Double recognition of Toleranzia AB

29 May - 2015

The company’s breakthrough therapy for the treatment of a rare neuromuscular disease have obtained Orphan Drug Designation, and in the same time the company have secured several millions in a combination of owner capital, a loan from ALMI as well as Eurostars funding.

Toleranzia AB is an R&D company that has developed a patent protected concept which can be used to treat several autoimmune diseases. The treatment acts on the root cause of the disease and does not only treat the symptoms. Toleranzia have recently obtained Orphan Drug Designation (ODD) for its Myasthenia Gravis therapy by the American Food and Drug Administration (FDA), as well as by the European Medicines Agency (EMA). This means that the product is considered to have the potential to be an Orphan Drug in the USA and in EU, with benefits like facilitated market entrance and regulatory advice as well as an extended exclusivity once on the market, 7 years in USA and 10 years in EU.

• The approvals by the FDA and EMA are acknowledgements for Toleranzia’s product for the treatment of Myasthenia Gravis but also a recognition of the company’s technology which can be adapted and developed for a portfolio of several autoimmune indications, says Toleranzia’s Chairman of the Board, Elisabet Litsmark Nordenstam.

The global market for Myasthenia Gravis therapy is today valued at 500 million SEK. However, todays therapies are only symptomatic, aimed at reducing the symptoms, muscle weakness and muscle fatigue, which in serious case can affect the breathing. Toleranzia’s product is expected to have a long-lasting and curing effect – not only symptomatic – as todays treatment options. If a curing therapy is introduced on the market it can be expected to be the first line treatment and would be worth at least 9 billion SEK.

The company received its second acknowledgement following penetration of the company by both private and public players. During the spring of 2015 the company have attracted several million SEK in a combination of new owner capital, a loan from ALMI as well as funding within the Eurostars program which is aimed at supporting small and medium sized enterprises with innovative projects with the potential to reach the market within two years following project completion. Within the Eurostars project ”Tolerize:MG” Toleranzia will coordinate and lead the work in collaboration with chosen partners in Denmark; Klifo and Bioneer, and in Lithuania; Biotechpharma.

• The budget for the Eurostars project totals 10,5 million SEK, where EU have granted appr. 6 MSEK, says Martina Verolin, Toleranzia’s Program Director and Chairman of the partner consortium that will carry out the Eurostars project. Toleranzia’s grant adds up to 2 MSEK and our partners will receive 4 MSEK to carry out work within the project. During this year we will together perform translational studies on human cells, establish new methods and plan for clinical trials, as well as produce therapeutic protein in the amounts and quality that is needed for the coming studies.

In the latest emission Toleranzia attracted about one million SEK from a number of Swedish Business Angels and GU Holding AB. Further, the company was approved for an innovation loan, 500 000 SEK, from ALMI Företagspartner Väst AB. The funds will be used as co-financing of the EUROSTARS project as well as for the recently initiated studies that will explore the effect of the therapy on human cells from Myasthenia Gravis patients. These studies are carried out with the support from the physicians at Sahlgrenska University Hospital, that are in charge of all Myasthenia Gravis patients within Västra Götalandsregionen.

• These news, by themselves and together, mean that we are now taking important steps towards the final drug that has a great potential to help people with Myasthenia Gravis. These patients are referred to drugs that can only alleviate the symptoms but does not have any permanent, curative effect. We are very happy and feel that this an acknowledgement that our clinically oriented research at Sahlgrenska Akademin within the University of Gothenburg have a strong support, says Professor Nils Lycke, the inventor of the concept.

Professor Nils Lycke have previously, together with research teams in Sweden and Italy, shown that this new therapy can reduce the severity of the disease with >85% in an experimental model of Myasthenia Gravis and the therapy also prevents the breakdown of the acetylcholine receptor (AChR) which is what is causing the muscle weakness in these patients. In addition to this, positive treatment effects have also been shown in models of Rheumatoid Arthritis, Multiple Sclerosis and Type 1 Diabetes.

For further information, please contact:

Elisabet Litsmark Nordenstam, Executive Chairman of the Board
tel: +46-70-550 86 17
e-mail: elisabet@partnerskapforframgang.se

About Toleranzia:

Toleranzia is an R&D company that builds on a research idea that started its commercial track in 2008 as a project within MIVAC Development, a vaccine company owned by researcher at the University of Gothenburg and GU Holding. Following a positive development the project was spun out and Toleranzia was founded at the end of 2011. The company has developed a patent protected concept for the treatment of several autoimmune diseases, both rare as well as more common indications. The vision is to treat the root cause of the disease and cure the patient with this unique technology. Professor Nils Lycke and his research group have made a unique breakthrough and demonstrated a way for the body to get re-educated to once again tolerate the body’s own cells and proteins through the concept of tolerance induction. The therapy will be given as a nasal spray. Toleranzia is on its way to develop a new, specific therapy for the treatment of Myasthenia Gravis (MG), a rare disease that affects about 200 000 people in the western world alone. The new nasal spray is expected to have a lasting and curing effect – not only symptomatic as today treatment options. Read more at: www.toleranzia.com.