- Neuren Pharmaceuticals’ (NEU) US partner, Acadia Pharmaceuticals, completes the enrolment of patients into its phase 3 trial of trofinetide for Rett Syndrome
- Rett Syndrome is a neurodevelopmental which can severely impact nearly every aspect of a child’s life, including the ability to speak, eat, walk and breathe
- If the results from the trial are positive and approved by the FDA, Neuren could earn revenue of up to $111 million, plus double-digit percentage royalties on net sales
- Acadia says it’s on track for top-line results in Q4 2021
- Neuren Pharmaceuticals last traded at $1.60 on August 4
Neuren Pharmaceuticals’ (NEU) US partner, Acadia Pharmaceuticals, has completed the enrolment of patients into its phase 3 trial of trofinetide in Rett Syndrome.
Rett Syndrome is a neurodevelopmental disorder which impacts one in 15,000 females worldwide, starting from infancy. Severe impairment can affect nearly every aspect of a child’s life: their ability to speak, walk, eat and even breathe. There is currently no medicine approved for Rett syndrome.
Acadia’s LAVENDER phase 3 trial is a randomised, double-blind, placebo-controlled study of around 18 patients over 12 weeks using trofinetide.
The trofinetide program has Fast Track, Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration (FDA).
The development and commercialisation of trofinetide in North America is fully funded by Acadia. Neuren is eligible to receive milestone payments of up to US$455 million (around A$616 million), plus royalties on net sales of the treatment in North America. Additionally, Neuren will be granted free and full access to all data for use in countries outside North America.
If the results from the trial are positive and the new drug application is approved by the FDA, Neuren could earn revenue over 2022 and 2023 of $111 million, plus double-digit percentage royalties on net sales.
Neuren CEO Jon Pilcher says the successful completion of enrolment is an important milestone for the company.
“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,” said Jon Pilcher.
Acadia says it’s on track for top-line results in Q4 2021.
Neuren Pharmaceuticals last traded at $1.60 on August 4.