– PharmaEssentia and AOP Orphan announce completion of recruitment of the pivotal European Phase III trial PROUD-PV (www.proud-pv.com) to support global marketing of P1101 (Ropeginterferon alfa-2b). This is the first registrational trial evaluating interferon in polycythemia vera (PV).
– US FDA has agreed that the data from this trial can be used for the BLA filing in the US.
– P1101 (Ropeginterferon alfa-2b), discovered by PharmaEssentia scientists, is a novel, long-acting, mono-pegylated interferon, administered only once every 14 days. After achieving response, PV patients can be switched to once monthly administrations with maintenance of efficacy.
– P1101 (Ropeginterferon alfa-2b) is expected to be better tolerated than other marketed pegylated interferons and to be effective in the majority of PV patients.
– In previous Phase I/II trials, P1101 (Ropeginterferon alfa-2b) treatment resulted in normalization of hematological parameters and molecular responses. In the extension of this trial (up to 4 years so far), there remain undetectable mutated JAK2 levels and complete normalization of the chromosomal make-up in some patients.
TAIPEI, March 11, 2015 /PRNewswire/ — PharmaEssentia (Taipei, Taiwan) and AOP Orphan (Vienna, Austria) announce the completion of recruitment for the Phase III trial PROUD-PV to support global marketing of P1101 (Ropeginterferon alfa-2b), a novel, long-acting, mono-pegylated interferon for the first line treatment of polycythemia vera.
The pivotal Phase III trial PROUD-PV commenced enrollment in October 2013, with over 260 PV patients now recruited in 14 countries and 50 centers all across Europe. Both naive and treatment-experienced patients have been enrolled and patients are either treated with P1101 or hydroxyurea, the current first line standard of care. The primary endpoint is a composite endpoint of hematocrit, platelets, leukocytes, and spleen size at 12 months.
Design and endpoints of this trial have been discussed and agreed upon with the European Medicines Agency. 12 month final primary endpoint data from the European PROUD-PV trial will also be used to form the basis of submission to US FDA, obviating the need for a US-based registrational trial. This submission to US FDA could potentially be earlier, depending on the data and ongoing discussions with US FDA.
Polycythemia vera is an orphan disease affecting 100,000 individuals in the United States with a similar sized population in the EU, with a potential market size in the billions. There is currently only one other drug approved in the US for the treatment of polycythemia vera — Incyte’s Jakafi, which is approved for second line treatment of PV. P1101 (Ropeginterferon alfa-2b) targets first line treatment of PV. PharmaEssentia estimates that ~65-75% of all PV patients are first line, with 25-35% comprising second line patients.
P1101 (Ropeginterferon alfa-2b) was discovered by scientists at PharmaEssentia and PharmaEssentia exclusively licensed the rights to P1101 (Ropeginterferon alfa-2b) for European, CIS, and Middle Eastern markets in the field of myeloproliferative neoplasms (MPN) to AOP Orphan in 2009, a multinational biotechnology company based in Vienna, Austria. PharmaEssentia retains the rights to P1101 (Ropeginterferon alfa-2b) in other major markets, such as North America, Asia, and South America.
In addition to polycythemia vera, PharmaEssentia is exploring usages for P1101 (Ropeginterferon alfa-2b) as an interferon with an improved side effect profile. These include the fields of essential thrombocythemia, chronic hepatitis B, chronic hepatitis C (genotype 2), and oncology (in combination with PD-1 targeted agents). Essential thrombocythemia is a field very similar to polycythemia vera and there have been no new FDA approvals in this indication for almost 2 decades. Chronic hepatitis B is an area of high unmet need, particularly in Asia, for which nucleoside analogues have several shortcomings. Among these include drug resistance and continuous treatment. Interferon, however, has a direct enhancing effect on the immunologic response to HBV, enabling short courses of therapy and low drug resistance. Pegylated interferon has also demonstrated as high as 90% response rates in Asian patients who carry the genotype 2 chronic hepatitis C virus. A once every two weeks Ropeginterferon will provide a better choice for these patients. Finally, PharmaEssentia is also exploring the potential synergistic effect of Ropeginterferon alfa-2b with immuno-check point inhibitors like PD-1 that it is developing in-house.
"Our collaboration with AOP was a strategic move, as early on we recognized the value of P1101 across several different fields of therapy, based on several publications in medical literature. We expect that if we enhance the compliance for patients when they take a more tolerable interferon, the expanding indications are very achievable to that of Humira, which is now approved in 8 indications with $12.5B of sales. Thus, the completion of recruitment of PROUD-PV marks a very important milestone for this asset. In particular, we are very thankful to AOP Orphan for this fruitful partnership as well as for the support of the US FDA in the process of bringing this important asset to the US market," remarks Ko-Chung Lin, CEO of PharmaEssentia.
In contrast to other pegylated interferons that require weekly administration, P1101 (Ropeginterferon alfa-2b) is administered only every other week. Ultimately, this is expected to result in improved tolerability, convenience and compliance and, as a consequence, better long-term treatment outcomes.
Results from a Phase I/II trial presented at ASH (American Society of Hematology) in 2012, 2013 and 2014 demonstrated positive results: the overall clinical response rate including reduction of red and white blood cells and platelets was approximately 90% and after 6 to 12 months of treatment, 45-50% of patients demonstrated complete responses, significantly higher than conventional PV treatments. Importantly, after one year all patients were completely independent from phlebotomies. After achieving therapeutic response (one year of treatment), administration frequency could be further reduced to monthly intervals with maintenance of efficacy.
Further, the JAK2 allelic burden was significantly reduced by treatment with P1101 (Ropeginterferon alfa-2b), providing evidence of the disease-modifying, potentially-curative properties of this therapy. PharmaEssentia and AOP Orphan believe that interferon is the only therapeutic option that has potential curative effects on polycythemia vera.
About Polycythemia Vera
Polycythemia vera 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.
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 in NanKang Science Park in Taipei, Taiwan, PharmaEssentia specializes in 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 market efficacious, safe and cost-effective therapies 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 construction of a world-class cGMP biologics production plant in Taichung Science Park in Taichung, Taiwan. The plant underwent a successful GMP inspection by TFDA in 2013 and is compliant with FDA and EMA requirements.
In March 2014, PharmaEssentia was listed on the Gretai Emerging Market Board and anticipates a formal IPO on the Gretai Exchange in early 2016.
Ko-Chung Lin, Ph.D.
Founder & CEO
Tel: +886-2-26557688 #7802
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
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 it provides individualized and customized services to meet and accommodate the needs of physicians and patients. AOP Orphan provides its services across all Central Europe, Middle East and Asia. Currently AOP Orphan focuses on orphan and complex diseases in hematology/oncology, cardiology, pulmonology, intensive care medicine, neurology and psychiatry.
AOP Orphan Pharmaceuticals AG
Dr. Georg Fischer
Wilhelminenstrasse 91/IIf, 1160 Vienna
T +43 1 503 72 44-42
F +43 1 503 72 44-61
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