FDA Grants Orphan Status for ATX-F8-117 for the Treatment of Haemophilia A Patients in the US

DIEPENBEEK, Belgium, June 9, 2015 /PRNewswire/ --

Prevention or treatment of haemophilia A patients with inhibitors or at risk of developing inhibitors to Factor VIII  

Apitope, the drug discovery and development company focused on disease-modifying treatments that reinstate immune tolerance, announced today that pre-clinical product candidate ATX-F8-117 has been granted Orphan Drug Status by the US Food and Drug Administration (FDA) for the prevention or treatment of inhibitors in haemophilia A patients with inhibitors or at the risk of producing inhibitors.

Commenting on the designation, Dr Keith Martin, CEO said: "Following the Orphan Drug Designation by the European Medical Agency last year, we are very pleased to receive designation from the US FDA for ATX-F8-117. These designations emphasise the need for an effective treatment for haemophilia A patients developing Factor VIII inhibitors that occurs in approximately 30 per cent of patients. This results in poor clotting of the blood leading to severe health issues. This orphan drug designation follows extensive pre-clinical evaluation and we look forward to advancing a clinical development programme for this important medical condition."

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 Factor VIII, they are often not fully tolerant to the replacement Factor VIII used to treat their condition. Consequently, 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.

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 

The Orphan status is granted to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. The Orphan Drug Designation program by the US Food and Drug Administration (FDA) provides incentives for sponsors to develop products for rare diseases or conditions. The program has successfully enabled the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983.

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
Tel: +44-117-370-7720
keith.martin@apitope.com

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