Audentes halts gene therapy trial after second patient death

A second patient in the ASPIRO clinical trial investigating AT132 (the investigational gene therapy product candidate) for X-linked myotubular myopathy (XLMTM) has tragically passed away, Audentes Therapeutics has revealed.

Prior to both of these deaths, Audentes, which was acquired last December in a $3 billion deal by Japan’s Astellas Pharma (TYO: 4503), in consultation with the ASPIRO Data Monitoring Committee (DMC), had halted further dosing of patients currently enrolled in the clinical trial. Subsequently, following interactions with the US Food and Drug Administration, the study was put on formal clinical hold. Per FDA’s guidance, it will continue to follow and actively monitor those patients who are enrolled in the trial

Preliminary findings indicate that the immediate cause of death was sepsis. This patient had progressive liver dysfunction characterized by hyperbilirubinemia that occurred within the first 4-6 weeks following AT132 dosing, and which did not respond to standard treatment. This patient received aggressive medical treatment for his liver dysfunction, including hospitalization and close clinical observation. Unfortunately, his condition worsened, and he ultimately succumbed to bacterial infection and sepsis. Additional information surrounding the death is being collected at this time.