Neuren commences enrolment new Phase 2 trials of NNZ-2591

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Neuren Pharmaceuticals (ASX:NEU) has announced its Phase 2 clinical trials of NNZ-2591 in children with each of Phelan-McDermid syndrome (PMS) and Pitt Hopkins syndrome (PTHS) are open for enrolment.

The company said the first subjects are expected to enter the trials imminently at Rush University Medical Center in Chicago.

The trials are being conducted in the US under approved Investigational New Drug applications (INDs) with the FDA. Top-line results from each trial are anticipated in the first half of 2023.

Neuren CEO Jon Pilcher said, “We are very excited to be working with the Phelan-McDermid and Pitt Hopkins communities in the United States to undertake these groundbreaking clinical trials. These seriously debilitating conditions have no approved medicines and we are eager to assess the potential impact of NNZ-2591, having observed highly encouraging effects in mouse models of each syndrome.”

The company said the open-label Phase 2 trials will each enrol a single group of up to 20 children to examine the safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591.

All subjects will receive NNZ-2591 as an oral liquid dose twice daily with titration up to the target dose during the first six weeks of treatment, subject to safety and tolerability. The treatment period is preceded by four weeks of observation to thoroughly examine the baseline characteristics prior to treatment against which safety and efficacy will be assessed for each child. A follow-up assessment will be made two weeks after the end of treatment.

The primary outcome measures for each trial are safety and tolerability, including the incidence, severity and frequency of adverse events, as well as measures of standard pharmacokinetic parameters. Secondary outcome measures include a range of exploratory efficacy measures for each syndrome, completed by clinicians and caregivers.