duchenne business to advance its pipeline without needing to raise equity. He acknowledged that 2025 was challenging but said the company had taken steps to address concerns, including focusing the indicated population for gene therapy ELEVIDYS on ambulatory patients.

For ELEVIDYS, Severino described current demand as a clean baseline, reflecting interest generated earlier in the year before a sales force expansion and the availability of newer data. He said the company believes the therapy has long-term growth potential but wants to see longer-term trends before predicting its timing or scale. An upcoming Cohort 8 data readout could also affect the trajectory, he said.

Sarepta’s exon-skipping therapies could face competition in the coming year from a Dyne program under FDA review for patients eligible for exon 51 treatment. Severino said he expects Sarepta’s established patient base to remain “sticky,” citing its product’s safety record, long-term evidence, reimbursement support and home dosing.

About 90% of patients participate in Sarepta’s in-home dosing program, he said. The company plans to compete for new patients on the attributes of its products, while the competitive picture will depend in part on the contents of any Dyne label, he added.

Upcoming DM1 and FSHD Readouts

For the upcoming DM1 update, Severino said investors should focus on DMPK knockdown and splicing correction, the measures the Phase 1b study was designed to assess. Sarepta also plans to present vHOT functional data, but he cautioned against drawing too much from changes in small patient groups or comparing them across programs.

Severino said there is no firmly established minimum threshold for splicing correction that would produce a clinically meaningful benefit. He described the 20% to 25% range as an area that starts to become interesting and said Sarepta’s goal is to exceed it.

In FSHD, Sarepta previously reported roughly 90% inhibition of DUX4-regulated genes across pooled cohorts in its single ascending dose study. Severino said the upcoming multiple-dose results would include higher doses and that inhibition above 70% would be an exceptional result, in his view.

Severino said accelerated approval could be possible for both programs, potentially using biomarkers, although the company would need to discuss its FSHD data with the FDA. He noted that a confirmatory FSHD study would need to be randomized and examine longer-term clinical endpoints.

Huntington’s Program Timeline

Sarepta expects to show proof of biological activity in Huntington’s disease around the middle of next year. Severino said the timing depends on how far the company needs to advance through dosing cohorts to generate convincing evidence.

He said the program’s subcutaneous delivery could offer an advantage over therapies delivered into cerebrospinal fluid. Sarepta is seeking to demonstrate that its systemically delivered treatment can reach the brain and reduce Huntington protein levels. Accelerated approval should be considered if the program produces the expected data, he said.

About Sarepta Therapeutics (NASDAQ:SRPT)

Sarepta Therapeutics is a biotechnology company focused on developing genetic medicines for rare diseases, with an emphasis on Duchenne muscular dystrophy (DMD). Its research includes RNA-based treatments designed to affect how genetic instructions are processed, as well as gene therapy approaches.

The company's DMD medicines include exon-skipping therapies such as eteplirsen (Exondys 51), golodirsen (Vyondys 53) and casimersen (Amondys 45). Each is intended for patients with a specific genetic mutation and works by helping cells produce a shortened form of dystrophin, a protein affected by DMD.

Sarepta is headquartered in Cambridge, Massachusetts.

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