Australian company Neuren Pharmaceuticals (ASX:NEU) has received the US$40 million milestone payment of US$40 million from Acadia for the US approval and first sale of its treatment for Rett syndrome.
The US FDA recently approved DAYBUE (trofinetide) is the first and only drug approved by the FDA for the rare genetic neurological and developmental disorder that affects brain development. This disorder primarily affects females and causes progressive motor skills and language loss.
Neuren is eligible to receive ongoing royalties on net sales of DAYBUE 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. Neurento estimates the one-third share to be valued at US$33 million.
No royalties or similar costs are payable by Neuren to third parties, which means that Neuren’s revenue from Acadia will flow through to pre-tax profit.
Neuren is conducting Phase 2 trials of its second drug candidate, NNZ-2591, for the treatment of Phelan-McDermid syndrome, Angelman syndrome, Pitt-Hopkins syndrome and Prader-Willi syndrome.
