Neuren Pharmaceuticals (ASX:NEU) has announced that the United States Patent and Trademark Office has allowed its patent application covering the use of NNZ-2591 to treat Pitt Hopkins syndrome (PTHS).
Neuren said the patent expiry date will be in April 2040, adding that related applications are pending in other territories.
There are currently no approved treatments for PTHS, which has a severely debilitating impact on the lives of patients, as well as their parents and siblings. 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 multi-centre Phase 2 clinical trial of treatment with NNZ-2591 for 13 weeks, improvements were seen in clinically important aspects of PTHS, including communication, social interaction, cognition and motor abilities. In the trial, 9 out of 11 children showed improvement, assessed by clinicians, and 8 out of 11 children showed improvement, assessed by caregivers.
Neuren said it plans to discuss the next steps in the development program with the FDA. Neuren is also currently preparing to commence a Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome, another severe neurodevelopmental disorder for which there are no approved treatments.