Neuren Pharmaceuticals (ASX:NEU) has reported a strong first-half performance for 2025, nearly doubling its profit after tax as royalties from its Rett syndrome therapy DAYBUE (trofinetide) continue to climb and its second drug candidate NNZ-2591 advances through late-stage development.
For the six months to 30 June 2025, Neuren posted a profit after tax of $15 million, up from $8 million in the same period last year. The result was driven by royalty income of $28.3 million from DAYBUE, marketed in the US by Acadia Pharmaceuticals, compared with $24.3 million a year earlier. Acadia reported DAYBUE net sales of US$180.7 million in the half, up from US$160.5 million for the corresponding period in 2024.
Acadia has guided for full-year DAYBUE US sales of US$380 to 405 million, implying Neuren will earn royalty income of $62–67 million in 2025, up from $56 million in 2024.
Neuren said the drug, approved in the US and Canada for Rett syndrome, still has significant growth potential, with two-thirds of diagnosed US patients yet to commence treatment. Outside the US, a marketing application has been filed in the European Union, with potential approval expected in early 2026. Named patient supply programs are also underway in Europe, Israel, and other international markets.
Neuren also bolstered shareholder returns, completing a $50 million share buyback at an average price of $12.27 per share. At 30 June, the company held $300 million in cash and short-term investments, up from $222 million at the end of 2024, highlighting its growing financial strength.
That balance sheet is supporting accelerated investment in NNZ-2591, Neuren’s wholly owned drug candidate being developed for a range of serious neurodevelopmental disorders. In the half-year, the company reached an agreement with the USFDA) on the design and primary endpoints of a pivotal Phase 3 trial in Phelan-McDermid syndrome (PMS), initiating its first trial site in the US. This marks the first-ever Phase 3 trial for PMS, a condition for which there are currently no approved treatments.
Momentum is also building across NNZ-2591’s broader pipeline. The candidate received FDA Fast Track designation for Pitt-Hopkins syndrome and secured a US patent valid until 2040. Development has begun for Hypoxic Ischemic Encephalopathy, a leading cause of neonatal death and long-term disability, while SYNGAP1-related disorder has been added to the pipeline following promising preclinical results. The incidence of SRD is estimated at one in 16,000 individuals, with no approved therapies currently available.
Basic earnings per share more than doubled from 6.3 cents in H1 2024 to 11.9 cents in H1 2025, reflecting both profitability and capital management initiatives. Research and development expenditure remained disciplined at $14.9 million, down from $17.9 million in the previous year.
Neuren’s CEO Jon Pilcher said the half-year performance reinforced both the commercial potential of DAYBUE and the long-term growth prospects of NNZ-2591. “With a strong cash position, growing royalty stream, and multiple late-stage programs underway, Neuren is uniquely positioned to advance treatments for rare and devastating neurological conditions that currently have no approved therapies,” he said.
The company’s near-term focus will be on supporting Acadia’s European regulatory push for DAYBUE and executing the pivotal Phase 3 trial of NNZ-2591 in PMS, while continuing to broaden its pipeline of indications.