Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration

Area of Science:

• Neuroscience
• Genetics
• Pharmacology

Background:

• Pantothenate kinase-associated neurodegeneration (PKAN) is a rare genetic disorder with no approved treatments.
• PKAN is characterized by progressive neurological decline, impacting daily functioning.

Purpose of the Study:

• To evaluate the efficacy of fosmetpantotenate in improving symptoms and stabilizing disease progression in PKAN patients.
• To assess the safety and tolerability of fosmetpantotenate in the PKAN population.

Main Methods:

• A randomized, double-blind, placebo-controlled, multicenter trial involving 84 patients with PANK2 pathogenic variants.
• Patients received either 300 mg oral fosmetpantotenate or placebo three times daily for 24 weeks.
• The primary endpoint was the change from baseline in the PKAN-Activities of Daily Living (PKAN-ADL) scale at week 24.

Main Results:

• No significant difference in PKAN-ADL scores was observed between the fosmetpantotenate and placebo groups at week 24 (P=0.9115).
• The incidence of treatment-emergent serious adverse events was comparable between the fosmetpantotenate (19.5%) and placebo (14.0%) groups.
• Six patients in the placebo group discontinued treatment due to disease progression or complications.

Conclusions:

• Fosmetpantotenate demonstrated a safe profile but did not show efficacy in improving functional outcomes in PKAN patients.
• The study concludes that fosmetpantotenate is not an effective treatment for PKAN based on the evaluated primary endpoint.