Major milestone for Australian pharmaceutical company, Neuren

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Australian company Neuren Pharmaceuticals (ASX:NEU) is set to receive a US$40 million milestone payment following the US launch of its treatment for Rett syndrome.

The company's North American partner Acadia Pharmaceuticals has announced that the recently approved treatment, DAYBUE (trofinetide), is now available as the first and only approved therapy for Rett syndrome.

Rett syndrome is a rare genetic neurological and developmental disorder affecting brain development. This disorder primarily affects females and causes progressive motor skills and language loss.

Under he agreement, Acadia will pay Neuren US$40 million following the first commercial sale of DAYBUE in the US. It is also eligible to receive ongoing royalties on net sales, 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. Neuren estimates this one-third share as US$33 million.

Neuren retains all rights to DAYBUE outside North America.

The company is also developing other therapies to treat serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. It is currently conducting Phase 2 trials of its second drug candidate, NNZ-2591, in PhelanMcDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome.