Project description
Innovative tolerance technology for the treatment of autoimmune disease
Myasthenia gravis (MG) is one of the most common chronic autoimmune neuromuscular diseases that causes weakness in the skeletal muscles responsible for breathing and moving parts of the body. Currently, there is no cure for MG, which belongs to the so-called orphan (rare) diseases with an unmet medical need. Toleranzia is a Swedish pharmaceutical company that develops therapies directed at the intrinsic resources of the immune system to treat autoimmune diseases. Under the EU-funded TOL2 project it will address the need for a safe and effective treatment to cure MG. The tolerance technology behind the TOL2 project has the potential to be applied to therapies for other autoimmune diseases.
Objective
Myasthenia gravis (MG) is a rare disease affecting 2/10.000 people. Although being a rare disease, MG is the most common chronic autoimmune neuromuscular disease that causes weakness in the skeletal muscles, which are responsible for breathing and moving parts of the body, including the arms and legs. MG restricts patients from having a full participation in everyday activities, frequently leading to unemployment and reducing their quality of life. Many patients may eventually experience a myasthenic crisis, the treatment of which is very expensive. Such crises can lead to the death of the patient. There is currently no cure for MG and neither is there any cure in sight. Even though there are over 30 MG-related clinical trials currently in progress, most of them are only symptomatic treatments.
Considering this urgent medical need, we are developing TOL2, an innovative therapy with potential to cure MG (not only symptomatic treatment). This will be the first therapy for MG that induces long-term remission. TOL2 is based on a tolerance-technology with potential to be developed for treatment of other autoimmune diseases. TOL2 has been granted Orphan Drug Designation by the US FDA.
Toleranzia is a Swedish pharmaceutical research and development company. Our therapies utilize the immune system’s own intrinsic power to treat autoimmune diseases. Our mission is to develop innovative therapies that will cure autoimmune diseases.
Our business strategy for TOL2 is to complete clinical phase IIa, and then to look for an agreement with a global pharma partner, who can or co-develop the therapy. Under this business model, revenue streams are obtained through upfront payments, milestone payments and royalties on product sales.
Considering total project cost of €5,6 m during years 2019-2021, upfront payments above €14 m and 20% royalties over sales, the NPV of the investment calculated (WACC 10%) will surpass €257m by 2029.
Fields of science
Programme(s)
Funding Scheme
SME-1 - SME instrument phase 1Coordinator
411 26 GOTEBORG
Sweden
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.