Neuren Pharmaceuticals’ (ASX: NEU) North America partner Acadia Pharmaceuticals received US Food and Drug Administration (FDA) approval of DAYBUETM (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Acadia expects DAYBUE to be available by the end of April 2023. DAYBUE is the first and only approved treatment for Rett syndrome.
Many people have shown great determination over the long journey to reach this historic outcome,” Neuren CEO, Jon Pilcher, said.
“The greatest has been shown by the Rett syndrome community and I am delighted for them. For Neuren, this is a transforming milestone that places us in a position to make the most of the opportunities ahead of us, as we work with the communities to make a difference in four other neuro developmental disorders.”
Neuren revenue outlook
In October 2022, Neuren received from Acadia a milestone payment of US$10 million following the acceptance of the NDA for review by the FDA. The next milestone payment to Neuren is US$40 million, payable following the first commercial sale of trofinetide in the United States, which is anticipated at the end of April 2023. Neuren is eligible to receive ongoing royalties on net sales of trofinetide in North America, plus milestone payments of up to US$350 million on achievement of a series of four thresholds of total annual net sales, plus one third of the market value of the Rare Pediatric Disease Priority Review Voucher that was awarded to Acadia by the FDA upon approval of the NDA, with the one third share estimated by Neuren as US$33 million.
In 2018, Neuren and Acadia entered into an exclusive license agreement for Acadia to develop and commercialise trofinetide for the treatment of Rett syndrome and other indications in North America (US, Canada and Mexico).
Neuren retains all rights to trofinetide for all countries outside North America and has a fully paid-up, irrevocable licence for use in those countries to all data generated by Acadia. Neuren intends to pursue registration and commercialisation of trofinetide through partners and is currently advancing discussions with a number of third parties.