What happens when a committed community of parents and donors makes a ton of noise and never gives up? Pharma takes notice. Thrilled to be in the official pipeline of Neuren Pharmaceuticals as of TODAY, May 17! Their compound reversed multiple symptoms in Pitt Hopkins mice! Clinical Trials coming in 2020!

Neuren’s NNZ-2591 drug candidate alleviates Pitt Hopkins and Angelman syndrome symptoms

By Lorna Nicholas – May 17, 2019

Neuren Pharmaceuticals (ASX: NEU) has revealed positive pre-clinical results for its NNZ-2591 drug candidate on Pitt Hopkins and Angelman syndromes.

According to Neuren, its NNZ-2591 drug is a synthetic analogue of the neurotrophic peptide cyclic glycine proline, which occurs natural in the brain.

In addition to having a positive effect on Pitt Hopkins and Angelman, Neuren has also demonstrated the drug’s efficacy in pre-clinical models of Parkinson’s disease, stroke, traumatic brain injury, peripheral neuropathy, Fragile X, Phelan-McDermid, memory impairment and multiple sclerosis.

Pitt Hopkins syndrome

Pitt Hopkins syndrome is a neurodevelopment condition in males and females and is caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18.

The syndrome is characterised by developmental delays with moderate to severe intellectual disability, behavioural differences, hyperventilation, breath holding, seizures, lack of speech, sleep disturbance, gastrointestinal issues, distinctive facial features and stereotypic hand movements.

In these latest pre-clinical test, NNZ-2591 was trialled in the TCF4 mutation mouse model, which exhibits features of Pitt Hopkins in humans.

Normal mice were compared to those with a disrupted TCF4 gene, and its was found after six weeks, NNZ-2591 normalised the deficits in all the tests of hyperactivity, daily living, learning and memory, sociability, motor performance and stereotypy.

“All positive confirmatory measures were statistically significant,” the company stated.

Read more on this exciting news here.