See Why Astellas Stopped Dosing In Neuromuscular Disease Gene Therapy Trial

Astellas Pharma Inc ALPMF has voluntarily paused screening and dosing of additional participants in its ASPIRO trial evaluating AT132 gene therapy in patients with X-linked Myotubular Myopathy (XLMTM).
The decision follows a serious adverse event (SAE) in a study participant due to abnormal liver function tests observed in the weeks following the AT132 dosing at a lower dose (1.3x1014 vg/kg).
To date, 24 ASPIRO participants have received AT132: seven at the 1.3x1014 vg/kg dose and 17 at the 3.5x1014 vg/kg dose.
Three participants previously treated at the 3.5x1014 vg/kg dose developed progressive cholestatic hepatitis and subsequent decompensated liver failure.
After that, these three participants died of either sepsis (2 participants) or a gastrointestinal bleed (1 participant), all of which were a consequence of liver failure.
In December 2020, the FDA lifted the clinical hold after modifications to the ASPIRO trial protocol, which included reducing dosing to the 1.3x1014 vg/kg dose level.
The participant associated with this current SAE was dosed in the summer of 2021 after the original clinical hold was lifted.
XLMTM is a rare neuromuscular disease characterized by extreme muscle weakness, respiratory failure, and early death.
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