Neuren Pharmaceuticals has achieved a significant regulatory milestone with the United States Patent and Trademark Office allowing its patent application for NNZ-2591 as a treatment for Pitt Hopkins syndrome (PTHS). The patent protection extends until April 2040, with related applications pending in other territories.
The development represents a crucial advancement for patients with PTHS, a severely debilitating neurodevelopmental disorder that profoundly impacts patients and their families. Currently, no approved treatments exist for this rare condition, highlighting the significant unmet medical need that NNZ-2591 aims to address.
Clinical Trial Results Show Promise
Neuren's multi-centre Phase 2 clinical trial demonstrated encouraging efficacy signals for NNZ-2591 in PTHS patients. During the 13-week treatment period, the compound showed improvements in clinically important aspects of the syndrome, including communication, social interaction, cognition and motor abilities.
The trial results revealed that 9 out of 11 children showed improvement as assessed by clinicians, while 8 out of 11 children demonstrated improvement according to caregiver assessments. These dual perspectives provide robust evidence of the treatment's potential therapeutic benefit.
Regulatory Support and Development Strategy
The US FDA has recognized the therapeutic potential of NNZ-2591 by granting both Orphan Drug designation and Fast Track designation to Neuren's development program for PTHS. These designations facilitate accelerated development pathways and provide regulatory advantages for rare disease treatments.
Neuren plans to engage with the FDA to discuss the next steps in the development program, building on the positive Phase 2 results to advance toward potential regulatory approval.
Expanding Neurodevelopmental Portfolio
Beyond PTHS, Neuren is advancing NNZ-2591 development in Phelan-McDermid syndrome, another severe neurodevelopmental disorder lacking approved treatments. The company recently secured Rare Pediatric Disease Designation from the FDA for this indication, which could qualify Neuren for a priority review voucher upon successful approval.
The company is preparing to commence a Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome and plans an end-of-phase 2 meeting with the FDA in the third quarter of 2024. Previous Phase 2 trial results in this indication showed significant improvement across multiple efficacy measures as assessed by both clinicians and caregivers.
Established Track Record
Neuren's expertise in neurodevelopmental disorders is demonstrated through its successful development of DAYBUE (trofinetide), which received FDA approval for treating Rett syndrome in adult and pediatric patients aged two and over. The company has granted Acadia Pharmaceuticals an exclusive worldwide license for trofinetide's development and commercialization.
NNZ-2591 represents Neuren's second drug candidate, currently in Phase 2 development for multiple neurodevelopmental disorders with positive results achieved in both Phelan-McDermid and Pitt-Hopkins syndromes.
