Idogen has a platform technology that enables development of tolerogenic vaccines in several areas. As the first indication Idogen has chosen to develop a treatment for patients with hemophilia A who are left with few treatment alternatives after having developed antibodies against their vital factor VIII-replacement. These patients have a large medical need and the treatment is based on a well-defined antigen, which means that technically there is good potential to develop a successful treatment. In January 2017, Idogen was granted an orphan drug designation in Europe for this indication, which means a number of advantages, such as less extensive clinical trials, support from authorities and prolonged market exclusivity. The company has adopted a progressive development strategy in which a tolerogenic vaccine for kidney transplantation will be developed in parallel with the first tolerogenic vaccine for the treatment of inhibiting anti-factor VIII antibodies in hemophilia A.
Hemophilia A – anti-drug antibodies
Hemophilia A is an inherited blood disorder caused by a lack of functioning coagulation factor VIII. The standard treatment for hemophilia A consists of lifelong factor VIII replacement. Unfortunately, up to one third of patients develop an immunological reaction against factor VIII, which causes patients great suffering and poses a major cost to society. This complication is currently treated with prolonged treatment with a high dose factor VIII to thus try to induce tolerance. In many cases it works out well, but for 20–30% of patients the antibodies against factor VIII remains and thus only few treatment options remains for the patient. Our aim is to offer these patients a sustainable and specific tolerance development with our tolerogenic vaccine.
By reprogramming the immune system Idogen has potential to reduce the risk of rejection of cells and organs in transplantation. The principle is the same as for treatment of autoimmune diseases with Idogen’s concept: to specifically silence the immune reaction towards the transplanted organ or cells. Ultimately, this could reduce the need for continuous, long-term immunosuppressive treatment after transplantations. Idogen’s second therapy area will therefore be a tolerogenic vaccine to prevent transplant rejection, primarily kidney transplantation. The development work with a tolerogenic vaccine for kidney transplantation has now commenced with the intention to initiate the first clinical study in 2019.
Autoimmune diseases – Rheumatoid arthritis
In autoimmune diseases such as rheumatoid arthritis and multiple sclerosis the body’s own immune response attack the tissue and cause disease. Current treatment for these conditions consists largely of drugs suppressing the immune response on a broad basis, thereby protecting the tissues against the immune system’s harmful attacks. These treatments, however, have serious disadvantages for the patient, such as increased susceptibility to infections and increased risk of cancer, since the entire immune system is suppressed. With Idogen’s concept an unwanted immune reaction might instead be specifically silenced. The preclinical development and customization of the treatment concept to suit rheumatoid arthritis is performed in close collaboration with an eminent research group at Oxford University. Idogen has achieved ”proof-of-principle” for treatment of rheumatoid arthritis with two follow-up molecules to Zebularine.