Neuren Pharmaceuticals wins DAYBUE STIX FDA approval

The Neuren Pharmaceuticals Ltd (ASX: NEU) share price is making headlines today after announcing US FDA approval for DAYBUE STIX, a new powder formulation of trofinetide for Rett syndrome. This milestone gives patients and caregivers more flexibility, and Neuren earns royalties from partner Acadia’s net sales.

What did Neuren Pharmaceuticals report?

• US FDA approved DAYBUE STIX (trofinetide) powder formulation for oral solution
• DAYBUE and DAYBUE STIX are the only FDA-approved Rett syndrome treatments
• Neuren receives royalties on all net sales of trofinetide
• Acadia holds exclusive worldwide commercialisation licence for trofinetide
• DAYBUE STIX expected to launch in the US from Q1 2026

What else do investors need to know?

The new DAYBUE STIX gives Rett syndrome patients a dye- and preservative-free powder, which can be mixed with drinks to improve taste and dosing flexibility. A bioequivalence study confirmed DAYBUE STIX matches the original oral solution in safety and efficacy, so doctors and patients can expect the same outcomes.

Neuren also holds potential for additional income through future commercial and development milestone payments, as outlined in its November 2025 investor presentation. Meanwhile, its second drug candidate, NNZ-2591, has shown positive results in Phase 2 trials for other neurological disorders.

What did Neuren Pharmaceuticals management say?

Neuren CEO Jon Pilcher said:

The Neuren team is excited about the approval of this new treatment option for Rett syndrome families and the continued investment and innovation for trofinetide by our global partner, Acadia. Caregivers can mix DAYBUE STIX with a variety of water-based liquids providing flexibility to modify the taste and volume of their loved-one’s dose. We look forward to seeing the impact as DAYBUE STIX becomes more broadly available during 2026.

What’s next for Neuren Pharmaceuticals?

Acadia plans to launch DAYBUE STIX in the US on a limited basis in the first quarter of 2026, expanding access from early in the second quarter. The powder formulation stands to boost convenience and potentially broaden uptake among Rett syndrome patients.

Looking ahead, Neuren continues to progress NNZ-2591 for several childhood neurodevelopmental disorders, all of which benefit from US and EU orphan drug designation. Milestone payments and royalties from DAYBUE offer Neuren ongoing revenue opportunities.

Neuren Pharmaceuticals share price snapshot

Over the past 12 months, Neuren Pharmaceuticals shares have climbed 43%, outperforming the S&P/ASX 200 Index (ASX: XJO) which has risen 5% over the same period.

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The post Neuren Pharmaceuticals wins DAYBUE STIX FDA approval appeared first on The Motley Fool Australia.

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Motley Fool contributor Laura Stewart has no position in any of the stocks mentioned. The Motley Fool Australia’s parent company Motley Fool Holdings Inc. has no position in any of the stocks mentioned. The Motley Fool Australia has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. This article contains general investment advice only (under AFSL 400691). Authorised by Scott Phillips. This article was prepared with the assistance of Large Language Model (LLM) tools for the initial summary of the company announcement. Any content assisted by AI is subject to our robust human-in-the-loop quality control framework, involving thorough review, substantial editing, and fact-checking by our experienced writers and editors holding appropriate credentials. The Motley Fool Australia stands behind the work of our editorial team and takes ultimate responsibility for the content published by The Motley Fool Australia.