Neuren's NNZ-259 Phase 2 Trial Demonstrates Significant Improvements In Pitt Hopkins Syndrome

(RTTNews) - Neuren Pharmaceuticals (NEU.AX) announced positive results from its Phase 2 clinical trial of NNZ-2591 in children with Pitt Hopkins syndrome (PTHS). Statistically significant improvement from baseline was observed by both clinicians and caregivers from treatment, across all 4 efficacy measures that were specifically designed to assess the core characteristics of PTHS.

The company noted that 9 out of 11 children that completed the trial showed improvement measured by the PTHS Clinical Global Impression of Improvement (CGI-I), an assessment by the clinician of the child's overall status compared with baseline. The mean CGI-I score was 2.6. Five children received a score of either 1 ("very much improved") or 2 ("much improved").

The company stated that 8 out of 11 children that completed the trial showed improvement measured by the PTHS Caregiver Overall Impression of Change (CIC), an assessment by the caregiver of the child's overall status compared with baseline. The mean CIC score was 3.0. Four children received a score of 2 ("much improved").

The company noted that 6 out of 11 children that completed the trial showed improvement measured by the PTHS Clinical Global Impression of Severity (CGI-S), an assessment by the clinician of the child's overall severity of illness, compared with the assessment at baseline. The CGI-S score improved from 6 to 5 for 3 children and from 5 to 4 for 3 children.

Eight out of eleven children that completed the trial showed improvement measured by the Caregiver Top 3 Concerns overall score, an individualised assessment by the caregiver of their child's most concerning symptoms. Language/Communication was the most commonly chosen concern, the company said.

The company noted that NNZ-2591 was well tolerated and demonstrated a good safety profile. All Treatment Emergent Adverse Events or TEAEs were mild to moderate and most were considered not related to study drug. There were no Serious TEAEs and no meaningful trends in laboratory values, electrocardiogram (ECG) or other safety parameters were observed during treatment.

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