With a phase 3 miss following a fatality in a related study and marking what analysts are calling the “final nail” in Pfizer’s Duchenne muscular dystrophy (DMD) gene therapy coffin, Sarepta Therapeutics and its Elevidys land in an even better position as the reigning DMD leader.

Pfizer on Wednesday disclosed that its fordadistrogene movaparvovec didn’t cause improvement in motor function among DMD patients aged 4 to 7 years old in a phase 3 study, nor did it score on secondary endpoints or make a difference compared to placebo. The data leave the company to evaluate “appropriate next steps” for the program, it said in a release.

That study readout comes after a young boy who received the therapy in a related phase 2 study of younger patients died of cardiac arrest in May. The recent phase 3 used a crossover trial design, but the company paused dosing associated with the crossover after the fatality.

With “no efficacy signals and a less-than-pristine safety profile,” analysts at Leerink consider it “unlikely” that the program will move forward considering the recent “final nail in the coffin,” the team wrote in a note to clients.

Given the rocky road for Pfizer’s program, some analysts, like those at William Blair, “never viewed fordadistrogene movaparvovec as a real competitive threat to Elevidys” anyway. Still, as Sarepta approaches an FDA decision date for a key expansion, the latest update “all but removes” Pfizer’s offering from the competitive landscape, the analysts said in a note.

Sarepta’s long-awaited news on a potential broader label and conversion to a full approval will come on June 21. Ever since Elevidys’ initial approval last June, the company has sought a label that will cover “the treatment of DMD patients with a confirmed mutation in the DMD gene,” or what CEO Douglas Ingram calls the “broadest possible label,” he said in a conference call earlier this year.

Currently, the therapy is approved to treat ambulatory patients ages 4 to 5, a restricted population that presents several “unique executional challenges,” chief customer officer Dallan Murray has said. Considering the average age of DMD diagnosis is 5 years old in the U.S., many patients in that age group are not yet diagnosed, meaning securing access to Elevidys before aging out of eligibility is “a race against time.”

Even though Elevidys has also suffered a primary endpoint miss in one of its phase 3 studies, analysts at William Blair believe that the totality of its data supports a conversion to a full approval and a broader expansion.

However, an indication to treat non-ambulatory patients is “more of a stretch,” the analysts noted. “We would not be surprised if these patients are excluded from the expanded label.”

Sarepta is steadily working through its relatively small eligible patient pool. It collected $200.4 million in Elevidys sales last year following its June 2023 approval and $133.9 million over this year’s first quarter.

Source:
https://www.fiercepharma.com/pharma/phase-3-miss-pfizers-dmd-candidate-no-threat-sareptas-elevidys-ahead-key-label-expansion
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