Neuren Expands Rare Disease Pipeline as DAYBUE™ Sales Hit Record High

Key Highlights:

• Q2 DAYBUE™ net sales reach US$96.1M, up 14% QoQ and YoY
• A$14.7M in Q2 royalties for Neuren; 2025 full-year estimate: A$62–67M
• New disorder added to NNZ-2591 pipeline: SYNGAP1-related disorder (SRD)
• Phase 3 trial preparations underway for Phelan-McDermid syndrome
• Global patient access initiatives expanded across Europe, Israel, and RoW
  
   

Neuren Pharmaceuticals Ltd (ASX: NEU) is making significant strides on multiple fronts. In a dual-update this week, the company reported strong Q2 2025 sales results from its partner Acadia Pharmaceuticals for DAYBUE™ (trofinetide), alongside the addition of SYNGAP1-related disorder (SRD) to its investigational pipeline for NNZ-2591.

DAYBUE™ Momentum Continues: Record Net Sales and Growing Patient Base

According to the Q2 2025 results released on August 7, Acadia Pharmaceuticals (Nasdaq: ACAD) reported US$96.1 million in DAYBUE™ net sales, marking a 14% increase year-on-year and quarter-on-quarter. Neuren earned A$14.7 million in royalties for the quarter—up 16% on Q2 2024 and 9% on Q1 2025.

With these gains, Acadia reiterated its 2025 guidance of US$380–405 million in DAYBUE™ net sales, which would translate to A$62–67 million in royalties for Neuren—assuming a stable AUD/USD exchange rate of 0.65.

Notably, the number of unique patients receiving DAYBUE™ shipments climbed to a record 987, up from 954 in Q1 and 920 in Q4 2024. Persistency also remained strong, with 70% of patients on therapy for over 12 months, signaling not only growing adoption but sustained treatment.

“Acadia has completed a 30% expansion of its field force, targeting untapped community segments beyond Rett syndrome centers of excellence,” noted Neuren in the update.

International rollout efforts are gaining momentum as well, with named patient supply programs now active in Europe (via Clinigen), Israel (Rafa), and the rest of the world (Farmamondo).

SYNGAP1-Related Disorder Joins NNZ-2591 Pipeline

On August 8, Neuren announced the addition of SYNGAP1-related disorder (SRD) to the development pipeline for NNZ-2591, its second drug candidate.

SRD is a rare neurodevelopmental condition caused by mutations in the SYNGAP1 gene. It leads to significant neurological and developmental challenges, including epilepsy, autism spectrum disorder, intellectual disability, motor skill delays, and speech and behavioral disorders. With no approved treatments currently available, the inclusion of SRD signals Neuren’s continued focus on unmet medical needs.

The company reported positive preclinical results, where treatment with NNZ-2591 reversed the neuronal dysfunction in an in-vitro human model derived from iPSC neurons affected by SYNGAP1 haploinsufficiency.

“This addition strengthens our commitment to address serious childhood-onset neurological conditions with no current treatment options,” said Jon Pilcher, CEO of Neuren.

NNZ-2591 Advancing Toward Phase 3

In parallel, Neuren is preparing for a Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome, following encouraging results from Phase 2 studies in Pitt Hopkins syndrome, Angelman syndrome, and Phelan-McDermid syndrome. All programs have received Orphan Drug Designation from the U.S. FDA, which offers regulatory incentives including market exclusivity.

What This Means for Investors

With a market cap of A$2.21 billion and a PE ratio of 16.16, Neuren is among the rare ASX-listed biopharma firms balancing commercial success with a robust development pipeline. The success of DAYBUE™ offers strong near-term financial visibility, while NNZ-2591 and future indications like SRD represent the longer-term growth engine.

Neuren’s multi-pronged strategy—expanding its commercial royalties while deepening its neurodevelopmental pipeline—continues to reinforce its position as a leader in rare disease drug development.

Outlook

Looking ahead, all eyes will be on:

• DAYBUE’s international approvals and broader market penetration
• Regulatory progress on NNZ-2591 Phase 3 trials
• Updates on SYNGAP1-related disorder studies and potential future trials
  
   

With record royalties, deep pipeline expansion, and continued strategic execution, Neuren remains a stock to watch in the global neurotherapeutics space.