Japan-based Astellas has voluntarily paused screening and dosing in a gene therapy clinical trial due to safety issues.
The decision was made after a study participant had abnormal liver function tests weeks after taking AT132, an investigational gene therapy developed for patients with X-linked Myotubular Myopathy, a rare neuromuscular disease. The patient’s liver tests were reportedly normal before entering the trial.
This setback is the latest for Astellas’ trial of the treatment. The trial was first put on hold after three out of 17 patients on a higher dose regimen developed cholestatic hepatitis and liver failure, and then later died. The trial hold was lifted in December 2020 after the company modified the design to only include the smaller dose (a dose that the patient in the current trial was on at the time of the failed liver tests).
Earlier this year, Astellas reported an impairment loss of about $540 million due to the first FDA clinical hold.
