Oral OGA Inhibitor Fast Tracked for Progressive Supranuclear Palsy

The Food and Drug Administration (FDA) has received Fast Track designation for FNP-223 for the treatment of progressive supranuclear palsy (PSP).

FNP-223, previously known as ASN90, is a reversible and substrate-competitive O-GlcNAcase enzyme inhibitor. The oral investigational drug is expected to rapidly increase O-GlcNAcylated tau proteins, thereby reducing abnormal aggregate tau proteins (neurofibrillary tangles [NFT]), commonly seen in PSP. 

The designation was supported by data from preclinical trials across human tauopathy mouse models. Findings showed that oral administration of FNP-223 reduced tau pathology (NFT formation), improved motor and breathing function, and increased survival in the brains of transgenic mice.  

FNP-223 is currently being investigated in the randomized, double-blind, phase 2 PROSPER trial (ClinicalTrials.gov Identifier: NCT06355531). The study is evaluating the safety, efficacy, and pharmacokinetics of FNP-223 in an estimated 220 adults aged 50 to 80 years old with possible or probable PSP-Richardson syndrome. 

Study participants will be randomly assigned to receive FNP-223 or matching placebo 3 times daily over 52 weeks. The primary endpoints include the change from baseline to week 52 in the Progressive Supranuclear Palsy Rating Scale score, as well as the number of participants experiencing treatment-emergent adverse events and serious adverse events.  

“Receiving Fast Track designation is a significant milestone in our journey to provide a transformative treatment for PSP,” said Oscar Pérez, Chief Scientific Officer of Ferrer. “We are excited to advance our research and hopefully offer a new therapeutic option earlier for patients living with this challenging condition.”

This article originally appeared on MPR