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news.Ferrer's FNP-223 has secured fast-track designation from the US Food and Drug Administration (FDA), intended to slow down the development of progressive supranuclear palsy (PSP), a neurodegenerative disease.
The company’s pharmaceutical production plant in Spain. Credit: Ferrer/Business Wire.
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In-licensed from Asceneuron, this new molecular entity is currently undergoing a Phase II trial.
The trial aims to assess the therapy’s efficacy, pharmacokinetics, and safety in adults diagnosed with possible/probable PSP-Richardson syndrome (PSP-RS), which is the common variant of this condition.
Individuals with PSP are said to experience a range of debilitating symptoms, including speech difficulties, imbalance, gait changes, and cognitive issues.
FNP-223 acts as a reversible and substrate-competitive inhibitor of the O-GlcNAcase (OGA) enzyme.
By binding to the active site of this enzyme, the therapy prevents the substrate from reaching the catalytic pocket.
This action hinders the removal of O-GlcNAc modifications from proteins like tau. Blocking O-GlcNAcase is anticipated to lead to an increase in glycosylated tau proteins, which could result in a decrease in abnormal tau aggregation as neurofibrillary tangles over time.
According to Ferrer, this orally administered chemical compound could be able to prevent the abnormal build-up of tau proteins in neurons, as observed in preclinical models.
The company is currently focused on demonstrating the effectiveness and safety of this molecule in PSP subjects.
The fast track status enables frequent interactions with the US regulator, potentially leading to accelerated approval and priority review if the drug meets necessary criteria.
Ferrer CEO Mario Rovirosa said: “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.”
