- Neuren Pharmaceuticals (NEU) receives approval from the US Food and Drug Administration to proceed with the Phase 2 trial of NNZ-2591 in Angelman Syndrome
- The trial will be conducted at three hospitals across Australia, enrolling up to 20 children aged between three and 17 to examine the safety, tolerability and efficacy of NNZ-2591
- Angelman Syndrome is a genetic disorder that causes developmental disabilities and nerve-related symptoms such as speech impairment and seizures
- Neuren is developing NNZ-2591, which aims to mimic the normal function of the brain and help control and normalise the biological process
- Shares in Neuren are up 3.49 per cent on the market and are trading at $3.85
Neuren Pharmaceuticals (NEU) has received approval from the US Food and Drug Administration to proceed with the Phase 2 trial of NNZ-2591 in Angelman Syndrome.
The Phase 2 trial will be conducted at three hospitals across Australia, enrolling up to 20 children aged between three and 17 to examine the safety, tolerability, efficacy and pharmacokinetics of NNZ-2591.
Beginning the trial is subject to Ethics Committee approval with results of the trial expected in H1 2023.
Neuren will also undertake the foundational work to prepare for phase three development.
“FDA approval of the first IND for NNZ-2591 is a huge step forward in Neuren’s plan to develop NNZ-2591 for four serious neurodevelopmental disorders,” CEO Joh Pilcher commented.
“The Angelman syndrome Phase 2 trial in Australia has been keenly awaited by the
Angelman community and we are very excited to be able to proceed.
“We also now anticipate receiving clearance next month of our IND applications for similar Phase 2 trials in Phelan-McDermid and Pitt Hopkins syndromes, subject to completion of those FDA reviews.”
Angelman Syndrome is a genetic disorder that causes developmental disabilities and nerve-related symptoms.
It isn’t usually detected until development delays become noticeable, usually when a baby is around six to 12 months old.
Symptoms include lack of crawling or babbling, minimal speech, frequent smiling or laughing and the inability to walk, move or balance well.
Other symptoms include drooling, seizures and sleep disorders.
Current treatments include anti-seizure medications as well as physical, behavioural and occupational therapies.
Neuren is developing NNZ-2591, which aims to mimic the normal function of the brain and help control and normalise the biological process.
Shares in Neuren were up 3.49 per cent on the market and were trading at $3.85 at 12:35 pm AEDT.