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Neuren rockets on positive study data

Neuren Pharmaceuticals soared on Tuesday after the small-cap biopharma reported that its partner, Acadia Pharmaceuticals, announced positive results from a study evaluating the use of Trofinetide for the treatment of Rett syndrome.


Acadia (Nasdaq: ACAD) released data from a pivotal, Phase 3 study evaluating the efficacy and safety of Trofinetide in 187 girls and young women aged 5-20 years with Rett syndrome.


Neuren is currently developing two new drug therapies to treat multiple serious neurological disorders that emerge in early childhood, none of which have any approved medicines.


The lead compound, Trofinetide, achieved positive results in a Phase 3 clinical trial and has also completed a Phase 2 clinical trial in Fragile X syndrome. Both programs have Fast Track designation from the US FDA.


Neuren has granted an exclusive licence to Acadia Pharmaceuticals for the development and commercialisation of Trofinetide in North America while retaining all rights outside North America. The development and commercialisation in North America are fully funded by Acadia, and Neuren is eligible to receive potential milestone payments of up to US$455 million, plus tiered escalating percentage royalties on net sales, plus one third of the market value of a Rare Pediatric Disease Priority Review Voucher if awarded by the FDA upon approval of a New Drug Application.


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


Neuren CEO Jon Pilcher commented: “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.”