• US patent allowed for NNZ-2591 to treat Pitt Hopkins syndrome
• No approved treatments currently available for this rare and serious disorder
• Positive results from Phase 2 clinical trial of NNZ-2591 in Pitt Hopkins syndrome

Neuren Pharmaceuticals (ASX: NEU) announced that the United States Patent and Trademark Office has allowed Neuren's patent application covering the use of NNZ-2591 to treat Pitt Hopkins syndrome (PTHS) for issuance as a patent. The patent will expire in April 2040. PTHS is a neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene, with characteristics including developmental delays, intellectual disability, behavioral differences, and lack of speech. There are currently no approved treatments for PTHS, which has a severely debilitating impact on patients and their families. The US FDA has granted Orphan Drug designation and Fast Track designation to Neuren's development program for NNZ-2591 in PTHS. In Neuren's Phase 2 clinical trial, improvements were seen in clinically important aspects of PTHS, including communication, social interaction, cognition and motor abilities, with 9 out of 11 children showing improvement assessed by clinicians and 8 out of 11 children showing improvement assessed by caregivers. Neuren plans to discuss the next steps in the development program with the FDA. Neuren is also preparing to commence a Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome, another serious neurodevelopmental disorder with no approved treatments.

Neuren plans to discuss the next steps in the development program for NNZ-2591 in Pitt Hopkins syndrome with the FDA.