Apitope Receives Orphan Designation in the European Union for ATX-F8-117 for the Treatment of Haemophilia A

DIEPENBEEK, Belgium, November 25, 2014 /PRNewswire/ --

Treatment for haemophilia A patients developing Factor VIII inhibitors  

Apitope, the drug discovery and development company focused on disease-modifying treatments for patients with autoimmune and allergic diseases, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan medicinal product designation to ATX-F8-117 for the treatment of haemophilia A.

Haemophilia A is a rare chronic bleeding disorder which leads to inadequate clotting of the blood in response to any type of injury or surgery. It is a genetic disorder that causes missing or defective Factor VIII, an essential blood-clotting protein. Haemophilia A patients are normally treated with Factor VIII to help with the clotting of their blood. However, since these patients' immune systems have had no or low exposure to normal Factor VIII, they are often not fully tolerant to the replacement Factor VIII used to treat their condition. As a consequence, up to 30 per cent of these patients develop Factor VIII inhibitor antibodies.

The development of these antibodies is the most serious complication that significantly limits the treatment of this disorder as well as increasing the cost burden. Apitope has, through its patented discovery platform, completed the research work to confirm that the two peptides in ATX-F8-117, derived from Factor VIII, have the potential to treat and prevent inhibitor development in haemophilia A patients treated with Factor VIII. Currently, there are few therapies available to help patients with inhibitors making the Apitope approach potentially life changing for patients.

Commenting on the designation, Dr Keith Martin, CEO said: "We are very pleased to receive Orphan Medicinal Product Designation by the EMA for ATX-F8-117 which underlines the need for an effective treatment for patients with haemophilia A. ATX-F8-117 is currently in preclinical development for the treatment of Factor VIII inhibitors which develop in approximately 30 percent of patients and results in poor clotting leading to severe health issues for patients. More specifically, the potential impact of this product could be an important step in the fight to help haemophilia A patients who cannot benefit fully from Factor VIII replacement therapy."

About Apitope  

Apitope International NV, based in Belgium and the UK, is a world-class drug developer of immunotherapies for the treatment of autoimmune and allergic diseases, including multiple sclerosis, Factor VIII intolerance, uveitis and Graves' disease. Apitope has a patented discovery platform which enables selection of potential disease-modifying peptide therapies for the autoimmune/allergic disease of interest; and has already generated a pipeline of 7 programmes in clinical and preclinical development, of which the lead programme in multiple sclerosis is partnered with Merck Serono. The discovery engine selects Apitopes^® - Antigen Processing Independent epiTOPES. Apitopes^® are soluble, synthetic peptides from the human sequence which can selectively suppress abnormal immune responses and reinstate the normal immune balance (Larche M, Wraith DC. Peptide-based therapeutic vaccines for allergic and autoimmune diseases. Nat Med 2005;11:S69-76). Stakeholders in the company include the Wellcome Trust, LRM, Vesalius Biocapital and the US MS charity, Fast Forward. For more information on the company, please visit: http://www.apitope.com.

About orphan designation 

Orphan medicinal product designation by the European Commission provides regulatory and financial incentives for companies to develop and market products that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. Orphan drug designation provides protocol assistance from the EMA during the product development phase, specific scientific advice and direct access to the centralised authorisation procedure. As a standard procedure, the maintenance of the European orphan drug status will require confirmation during the review of a future Marketing Authorisation Application.

About haemophilia A and Factor VIII intolerance 

Haemophilia A is a rare chronic bleeding disorder with an incidence of 1 in 5000 which leads to inadequate clotting of the blood in response to any type of injury or surgery. 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 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 around 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 inhibitors.

For further information:

Apitope International N.V.
Dr. Keith Martin, CEO
+44-117-370-7720
keith.martin@apitope.com

Hume Brophy
Mary Clark, Supriya Mathur, Hollie Vile
Tel: +44-20-3440-5653
apitope@humebrophy.com