Sarepta reveals third patient death

A patient in a phase 1 study of an investigational gene therapy from Sarepta Therapeutics has died, marking the third patient death this year.

In an analyst Q&A session held this morning with Sarepta senior management, the company clarified that the fatal adverse event occurred in a 51-year-old patient with limb-girdle muscular dystrophy type 2E who was participating in a four-person phase I study of SRP-9004. The patient, who was non-ambulatory, died from acute liver toxicity 11 weeks after being dosed with SRP-9004, a gene therapy designed to deliver a healthy copy of the SGCA gene to muscle cells.

The news comes two days after Sarepta revealed a strategic re-org that included 500 layoffs and pipeline cuts. As a result of the reprioritization, most of the gene therapies in development for limb-girdle muscular dystrophy were cut, including the program for SRP-9004.

It was revealed during today’s Q&A that the fatal serious adverse event happened about a month ago. Sarepta has already informed the FDA. During the call, Sarepta CEO Douglas Ingram reiterated that the decision to halt the SRP-9004 program was previously determined and was not related to the recent patient death.

When pushed to explain why the third patient death wasn’t disclosed along with the re-org, Ingram said the discussion of the death “wasn’t salient to the discussions on Wednesday” and will occur in the context of the entire study results at an upcoming medical meeting, as per usual practice.

Last month, Sarepta revealed that was halting the commercial and clinical use of Elevidys gene therapy for non-ambulatory Duchenne muscular dystrophy patients, following a second case of fatal acute liver failure.