Toleranzia AB (publ) announced that the results of preclinical studies with the Company's most advanced drug candidate TOL2, which is being developed for the autoimmune disease myasthenia gravis, have been published in the scientific journal Frontiers in Immunology. The study results show that intravenous treatment with TOL2 effectively and sustainably reduces all disease symptoms in a preclinical myasthenia gravis model. A comparative analysis also showed that the treatment effect of the drug candidate far exceeded that of two established treatments for myasthenia gravis.

Myasthenia gravis is a progressive autoimmune disease that occurs when the body mistakenly attacks and breaks down the acetylcholine receptors in the body's muscles. When the receptors are destroyed, signal transmission between the nervous system and the muscles is affected, and the patient's muscle function gradually deteriorates. In severe cases, the disease can lead to life-threatening respiratory muscle weakness.

Current treatments are only symptomatic and the medical need for safe and effective therapies is therefore huge. Toleranzia is developing the drug candidate TOL2, which is a modified form of the acetylcholine receptor. By adding the modified form of the acetylcholine receptor, the patient's immune system is stimulated to re-tolerate the receptors and thus stop attacking them.

In the current study, researchers administered TOL2 intravenously in an experimental autoimmune disease model of myasthenia gravis (EAMG) to investigate the effect on muscle strength, muscle levels of acetylcholine receptors and signal transduction. The study results showed that the therapy generated a normalization of all endpoints tested, with a robust and sustained reduction in disease symptoms over time as a result. Treatment response was evident in early as well as established and advanced disease.

A comparative analysis also showed that Toleranzia's drug candidate provides a superior treatment effect compared to the two therapies that make up the current standard of care for myasthenia gravis. The study also documented a favorable safety profile of the drug candidate, in terms of good tolerability and absence of toxicity.