WINNIPEG, CANADA – (September 23, 2025) Medicure Inc. (“Medicure” or the “Company“) (TSXV:MPH, OTC: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 an update on enrollment in the MEND-PNPO study (ClinicalTrials.gov NCT04706013), with six patients now enrolled including one having completed the 12-month study period. Medicure plans to enroll 10-15 patients in total and is targeting having all patients start enrollment before the end of 2025.
Medicure’s R&D focus is primarily on its Phase 3 study to seek approval of MC-1 as the first FDA-approved therapy for patients with Pyridox(am)ine 5′-Phosphate Oxidase (PNPO) deficiency, which is a rare neurometabolic disorder often leading to neonatal onset seizures that are resistant to other antiseizure medications, resulting in severe neurological dysfunction and ultimately death if untreated.
The FDA has granted both a Rare Pediatric Disease Designation and an Orphan Drug Designation to MC-1 for the treatment of seizures associated with PNPO deficiency. Additionally, the European Medicines Agency (“EMA”) has granted an Orphan Drug Designation to MC-1 for the treatment of PNPO deficiency. Medicure also received fast track designation for use of MC-1 for the treatment of PNPO deficiency, which will facilitate the review of Medicure’s new drug application (“NDA”) by the FDA.
Upon approval of the NDA for MC-1 for patients with PNPO deficiency, Medicure is eligible to receive a priority review voucher from the FDA, which can be redeemed or sold, and provides significant value.
Elements of the MEND-PNPO study as described below will be presented at the 54th Child Neurology Society Conference in Charlotte, North Carolina on October 8, 2025 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) and co-authored by the study principal investigators.
The presentation includes the study design, primary and secondary endpoints, dosing of MC-1 for patients enrolled, and baseline steady state pyridoxal 5′-phosphate (P5P) levels. Based on enrolled patients, baseline P5P treatment regimens ranged between 14 mg/kg/day to 58 mg/kg/day, and baseline steady state P5P levels were also variable between subjects ranging from 4.84 ng/mL to 136 ng/mL.
“We sincerely thank all patients and their families, along with our clinical investigators for their participation in the MEND-PNPO study. The enrollment to date, along with the presentation of the initial data at the 54th Child Neurology Society conference, represent significant milestones as we drive towards completion of the study and our NDA filing”, said Albert D. Friesen, Chief Executive Officer of Medicure and Chair of its Board of Directors.
About Medicure Inc.
Medicure is a company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in the United States market. The present focus of the Company is the marketing and distribution of AGGRASTAT® (tirofiban hydrochloride) injection and ZYPITAMAG® (pitavastatin) tablets in the United States, where they are sold through the Company’s U.S. subsidiary, Medicure Pharma Inc. Medicure also operates Marley Drug Inc. (“Marley Drug”), a pharmacy subsidiary servicing all 50 states, Washington D.C. and Puerto Rico. Marley Drug® is committed to improving access to medications for all Americans together with exceptional customer service and free home delivery. Medicure also operates Gateway Medical Pharmacy (“Gateway”), located in Portland, Oregon in a medical office building near major transportation lines and multiple healthcare clinics and centers. In addition to regular customers, the pharmacy services multiple long-term care facilities and provides non-sterile compounding services. Medicure also operates West Olympia Pharmacy (“West Olympia”), located in Olympia, Washington in a medical office complex near multiple clinics. For more information visit www.marleydrug.com. For more information about Medicure please visit www.medicure.com. For additional information about AGGRASTAT®, please visit www.aggrastat.com or refer to the full Prescribing Information. For additional information about ZYPITAMAG®, please visit www.zypitamag.com or refer to the full Prescribing Information.