Neuren Pharmaceuticals (ASX:NEU) has reported positive results from a Phase 2 trial of NNZ-2591 in children with Pitt-Hopkins syndrome.
In a statement to investors, Neuren said NNZ-2591 achieved a statistically significant improvement from the baseline across all four efficacy measures designed to assess the core characteristics of Pitt-Hopkins syndrome (PTHS).
Despite its severely debilitating impact on patients, their parents, and siblings, no approved treatments for PTHS exist.
Neuren CEO Jon Pilcher said, "We are very excited about the results of this first clinical trial in Pitt Hopkins patients. This underserved community has such urgent unmet need and we can now continue towards our goal of developing a first approved treatment. We are very grateful to the people in the Pitt Hopkins community and at the trial sites in the United States who enabled the successful completion of this extremely challenging, but groundbreaking trial.
Dr Nancy Jones, Neuren's vice president of clinical development, added, "The consistent results on the PTHS specific assessments affirm the need for syndrome specific measurements in severe neurodevelopmental disorders where measures that were developed for broader populations may not be as appropriate. Neuren appreciates the collaboration of Dr Cassandra Newsom, Associate Professor at the University of Alabama, Birmingham (an investigator in the trial), the Pitt-Hopkins Research Foundation, and others who contributed to the development of these important measures."
