Neuren Pharmaceuticals completes enrolment in Pitt Hopkins syndrome Phase 2 trial

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Neuren Pharmaceuticals (ASX:NEU) has announced that the enrolment of subjects in its Phase 2 clinical trial of NNZ-2591 in Pitt Hopkins syndrome has been completed.

The company said it expects top-line results from the trial in the second quarter of 2024.

Neuren CEO Jon Pilcher said, “We are grateful to all the people who have contributed to achieving this important milestone as we strive to develop a potential first therapy for Pitt Hopkins syndrome, including the Pitt Hopkins community and the trial site teams in the United States. We now look forward to the remaining patients completing the trial and to releasing the first results of treatment with NNZ-2591 in children with Pitt Hopkins syndrome.”

The Phase 2 trial in children aged 3 to 17 at five US hospitals examines the safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591. All subjects receive NNZ-2591 as an oral liquid dose twice daily.

Pitt Hopkins syndrome is a neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18. It results in a range of developmental delays with moderate-to-severe intellectual disability and behavioural differences, hyperventilation and breath-holding while awake, seizures, gastrointestinal issues, lack of speech, sleep disturbance, stereotypic hand movements and distinctive facial features.

Neuren is also conducting Phase 2 clinical trials of NNZ-2591 in children with three other neurodevelopmental disorders – Phelan McDermid syndrome, Angelman syndrome and Prader-Willi syndrome.