Neuren says Rett syndrome Phase 3 trial enrolment successfully completed

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Neuren Pharmaceuticals (ASX: NEU) today announced that its US partner Acadia Pharmaceuticals has successfully completed enrolment in the LAVENDER Phase 3 trial of trofinetide in Rett syndrome.

The company said the trial is on track for top-line results in the fourth quarter of 2021.

LAVENDER is a randomised, double-blind, placebo-controlled Phase 3 trial testing treatment of approximately 180 patients for 12 weeks with trofinetide or placebo.

Rett syndrome is a debilitating neurodevelopmental disorder estimated to affect up to 1-in-15,000 females worldwide. A range of severe impairments emerge in infancy, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat, and even breathe.

There are currently no medicines approved for Rett syndrome.

The trofinetide program has Fast Track, Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration.

Neuren CEO Jon Pilcher said: “This successful completion of enrolment is a very important milestone for Neuren and we are now excited to await results from the Phase 3 trial before the end of 2021. Given the many challenges presented by the pandemic over the last 18 months, this is a great achievement by Acadia and reflects the remarkable commitment and determination of the Rett syndrome community in the United States.”

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 FDA upon approval of a New Drug Application for trofinetide.