Neuren Pharmaceuticals (ASX: NEU)Share Price and News
About Neuren Pharmaceuticals
Neuren Pharmaceuticals is an Australian biopharmaceutical company specialising in developing therapies for serious neurodevelopmental disorders that manifest in early childhood.
Neuren's flagship product, DAYBUE™ (trofinetide), is a synthetic analogue of the neuropeptide IGF-1 and has been approved by the U.S FDA for Rett Syndrome.
Rett syndrome is a rare brain disorder that leads to severe impediments, such as a loss of motor skills and language. Rett syndrome becomes apparent after 6-18 months of age and almost exclusively impacts females. There are currently no other FDA-approved medicines for this disease.
The company is seeking to expand the use of trofinetide in other jurisdictions and for other indications. The company is also advancing other assets at the R&D phase, particularly NNZ-2591.
Neuren's Company History
Neuren Pharmaceuticals was founded in 2001 and initially focused on developing Glypromate to prevent cognitive impairment after cardiac surgery. However, after the Phase 3 trial of Glypromate failed in 2008, the company strategically pivoted to concentrate on neurodevelopmental disorders, leading to the development of trofinetide.
In 2018, Neuren signed a deal with San Diego-based company Acadia Pharmaceuticals, giving Arcadia the North American rights to Trofinetide in the event of commercialisation. In return, Acadia agreed to fund a phase III trial for Rett Syndrome and commercialisation costs if and when it entered the market.
The Arcadia-funded Phase III trial was a complete success, meeting the primary endpoints on a statistically significant basis. After the trial was concluded in late 2021, Neuren and Acadia made their case to the FDA and the green light was given in March 2023.
Trofinetide thus became the only approved treatment for Rett Syndrome. It also gave Neuren a Rare Pediatric Disease Priority Review Voucher (PRV) as well as US$40m a month later when the first sale was made, along with royalties on net sales and one third of the market value of the PRV.
In CY23, Daybue made US$177.2m in sales, and followed this up with US$348.4m in CY24. This led to over A$85m in royalties (A$27m in CY23 and A$56.2m in CY24) to Neuren along with A$205m in milestone and upfront payments in CY23 and a further A$80.5m in CY24 from achieving US$250m+ in sales, plus A$76.5m from Neuren’s one third of net proceeds from the sale of its PRV. Neuren made an A$156.9m profit in 2023 and a $145.6m profit in 2024.
Future Outlook of Neuren Pharmaceuticals (ASX: NEU)
For CY25, Arcadia has guided to US$380-405m in the US alone, which would lead to A$62-67m in royalties. With Canadian sales due to start in Q3 of 2025, the global sales figure could be higher. European approval is expected in Q1 of 2026. Plus, there could be even more milestone payments to come, the most likely next one will be US$50m for exceeding US$500m in sales.
Neuren is not just reliant on the success of Trofinetide. It has another asset, NNZ-2591, that has undergone multiple clinical trials for multiple neurological diseases that are larger than the market for Rett syndrome. These include Pheland McDermind Syndrome (PMS), Angelman syndrome and Pitt Hopkins syndrome. The company has obtained top-line Phase 2 results for all three in the last 24 months.
Neuren investors got excited in April 2025 when the company, having indicated it could target PMS first, confirmed the primary endpoints for a Phase 3 trial of NNZ-2591 in PMS. In other words, it knows which targets it will need to set in order to deem the trial a success and is now nearly able to proceed, although the FDA still needs to review the final protocol.
Is NEU a Good Stock to Buy?
In our view, yes. Neuren's breakout in the US along with its expanding sales pipeline, and potential for international market approvals suggest a strong foundation for sustained growth.
Our Stock Analysis
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Frequently Asked Questions
Neuren develops therapies for serious neurodevelopmental disorders, including Rett syndrome, Phelan-McDermid, Angelman, Pitt-Hopkins, and Prader-Willi syndromes. Its flagship asset Trofinetide is FDA-approved in the USA for Rett Syndrome.