Medicure presents PNPO deficiency trial data at epilepsy congress

Medicure Inc., a company traded on the TSXV and OTC markets, showcased data from its Phase 3 MEND-PNPO study at the European Epilepsy Congress in Athens, Greece. The trial assesses MC-1, a pharmaceutical-grade oral P5P formulation, for treating PNPO deficiency in individuals aged 2 and above with a genetically confirmed diagnosis.

Dr. Phillip Pearl, a prominent figure in pediatric epilepsy, presented the study alongside colleagues from various international research centers. Enrollment began in January 2023, and as of now, nine patients have joined, with three completing the one-year study period. Baseline P5P levels ranged from 59.8 to 165 ng/mL, while treatment regimens varied from 14 to 58 mg/kg/day.

Medicure aims to recruit around 10 more participants. PNPO deficiency, an exceedingly rare genetic disorder, often manifests in infancy with severe seizures and potentially leads to developmental delays, neurological issues, and death if left untreated. Currently, no FDA-approved P5P products exist for this condition. Medicure plans to present the study at upcoming conferences in Montreal and Denver.

The company's mission is to develop and commercialize pharmaceuticals and healthcare products for the U.S. market.