Medicure presents MEND-PNPO Phase 3 study design at 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.
Medicure will also present the MEND-PNPO study at two North American meetings later this year: the Child Neurology Society (CNS) Annual Meeting, taking place October 14-17, 2026 at the Palais des congrès de Montréal in Montréal, Québec, Canada, and the American Epilepsy Society (AES) Annual Meeting, taking place December 4-8, 2026 at the Colorado Convention Center in Denver, Colorado, USA.
"Presenting MEND-PNPO to the European epilepsy community is an important step in building awareness of PNPO deficiency and of the need for a standardized, pharmaceutical-grade P5P therapy," said Albert D. Friesen, President, Chief Executive Officer of Medicure and Chair of its Board of Directors. "We are grateful to the patients, families and investigators taking part in this study as we work to bring the first approved therapy for PNPO deficiency to patients in need."
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.
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AGGRASTAT ® (tirofiban hydrochloride) injection, ZYPITAMAG ® (pitavastatin) tablets, and Marley Drug ® are registered trademarks.
For more information, please contact:
Dr. Albert D. Friesen Chief Executive Officer Tel. 888-435-2220 Fax 204-488-9823 E-mail: info@medicure.com www.medicure.com
SOURCE: Medicure, Inc.
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