FDA grants orphan drug designation to treatment of progressive supranuclear palsy

The US Food and Drug Administration (FDA) has granted orphan drug designation to Asceneuron’s treatment of progressive supranuclear palsy (PSP) — which is a rapidly progressing rare neurodegenerative disorder.

ASN120290 is a selective inhibitor of the O-GlcNAcase enzyme with a unique mechanism of action, giving it the potential to become a first-in-class treatment for PSP and other tau-related dementias.

Recent data from a randomised, double-blind, placebo-controlled Phase I study assessing the safety and tolerability of single and multiple doses of the inhibitor in healthy volunteers will be presented at the Alzheimer’s Association International Conference taking place in Chicago between 22–26 July.

“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,” commented Dirk Beher, chief executive officer and a founder of Asceneuron. “The granting of orphan drug designation for ASN120290 by the FDA is an important milestone for the team and the company. It strengthens our commitment to serving this important unmet medical need and bringing this molecule to patients.”

Asceneuron is an emerging, clinical stage biotech company focusing on the development of orally bioavailable therapeutics for debilitating neurodegenerative disorders with high unmet medical need, such as orphan tauopathies, Alzheimer’s and Parkinson’s diseases.