Another patient has died after receiving an investigational gene therapy from Sarepta Therapeutics, the company confirmed to BioSpace.
In an email Thursday evening, a spokesperson said the death occurred in June and was associated with a case of acute liver failure, the same complication that led to two previously reported fatalities linked to Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys.
Sarepta’s stock is down 27% in pre-market trading.
The newly revealed death was that of a 51-year-old man who had been participating in a Phase I study for limb-girdle muscular dystrophy (LGMD) and was treated with Sarepta’s SRP-9004, a gene therapy that, like Elevidys, uses an adeno-associated virus (AAV) vector. Sarepta has previously pinned these fatal liver complications on AAVs.
“While we do everything possible to ensure patient safety, there is inherent risk in clinical trials,” the spokesperson wrote. “Our deepest condolences go to the family and all those involved in his care.”
Analysts noticed that the company did not report the death in its corporate update earlier this week.
“We think the LGMD patient death could amplify patient hesitancy to use commercial Elevidys and increase investor distrust since the company did not disclose the event on its call,” William Blair wrote in a note to investors early Friday morning.
The company announced Wednesday that it would lay off some 500 employees—about 36% of its workforce—pare down its pipeline to realize some $420 million in savings and focus on its siRNA programs instead of gene therapies. Work on “most of the gene therapies in development for limb-girdle muscular dystrophy” will be suspended. Sarepta shares surged some 20% after the announcement and analysts lauded the business overhaul. BMO Capital Markets in a Wednesday note said the pipeline pivot “can give a fresh/[positive] spin to the story.”
“The only risk we see moving forward is the death of a third Elevidys patient,” BMO said.
That pipeline reshuffle includes SRP-9004, the development for which has been halted along with most of the company’s LGMD program, with the exception of SRP-9003, another gene therapy for LGMD for which the company plans to submit an application to the FDA in the second half of this year.
When asked why Sarepta left out the news of the LGMD study death in its corporate update, the spokesperson stated that the company had been transparent about this development. “The event had already been communicated to regulators and investigators in an appropriate and timely manner. We have also transparently disclosed this tragic event to the community with whom we have partnered closely throughout the development process.”
The two Elevidys patient deaths earlier this year pushed Sarepta to launch a series of safety initiatives for the gene therapy. The biotech last month announced that it would organize an independent panel of experts to assess an updated immunosuppressive regimen for patients. Sarepta also paused Elevidys shipments for non-ambulatory patients.
On Wednesday, the company bolstered these efforts by adding a boxed warning to Elevidys’ label for acute liver injury and acute liver failure.