AOP Orphan Pharmaceuticals AG (AOP Orphan) today announced that based on promising phase II data it will initiate a phase III trial to support European Marketing Authorization of a novel mono-pegylated Interferon alpha 2b (AOP2014/P1101) for treatment of Polycythemia Vera (PV).
AOP Orphan has exclusively licensed AOP2014/P1101 for development and commercialization in the field of Myeloproliferative Disorders (MPDs) with the territory of Central Europe, CIS and Middle East from Pharmaessentia Corp. a biotech company based in Taiwan. In contrast to other pegylated Interferons that require weekly administration, AOP2014/P1101 is applied only every other week, resulting in higher convenience and potentially improved tolerability, compliance and long term treatment outcomes.
Results from a phase II trial sponsored and conducted by AOP Orphan were presented yesterday Dec 9, 2012 in an oral presentation by Prof. Dr. Heinz Gisslinger from Vienna, Austria at this year´s prestigious annual ASH (American Society of Hematology) meeting held in Atlanta, U.S.A.
“Pegylated Interferons represent the most promising treatment modality for patients suffering from MPDs, even providing a chance for cure.”, comments Prof. Gisslinger, University Hospital for Internal Medicine I, Hematology, General Hospital Vienna, Austria.
Data from 41 PV patients showed no dose limiting toxicities in a dose range from 50-540 μg given every two weeks, with a side effect profile in line with expectations based on other pegylated Interferons. The overall response rate exceeded 90%, at 12 months of treatment 45-50% of patients showed a complete response based on normalization of hematological parameters. A trend for normalization of spleen size was also observed at this relatively early time point. Importantly, after one year all patients were completely independent from phlebotomies. Furthermore, JAK2 allelic burden was reduced significantly and sustained starting from week 28 of treatment. Molecular responses are regarded as important disease modification with the ultimate potential for cure.
“We are extremely happy with these promising results of AOP2014/P1101, and will take all efforts to make this novel treatment available to patients”, said Dr. Rudolf Widmann, CEO of AOP Orphan.
Based on these results AOP Orphan is initiating a pivotal phase III trial involving Polycythemia Vera patients. The trial termed PROUD-PV will commence early in 2013 and will include centers all across
Europe. Design and endpoints of this trial have been discussed between the European Medicines Agency EMA and AOP Oprhan to support a European Marketing Authorization, using EMAs centralized procedure. AOP2014/P1101 has received Orphan Drug designation already in 2011.
About Polycythemia Vera
Polycythemia Vera (PV) is a Rare Disease of the blood-building cells in the bone marrow primarily resulting in a chronic increase of red blood cells (erythrocytes), thereby having adverse impact on blood rheology. Circulatory disorders such as thrombosis and embolism, and also malignant transformation to myelofibrosis or leukemia, are possible outcomes of the disease.With proper treatment, the majority of PV patients have a near-normal life expectancy.
About AOP Orphan Pharmaceuticals AG (AOP Orphan)
AOP Orphan is a multinational Austrian company with a strong focus on clinical research , development and distribution of medicines for rare and complex diseases. Supplying patients and medical specialists with such medications requires the provision of extended services. Big global companies encounter frequent problems in serving such market segments optimally, a fact reflected in the rapid growth of AOP Orphan as we provide individualized and customized services to meet and accommodate for the needs of physicians and patients. AOP Orphan provides its services across all Central Europe, Middle East & Asia. Currently AOP Orphan is concentrating on orphan and complex diseases in hematology / oncology, cardiology, pulmonology, intensive care medicine, neurology and psychiatry.
About PharmaEssentia Corp.
PharmaEssentia Corporation is a fully integrated biopharmaceutical company established by a group of Taiwanese-American scientists. Headquartered at the state-of-the art facility —NanKang Science Park— Taipei, Taiwan, creating and providing products to improve the quality of life for patients suffering from various diseases. Our mission is to discover, develop, and bring to the market efficacious, safe and cost-effective therapeutic products for human disease. Our strategy is to leverage our resources in a flexible and dynamic way for the benefit of patients and the best return for our shareholders.
Shu-Fen Li , MBA
Director, Strategic Planning and Business Development
T +886-2-26557688 #7812
Address: 13F, No.3, YuanQu St. NanKang Dist. Taipei 115, Taiwan