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Astellas Pharma Says Rare Neuromuscular Clinical Trial Participant Passed Away

Astellas Pharma Inc. (ALPMY,ALPMY) said Tuesday that the ASPIRO clinical trial?participant, who?developed a serious adverse event, passed away on September 9. The cause of death is still pending.

The ASPIRO clinical trial had evaluated AT132 in patients with X-linked Myotubular Myopathy. X-linked Myotubular Myopathy is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure and early death.

Astellas noted that it is gathering the relevant clinical information and incorporating such information into the ongoing investigation regarding other serious adverse events observed to date in the ASPIRO trial. It was the first and only participant dosed following the December 23, 2020, release of the earlier FDA clinical hold.

September 1, Astellas said that it had paused screening and dosing of additional participants in its ASPIRO clinical trial evaluating AT132 in patients with X-linked Myotubular Myopathy.

The move had come after the reporting of a recent serious adverse event 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.

The company then said the participant had a history of intermittent cholestasis. However, prior to dosing, the participant had a normal liver ultrasound; and the participant's liver function tests, were within eligibility criteria.

The U.S. Food and Drug Administration had informed Astellas via email that the ASPIRO trial had been placed on clinical hold and a written letter is expected over the next several weeks.

Astellas said that, upon receipt of the official FDA clinical hold letter, it will review its contents and have further dialogue with regulators on the path forward.

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