Australian biopharmaceutical company Neuren Pharmaceuticals has announced positive top-line data from its Phase II trial of NNZ-2591, a treatment for children with Pitt Hopkins syndrome (PTHS).
PTHS is a neurodevelopmental condition caused by the loss in a single copy or a mutation of the TCF4 gene on chromosome 18.
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The open label trial enrolled 16 children aged three to 17 years at five US hospitals.
It assessed NNZ-2591’s safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment.
The medication was administered orally twice daily, with dosage increased to 12mg/kg during the first six weeks subject to safety and tolerability reviews.
The trial began with a minimum four-week screening period to establish baseline characteristics before the treatment phase. A follow-up assessment was held two weeks post-treatment.
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By GlobalDataSafety, tolerability and pharmacokinetics were the trial’s primary endpoints, while its secondary endpoints included efficacy measures tailored to PTHS and others used in various neurodevelopmental conditions.
Clinicians and caregivers observed statistically significant improvements from baseline across all four PTHS-specific efficacy measures. The mean improvement for each measure was also statistically significant.
Nine out of 11 children showed improvement on the PTHS Clinical Global Impression of Improvement (CGI-I), while eight demonstrated improvement on the PTHS Caregiver Overall Impression of Change (CIC).
In addition, six out of 11 children showed improvement on the PTHS Clinical Global Impression of Severity (CGI-S).
NNZ-2591 was well-tolerated and had a good safety profile, with all treatment-emergent adverse events being mild to moderate and most deemed unrelated to the study drug.
Neuren CEO Jon Pilcher said: “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.”
