The Neuren Pharmaceuticals Ltd (ASX: NEU) The share price is en route to finish the week on a very positive note.
In the morning trade, the shares of the biopharmaceutical company exceeded 50% more at $ 1.55 after 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 candidate drug NNZ-2591 normalized all deficits in a pre-clinical model of Angelman syndrome (AS) and Pitt Hopkins syndrome of developmental disorder (PTHS).
Management expects that both AS and PTHS meet the criteria for the designation of orphan drugs. It also notes that there are currently no specific treatments for both syndromes.
Pitt Hopkins syndrome.
PTHS is a condition of neurological development that affects both males and females and is caused by the loss of a copy or mutation of the TCF4 gene on chromosome 18. It is estimated to have an impact on 1 in 11,000 people.
The communiqué explains that NNZ-2591 was tested in the tcf4 mutant mouse model, which presents characteristics of PTHS in humans, comparing mice and normal mice with a perturbed tcf4 gene.
In mice with a destroyed gene, treatment with NNZ-2591 for six weeks normalized deficits in all tests of hyperactivity, daily life, learning and memory, sociability, motor performance and stereotypy. All positive confirmation measures were statistically significant.
Angelman syndrome.
AS is a condition of neurodevelopment estimated at 1 in 15,000 people, both male and female. It is caused by a deletion or mutation in the E3A (UBE3A) gene of the ubiquitin ligase protein 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 mice and normal mice with a destroyed gene. In knockout mice, treatment with NNZ-2591 for six weeks normalized deficits in all tests of anxiety, daily life, sociability, motor performance and cognition. He also eliminated convulsions. Again, all positive confirmation measures were statistically significant.
Neuren is not the only pharmaceutical company that pushes higher today. In the late morning trade on CSL Limited (ASX: CSL) the share price has risen by almost 2% and the Clinuvel Pharmaceuticals Limited (ASX: CUV) The share price has increased by 3.5%.
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James Mickleboro has no position in any of the stocks mentioned. The Motley Fool Australian mother company Motley Fool Holdings Inc. owns shares in CSL Ltd. The Motley Fool Australia has no position in any of the securities mentioned. We Fools may not all have the same opinions, but we are all convinced that, considering a wide range of insights, we have become better investors. The Motley Fool has a disclosure policy. This article contains only general investment recommendations (with AFSL 400691). Authorized by Scott Phillips.
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