藥華林國鐘….長效α干擾素 進入 pivotal phase III trial (PROUD-PV)

新藥後年上市 藥華行情看漲 2013-8-30〔記者陳永吉／台北報導〕國發基金持股13％的新藥公司—藥華醫藥，將在今年底登錄興櫃，由於藥華授權出去的產品P1101預計2015年底就能上市，屆時可分得權利金，且因藥品潛力佳，目前藥華在未上市的行情每股超過140元。藥華在2003年成立，創辦人為現任公司總經理林國鐘，藥華在2007年以PEGylation技術平台開發成功新一代（Next Generation） PEG長效型α干擾素藥物 （產品代號P1101），並在2009年時授權給奧地利廠商AOP，不過授權金額及未來產品上市後可分到的權利金比例，林國鐘表示基於保密原則，不便透露。林國鐘指出，P1101目前進行的人體試驗包括罕見血液增生疾病的真性紅血球增多症、B型肝炎及C型肝炎等，其中合作夥伴AOP已經在歐洲完成真性紅血球增多症的第二期臨床試驗，結果在去年底亞特蘭大的ASH年會發表，數據顯示P1101比現有的藥物副作用小，且效果更佳。林國鐘表示，根據AOP的規劃，P1101將在今年下半年啟動第三期臨床試驗，由於P1101在2011年及2012年已被歐洲醫藥品管理局及美國食品藥物管理局授予孤兒藥資格認定，所以三期臨床試驗估計可在2014年完成，2015年底可將藥品推上市，由於藥華授權給AOP的區域為歐洲及中東還有前蘇聯獨立國，內部估計光是歐洲就有5億美元的市場價值。由於AOP的二期臨床數據藥華也可使用，因此P1101在美國的臨床試驗，將由藥華自己規劃，林國鐘表示，9月26日就會跟美國FDA討論第三期臨床試驗還要做哪些部分，不過也因P1101獲得美國FDA孤兒藥的認定，估計臨床試驗時間也不會太久。藥華今年成立10週年，將在下週舉辦大型罕見血液疾病研討會，林國鐘說，慶祝10週年方式用學術交流更有意義，並且也讓國外知道，台灣有發展新藥的實力。

AOP Orphan Pharmaceuticals Reports Positive Phase II Data of Novel Mono-Pegylated Interferon Alpha 2b for Treatment of Polycythemia Vera (PV) Monday, 10 December 2012 15:45 AOP Orphan announces results from phase II trial in PV at ASH Annual Meeting in Atlanta, U.S.A. Promising efficacy and safety data support further development of AOP2014/P1101  AOP2014/P1101 received Orphan Drug designation in 2011

AOP Orphan is initiating a European phase III trial PROUD-PV to support European Marketing Authorization  Vienna, Austria, December 10, 2012 / B3C newswire / - 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.