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Neuren Pharmaceuticals (ASX: NEU), a Melbourne-based biotech that is developing therapies for neurodevelopmental disorders in early childhood, has scored a cash windfall from the US$150 million ($229 million) sale of a Rare Paediatric Disease Priority Review Voucher (PRV) by its US partner Acadia Pharmaceuticals (NASDAQ: ACAD).
Neuren will receive a third of the proceeds of the sale, or US$50 million ($76.4 million), which is a significant premium to the company’s US$33 million ($50.4 million) estimate of the value of the voucher as reported in its FY23 annual report.
The PRV was granted by the US Food and Drug Administration (FDA) following the approval of DAYBUE (trofinetide) for the treatment of Rett syndrome, a rare genetic neurological and developmental disorder that affects the way the brain develops.
The FDA established the PRV program to provide incentives for biotechs developing new drugs for rare paediatric diseases. Through a PRV, the FDA cuts the review time of a drug application from the usual 10 months to six months.
Under the terms of the PRV program, a voucher can be redeemed to allow for a company to receive priority review for a different product by transferring or selling the voucher to another sponsor.
In June last year, Neuren secured a milestone payment of US$40 million ($61 million) from Acadia from the first commercial sale of DAYBUE.
Last month, Acadia announced it had gained Health Canada approval for DAYBUE to treat Rett syndrome in adult and paediatric patients aged from two years and older under the Priority Review process.
DAYBUE is the first and only drug approved in Canada for the treatment of Rett syndrome and it is the first time the drug has been approved for use outside of the US.
Neuren granted Acadia the exclusive worldwide licence for the development and commercialisation of trofinetide.
The company’s second drug candidate, NNZ-2591, is in Phase 2 development for multiple neurodevelopmental disorders, with the company saying it has achieved positive results in Phase 2 clinical trials for Phelan-McDermid syndrome, Pitt Hopkins syndrome and Angelman syndrome.
Neuren reported royalty revenue from Acadia of $24.3 million in the six months to the end of June this year, up from $3.5 million reported a year earlier.
Canada is included as part of the North America region under Neuren’s agreement with Acadia. This means that any sales in Canada will be added to US sales for the calculation of Neuren’s royalties and sales milestone payments.
Shares in Neuren Pharmaceuticals surged to a high $13.39, up $1.12 from yesterday's close, following the announcement.
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