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Asceneuron SA passes US FDA's ODD for ASN120290 for treating progressive supranuclear palsy (PSP).

M2 PHARMA-July 19, 2018-Asceneuron SA passes US FDA's ODD for ASN120290 for treating progressive supranuclear palsy (PSP)


Biotech company Asceneuron SA reported on Wednesday the receipt of the US Food and Drug Administration's (FDA) Orphan Drug Designation for ASN120290 for the treatment of progressive supranuclear palsy (PSP).

PSP is a rare neurological condition that causes severe problems with walking, balance, speech, swallowing and vision as a result of the accumulation of aggregates of the tau protein in the brain. It is estimated that three to six people per 100,000 will develop PSP and there is currently no cure for the disease.

According to the company, ASN120290 is a selective inhibitor of the O-GlcNAcase enzyme. Based on its unique mechanism of action, the molecule has the potential to become a first in class treatment for PSP and other tau-related dementias. The neurofibrillary tangles are widely recognized as a key driver of neurodegeneration and clinical symptoms in the majority of dementia cases, including Alzheimer's disease.

In conjunction, the company has completed a randomized, double-blind, placebo-controlled phase I study to assess its safety and tolerability of single and multiple doses of ASN120290 in healthy young and elderly volunteers.

Additionally, the data from that study will be presented at the upcoming Alzheimer's Association International Conference (AAIC) to be held in Chicago from 22-26 July 2018.

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Publication:M2 Pharma
Date:Jul 19, 2018
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