Investigational Tau Modifier Receives Orphan Drug Status for Progressive Supranuclear Palsy


The Food and Drug Administration (FDA) has granted an orphan drug designation to ASN120290 (Asceneuron SA, Lausanne, Switzerland) for the treatment of progressive supranuclear palsy (PSP). In preclinical studies, ASN120290 substantially reduced the build-up of toxic tau aggregates and formation of neurofibrillary tangles, thought to be a key driver of neurodegeneration in multiple forms of dementia, including Alzheimer’s disease and Parkinson’s disease.

ASN120290 is a selective inhibitor of the glycoside hydrolase, O-GlcNAcase, with the potential to become a first-in class-treatment for PSP and other neurodegenerative diseases in which tau plays a pathophysiology role.

A randomized, double-blind, placebo-controlled phase 1 trial of ASN120290 study assessing safety and tolerability of single and multiple doses in healthy volunteers has been completed. Data from that study will be presented at the upcoming Alzheimer's Association International Conference (AAIC) in Chicago July 22-26, 2018.

Dirk Beher, Chief Executive Officer and a founder of Asceneuron, commented: "PSP is a rare neurological condition for which there is currently no treatment available. ASN120290 is an orally bioavailable molecule that has the potential of treating the root cause of the neurodegeneration. (This) is an important milestone for the team and the company."

Orphan drug status is intended to advance drug development for rare diseases and is granted to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases. Orphan drug development includes FDA assistance in clinical trial design and an exemption from FDA user fees.


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