Neuren shows improvements in Pitt Hopkins syndrome study

2024-05-27

临床结果临床2期临床1期

Neuren Pharmaceuticals announced Monday that top-line results from its Phase II trial of NNZ-2591 in children with Pitt Hopkins syndrome (PTHS) showed improvement from baseline across all four efficacy measures designed to assess the core characteristics of the neurodevelopmental condition. "We can now continue towards our goal of developing a first approved treatment" for PTHS, remarked CEO Jon Pilcher.

The open label trial in 16 children with a mean age of nine years examined the safety, tolerability, pharmacokinetics and efficacy of NNZ-2591 treatment as an oral liquid dose twice daily for 13 weeks. Baseline characteristics were compared with those at two weeks post-treatment. The primary endpoints were safety, tolerability and pharmacokinetics. Secondary endpoints included four efficacy measures designed for PTHS assessed by both clinicians and caregivers, as well as ten efficacy measures that have been used in other neurodevelopmental conditions.

Eleven participants completed the trial. NNZ-2591 was well tolerated and showed a good safety profile. All treatment emergent adverse events (TEAEs) were mild to moderate and most were not related to study drug, with no serious TEAEs. Neuren noted "no meaningful trends in laboratory values or other safety parameters during treatment."

For efficiency, the mean improvement from baseline was significant for the four measures designed for PTHS but not for the ten measures used in other neurodevelopmental conditions. The mean Clinical Global Impression of Improvement (CGI-I) was 2.6, with nine of the 11 children showing improvement assessed by clinicians. The mean Caregiver Overall Impression of Change (CIC) score was 3.0, with eight of 11 children showing improvement as assessed by clinicians. The PTHS Clinical Global Impression of Severity (CGI-S) improved from 6 to 5 for three children and from 5 to 4 for three children. Meanwhile, eight children showed improvement measured by the Caregiver Top 3 Concerns overall score, which included communication, self-care, behaviour and motor skills.

Investigator Elliott Sherr said that "I am optimistic about the results in the PTHS specific measures. The mechanism of action of NNZ-2591 supports this response seen in PTHS and its potential in other highly impactful neurodevelopmental disorders."

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