US Patent Granted for NNZ-2591 with Expiry in 2040 Following Positive Phase 2 Results
Neuren Pharmaceuticals has been granted a US patent for NNZ-2591 to treat Pitt Hopkins syndrome, bolstered by promising Phase 2 clinical trial results and FDA orphan drug status.
- US patent allowed for NNZ-2591 with expiry in April 2040
- Positive Phase 2 trial outcomes in Pitt Hopkins syndrome patients
- FDA granted Orphan Drug and Fast Track designations
- Next development steps to be discussed with FDA
- Phase 3 trial preparation underway for Phelan-McDermid syndrome
Patent Milestone Strengthens Intellectual Property
Neuren Pharmaceuticals (ASX, NEU) has achieved a significant regulatory milestone with the United States Patent and Trademark Office allowing its patent application for NNZ-2591, a drug candidate targeting Pitt Hopkins syndrome (PTHS). The patent, which will expire in April 2040, provides Neuren with a robust intellectual property foundation in the US, a critical market for rare disease therapies. Related patent applications remain pending internationally, indicating a broader strategy to protect this asset globally.
Addressing an Unmet Medical Need
Pitt Hopkins syndrome is a rare and severely debilitating neurodevelopmental disorder characterized by intellectual disability, communication challenges, and distinctive physical symptoms. Currently, no approved treatments exist, underscoring the urgent need for effective therapies. NNZ-2591 has shown encouraging results in a multi-centre Phase 2 clinical trial, where improvements were observed in communication, social interaction, cognition, and motor skills among children with PTHS. These findings, although from a small cohort, suggest potential clinical benefits that could transform patient outcomes.
Regulatory Progress and Future Development
The US Food and Drug Administration has granted NNZ-2591 both Orphan Drug and Fast Track designations for PTHS, recognizing the drug’s potential to address a rare and serious condition. These designations facilitate a more expedited development and review process. Neuren plans to engage with the FDA soon to discuss the next steps in the clinical development pathway, which will be closely watched by investors and the rare disease community alike.
Expanding Pipeline in Neurodevelopmental Disorders
Beyond PTHS, Neuren is preparing to initiate a Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome, another rare neurodevelopmental disorder lacking approved treatments. This expansion highlights Neuren’s broader commitment to addressing unmet needs in childhood neurological conditions. The company’s existing FDA-approved drug, DAYBUE™ (trofinetide), for Rett syndrome, further cements its position in this specialized therapeutic area.
Looking Ahead
While the patent allowance and positive clinical data mark important progress, the path to regulatory approval and commercialisation remains complex. The upcoming discussions with the FDA and the initiation of Phase 3 trials will be pivotal in determining NNZ-2591’s future. For patients and families affected by PTHS and related disorders, Neuren’s advances offer a hopeful glimpse of potential new treatment options on the horizon.
Bottom Line?
Neuren’s patent win and clinical progress set the stage for critical FDA talks and next-phase trials that could reshape rare neurodevelopmental disorder treatment.
Questions in the middle?
- When will Neuren initiate the Phase 3 trial for NNZ-2591 in Pitt Hopkins syndrome?
- What specific endpoints will Neuren target in upcoming regulatory discussions with the FDA?
- How might international patent approvals impact Neuren’s global development and commercial strategy?