Neuren Pharmaceuticals (ASX:NEU) is set to receive a substantial payout following the sale of a Rare Pediatric Disease Priority Review Voucher (PRV) by its US-based partner, Acadia Pharmaceuticals (NASDAQ: ACAD), for $US150m. Under the agreement, Neuren will receive one-third of the net proceeds, estimated to be approximately $US50m.
The PRV was issued by the US Food and Drug Administration (FDA) following its approval of DAYBUE (trofinetide), a treatment for Rett syndrome in patients aged two years and older. PRVs are awarded as part of the FDA's Rare Pediatric Disease Program, an initiative aimed at encouraging the development of therapies for severe 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 months to six months, potentially accelerating a drug’s market entry. Companies that receive a PRV may apply it to their own drug pipeline or sell it to other companies seeking faster FDA review.
The voucher awarded for DAYBUE reflects its impact on treating Rett syndrome, a rare neurological disorder that almost exclusively affects females. Rett syndrome typically manifests in early childhood and is characterised by a period of normal development followed by a gradual loss of motor and cognitive skills. Symptoms include difficulty with movement, loss of speech, repetitive hand movements and breathing irregularities. Most individuals with Rett syndrome require lifelong care, and there is no known cure. Trofinetide, now marketed as DAYBUE, addresses some of the disorder’s symptoms by targeting underlying inflammatory and neurodevelopmental processes.
Neuren’s licensing agreement with Acadia grants Acadia the global rights to develop and commercialise DAYBUE.
Neuren’s CEO, Jon Pilcher, highlighted the partnership’s success, emphasising that the proceeds from the PRV sale will enable continued investment in Neuren’s neurodevelopmental pipeline.
Shares in Neuren Pharmaceuticals are trading 5.13% higher at $12.90.
