Medicure Unveils Phase 3 Study Data at European Epilepsy Congress

Medicure Inc. has revealed the design and baseline data of its Phase 3 MEND-PNPO study at the 16th European Epilepsy Congress. This pivotal trial investigates MC-1, a pharmaceutical-grade oral formulation of pyridoxal 5'-phosphate (P5P), for treating Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency. MC-1 is not FDA-approved, and its safety and effectiveness remain unconfirmed.

The phase 3, open-label study spans 52 weeks across multiple centers, aiming to enroll about 10 patients with genetically confirmed PNPO deficiency. Current enrollment stands at nine patients, with three having completed the one-year trial. Baseline P5P levels in participants range from 59.8 to 165 ng/mL.

PNPO deficiency, a rare inherited neurometabolic disorder, presents severe seizures in infants. Lifelong P5P therapy is required, but no approved pharmaceutical-grade product exists. Medicure will also share its findings at upcoming conferences in North America.