Acadia submits Rett Syndrome New Drug Application to the FDA 

Posted: 19 July 2022

Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) is pleased to report that its US partner Acadia Pharmaceuticals (Nasdaq: ACAD) has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older.

Acadia has exclusive rights to develop and commercialize trofinetide in North America. Under the terms of Neuren’s agreement with Acadia, the development and commercialisation of trofinetide in North America is fully funded by Acadia and Neuren may receive potential milestone payments of up to US$455 million, plus double-digit percentage royalties on net sales of trofinetide in North America, plus one third of the market value of a Rare Pediatric Disease Priority Review Voucher if awarded by the FDA upon approval of a NDA for trofinetide.

If the NDA is approved by the FDA, Neuren expects to earn revenue over 2022 and 2023 for Rett syndrome in the US alone of A$118 million plus double-digit percentage royalties on net sales. The expected revenue in addition to the royalties comprises:

  • A milestone payment in 2022 of US$10 million (A$14 million at assumed exchange rate of 0.70) following acceptance of the NDA for review by the FDA
  • A milestone payment in 2023 of US$40 million (A$57 million), following the first commercial sale of trofinetide in the United States
  • US$33 million (A$47 million) in 2023 as Neuren’s one third share of the market value of a Priority Review Voucher, estimated as US$100 million.

Neuren’s additional ongoing revenue from potential sales has two components:

  • Double digit percentage royalties on net sales of trofinetide in all indications. The annual net sales are recorded in tiers and an escalating percentage is applied to each successive tier.
  • Payments of up to US$350 million (approximately A$500 million) on achievement of a series of 4 thresholds of total annual net sales for all indications.

No royalties or similar costs are payable by Neuren to third parties, which means that Neuren’s revenue from Acadia will flow through to pre-tax profit.

Neuren retained all rights to trofinetide for all countries outside North America and has a fully paid-up, irrevocable licence for use in those countries to all data generated by Acadia. Rett syndrome is a devastating condition with no approved therapies and there is urgent unmet need around the world for a treatment. Neuren has received strong interest for potential commercial partnerships and discussions are continuing under a process to secure the optimum outcome for shareholders and for patients.

About Neuren

Neuren is developing two new drug therapies to treat multiple serious neurological disorders that emerge in early childhood, none of which have any approved medicines.

The lead compound, trofinetide, achieved positive results in a Phase 3 clinical trial for Rett syndrome and has also completed a Phase 2 clinical trial in Fragile X syndrome. Both programs have Fast Track designation from the US Food and Drug Administration (FDA). Neuren has granted an exclusive licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide in North America, while retaining all rights outside North America.

Neuren is initiating Phase 2 trials of its second drug candidate, NNZ-2591, for each of Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome.

Recognising the urgent unmet need, all six programs have been granted “orphan drug” designation in the United States. Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases.

Jon Pilcher, CEO
+61 438 422 271


News & opinion

Member Directory