Astellas voluntarily halts patients dosing in AT132 trial in X-linked Myotubular Myopathy

Sep. 01, 2021 3:25 AM ETAstellas Pharma Inc. (ALPMF)ALPMYBy: Mamta Mayani, SA News Editor
  • Astellas Pharma (OTCPK:ALPMF) has voluntarily paused screening and dosing of additional participants in its ASPIRO clinical trial evaluating AT132 in patients with X-linked Myotubular Myopathy (XLMTM).
  • This decision follows the reporting of a recent serious adverse event (SAE) in a study participant due to abnormal liver function tests observed in the weeks following dosing of the AT132 investigational gene therapy product at a lower dose (1.3x1014 vg/kg).
  • The company voluntarily halted screening and dosing, reported the SAE to regulatory agencies, and is engaged in dialogue with regulators.
  • At this time, an IND clinical hold has not been issued.
  • The participant has a history of intermittent cholestasis. However, prior to dosing, liver ultrasound was normal; and the participant's LFTs, reflecting normal bilirubin levels, were within eligibility criteria.
  • In December of 2020 the clinical hold was lifted after the FDA reviewed the modifications to the ASPIRO trial protocol, which included a reduction of 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 serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure and early death.

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