Positive late-stage trial results for Neuren Pharmaceuticals' trofinetide

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Neuren Pharmaceuticals (ASX:NEU) has reported that its partner for trofinetide in North America, Acadia Pharmaceuticals, has announced positive top-line results from a Phase 3 study.

The Lavender study is evaluating the efficacy and safety of trofinetide in 187 girls and young women aged 5-20 years with Rett syndrome. 

Neuren CEO Jon Pilcher said, “We are delighted with these robustly positive results and are now eager to see trofinetide progress through the regulatory approval process. We are very grateful to the Rett syndrome community – the patients, their caregivers, study site personnel, physicians and everyone who participated in the Lavender study, as well as in Neuren’s two Phase 2 studies that paved the way.”

The development and commercialisation of trofinetide in North America is fully funded by Acadia. Neuren is eligible to receive potential milestone payments of up to US$455 million, plus tiered escalating double-digit percentage royalties on net sales of trofinetide in North America, plus one-third of the market value of a Rare Pediatric Disease Priority Review Voucher if awarded by the US FDA upon approval of a New Drug Application for trofinetide.

Neuren said it would earn revenue over 2022 and 2023 for Rett syndrome in the US alone of A$111 million plus double-digit percentage royalties on net sales if a New Drug Application is approved by the FDA and trofinetide is launched in the US.

Neuren said it retains all rights to trofinetide in countries outside North America and has free and full access to all data from the US development program. Neuren expects to engage commercial partners for Europe and Asia.