Neuren set for FDA talks on trial to treat developmental genetic disorders

Neuren Pharmaceuticals (ASX: NEU) has been granted the chance to talk to the U.S. Food and Drug Administration (FDA) about some of the positive indicators that may be expected from its upcoming Phase 3 clinical trial program for a drug to treat multiple childhood genetic conditions.

The FDA has granted Neuren a Type C meeting – set for early April 2025 – to explore the efficacy endpoints of the trial program for candidate NNZ-2591.

This comes after a Type B End of Phase 2 meeting with the government agency, in which key features of the trial were discussed and agreed to.

NNZ-2591 is the company’s second drug candidate, which is in development with a focus on treating some neurodevelopmental disorders. During the Phase 2 clinical trial, it revealed positive results regarding Phelan-McDermid syndrome, Pitt-Hopkins syndrome, and Angelman syndrome.

CEO Jon Pilcher said Neuren was looking forward to a positive discussion with the FDA.

“Having a confirmed Type C meeting now establishes a clear timetable and in parallel we are continuing all our preparations, planning for mid-year commencement of the first ever Phase 3 trial for children with Phelan-McDermid syndrome,” he aid.

Neuren shares have been trading at $13.93.

Join the discussion: See what HotCopper users are saying about Neuren Pharmaceuticals and be part of the conversations that move the markets.

The material provided in this article is for information only and should not be treated as investment advice. Viewers are encouraged to conduct their own research and consult with a certified financial advisor before making any investment decisions. For full disclaimer information, please click here.