Clinical Testing of Drug NNZ-2591 for Angelman Syndrome Commences in Australia (Phase 2)
Neuren Pharmaceuticals, a biopharmaceutical company, is conducting a Phase 2 trial for their investigational oral therapy NNZ-2591, aimed at treating Angelman syndrome. The trial, which is currently recruiting pediatric patients, is being held at three hospitals: one each in Queensland, New South Wales, and Victoria.
The open-label and dose-escalating study (NCT05011851) plans to enroll up to 20 children, ages 3 to 17, with a confirmed diagnosis of Angelman syndrome. The trial's main goal is to assess NNZ-2591's safety and tolerability, and its pharmacokinetics.
NNZ-2591 has received regulatory clearance in both Australia and the U.S. Dosing will gradually increase during the trial's first six weeks, subject to safety and tolerability, until a target dose of 50 milligrams per milliliters (mg/mL) is reached. Following an initial four-week observation and assessment period, all participants will be given NNZ-2591 as an oral solution twice daily for 13 weeks (about three months).
Professor Sarah H. B. Wright, the head of the Center for Clinical Studies in Rare Neurodevelopmental Disorders at Children's Health Queensland Hospital in South Brisbane, Queensland, is leading the study.
Findings in the Phase 2 trial are expected to aid in the design of a larger and registrational trial. Top-line results from the trial are expected in May 2023.
In preclinical studies with genetically engineered mice lacking the UBE3A gene, treatment with NNZ-2591 improved cognition and motor function, reduced anxiety, and prevented seizures. Additional or secondary goals are exploratory measures of treatment efficacy, completed by clinicians and caregivers. These include whether the patient has improved or worsened with treatment using various Angelman syndrome-specific Clinical Global Impression scales, and caregivers' impressions of change.
NNZ-2591 is a compound with a chemical structure similar to the cyclic glycine proline. It is intended to restore normal communication and signaling between brain cells. Any future application seeking NNZ-2591's approval to treat Angelman syndrome will require a registrational trial.
This follow-up assessment will be conducted two weeks after finishing treatment. The trial will not be a placebo-controlled study, meaning all participants will receive the drug.
Angelman syndrome is a rare genetic disorder characterised by severe intellectual disability, speech impairment, and frequent seizures. There is currently no cure for the condition. The development of NNZ-2591 offers hope for families affected by this disorder.
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