Neuren delivers A$65 million in royalty income as DAYBUE revenue grows 15%
Neuren Pharmaceuticals (ASX: NEU) has reported Neuren Pharmaceuticals 2025 Financial Results featuring A$65 million in royalty income and A$30 million profit after tax for the full year ending 31 December 2025. The biotech’s royalty income climbed 15% from A$56 million in 2024, driven by continued uptake of DAYBUE (trofinetide) for Rett syndrome treatment in the United States.
Since DAYBUE’s April 2023 launch, Neuren has earned cumulative income of A$510 million from its exclusive licensing agreement with Acadia Pharmaceuticals. The company closed 2025 with A$296 million in cash and short-term investments, positioning it to self-fund ongoing clinical development programmes without shareholder dilution.
- A$65 million royalty income (up 15% year-on-year)
- A$30 million profit after tax
- A$296 million cash position at 31 December 2025
- A$510 million cumulative DAYBUE income since commercial launch
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DAYBUE sales momentum continues with new powder formulation launched
DAYBUE generated net sales of US$391 million in 2025, representing 12% growth from the prior year. Unique patients receiving DAYBUE shipments exceeded 1,000 for the first time, whilst the 12-month persistency rate improved to approximately 55%.
In December 2025, the US Food and Drug Administration approved DAYBUE STIX, a dye- and preservative-free powder formulation designed to offer greater flexibility in dose volume and taste. The new formulation launched on a limited basis in Q1 2026 and is expected to roll out more broadly early in Q2 2026. Management anticipates DAYBUE STIX may facilitate treatment for patients whose families previously declined or discontinued the liquid version.
Despite 76% of Q4 2025 new prescriptions originating from community physicians outside Rett syndrome centres of excellence, approximately two-thirds of the 6,000 diagnosed US Rett patients are yet to try DAYBUE. Acadia Pharmaceuticals expanded its US field force by approximately 30% during 2025 to accelerate community penetration.
Acadia has provided 2026 net sales guidance of US$460-490 million, which excludes any potential European commercial sales. Based on this guidance and an exchange rate range of 0.70-0.72, Neuren anticipates earning full-year royalties of A$70-77 million in 2026.
| Year | DAYBUE Net Sales (US$m) | YoY Growth | Royalty to Neuren (A$m) |
|---|---|---|---|
| CY2023 (Apr-Dec) | 348 | N/A | 48 |
| CY2024 | 349 | N/A | 56 |
| CY2025 | 391 | +12% | 65 |
| CY2026 (Guidance) | 460-490 | +18-25% | 70-77 |
International expansion and regulatory update
Israel’s Ministry of Health approved DAYBUE oral solution in January 2026. Named patient supply programmes continue to expand across Europe, the Middle East, and Latin America.
In February 2026, Acadia was informed of a negative trend vote by the Committee for Medicinal Products for Human Use of the European Medicines Agency regarding the Marketing Authorisation Application for trofinetide. Acadia intends to request a re-examination following formal adoption in late February, with the re-examination opinion expected at the end of Q2 2026.
In Japan, trofinetide received Orphan Drug Designation and Acadia commenced a clinical trial to support a marketing application. Results are anticipated in Q4 2026 or Q1 2027, facilitating the application in 2027.
What is a royalty-based biotech business model?
Neuren operates a capital-efficient model by licensing its intellectual property to pharmaceutical partners in exchange for royalties and milestone payments. Under the licensing agreement with Acadia, Neuren earns tiered royalties on DAYBUE net sales without bearing manufacturing, commercialisation, or sales infrastructure costs.
The royalty structure escalates as sales increase:
- 10% on the first US$250 million in annual net sales
- 12% on sales between US$250 million and US$500 million
- 14% on sales between US$500 million and US$750 million
- 15% on sales exceeding US$750 million
This model generates high-margin recurring revenue that flows directly to the bottom line with minimal incremental cost. Neuren retains 100% ownership of its pipeline assets and development programmes, allowing the company to fund clinical trials without raising equity capital or diluting existing shareholders. Investors benefit from leverage to sales growth whilst Acadia bears the operational execution risk.
Phase 3 Koala trial underway as NNZ-2591 pipeline advances
Neuren commenced its Koala Phase 3 clinical trial evaluating NNZ-2591 (ercanetide) in Phelan-McDermid syndrome (PMS) during the second half of 2025. The first two participants began dosing in February 2026.
The trial is a randomised, double-blind, placebo-controlled study enrolling approximately 160 children aged 3-12 years with PMS. Following a screening period of up to four weeks, participants receive 13 weeks of treatment with NNZ-2591 or placebo, with eligibility to continue in a 12-month open-label extension.
In October 2025, the FDA granted Fast Track designation for the PMS programme. The agency previously aligned with Neuren on using a single Phase 3 trial to support a New Drug Application, streamlining the regulatory pathway.
In February 2026, the United States Congress reauthorised the Rare Pediatric Disease Priority Review Voucher programme to 30 September 2029. Marketing approval of NNZ-2591 for PMS would qualify Neuren for a voucher, of which it would retain 100% ownership. Recent voucher sales have been announced at US$200 million and US$205 million, representing a potential non-dilutive funding source.
- Phelan-McDermid syndrome (PMS): Phase 3 Koala trial initiated with Fast Track designation and FDA alignment on single pivotal trial. Rare Pediatric Disease designation positions Neuren for a Priority Review Voucher upon approval.
- Pitt Hopkins syndrome (PTHS): Fast Track designation granted by FDA in 2025. A new US patent covering NNZ-2591 to treat PTHS was issued with expected expiry in 2040.
- Hypoxic ischemic encephalopathy (HIE): Development initiated during 2025 in partnership with Hope for HIE, the global organisation representing families impacted by brain injury at birth. Many thousands of babies experience HIE annually worldwide.
CEO Jon Pilcher
“In 2025 we achieved a critical milestone for Neuren’s value creation strategy with the commencement of our Koala Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome. There is so much to look forward to this year as we continue to execute that program towards a New Drug Application and in parallel advance NNZ-2591 for Pitt Hopkins syndrome and HIE.”
During 2025, Neuren also added SYNGAP1-related disorder to its neurodevelopmental disorders pipeline. In an in-vitro model using human iPSC-derived neurons, treatment with NNZ-2591 reversed neuronal dysfunction caused by SYNGAP1 haploinsufficiency.
Capital management signals board confidence
During 2025, Neuren completed the on-market share buy-back programme commenced in 2024, deploying A$50 million to repurchase shares at an average price of A$12.27 per share. A new share buy-back programme commenced on 2 March 2026, limited to 5% of shares on issue.
The Board views the current share price as materially undervaluing Neuren’s assets relative to internal analyses and published analyst valuations. All NNZ-2591 development programmes for PMS, PTHS, and HIE remain well-funded alongside the buy-back, which will occur at management’s discretion. Neuren may vary, suspend, or terminate the programme at any time based on market conditions, operational performance, and investment opportunities.
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What to watch in 2026
- Koala trial enrolment progress as sites activate globally and dosing continues through Q1-Q2 2026
- DAYBUE STIX commercial launch impact following broader rollout early in Q2 2026
- EMA re-examination opinion on the Marketing Authorisation Application expected at the end of Q2 2026
- Japan trial results for DAYBUE anticipated in Q4 2026 or Q1 2027, supporting a 2027 marketing application
- Royalty income trajectory with 2026 guidance of A$70-77 million representing potential 18-25% growth from 2025
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