AOP Orphan Pharmaceuticals Announces Progress of Pivotal Phase III Trial PROUD-PV Applying Ropeginterferon ALFA 2B, a Novel, Long-Acting, Mono-Pegylated Interferon for Treatment of Polycythemia Vera

AOP Orphan announces completion of recruitment of its pivotal phase III trial PROUD-PV ( to support global licensure of Ropeginterferon alfa 2b.Ropeginterferon alfa 2b is a novel, long-acting, mono-pegylated Interferon, administered only once every 14 days, after achieving therapy response even only monthly.Based on previous phase I/II trials, Ropeginterferon alfa 2b is expected to be safe and effective in the majority of PV patients.Hematological and molecular responses occured in most patients; moreover, several patients achieved undetectable mutated JAK2 levels and a complete normalization of their chromosomal make-up

AOP Orphan Pharmaceuticals AG (AOP Orphan) today reported the completion of recruitment for its phase III trial PROUD-PV to support global licensure of Ropeginterferon alfa 2b (AOP2014/P1101), a novel, long-acting, mono-pegylated Interferon for the treatment of Polycythemia Vera (PV). Importantly, Ropeginterferon alfa 2b is administered only every other week. After achieving therapy response, administration frequency may be further extended to monthly intervals. Ultimately, this is expected to result in improved tolerability, convenience and compliance and, as a consequence, better long-term treatment outcomes.

AOP Orphan has exclusively licensed, Ropeginterferon alfa 2b for development and commercialization in the field of Myeloproliferative Neoplasms (MPNs) from Pharmaessentia Corp. a biotech company based in Taiwan for European, CIS, and Middle Eastern markets. Pharmaessentia retains the rights for development and commercialization of P1101 in other major markets, such as North America, Asia, and South America.
Based on very encouraging phase I/II data (see below), AOP Orphan has set up the pivotal phase III trial PROUD-PV. Design and endpoints of this trial have been discussed and agreed with both the European Medicines Agency EMA and the U.S. FDA, to support global licensure of Ropeginterferon alfa 2b, which also has Orphan Drug status in both Europe and the USA.

Since its commencement in October 2013 over 260 PV patients have been recruited in 50 centers all across Europe. Enrollment of patients has been successfully completed in February 2015. Patients are either treated with Ropeginterferon alfa 2b, or hydroxyurea, which is a registered treatment for PV for one year. Throughout the trial, a number of clinical and hematological parameters are assessed. Treatment with Ropeginterferon alfa 2b is expected to be safe and effective in the majority of patients and to be superior to hydroxyurea.

“We already know from several smaller studies that interferons work effectively against myeloproliferative diseases”, remarked both Professors Jean-Jacques Kiladjian from Paris and Heinz Gisslinger from Vienna.

“The unparalleled quick uptake and progress of PROUD-PV, completing enrollment of over 260 patients in around 15 months, proves the eagerness of both patients and physicians for this new treatment paradigm”, said Dr. Rudolf Widmann, CEO of AOP Orphan.

Results from a phase I/II trial sponsored and conducted by AOP Orphan presented at ASH (American Society of Hematology) in 2012, 2013 and 2014 appear very encouraging: the overall clinical response rate including reduction of red and white blood cells and platelets was around 90% and after 6 to 12 months of treatment, 45-50% of patients even showed complete response. Importantly, after one year all patients were completely independent from phlebotomies. In addition, all patients remaining in this trial could be switched to an even more convenient monthly treatment schedule.

Hematological responses correlated with molecular responses: JAK2 is the driver mutation of PV and the JAK2 allelic burden was significantly reduced by treatment with Ropeginterferon alfa 2b. Several patients even achieved undetectable mutated JAK2 levels. The clinical relevance of the JAK2 molecular responses was further substantiated by genome wide analysis revealing a complete normalization of the chromosomal make-up in several patients (Them et al. American J. Hematology 2015). These encouraging finding provide the hope for a cure of at least some patients.

About Polycythemia Vera
Polycythemia Vera (PV) is a cancer of the blood-building cells in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. This condition may result in circulatory disorders such as thrombosis and embolism, as well as malignant transformation to myelofibrosis or leukemia.

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.

About PharmaEssentia Corporation (PEC)
PharmaEssentia Corporation is a fully integrated global biopharmaceutical company based in Taiwan 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.
In 2013, PharmaEssentia Corp. completed the construction of a world-class cGMP biologics production plant in Taichung, Taiwan to produce quantities safe, efficacious, and technologically advanced drugs. The plant underwent a successful GMP certificate by TFDA in 2013 and is compliant with FDA and EMA requirements.

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