Barcelona, June 17, 2025 – We are pleased to announce that FNP-223, an innovative therapy licensed from Asceneuron and intended to slow the progression of progressive supranuclear palsy (PSP), has received the designation of an accelerated evaluation procedure or "fast-track" by the U.S. Food and Drug Administration (FDA). FNP-223, a new chemical entity in active development for PSP, is currently in a Phase 2 study to evaluate its safety, efficacy, and pharmacokinetics in adult patients with probable or possible PSP-Richardson (PSP-RS), the most common clinical variant of this neurodegenerative disease¹.
"We are thrilled to receive the FDA's Expedited Evaluation Procedure designation for FNP-223 in the treatment of PSP. In line with our purpose of using the business to fight for social justice, we are committed to advancing this promising therapy as quickly as possible to benefit the greatest number of patients," stated Mario Rovirosa, CEO of Ferrer.
The accelerated evaluation procedure designation represents an important milestone in the drug development process. This is a program that allows for more frequent meetings with the FDA to address drug development, as well as access, if certain criteria are met, to accelerated approval and priority review.
"This designation underscores the importance of accelerating the development and review of FNP-223 to address critical unmet needs in patients with this rare and debilitating disease," said Marta Parmar, Ferrer's Director of Quality, Regulatory Affairs and Pharmacovigilance.
Progressive supranuclear palsy manifests itself with symptoms such as speech difficulties, loss of balance, gait disturbances and cognitive problems²,⁴. PSP has a prevalence of approximately 5 cases per 100,000 people and mainly affects people over 60 years of age³. Its etiology is thought to be related to the abnormal accumulation of Tau proteins in certain areas of the brain, leading to neurodegeneration³,⁴. Preclinical models have shown that FNP-223 can prevent such abnormal accumulation of Tau in neurons⁵. Ferrer is now seeking to demonstrate that this molecule is safe and effective in patients with PSP.
Oscar Pérez, Ferrer's Chief Scientific Officer, also expressed his enthusiasm: "Receiving the 'fast-track' designation is an important milestone in our journey to deliver a transformative treatment for PSP. We are excited to continue advancing in our research and hope to offer, as soon as possible, a new therapeutic option to people living with this complex disease".
About FNP-223
FNP-223 is a novel orally administered chemical compound that functions as a reversible inhibitor and substrate-competitive of the O-GlcNAcase (OGA) enzyme⁵. As for its mechanism of action, FNP-223 binds to the active site of the OGA enzyme. As a result, the inhibitor prevents the substrate from accessing the catalytic pocket, preventing the removal of O-GlcNAc modifications from natural client proteins, such as Tau protein. Inhibition of O-GlcNAcase is expected to cause a rapid increase in Tau O-GlcNAcylated (glycosylated) proteins, ultimately leading to a reduction of abnormal aggregated tau in the form of neurofibrillary tangles (NFTs) over a certain period⁵.
Bibliography:
- ClinicalTrials.gov A Randomized, Double-blind, Placebo-controlled, Phase 2 Study to Assess the Efficacy, Safety, and Pharmacokinetics of FNP-223 (Oral Formulation) to Slow the Disease Progression of Progressive Supranuclear Palsy (PSP) (PROSPER). ClinicalTrials.gov [Internet]. Available from: https://www.clinicaltrials.gov/study/NCT06355531.
- Coughlin DG, Litvan I. Progressive supranuclear palsy: Advances in diagnosis and management. Parkinsonism Relat Disord. 2020 Apr;73:105-116. doi: 10.1016/j.parkreldis.2020.04.014. Epub 2020 May 25.
- Agarwal S, Gilbert R. Progressive Supranuclear Palsy. StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024. Available from:https://www.ncbi.nlm.nih.gov/books/NBK526098/
- Rowe JB, Holland N, Rittman T. Progressive supranuclear palsy: diagnosis and management.
- Permanne B, Sand A, Ousson S, Nény M, Hantson J, Schubert R, et al. D. O-GlcNAcase Inhibitor ASN90 is a Multimodal Drug Candidate for Tau and α-Synuclein Proteinopaties. ACS Chem Neurosci. 2022 Apr 20; 13(8):1296-1314. DOI: 10.1021/acschemneuro.2C00057.
