Neuren Pharmaceuticals (ASX:NEW) is set to receive a substantial payout following the sale of a Rare Pediatric Disease Priority Review (PRV) voucher by its US partner, Acadia Pharmaceuticals (NASDAQ: ACAD), for $150 million. Under the agreement, Neuren will receive one-third of the net proceeds, estimated to be approximately $50 million.
The PRV was issued by the U.S. Food and Drug Administration (FDA) following the approval of DAYBUE (trofinetide), a treatment for Rett syndrome in patients two years of age and older. PRVs are awarded under the FDA’s Rare Pediatric Diseases Program, an initiative to encourage the development of therapies for serious and life-threatening pediatric diseases with limited treatment options. These vouchers allow companies to receive an expedited review for a future drug, reducing the FDA’s standard review time from ten to six months, potentially speeding a drug’s entry to market. Companies that receive a PRV can apply it to their own drug pipeline or sell it to other companies seeking faster FDA review.
The voucher awarded to DAYBUE reflects its impact on the treatment of Rett syndrome, a rare neurological disease that almost exclusively affects women. Rett syndrome typically occurs in early childhood and is characterized by a period of normal development followed by a gradual loss of motor and cognitive abilities. Symptoms include difficulty moving, loss of speech, repetitive hand movements, and breathing irregularities. Most people with Rett syndrome require lifelong treatment and there is no known cure. Trophinetide, now marketed as DAYBUE, addresses some of the symptoms of the disorder by targeting the underlying inflammatory and neurodevelopmental processes.
Neuren’s licensing agreement with Acadia grants Acadia global rights to develop and commercialize DAYBUE.
Neuren CEO Jon Pilcher highlighted the success of the partnership, noting that proceeds from the sale of PRV will allow continued investment in Neuren’s neurodevelopment journey.
Neuren Pharmaceuticals shares are trading 5.13% higher at $12.90.