Opening the first session of the 18th Bioshares Biotech Summit, Neuren Pharmaceuticals' CEO Jon Pilcher spoke of Neuren's success with trofinetide and NNZ-2591, and the commercial options on the table.

Neuren Pharmaceuticals (NEU: $15.57) announced last week top-line Phase II results for NNZ-2591 in Angelman syndrome (AS). NNZ-2591 is a synthetic peptide that maintains the bioavailability of IGF-1 which promotes synaptic connections in the brain. It is the follow-on compound from trofinetide (DAYBUE) which has been approved and is being sold by Acadia Pharmaceuticals.

Neuren Pharmaceuticals (NEU: $19.02) announced top-line results for its phase II trial of drug candidate NNZ-2591 in 16 patients with Pitt-Hopkins Syndrome (PTHS).

Last month, Neuren Pharmaceuticals (NEU; $24.65) announced impressive top-line data from its Phase II trial of NNZ-2591 conducted in 18 patients with Phelan-McDermid Syndrome (PMS).

Over the last two months, there have been some strong price movements in several major and minor biotech stocks. Following a downturn that has lasted almost two years, has the Australian sector finally bottomed?

We reviewed Clinuvel Pharmaceuticals, Dimerix, Imugene, and Neuren.

In additional to the lucrative royalty stream and milestone payments Neuren Pharmaceuticals (NEU: $14.68) is receiving from its first drug DAYBUE, the company is also conducting four clinical studies in four separate neurodevelopmental disorders with its next drug candidate, NNZ-2591.

–3 November 2023–

Neuren  Pharmaceuticals' (NEU: $11.29) partner Acadia Pharmaceuticals has reported a strong second quarter of sales of its drug DAYBUE for Rett syndrome after having been launched in the US in April this year.

Neuren Pharmaceuticals (NEU: $14.20) announced this week a significant expansion of its partnership with US biopharmaceutical company Acadia Pharmaceuticals (NASDAQ: ACAD). The agreement not only sees Acadia's exclusive licence for trofinetide in North America expand to a worldwide licence, but also Acadia's acquisition of the exclusive worldwide licence for NNZ-2591 in Rett Syndrome and Fragile X Syndrome.

Terms: Trofinetide brand name is DAYBUE in North America.

A book was written in 1994 about US biotech company Vertex Pharmaceuticals called The Billion Dollar Molecule. Last week Neuren Pharmaceuticals (NEU: $13.23) may have been successful in achieving that quest - with approval in the US, through its partner Acadia Pharmaceuticals, of the drug which has been renamed DAYBUE (formerly trofinetide) for the treatment of Retts syndrome.

Arguably the most significant event for the Australian biotech sector is approaching with a decision expected from the FDA on the approval of trofinetide, developed by Neuren Pharmaceuticals (NEU: $7.16), on 12 March.

Neuren Pharmaceuticals (NEU: $5.45) has some very clear objectives set for the next 17 months. The most significant of these is gaining US approval of its lead drug candidate, trofinetide, which is being commercialised in North America under license from Neuren by Acadia Pharmaceuticals for the treatment of Rett syndrome in children and adults.

Acadia filed trofinetide for approval last month. The next stage is notice from the FDA that the application is in order and has been accepted (in September), which will trigger a US$10 million payment to Neuren from Acadia.