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- Neuren Pharmaceuticals (NEU) is granted ethics approval to move ahead with its phase two clinical trial for a drug to treat Angelman syndrome
- The Human Research Ethics Committee’s approval covers three sites in Australia for the clinical trial of NNZ-2591 in Sydney, Brisbane and Victoria
- The phase two trial will enrol up to 20 children aged 3 to 17 years to examine safety, tolerability and efficacy of the drug over 13 weeks
- Separate research governance authorisation is now required from each site before they can be initiated and commence enrolment, with results expected in the first half of 2023
- Neuren Pharmaceuticals shares were down 0.9 per cent at $4.14
Neuren Pharmaceuticals (NEU) has been granted ethics approval to move ahead with its phase two clinical trial in Australia of a therapy for Angelman syndrome.
Angelman syndrome is characterised by severe developmental delay and learning disabilities, resulting in balance issues, motor impairment and debilitating seizures. There are currently no approved medicines for the disease, and patients require continuous, around-the-clock care.
The Human Research Ethics Committee’s approval covers three sites in Australia for the clinical trial of the drug NNZ-2591 based at hospitals in Sydney, Brisbane and Victoria.
The phase two trial will enrol up to 20 children aged 3 to 17 years with the disease to examine safety, tolerability, pharmacokinetics, and efficacy over 13 weeks of treatment, with the treatment administered orally.
“This is another important step achieved for the first clinical trial of NNZ-2591 in patients and Neuren’s first in Australia,” said Neuren CEO Jon Pilcher.
“We are very excited to be working closely with the local Angelman syndrome community and are eager to accelerate development of this potential therapy which has shown such promise to date.”
Separate research governance authorisation is now required from each site before they can be initiated and commence enrolment of subjects.
Results from the trial are expected in the first half of 2023.
Meanwhile, Neuren is also awaiting clearance from the US Food and Drug Administration for phase two trials of NNZ-2591 for both Phelan McDermid syndrome and Pitt Hopkins syndrome.
In parallel with the trials, Neuren is executing the foundational work to prepare for phase three development of NNZ-2591 across these neurodevelopmental disorders and Prader-Willi syndrome.
The company says the lead compound in NNZ-2591, trofinetide, has achieved positive results in a phase three clinical trial for Rett syndrome and has also completed a phase two clinical trial in Fragile X syndrome.
Neuren Pharmaceuticals shares were trading for $4.14, down 0.9 per cent, at 10:20am AEDT.
