- AOP Orphan announces progress of pan-European phase III trial PROUD-PV to support European Marketing Authorization of AOP2014/P1101, a next-generation, mono-pegylated Interferon alpha 2b.
- AOP2014/P1101 is administered only once every 14 days; in some cases only monthly. Hence, it is expected to be better tolerated than other marketed pegylated interferons
- Promising efficacy and safety data from previous phase II support phase III development
- AOP2014/P1101 received Orphan Drug status in 2011
AOP Orphan Pharmaceuticals AG (AOP Orphan) today reported on the progress of its phase III trial PROUD-PV to support European Marketing Authorization of a novel mono-pegylated Interferon alpha 2b (AOP2014/P1101) for the treatment of Polycythemia vera (PV).
Based on earlier promising results (see PR Dec 2012), AOP Orphan has set up a pivotal phase III trial with Polycythemia vera patients. The trial termed PROUD-PV includes centers all across Europe. Study activities are ongoing, and the recruitment of patients has successfully started. PV patients are either given mono-pegylated Interferon alpha 2b (AOP2014/P1101) or hydroxyurea, which is a registered treatment for PV. Patients will stay on the course of treatment for one year.
Importantly, in contrast to other pegylated Interferons that require weekly administration, AOP2014/P1101 is administered only every other week with the potential for monthly administrations. Ultimately, this is expected to result in improved tolerability, convenience and compliance and, as a consequence, better long-term treatment outcomes.
Throughout the trial, a number of clinical and hematological parameters are assessed. Treatment with AOP2014/P1101 is expected to be effective in the majority of patients and to be superior to hydroxyurea.
Design and endpoints of this trial have been discussed and agreed with the European Medicines Agency EMA, to support a European Marketing Authorization, using EMA’s centralized procedure. AOP2014/P1101 was already awarded Orphan Drug status in 2011.
“We already know from small studies that interferons work effectively against Myeloproliferative Diseases”, remarked Professor Jean-Jacques Kiladjian from Paris. “It is a great relief that finally a pharmaceutical company like AOP Orphan is developing an even further improved interferon, that was shown to be effective, safe and more convenient for patients in early phase studies, specifically for patients with polycythemia vera in a large international study.”
“PROUD-PV is a landmark endeavor for AOP Orphan and a great opportunity to make an improved, effective treatment available for patients in the orphan sector”, said Dr. Rudolf Widmann, CEO of AOP Orphan.
Results from a phase II trial sponsored and conducted by AOP Orphan were presented at ASH (American Society of Hematology) in 2012. The overall response rate exceeded 90 %: After 12 months of treatment, 45-50 % of patients showed a complete response based on the normalization of hematological parameters. Importantly, after one year all patients were completely free from phlebotomies. Furthermore, JAK2 allelic burden was significantly reduced on a sustained basis as of week 28 of treatment. Molecular responses are regarded as an important disease modification with the ultimate potential for cure.
AOP Orphan has exclusively licensed AOP2014/P1101 for development and commercialization in the field of Myeloproliferative Disorders (MPDs) from Pharmaessentia Inc., a biotech company based in Taiwan.
To follow updates on PROUD-PV check our website: www.proud-pv.com
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 an adverse impact on blood rheology. Circulatory disorders such as thrombosis and embolism, as well as 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 medication 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.
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 diseases. Our strategy is to leverage our resources in a flexible and dynamic way to the benefit of patients and to achieve the best return for our shareholders.
Ko-Chung Lin, Ph.D.
Founder & CEO
Shu-Fen Li , MBA
Director, Strategic Planning and Business Development
Tel: +886-2-26557688 #7812
Address: 13F, No.3, YuanQu St. NanKang Dist. Taipei 115, Taiwan