- Neuren Pharmaceuticals (NEU) has added Prader-Willi syndrome to its development pipeline for its NNZ-2591 treatment
- This announcement follows a study in a pre-clinical model that showed compelling effects of treatment on key symptoms of Prader-Willi syndrome
- Prader-Willi syndrome is a rare genetic condition, characterised by insatiable hunger, obesity, diabetes, weak muscles, intellectual disabilities and behavioural problems
- The study was conducted with mice which mimic the behaviour and metabolic profile of Prader-Willi syndrome in humans
- With the help of NNZ-2591, behavioural deficits in the mice were eliminated, and issues of obesity and excessive insulin were also reduced to normal
- The company plans to submit applications for orphan drug designation in the U.S. and Europe
- Company shares are down 3.57 per cent, trading at $1.45
Neuren Pharmaceuticals (NEU) has added Prader-Willi syndrome to its development pipeline for its NNZ-2591 treatment.
Prader-Willi syndrome is a rare genetic condition that affects between one in 10,000 to 30,000 people worldwide, characterised by insatiable hunger, obesity, diabetes, weak muscles, intellectual and learning disabilities and behavioural problems.
This announcement follows a study in a pre-clinical model that showed compelling effects of treatment on key symptoms of Prader-Willi syndrome.
The study was conducted with mice which mimic the behaviour and metabolic profile of Prader-Willi syndrome in humans. Six groups of ten mice were each dosed orally every day for six weeks with either NNZ-2591 or a placebo.
Behavioural tests examined hypoactivity, daily living, social interaction, cognition and anxiety. Obesity was examined by measuring fat mass and blood levels of insulin.
With the help of NNZ-2591, behavioural deficits in the mice were eliminated, and issues of obesity and excessive insulin were also reduced to normal.
NNZ-2591 had no impact on normal mice but consistently treated impaired animals successfully.
“These excellent results have once again reinforced the potential for NNZ-2591 to make a difference across multiple neurodevelopmental disorders in which signalling between brain cells and IGF-1 metabolism are impaired,” said Neuren CEO Jon Pilcher.
“We look forward to working with Prader-Willi families and clinicians, as we are already doing with PhelanMcDermid, Angelman and Pitt Hopkins, to bring this potential treatment to fruition,” he added.
The company plans to submit applications for orphan drug designation in the U.S. and Europe after receiving designation for Phelan-McDermid, Angelman and Pitt Hopkins syndromes in both areas following similarly compelling pre-clinical model results.
Foundational development already completed for the treatment should enable phase two trials to go ahead for Prader-Willi syndrome later in 2021.
Company shares are down 3.57 per cent, trading at $1.45 at 10:45 am AEDT.