Toleranzia

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.
 
"We are pleased that the positive research results for our drug candidate TOL2 have now been published in a reputable scientific journal. The fact that we see a therapeutic effect in this preclinical trial model even in the late stages of myasthenia gravis, when the condition is fully established and progressive, is crucial for TOL2 to be developed into a drug that meets the huge medical need that the affected patients still have. The study results provide an important basis for the planning of the clinical trial that we intend to initiate next year," says Charlotte Fribert, CEO, Toleranzia AB.
 
The studies were conducted in collaboration with Toleranzia's research partners at the Hellenic Pasteur Institute, Greece, and Tzartos NeuroDiagnostic, Greece. The full article is available at the following link: research article.
 
For further information, please contact:
Charlotte Fribert - CEO, Toleranzia
Tel: +46 763 19 98 98
Email: charlotte.fribert@toleranzia.com
 
About Toleranzia AB (publ)
Toleranzia AB (publ) develops drugs that harness the power of the immune system for the treatment of autoimmune orphan diseases. The drugs, which target the cause of the disease, can cure or significantly alleviate the disease and not, like current treatments, merely reduce the symptoms. They have the potential to be the first long-acting or curative therapies that act specifically on the underlying cause of the autoimmune orphan disease for which they are being developed. Toleranzia's shares are listed on the Nasdaq First North Growth Market and Mangold Fondkommission AB, 08-503 015 50, CA@mangold.se, is the Company's Certified Adviser.