TOL2 | An effective and safe targeted therapy for treatment of Myasthenia gravis

Summary
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.
Unfold all
/
Fold all
More information & hyperlinks
Web resources: https://cordis.europa.eu/project/id/854642
Start date: 01-12-2018
End date: 31-05-2019
Total budget - Public funding: 71 429,00 Euro - 50 000,00 Euro
Cordis data

Original description

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.

Status

CLOSED

Call topic

EIC-SMEInst-2018-2020

Update Date

27-10-2022
Images
No images available.
Geographical location(s)
Structured mapping
Unfold all
/
Fold all
Horizon 2020
H2020-EU.2. INDUSTRIAL LEADERSHIP
H2020-EU.2.1. INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies
H2020-EU.2.1.0. INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies - Cross-cutting calls
H2020-EIC-SMEInst-2018-2020
H2020-SMEInst-2018-2020-1
H2020-EU.2.3. INDUSTRIAL LEADERSHIP - Innovation In SMEs
H2020-EU.2.3.0. INDUSTRIAL LEADERSHIP - Innovation In SMEs - Cross-cutting calls
H2020-EIC-SMEInst-2018-2020
H2020-SMEInst-2018-2020-1
H2020-EU.3. SOCIETAL CHALLENGES
H2020-EU.3.0. Cross-cutting call topics
H2020-EIC-SMEInst-2018-2020
H2020-SMEInst-2018-2020-1