Neuren’s Record Year Sparks Bold Phase 3 Push for Neurodevelopmental Drug

Strong Financial Foundation

Neuren Pharmaceuticals has reported a landmark year for 2024, posting a total comprehensive income of A$166 million, underpinned by robust royalty income from its partner Acadia Pharmaceuticals’ commercialisation of DAYBUE™ (trofinetide). The US net sales of DAYBUE surged to US$348 million, nearly doubling year-on-year, and driving royalties of A$56 million to Neuren. This financial strength has translated into a cash position of A$341 million as of March 2025, all generated organically without the need for fresh capital raising.

Clinical Progress and Pipeline Expansion

Beyond financials, Neuren is advancing its promising drug candidate NNZ-2591, targeting rare neurodevelopmental disorders including Phelan-McDermid syndrome, Pitt Hopkins syndrome, and Angelman syndrome. Positive Phase 2 trial results across these indications have validated the drug’s potential broad impact. The company has secured FDA alignment on efficacy endpoints, a critical regulatory milestone, enabling it to initiate the first-ever Phase 3 trial for Phelan-McDermid syndrome later this year. Additionally, Neuren is expanding NNZ-2591’s scope to include hypoxic-ischemic encephalopathy (HIE), a devastating neonatal brain injury, aiming to pioneer a novel treatment paradigm.

Market Dynamics and Investor Sentiment

Despite these achievements and unanimous “buy” ratings from nine covering analysts with an average target price of $26, Neuren’s share price has experienced a notable decline from its peak. The chairman attributed this to volatile market conditions, momentum trading, and a short-term focus on quarterly sales rather than long-term growth potential. The company’s confidence is underscored by its ongoing $50 million share buy-back program initiated in late 2024, signaling management’s belief in the sustainability of cash flows and future value creation.

Global Expansion and Patient Impact

Acadia’s efforts to expand DAYBUE’s reach beyond the US into Canada, Europe, and Japan are progressing, with regulatory submissions and managed access programs underway. The patient base in the US has grown steadily, with 954 active patients reported in Q1 2025, 65% of whom have been on therapy for over 12 months, indicating strong treatment adherence. Real-world evidence and caregiver testimonials highlight meaningful improvements in quality of life for individuals with Rett syndrome, reinforcing the drug’s clinical value.

Looking Ahead

Neuren’s transition from Phase 2 to Phase 3 development has involved bolstering its internal capabilities and quality systems, positioning the company to execute pivotal trials efficiently. The upcoming milestones include commencing the Phase 3 trial for Phelan-McDermid syndrome, pursuing regulatory pathways for other indications, and supporting Acadia’s global commercial expansion of DAYBUE. With a strong balance sheet and a diversified pipeline, Neuren is poised to deliver significant shareholder value while addressing critical unmet needs in pediatric neurological disorders.