Fourth child dies in gene therapy trial

Sept. 14, 2021

A fourth patient has died in an Astellas-Audentes Therapeutics trial testing a gene therapy for a rare neuromuscular disorder.

The trial — called ASPIRO — was first paused last year after three patients suffered liver failure and later died. At the time, the treatment was being administered at two different doses. Because all of the patients who fell ill were in the higher-dose group, Astellas was later given the FDA’s blessing to restart the trial using only the lower dose.

Then, earlier this month, Astellas announced that a fourth patient — who had received the lower dose — had abnormal liver tests. Today, the company said that this fourth patient has died. The company said that the exact cause of death is still being investigated.

“Astellas is gathering the relevant clinical information and incorporating such information into the ongoing investigation regarding other serious adverse events observed to date in the ASPIRO trial,” the company said in a statement. “This was the first and only participant dosed following the December 23, 2020, release of the earlier FDA clinical hold.”

The ASPIRO trial is studying AT132, a gene therapy for X-linked myotubular myopathy (XLMTM). Astellas said that all told, 24 patients — all aged 5 or less — have received the lower dose of the treatment.

Astellas took over the asset as part of its $3 billion buyout of Audentes — an acquisition at risk of not generating returns. Earlier this year, Astellas reported that it already had to take a $540 million impairment charge related to AT132.