Medicure Inc. presented the design, rationale, and baseline data from its MEND-PNPO Phase 3 study at the 16th European Epilepsy Congress in Athens, Greece. The pivotal trial evaluates MC-1, a pharmaceutical-grade oral formulation of pyridoxal 5'-phosphate (P5P), for treating Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency.

The poster presentation, led by Dr. Phillip Pearl of Boston Children's Hospital and Harvard Medical School, outlines the study's primary and secondary endpoints, dosing regimens, and patient baseline metrics. Co-investigators from sites across the United States, Australia, and Europe contributed to the analysis.

Study Enrollment and Baseline Metrics

MEND-PNPO is a 52-week, open-label, multi-center, externally controlled study targeting patients aged 2 years and older with genetically confirmed PNPO deficiency. Enrollment commenced in January 2023 and is projected to conclude in 2026. The company aims to enroll approximately 10 patients in total.

As of the presentation, nine patients had been enrolled, with three completing the one-year study duration. Baseline data revealed significant variability in prior treatment regimens and physiological markers among participants.

PNPO deficiency is an ultra-rare inherited neurometabolic disorder caused by mutations in the PNPO gene. This mutation prevents adequate production of P5P, the active form of vitamin B6. The condition typically manifests in newborns and infants as severe, difficult-to-control seizures, potentially leading to developmental impairment, neurological dysfunction, or death if untreated. Lifelong P5P therapy is standard, yet no pharmaceutical-grade P5P product is currently approved.

What the Numbers Show

The wide disparity between baseline treatment regimens (14-58 mg/kg/day) and resulting steady-state P5P levels (59.8-165 ng/mL) highlights the clinical challenge of managing PNPO deficiency without standardized pharmaceutical-grade therapy. This variability underscores the rationale for MC-1, which seeks to provide consistent dosing and absorption compared to current non-pharmaceutical alternatives.

Forward-Looking Developments

Medicure plans to present the MEND-PNPO study at two additional North American conferences later this year. These include the Child Neurology Society Annual Meeting in Montreal, Canada, from October 14-17, 2026, and the American Epilepsy Society Annual Meeting in Denver, Colorado, from December 4-8, 2026.

Albert D. Friesen, President and CEO of Medicure, stated that presenting to the European epilepsy community builds awareness of the need for standardized P5P therapy. He emphasized the company's goal to bring the first approved therapy for PNPO deficiency to patients.

MC-1 is not FDA-approved, and its safety and effectiveness have not been established.

Poly Medicure Limited shareholders approved all five resolutions at its 31st Annual General Meeting (AGM) held on September 5, 2026. The company conducted the meeting via video conference and other audio-visual means in compliance with the Companies Act, 2013.

The Board of Directors appointed Pawan Kumar Mishra, a practicing Company Secretary, as the scrutinizer for the remote e-voting and e-voting processes. Shareholders voted between September 2 and September 4, 2026, for remote e-voting, with additional voting available during the AGM.

Voting Participation

As of the record date, the company had 76,353 equity shareholders. A total of 91 shareholders attended the meeting through video conferencing. This included four promoters and promoter group members and 87 public shareholders.

Resolutions Approved

Shareholders approved the following agenda items:

• Adoption of audited standalone and consolidated financial statements for the year ended March 31, 2026.
• Declaration of dividend on equity shares for FY26.
• Reappointment of Rishi Baid as a director retiring by rotation.
• Reappointment of Devendra Raj Mehta as a non-executive non-independent director, having attained the age of 75 years.
• Approval of remuneration for M/s Jai Prakash & Company, Cost Accountants, as the cost auditor for FY27.

Voting Results Overview

The table below summarizes the voting outcomes for key resolutions:

All ordinary resolutions received requisite majority support. The special resolution regarding the reappointment of Devendra Raj Mehta also passed with the required special majority under Section 114(2) of the Companies Act, 2013.