The University of Alabama at Birmingham (UAB) in the US has announced the completion of a Phase 2 trial of Neuren Pharmaceuticals’ NNZ-2591, demonstrating promising outcomes in treating Pitt-Hopkins syndrome (PTHS).

The mid-stage clinical trial was directed by the university’s researchers.

NNZ-2591, which targets IGF-1 receptors, showed significant improvements in participants as observed by clinicians and caregivers.

Specifically, enhancements in communication, social interaction, cognition, and motor abilities were observed.

Cassandra Newsom, director of the translational research core at Civitan International Research Center, UAB, spearheaded the Phase 2 trial of NNZ-2591.

The open-label study included 11 children aged three to 17 from five hospitals in the US, including UAB. It evaluated safety, tolerability, and pharmacokinetics throughout a 13-week treatment period.

NNZ-2591 was administered orally as a liquid twice daily, with a gradual increase to a target dose of 12mg/kg over the first six weeks of treatment.

The trial started with at least four weeks of screening and observation, followed by a treatment period of 13 weeks.

At UAB, the first site for screening and enrolling participants in the drug trial, all four qualifying participants completed the study.

The study involved the alignment between caregiver ratings of improvement and those of clinicians.

The primary endpoints of this trial were to evaluate the safety, tolerability, and pharmacokinetics of NNZ-2591 in children with PTHS.

Secondary endpoints included four efficacy measures specifically tailored for PTHS, assessed by both clinicians and caregivers.

According to Newsom, the positive findings will help develop the first approved treatment for PTHS.

The researcher’s team is now planning to get approval for a larger Phase 3 double-blind, placebo-controlled trial.

Newsom anticipates that UAB will again serve as a trial site in this next phase of research.

Australia-based Neuren Pharmaceuticals is advancing NNZ-2591 for several severely debilitating neurological disorders with no or limited approved treatment options.

In December 2023, Neuren Pharmaceuticals reported encouraging outcomes from a Phase 2 trial focused on Phelan-McDermid syndrome.

The company anticipates releasing top-line results from a Phase 2 trial investigating NNZ-2591’s efficacy in Angelman syndrome in Q3 2024.

Neuren Pharmaceuticals’ first drug, Daybue (trofinetide), is US Food and Drug Administration (FDA)-approved for Rett syndrome.