Toleranzia AB has announced the submission of a clinical trial application (CTA) for its drug candidate TOL2, aimed at treating myasthenia gravis. This marks a significant milestone for the company as it seeks to address the underlying cause of this rare autoimmune disease. The planned trial is a multi-center, placebo-controlled, first-in-human (FiH) study designed to evaluate the safety, tolerability, and preliminary efficacy of TOL2 in patients with myasthenia gravis.
Trial Design and Objectives
The Phase 1 trial will involve administering single and multiple ascending doses of TOL2 to myasthenia gravis patients. Clinical centers in Sweden, Denmark, and Germany will participate, with Clinical Trial Consultants (CTC) managing the trial. The primary objectives include assessing the safety and tolerability of TOL2, while secondary endpoints will explore preliminary efficacy signals. The trial aims to provide critical data on TOL2's potential as a disease-modifying therapy.
Manufacturing and Logistics
Large-scale manufacturing of lyophilized TOL2 drug product for the trial is currently underway at Curia Global in Scotland. ClinStorage AB will manage the labeling, storing, and distribution of the drug to the participating clinics, ensuring seamless logistics for the trial execution.
Management Commentary
"With this clinical trial application for TOL2, Toleranzia reaches its most important milestone to date," said Charlotte Fribert, CEO of Toleranzia. "Following regulatory approval, we are ready and eager to start the clinical trial as soon as possible in patients with myasthenia gravis, a patient group that is in great need of safer and more effective treatments."
Myasthenia Gravis: An Unmet Need
Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Current treatments primarily focus on managing symptoms rather than addressing the underlying cause. Toleranzia's TOL2 aims to provide a long-acting or curative treatment that specifically targets the autoimmune response driving the disease, potentially offering a more effective and sustainable solution for patients.
