Acadia Pharmaceuticals’ DAYBUE revenue stream has grown by 14% (QoQ and YoY) to US$96.1 million. Acadia has provided DAYBUE full-year 2025 guidance of US$380–405 million, which would imply revenue to Neuren Pharmaceuticals (NEU: $17.17) of US$14.7 million for this quarter and US$62–67 million for the year.
Neuren CEO Jon Pilcher noted that Q1 usually shows a drop from the preceding Q4 due to ordering anomalies.
Upside for DAYBUE
At this year’s Summit, Pilcher said DAYBUE still has strong upside, with only one-third of diagnosed patients treated. US health care providers expect that around 70% of eligible Rett syndrome patients can be reached within two years.
Growth drivers:
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Diagnosed Rett patients increased from 4,500 at launch to 5,500 today, with expectations of 6,000–9,000 in the US.
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Patient numbers on DAYBUE rose from 917 (mid-2024) to 987 (June 2025).
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Persistence is improving: 70% of patients have been on treatment >12 months, as GIT side effects are better managed.
Acadia is accelerating growth with:
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30% increase in field reps
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New CEO and Head of Commercial Operations
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More sophisticated marketing approaches
So far, uptake has been strongest in US “Centres of Excellence,” but 70% of patients live outside these zones, which is now a focus. Pilcher sees strong potential to double existing sales.
Milestones:
Ex-US expansion:
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Approval received in Canada (pricing negotiations ongoing).
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Europe approval possible in early 2026.
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Japan: bridging study planned.
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Higher royalties outside North America (~15% in EU vs. 10% in NA).
All additional royalties now flow straight to pre-tax profit for Neuren.
NNZ-2591: Second Growth Engine
Neuren also sees major upside from NNZ-2591, which has shown positive results in three Phase II trials (Phelan McDermid, Pitt Hopkins, Angelman syndromes). Demand for Phase III candidates is far stronger than for Phase II, and Pilcher anticipates a more competitive licensing process than trofinetide (DAYBUE).
Phase III in PMS:
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First-ever Phase III trial in Phelan McDermid Syndrome (commenced 25 August 2025).
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160 patients, 13-week duration, cost US$80–90m (~two years of DAYBUE royalties).
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Mechanistically linked to DAYBUE, with Phase II results providing confidence.
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Appears more clinically powerful with a better side effect profile.
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Functions as a disease-modifying therapy, not just symptomatic.
New Indications
Syngap1 syndrome — Pilcher unveiled this new indication at the Summit.
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Rare genetic disorder (loss-of-function mutation in SYNGAP1).
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Features epilepsy, seizures, hyperexcitable neurons.
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~500 diagnosed worldwide, but prevalence estimated 1–4 per 10,000.
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Incurable without gene editing.
Hypoxic-Ischaemic Encephalopathy (HIE) also recently added as a preclinical indication.
Advantages across all programs:
Financials
Bioshares recommendation: Accumulate
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