Benzinga | Sep 1, 2021 07:23AM EDT

See Why Astellas Stopped Dosing In Neuromuscular Disease Gene Therapy Trial

* Astellas Pharma Inc (OTC: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.