Therapeutic product candidates
Factor VIII Intolerance
Haemophilia A (HA) is a rare chronic bleeding disorder which leads to inadequate clotting of the blood in response to any type of injury or surgery. The most common form of HA is congenital HA, which mostly affects males due to its X-linked recessive inheritance pattern and has an incidence of 1 in 5000.
There are approximately 33,000 HA patients under active management in the USA and Europe. The mainstay of HA treatment is the life-long replacement therapy with synthetic FVIII to achieve haemostasis using either plasma derived or recombinant FVIII products.
The most challenging complication of therapy is the development of anti-FVIII neutralising antibodies which occurs in 20-30% of patients. These antibodies block (inhibit) the pro-coagulant function of FVIII and are therefore termed inhibitors. The development of such inhibitors sharply decreases the efficacy of the FVIII and quickly renders the FVIII replacement ineffective and can result in joint damage, brain damage and, ultimately, death. There are currently no treatments available to prevent or treat the development of neutralising antibodies.
The ATX-F8-117 discovery programme has identified two Apitopes® which have the potential to treat and prevent Factor VIII inhibitor formation by as much as 96%.