Neuren Pharmaceuticals (ASX:NEU) today reported that its US partner for trofinetide, Acadia Pharmaceuticals, will be conducting a clinical trial in girls with diagnosed Rett syndrome.
The DAFFODIL clinical of trofinetide will include approximately ten girls aged two to five with diagnosed Rett syndrome. It will be a 12-week, multicentre, open-label safety, tolerability and pharmacokinetics study in this younger age group, followed by a possible open-label extension for up to an additional 21 months.
Acadia also recently completed enrolment in the LAVENDER phase three trial of trofinetide in Rett syndrome that remains on track to deliver top-line results in the fourth quarter of 2021.
LAVENDER is a randomised, double-blind, placebo-controlled phase three trial testing treatment of approximately 180 patients aged five to 20 years for 12 weeks with trofinetide or placebo.
Rett syndrome is a debilitating neurodevelopmental disorder estimated to affect between one in 10,000 and one 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 FDA.
The development and commercialisation of trofinetide in North America are 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. Further, Neuren has free and full access to all data for use in countries outside North America.