Neuren Pharmaceuticals (ASX:NEU) - CEO, Jon Pilcher
CEO, Jon Pilcher
Source: Neuren Pharmaceuticals
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  • Neuren Pharmaceuticals’ (NEU) partner has announced positive top-line results from a phase three study on Rett syndrome
  • Acadia Pharmaceuticals’ phase three “Lavender” study evaluated the efficacy and safety of investigational drug trofinetide in 187 girls and young women with the rare disorder
  • Neuren could earn up to $111 million in revenue over the next two years if a new drug application is approved by the FDA and trofinetide is launched in the US
  • The company says its next step will be to engage commercial partners from Europe and Asia
  • Neuren Pharmaceuticals was up 94.7 per cent, trading at $3.31 at 11:45 am AEDT

Neuren Pharmaceuticals’ (NEU) shares have soared following positive top-line results from a phase three study on Rett syndrome.

Acadia Pharmaceuticals’ phase three “Lavender” study evaluated the efficacy and safety of investigational drug trofinetide in 187 girls and young women aged 5-20 years with the rare genetic neurological and developmental disorder.

Rett syndrome causes problems in brain function and can result in seizures, disorganised breathing patterns, an abnormal side-to-side curvature of the spine (scoliosis), and sleep disturbances.

The development and commercialisation of trofinetide in North America is fully funded by Acadia, while Neuren is eligible to receive potential milestone payments of up to US$455 million (roughly A$645 million), plus tiered escalating double-digit percentage royalties on net sales of trofinetide in North America.

Neuren said it has the potential to earn up to $111 million in revenue plus royalties over the next two years if a new drug application is approved by the FDA and trofinetide is launched in the US.

“We are delighted with these robustly positive results and are now eager to see trofinetide progress through the regulatory approval process,” said Neuren CEO, Jon Pilcher.

“We are very grateful to the Rett syndrome community – the patients, their caregivers, study site personnel, physicians and everyone who participated in the Lavender study, as well as in Neuren’s two Phase 2 studies that paved the way.”

Neuren will retain all rights to trofinetide in countries outside North America and has free and full access to all data from the US development program.

Neuren said its next step will be to engage commercial partners from Europe and Asia.

Neuren Pharmaceuticals was up 94.7 per cent, trading at $3.31 at 11:45 am AEDT.  

NEU by the numbers
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