Neuren Pharmaceuticals (ASX:NEU) is advancing preparations for a phase 3 trial of its drug candidate NNZ-2591 in children with Phelan-McDermid syndrome, according to a Monday filing with the Australian Securities Exchange.
The Phelan-McDermid syndrome is a rare genetic disorder characterized by the loss or structural change in a portion of chromosome 22.
The announcement follows a meeting with the US Food and Drug Administration in which the company and the US FDA reached alignment on key aspects of the program, with the company submitting additional information to confirm endpoints for the primary efficacy assessment
The 13-week program is a single randomized, double-blind, placebo-controlled trial, the filing said.
Shares rose almost 3% in recent Monday trade.
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