AveXis Phase I/II SMA Type 2 gene therapy trial will include 20 patients across 12 sites, same endpoints as Phase I – source

20 Oct 2016

AveXis’ (NASDAQ:AVXS) Phase I/II trial of its gene therapy, AVXS-101, in Type 2 spinal muscular atrophy (SMA) will include around 20 patients across 12 sites, according to a source familiar with the situation. A second source noted the same endpoints as an earlier study are likely to be adopted in the Phase I/II.

The company indicated in an August webcast that the trial would begin before YE16, but has not disclosed its trial design.

The second source confirmed the Phase I/II trial planning is in progress but noted the study protocol had not been finalised. The first noted that as gene therapy is virally mediated AVXS-101 has raised some regulatory concerns and there is extra FDA trial scrutiny before the trial could begin.

A company spokesperson noted AveXis plans to provide an update on the development pathway of AVXS-101 once it receives minutes from an FDA Type B meeting. The spokesperson did not confirm when the meeting had occurred. Type B meetings include end-of-Phase I meetings, according to FDA information.

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