MEDICURE ANNOUNCES UPDATE ON ENROLLMENT IN ITS MEND-PNPO PHASE 3 CLINICAL TRIAL AND PRESENTATION OF THE TRIAL DESIGN AND BASELINE DATA AT THE 2025 CHILD NEUROLOGY SOCIETY CONFERENCE

September 23, 2025

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 54^th 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 54^th 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.

To be added to Medicure's e-mail list, please visit:
http://medicure.mediaroom.com/alerts

Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward Looking Information: Statements contained in this press release that are not statements of historical fact, including, without limitation, statements containing the words "believes", "may", "plans", "will", "estimates", "continues", "anticipates", "intends", "expects" and similar expressions, may constitute "forward-looking information" within the meaning of applicable Canadian and U.S. federal securities laws (such forward-looking information and forward-looking statements are hereinafter collectively referred to as "forward-looking statements"). Forward-looking statements, include estimates, analysis and opinions of management of the Company made in light of its experience and its perception of trends, current conditions and expected developments, as well as other factors which the Company believes to be relevant and reasonable in the circumstances. Inherent in forward-looking statements are known and unknown risks, uncertainties and other factors beyond the Company's ability to predict or control that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements, and as such, readers are cautioned not to place undue reliance on forward-looking statements. Such risk factors include, among others, the Company's future product revenues, expected results, including future revenue from P5P, the likelihood of receiving a priority review voucher from the United State Food and Drug Administration, expected future growth in revenues, stage of development, additional capital requirements, risks associated with the completion and timing of clinical trials and obtaining regulatory approval to market the Company's products, the ability to protect its intellectual property, dependence upon collaborative partners, changes in government regulation or regulatory approval processes, and rapid technological change in the industry. Such statements are based on a number of assumptions which may prove to be incorrect, including, but not limited to, assumptions about: general business and economic conditions; the impact of changes in Canadian-US dollar and other foreign exchange rates on the Company's revenues, costs and results; the timing of the receipt of regulatory and governmental approvals for the Company's research and development projects; the availability of financing for the Company's commercial operations and/or research and development projects, or the availability of financing on reasonable terms; results of current and future clinical trials; the uncertainties associated with the acceptance and demand for new products and market competition. The foregoing list of important factors and assumptions is not exhaustive. The Company undertakes no obligation to update publicly or otherwise revise any forward-looking statements or the foregoing list of factors, other than as may be required by applicable legislation. Additional discussion regarding the risks and uncertainties relating to the Company and its business can be found in the Company's other filings with the applicable Canadian securities regulatory authorities or the US Securities and Exchange Commission, and in the "Risk Factors" section of its current Form 20F.

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

 

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