The Neuren Pharmaceuticals Ltd (ASX: NEU) share price is on course to finish the week on a very positive note.
In morning trade the biopharmaceutical company's shares rocketed a massive 50% higher to $1.55 following the release of two announcements.
What was announced?
This morning the company provided an update on its drug candidate NNZ-2591.
According to the update, treatment with its drug candidate NNZ-2591 has normalised all deficits in a pre-clinical model of both Angelman syndrome (AS) and the neurodevelopmental disorder Pitt Hopkins syndrome (PTHS).
Management anticipates that both AS and PTHS meet the criteria for Orphan Drug designation. It also notes that there are currently no treatments specifically for either syndrome.
Pitt Hopkins syndrome.
PTHS is a neurodevelopmental condition affecting both males and females and is caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18. It has been estimated to impact as many as 1 in 11,000 people.
The release explains that NNZ-2591 was tested in the tcf4 mutation mouse model, which exhibits features of PTHS in humans, comparing normal mice and mice with a disrupted tcf4 gene.
In the mice with a disrupted gene, treatment with NNZ-2591 for six weeks 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.
Angelman syndrome.
AS is a neurodevelopmental condition estimated to affect 1 in 15,000 people, both males and females. It is caused by a deletion or mutation in the ubiquitin protein ligase E3A (UBE3A) gene on chromosome 15.
NNZ-2591 was tested in the ube3a knockout mouse model. This resembles features of Angelman syndrome in humans and includes motor deficits, learning problems, and alterations in synaptic connectivity and plasticity.
The study compared normal mice and mice with a disrupted gene. In the knockout mice, treatment with NNZ-2591 for six weeks normalised the deficits in all the tests of anxiety, daily living, sociability, motor performance, and cognition. It also eliminated seizures. Once again, all positive confirmatory measures were statistically significant.
Neuren isn't the only pharmaceutical company pushing higher today. In late morning trade the CSL Limited (ASX: CSL) share price is up almost 2% and the Clinuvel Pharmaceuticals Limited (ASX: CUV) share price is up 3.5%.
