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An effective and safe targeted therapy for treatment of Myasthenia gravis

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

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
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.
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.