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  • Neuren Pharmaceuticals (NEU) submits a new drug application (NDA) to the US FDA for its Rett syndrome treatment through its partner, Acadia Pharmaceuticals
  • If the NDA is approved by the FDA, Neuren expects to earn revenue over 2022 and 2023 for Rett syndrome of $118 million plus double-digit percentage royalties on net sales in the US
  • Acadia CEO Steve Davis says the application is an important step forward for members of the Rett community, who face a disease with no approved therapies
  • Under the partnership agreement signed in 2018, Acadia continues to fully fund the development and commercialisation of trofinetide in North America, which Neuren may receive potential milestone payments of up to US$455 million (A$668 million)
  • Shares in Neuren are up 6.27 per cent, trading at $4.16 as of 2:16 pm AEST

Neuren Pharmaceuticals (NEU) has submitted a new drug application (NDA) to the US Food and Drug Administration (FDA) for its Rett syndrome treatment through its partner, Acadia Pharmaceuticals.

Rett syndrome is a rare genetic neurodevelopmental disorder caused by mutations on the X chromosomes.

The company’s trofinetide investigational drug is aimed at treating the syndrome in adults and pediatric patients two years of age and older by potentially reducing neuroinflammation and supporting synaptic function.

If the FDA approves the application, Neuren expects to earn revenue at least $118 million over 2022 and 2023 for Rett syndrome in the US alone.

Under the partnership agreement signed in 2018, Acadia has and will continue to fully fund the development and commercialisation of trofinetide in North America, which Neuren may receive potential milestone payments of up to US$455 million (A$668 million).

Additionally, Neuren may receive double-digit percentage royalties on trofinetide net sales in the continent with one third of the market value of a Rare Pediatric Disease priority review voucher if FDA approval from this NDA is received.

Acadia CEO Steve Davis said the application is an important step forward for members of the Rett community, who face a disease with no approved therapies.

“We are grateful to the patients, their families and the physicians who have participated in the trofinetide clinical studies, including our pivotal Phase 3 Lavender study.

“We look forward to working with the FDA as it evaluates the NDA.”

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.

The company has 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.

Further, the company said it has received strong interest for potential commercial partnerships, and discussions are continuing.

Shares in Neuren were up 6.27 per cent, trading at $4.16 as of 2:16 pm AEST.

NEU by the numbers
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