Neuren gains FDA feedback on Pitt Hopkins and HIE drug trial
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The news: Neuren Pharmaceuticals received written feedback from the US Food and Drug Administration (FDA) regarding next steps on its drug NNZ-2591 for Pitt Hopkins syndrome and hypoxic-ischemic encephalopathy (HIE).
The context: In an ASX filing on Wednesday evening, Neuren said the FDA validated its planned IND-opening study for NNZ-2591 in HIE, on the condition that Neuren provides additional juvenile animal study data which it plans to submit before commencing the study later in 2026.
The FDA said a Pitt Hopkins syndrome-specific clinical global impression scale may be used as a co-primary endpoint – taking a similar approach that is currently being implemented in Neuren’s ongoing Phase 3 trial in Phelan McDermid syndrome.
In the filing, Neuren CEO Jon Pilcher said while the company was “disappointed that in both cases the guidance was received as Written Responses Only and was delayed relative to FDA’s goal dates…We have a clear path forward and remain well positioned to fund the programs.”
Pilcher added that the feedback will bear a minimal financial impact on the company.
Neuren had entered a trading halt earlier on Wednesday ahead of the announcement, which it said would remain in place until the earlier of the announcement or the start of trading on Friday.
The FDA granted fast track designation for the treatment of NNZ-2591 in Angelman syndrome, Pitt Hopkins syndrome and Phelan-McDermid syndrome last year. The HIE program is currently in the pre-clinical development phase.
Neuren shares tanked 10% on Tuesday after the European Medicines Agency (EMA) gave the marketing authorisation application for Neuren's Rett syndrome treatment, trofinetide, a "negative trend vote".
The source: ASX
