Medicure Presents MEND-PNPO Phase 3 Study Design and Baseline Data at the 16th European Epilepsy Congress

WINNIPEG, MB / ACCESS Newswire / September 8, 2026 / Medicure Inc. ("Medicure" or the "Company") (TSXV:MPH)(OTC PINK:MCUJF), a company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in the United States market, today announced that the design, rationale and baseline data from its MEND-PNPO Phase 3 study (ClinicalTrials.gov NCT04706013) are being presented at the 16th European Epilepsy Congress (EEC), taking place September 5-9, 2026, at the Megaron Athens International Conference Centre in Athens, Greece. MEND-PNPO is a pivotal trial evaluating MC-1 for the treatment of Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency.

The poster, titled "Pharmaceutical Grade Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency: MEND-PNPO Study Design and Rationale," is presented by lead investigator Dr. Phillip Pearl (Director of Epilepsy and Clinical Neurophysiology at Boston Children's Hospital and William G. Lennox Chair and Professor of Neurology at Harvard Medical School, Boston, MA), together with co-investigators from study sites in the United States, Australia and Europe. The presentation outlines the study design, primary and secondary endpoints, MC-1 dosing regimens, and baseline pyridoxal 5'-phosphate (P5P) levels among enrolled patients.

MEND-PNPO is a 52-week, Phase 3, open-label, multi-center, externally controlled study evaluating MC-1, a pharmaceutical-grade oral formulation of P5P, in patients aged 2 years and older with genetically confirmed PNPO deficiency. Enrollment began in January 2023 and is expected to be completed in 2026. As presented at EEC, 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. Medicure aims to enroll approixamtely 10 patients with confirmed PNPO deficiency in the trial. Please note, MC-1 is not FDA-approved. The safety and effectiveness of MC-1 has not been established.

PNPO deficiency is an ultra-rare inherited neurometabolic disorder caused by mutations in the PNPO gene, resulting in an inability to produce adequate levels of pyridoxal 5'-phosphate (P5P), the active form of vitamin B6. The condition commonly presents in newborns and infants with severe, difficult-to-control seizures and can lead to significant developmental impairment, neurological dysfunction, and death if untreated. Lifelong P5P therapy is typically required, yet no pharmaceutical-grade P5P product is approved.

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