The Neuren Pharmaceuticals, one of the top growth stocks witnessed its share price rising by 7.6%, till the time of writing, upon release of its sales data showing significant jump.
Neuren Pharmaceuticals Limited (ASX: NEU)
Neuren Pharmaceuticals Limited has made significant strides in drug development and commercialization, with major achievements in regulatory approvals and financial performance. The company received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for NNZ-2591, its investigational treatment for Pitt Hopkins syndrome (PTHS) and Angelman syndrome (AS). This designation, already granted for Phelan-McDermid syndrome (PMS), strengthens Neuren’s position in developing breakthrough therapies for rare neurodevelopmental disorders.
The Rare Pediatric Disease Designation offers Neuren a potential Priority Review Voucher (PRV) if NNZ-2591 secures marketing approval. The PRV program incentivizes drug development for serious childhood disorders, allowing recipients to either fast-track another drug review or sell the voucher. Neuren’s partner, Acadia Pharmaceuticals, previously sold a PRV for US$150 million after obtaining FDA approval for DAYBUE™ (trofinetide) in Rett syndrome, demonstrating the significant financial potential of this designation.
Neuren’s commercial success through its partnership with Acadia Pharmaceuticals continues to grow. DAYBUE™ (trofinetide), an FDA-approved treatment for Rett syndrome, generated US$348 million in net sales in 2024, reflecting a 97% increase from 2023. The fourth quarter (Q4) of 2024 alone saw record sales of US$96.7 million, signaling continued market adoption.
Despite strong growth, 70% of diagnosed Rett syndrome patients in the U.S. have yet to try DAYBUE™, presenting significant untapped potential. To accelerate adoption, Acadia is:
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Expanding its field force by 30% to reach more physicians.
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Launching branded Direct-to-Consumer (DTC) campaigns to increase awareness.
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Enhancing patient support programs to improve treatment accessibility.
For 2025, Acadia forecasts DAYBUE™ sales between US$380 million and US$405 million, with first sales in Canada expected in Q3 2025, pending price negotiations. Additionally, the company anticipates initial managed access program revenues in select European Union (EU) countries, with full EU approval expected in Q1 2026.
Neuren is among the best growth stocks to buy now as its royalty revenue from DAYBUE™ soared to A$56.2 million in 2024, a 110% increase from 2023. Based on Acadia’s 2025 guidance, Neuren is expected to receive A$62 million to A$67 million in royalties, further strengthening its financial position for ongoing research and development efforts.
With NNZ-2591 advancing through Phase 2 trials and potential PRV opportunities, Neuren is well-positioned to expand its global footprint in rare pediatric disease treatments. The strong commercial trajectory of DAYBUE™, increasing royalty revenues, and regulatory advancements for NNZ-2591 solidify Neuren’s leadership in neurodevelopmental drug innovation.
Source: Company’s Report
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