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

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

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