Neuren reports progress in Pitt Hopkins syndrome trial

Neuren Pharmaceuticals revealed promising findings from its Phase II trial of NNZ-2591 for children with Pitt Hopkins syndrome (PTHS). The study indicated significant improvements from baseline across all four efficacy measures specifically tailored to evaluate PTHS's core characteristics. CEO Jon Pilcher stated this marks a crucial step towards developing the first approved treatment for PTHS.

The open-label trial included 16 children, averaging nine years old, who received NNZ-2591 as an oral liquid dose twice daily over 13 weeks. Researchers assessed safety, tolerability, pharmacokinetics, and efficacy, comparing baseline characteristics with those observed two weeks post-treatment. Primary endpoints focused on safety, tolerability, and pharmacokinetics, while secondary endpoints encompassed four PTHS-specific efficacy measures and ten additional measures used in other neurodevelopmental conditions.

Out of the 16 participants, 11 completed the trial. Results showed NNZ-2591 was well tolerated, featuring a commendable safety profile. Treatment-emergent adverse events (TEAEs) were generally mild to moderate, with most unrelated to the study drug. Importantly, no serious TEAEs were reported, and there were no significant trends in laboratory values or other safety parameters throughout the treatment period.

Regarding efficacy, the trial demonstrated significant improvement in the four measures designed specifically for PTHS, contrasting with the ten measures applicable to other neurodevelopmental conditions, which did not show similar significant improvements. Clinically, the mean Clinical Global Impression of Improvement (CGI-I) was 2.6, with nine out of eleven children showing improvement according to clinician assessments. The Caregiver Overall Impression of Change (CIC) had a mean score of 3.0, with eight of eleven children exhibiting improvement as noted by clinicians.

Additionally, the PTHS-specific Clinical Global Impression of Severity (CGI-S) improved for six children, with three improving from a severity score of 6 to 5, and three from 5 to 4. Caregivers also observed positive changes; eight children showed improvement in the Caregiver Top 3 Concerns overall score, which covers areas such as communication, self-care, behavior, and motor skills.

Investigator Elliott Sherr expressed optimism about the results specific to PTHS. He highlighted that the mechanism of action of NNZ-2591 aligns with the positive responses observed in PTHS patients, suggesting its potential efficacy in treating other significant neurodevelopmental disorders.

The study's outcomes are encouraging, signaling a potential breakthrough in treating Pitt Hopkins syndrome and possibly other neurodevelopmental conditions. Neuren Pharmaceuticals aims to continue their work towards securing approval for NNZ-2591, thereby providing a much-needed therapeutic option for individuals affected by PTHS.