Neuren Pharmaceuticals (ASX:NEU) has said it’s received a “positive” response from the FDA regarding a potential approval for its Phase 3 trial to treat children with Phelan-McDermid Syndrome (PMS.)

Using Neuren’s in-house drug candidate of interest, NNZ-2591, the company and the FDA have agreed a Phase 3 trial would be a single randomised double-blind and placebo-controlled 13 week trial looking at children aged 3-12 presenting with PMS.

The company believes its current plans for Phase 3 testing would be more streamline than Phase 2 with a “less burdensome safety monitoring plan” appearing to be acceptable for the FDA.

To that end, the company is currently preparing for Phase 3 trials with service providers and trial sites being identified.

Its drug of interest, NNZ-2591, has reportedly unlocked repeated success in mice trials.

PMS is a severe condition affecting chromosome 22 in the human body which ultimately disrupts a gene that controls proper formation of the brain.

“There are no medications, drugs, or therapies specifically for PMS, which has an overwhelming unmet medical need,” Neuren wrote on Monday.

“The most common characteristics are moderate to severe developmental and intellectual impairment … delayed or absent speech, symptoms of autism, low muscle tone, motor delays, mild to severe epilepsy,” and social behavioural issues.

HotCopper users were net positive on the news on Monday – but none mentioned that Neuren was the subject of a short sellers’ report back in February of 2024 over claims its other ‘wonder drug’ Daybue could ‘make things worse’ for child patients.

NEU last traded at $14.05.

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