Neuren Pharmaceuticals (ASX:NEU) has received the initial payment of US$10 million (approximately A$13.6 million) under the recently announced license agreement with ACADIA Pharmaceuticals.
Under the agreement, ACADIA has been granted exclusive rights to develop and commercialise trofinetide for all clinical indications in North America.
Neuren has retained all commercial rights to trofinetide outside North America and has free access and rights to use all the technical, clinical and regulatory data that will be generated by ACADIA in the US.
ACADIA has assumed responsibility for funding and executing all remaining development activities for trofinetide in all indications, including the single Phase 3 trial for Rett syndrome which is due to commence in the second half of 2019.
Neuren said it is already working closely with ACADIA’s clinical, regulatory and manufacturing teams, as drug supplies for the Phase 3 trial are manufactured and clinical protocols, sites and logistics are prepared.
ACADIA’s investment for Rett syndrome prior to a US marketing application is expected to be approximately US$55 million, said Neuren in a statement.
Rett syndrome has been estimated to occur in 1-in-10,000 to 15,000 live female births worldwide.
For North America, ACADIA has indicated the peak annual sales potential for trofinetide in Rett syndrome as more than US$500 million. ACADIA will pay to Neuren double-digit percentage royalties on net sales of trofinetide across all indications in North America.
Trofinetide in Rett syndrome and Fragile X syndrome is covered by issued patents to 2032, said Neuren, as well as by orphan drug exclusivity periods following marketing approval of up to 7.5 years in the US and up to 12 years in the EU.
In addition to the royalties on trofinetide sales in North America, Neuren is eligible to receive a number of other milestone-related payments, including up to US$350 million on achievement of thresholds of total annual net sales of trofinetide in North America.
Neuren executive chairman Richard Treagus said, “ACADIA has demonstrated strong strategic intent and commitment to trofinetide and we have full confidence in their development and commercialization capabilities, as well as their funding capacity. The partnership has secured the capabilities and funding required to bring trofinetide to market, while retaining for Neuren a very significant participation in the value of trofinetide in North America, all the value of trofinetide outside North America and the ability to advance NNZ-2591.”