Arno Therapeutics, Inc., a clinical stage biopharmaceutical company primarily focused on the development of therapeutics for the treatment of cancer and other life threatening diseases, today announced that the European Commission, acting on the recommendation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has designated AR-12 as an orphan medicinal product for the treatment of two separate infectious diseases, cryptococcosis and tularaemia.
Alex Zukiwski, MD, Chief Executive Officer of Arno Therapeutics, commented, "Following positive recommendation by the EMA's Committee for Orphan Medicinal Products, we are very pleased with the European Commission's designation of AR-12 as an orphan drug to treat two infectious diseases that continue to affect people across the globe and for which new therapeutic options are needed. This designation marks an important milestone that supports Arno's global regulatory and development strategy. We believe AR-12 has potential as a promising approach to address unmet medical needs of patients infected with cryptococcosis and tularaemia and look forward to further investigating AR-12 in these two disease areas."
The AR-12 orphan medicinal product designation was based on application packages that contained relevant data from pre-clinical models for each condition. In the application regarding cryptococcosis, results of an animal model evaluating AR-12 showed a reduction in brain fungal burden in combination with fluconazole, an antifungal medication.1 In the application regarding tularaemia, results of an animal model evaluating AR-12 showed improved survival in combination with gentamicin.1
The European Commission grants orphan designations for medicines that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.
Preliminary data demonstrate that the mechanism of action of AR-12 may include induction of host cell autophagy and inhibition of fungal acetyl coenzyme A synthetase. Additional work is ongoing to further understand the mechanism of action. Based on pre-clinical research, AR-12 may have potential as an orally-available antimicrobial agent in a number of infectious diseases.
About Cryptococcosis
Cryptococcosis is an infectious disease of the lungs caused by the fungus Cryptococcus neoformans, typically found in soil and breathed in. It is most often seen in people with a weakened immune system, including those with HIV infection, organ transplants receiving immunosuppressive therapy, receiving cancer chemotherapy, or who have Hodgkin's disease. Cryptococcus is one of the most common life-threatening fungal infections in people with AIDS.2
About Tularaemia
Tularaemia is an infection which can be spread from animals to humans that is caused by the bacterium Francisella tularensis. Human infection can occur through a variety of mechanisms, most often of which is through bites of infected insects such as ticks, mosquitoes and flies. Following an incubation period of about 3-5 days in humans, symptoms start abruptly with high fever. Other symptoms vary with the portal of entry, and include swollen lymph glands, eye infection, throat infection, pneumonia and severe infection with blood stream infection.3 The condition may become debilitating due to the development of lymphadenopathy, pneumonia, diarrhea, and cutaneous ulcers, with a protracted recovery.1 Francisella tularensis (tularemia) is a Category A Priority Pathogen on the National Institute of Allergy and Infectious Disease (NIAID) list of Biodefense and Emerging Infectious Diseases.
About AR-12
AR-12 is an orally-available small molecule. Preliminary data demonstrate that the mechanism of action may include induction of host cell autophagy and inhibition of fungal acetyl coenzyme A synthetase. Previously, AR-12 has completed Phase 1 clinical trials in patients with cancer. Additional pre-clinical research indicates that AR-12 may have potential as an antimicrobial agent in various infectious diseases.