In a Sept. 8, 2026 update, Medicure Inc. (TSXV: MPH; OTC Pink: MCUJF) presented data from its MEND-PNPO Phase 3 study at the 16th European Epilepsy Congress in Athens, Greece, held Sept. 5-9, 2026. The company also listed the Child Neurology Society Annual Meeting in Montreal, Quebec, Oct. 14-17, and the American Epilepsy Society Annual Meeting in Denver, Colorado, Dec. 4-8, as additional presentation venues.
MEND-PNPO is a 52-week, open-label, multi-center study evaluating MC-1, a pharmaceutical-grade oral formulation of pyridoxal 5'-phosphate, or P5P, in patients aged 2 years and older with genetically confirmed PNPO deficiency. The condition is an ultra-rare inherited neurometabolic disorder caused by mutations in the PNPO gene.
The study began enrollment in January 2023 and is expected to complete enrollment in 2026, with a target of about 10 patients. Medicure said nine patients have been enrolled to date and three have completed the one-year study. Baseline P5P treatment regimens ranged from 14 to 58 mg/kg/day, and baseline steady-state P5P levels ranged from 59.8 to 165 ng/mL.
MC-1 has not been approved by the U.S. Food and Drug Administration, and its safety and effectiveness have not been established. The company noted that no pharmaceutical-grade P5P product is currently approved.
The update named Dr. Phillip Pearl, director of epilepsy and clinical neurophysiology at Boston Children's Hospital and professor of neurology at Harvard Medical School. Study sites and investigators are located in the United States, Australia and Europe.
