Sarepta could be ‘potential turnaround story’ with catalysts lined up into 2027

 

Jefferies analysts made the prediction after Sarepta Therapeutics, which has endured nothing short of a saga with the Duchenne muscular dystrophy gene therapy Elevidys, presented data this week showing its clinically meaningful benefit in older patients who are able to walk.

Sarepta Therapeutics could turn things around yet, Jefferies analysts declared after the embattled biotech presented data showing “durable and clinically meaningful benefits” of its gene therapy Elevidys in older patients with Duchenne muscular dystrophy who are able to walk.

The late-breaking poster, which showcased two-year data from 25 patients aged 8–12 years treated with Elevidys in Sarepta’s Phase 3 EMBARK and Phase 1b ENDEAVOR trials, was presented at the 31st Annual Congress of the World Muscle Society (WMS) in Hiroshima, Japan on Wednesday.

The analysis, which compared the trial participants to “well-matched” external controls, showed clinically meaningful efficacy on key scales measuring ambulatory motor function, time to rise from the floor and gait speed, Sarepta reported. The results showed a “sustained treatment effect and slowing of disease progression,” supporting Elevidys’ use in 8- to 12-year-old ambulatory patients with Duchenne muscular dystrophy (DMD).

Jefferies seemed to agree, telling investors in a Wednesday note that the analysis gives Sarepta’s expanded salesforce “new data to support Elevidys’ (+) benefit/risk profile (even for older ambulatory DMD patients).”
Elevidys’ original accelerated approval in 2023 was for patients 4-5-years old based on data showing increased expression of the Elevidys micro-dystrophin protein in this age group.

“The period between 8 and 12 years of age represents a critical phase of Duchenne when many boys transition from relative stability to progressive functional decline,” Louise Rodino-Klapac, president of Research & Development and Technical Operations at Sarepta, said in a prepared statement.

Elevidys’ safety profile was manageable and consistent with what has been seen with the gene therapy to date, according to Sarepta. Treatment-related adverse events occurred in 20% of patients, Jefferies noted, with the most common being nausea and vomiting. There were three liver-related events within 60 days, but no treatment-related deaths, discontinuations or muscle inflammation, the firm noted.

The new results follow Sarepta’s “annus horribilis,” during which multiple patients who’d received Elevidys died. The deaths, which occurred in patients who could no longer walk, were linked to a viral vector that delivers Elevidys as well as Sarepta’s experimental gene therapy for limb-girdle muscular dystrophy (LGMD). After initially refusing, the biotech in July 2025 capitulated to the FDA’s request to suspend U.S. shipments of Elevidys. That hold lasted all of a week before the FDA recommended it be lifted for ambulatory patients with DMD.

In November, the agency added a black box warning to Elevidys’ label to alert patients and prescribers to a risk of serious liver toxicities and death. The FDA also narrowed the gene therapy’s use to ambulatory Duchenne patients 4 years and older with a confirmed mutation in the DMD gene. Elevidys previously had accelerated approval for non-ambulatory patients.

And if the Elevidys setbacks weren’t enough, the biotech lost its long-time leader, Doug Ingram, in February of this year after the CEO stepped down due to personal reasons. Ingram was succeeded by former AbbVie R&D exec Michael Severino in July.

A critical stretch

Certainly, it’s been an upward battle for Sarepta, but Jefferies expressed confidence in the company’s ability to rebound.

“[Sarepta] could be a pot’l turnaround story as Elevidys sales potentially recover and the pipeline generates compelling data,” the analysts wrote on Wednesday.

Still to come this year, Sarepta is expecting multiple-ascending dose readouts from two Phase 1/2 trials in myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy. The assets—both from the company’s siRNA platform—could generate sales of $1 billion a piece, Jefferies said in a Sept. 24 note.

Meanwhile, in the first half of 2027, data from Cohort 8 of the ENDEAVOR trial—which is investigating Elevidys’ safety and expression in non-ambulatory patients with DMD—“could restore DMD and even LGMD prospects,” Jefferies said Wednesday.

Another key inflection point is the FDA’s upcoming decision on whether to grant full approval to Sarepta’s exon skippers Amondys 45 and Vyondys 53, which both failed to improve motor function in a confirmatory trial in Nov. 2025. The FDA has set a target action date of Feb. 28 for both therapies. A positive decision could help to stabilize the DMD franchise, Jefferies said.

https://www.biospace.com/drug-development/sarepta-could-be-potential-turnaround-story-with-catalysts-lined-up-into-2027