Ferrer Receives FDA Fast Track Designation for FNP-223 in Progressive Supranuclear Palsy (PSP)

Ferrer, a B Corp-certified international pharmaceutical company, has announced that FNP-223, a novel therapy in-licensed from Asceneuron and aimed at slowing the development of progressive supranuclear palsy (PSP), has received Fast Track designation from the US Food & Drug Administration (FDA). FNP-223, a new molecular entity in active development for PSP, is in an ongoing Phase 2 study to evaluate its safety, efficacy, and pharmacokinetics in adult patients with possible or probable PSP-Richardson syndrome (PSP-RS), the most common clinical variant of this neurodegenerative disease¹.

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Ferrer's pharmaceutical production plant in Sant Cugat del Vallés, Barcelona, Spain.

“We are thrilled to receive Fast Track designation from the FDA for FNP-223 in the treatment of PSP. Consistent with our purpose of using business to fight for social justice, we are committed to advancing this promising therapy as quickly as possible to benefit as many patients as possible,” said Mario Rovirosa, Chief Executive Officer of Ferrer.

Fast Track designation is a significant milestone in the drug development process. It is a program that offers the possibility of having more frequent meetings with the FDA to discuss the drug’s development, eligibility for Accelerated Approval and Priority Review if relevant criteria are met.

“This designation underscores the importance of expediting the development and review of FNP-223 to address critical unmet needs in patients with this rare and devastating disease,” said Marta Parmar, Ferrer’s Chief Quality, Regulatory and Pharmacovigilance Officer.

Progressive supranuclear palsy manifests in patients with symptoms such as difficulty speaking, imbalance, changes in gait, cognitive problems^2-4. PSP has a prevalence of approximately 5 cases per 100,000 people and primarily affects individuals over the age of 60³. The disease's etiology is believed to be related to the abnormal accumulation of tau proteins in certain areas of the brain, leading to neurodegeneration^3,4. Preclinical models have demonstrated that FNP-223 can prevent the abnormal accumulation of tau proteins in neurons⁵. Ferrer now aims to show that this molecule is safe and effective in patients with PSP.

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

About FNP-223

FNP-223 is a new orally administered chemical compound that functions as a reversible and substrate-competitive inhibitor of the O-GlcNAcase (OGA) enzyme⁵. Mechanistically, FNP-223 binds to the active site of OGA enzyme. As a result, the inhibitor prevents the substrate from accessing the catalytic pocket, thereby impeding the removal of O-GlcNAc modifications from natural client proteins such as the tau protein. Inhibiting O-GlcNAcase is expected to cause a rapid increase of O-GlcNAcylated (glycosylated) tau proteins, ultimately leading to a reduction in abnormal aggregated tau as neurofibrillary tangles (NFT) over a certain period⁵.

Bibliography:

1. 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.

2. 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.

3. 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/.

4. Rowe JB, Holland N, Rittman T. Progressive supranuclear palsy: diagnosis and management. Pract Neurol. 2021;21(5):376-383. doi: 10.1136/practneurol-2020-002794.

5. 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 Proteinopathies. ACS Chem Neurosci. 2022 Apr 20;13(8):1296-1314. doi: 10.1021/acschemneuro.2c00057.

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