- 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.
- Related content: Benzinga's Full FDA Calendar
- Price Action: ALPMY stock closed at $16.78 on Tuesday.
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