An effective and safe targeted therapy for treatment of Myasthenia gravis

Project Information

TOL2

Grant agreement ID: 854642

DOI

10.3030/854642

Project closed

EC signature date 28 January 2019

Start date 1 December 2018

End date 31 May 2019

Funded under

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Total cost

€ 71 429,00

EU contribution

€ 50 000,00

Coordinated by

TOLERANZIA AB
Sweden

Periodic Reporting for period 1 - TOL2 (An effective and safe targeted therapy for treatment of Myasthenia gravis)

Reporting period: 2018-12-01 to 2019-05-31

Summary of the context and overall objectives of the project

Myasthenia gravis (MG) is an orphan autoimmune disease affecting about 2 in 10 000 people. MG patients suffer from different degrees of muscular weakness that restricts them in performing everyday activities and drastically reduces their quality of life. At present there is no cure for MG and current available treatments are only symptomatic and accompanied by severe side effects. Considering the urgent medical need for a safe and effective therapy, Toleranzia is developing TOL2, an innovative therapy that will not only ameliorate MG symptoms but will induce long-term remission and potentially cure the disease. Based on the very positive results obtained in this project, Toleranzia will continue the development of TOL2 towards commercialisation.

In this project, we performed a feasibility study with the following objectives:
- To demonstrate market viability of TOL2
- To apply for Orphan Drug Designation (ODD) for TOL2 from the European Medicines Agency (EMA)
- To develop a business plan

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

To investigate the market viability of TOL2 a market analysis was performed. This generated a greater understanding of the market potential of TOL2 for treatment of MG through the identification and mapping of relevant stakeholders. Toleranzia established contact with some of these groups to inform them about the benefits of TOL2, raise their awareness, create a scientific committee and promote market acceptance. This greatly increased our network. In addition, the execution of an in-depth competition analysis provided a detailed picture of our competitors including the level of threat they pose to the TOL2 project.
An application to obtain ODD from EMA was prepared and submitted. If granted, Toleranzia will benefit from the range of incentives offered to ODD holders such as market exclusivity, scientific advice and fee reductions for regulatory activities.
Finally, a detailed business plan including a financial model was developed. Through this document the goals of the company and the best ways to accomplish them were clearly defined.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

The ultimate goal of the project is the commercialization of TOL2 in partnership with a major pharmaceutical company. Treatment with TOL2 will have a long-term effect or be curative, thus greatly improve patients’ quality of life and reducing public costs associated with hospitalizations and unemployment. Moreover, the technology behind TOL2 can be applied to develop treatments for other autoimmune diseases.

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