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AOP Orphan and PharmaEssentia announce pivotal phase III results for ropeginterferon alfa-2b in Polycythemia Vera


  • At 12 months of treatment ropeginterferon alfa-2b showed non-inferiority to hydroxyurea (HU) in Complete Hematologic Response (CHR)
  • PROUD-PV demonstrated a significantly better safety and tolerability profile of ropeginterferon alfa-2b versus HU
  • AOP will submit the data from PROUD-PV and the ongoing long-term follow-up trial CONTINUATION-PV to obtain European marketing authorization in the coming months
  • PharmaEssentia intends to present this data to the FDA as it seeks approval for commercialization in the U.S.
  • PharmaEssentia discovered ropeginterferon alfa-2b and has exclusively licensed the rights for development and commercialization in MPNs to AOP Orphan in Europe, CIS and Middle Eastern markets

Vienna and Taipei, 5 December, 2016: AOP Orphan Pharmaceuticals AG (AOP Orphan) and PharmaEssentia Corporation (Taipei Exchange: 6446) announced results from the PROUD-PV study, a pivotal phase III clinical study in Polycythemia Vera (PV) presented at ASH 2016.
The PROUD-PV is a phase III, randomized, open-label, multicenter, controlled, parallel arm study assessing the efficacy and safety of ropeginterferon alfa-2b versus HU in patients with Polycythemia Vera. This study is part of the development program to support marketing authorizations of ropeginterferon alfa-2b (AOP2014/P1101) in Europe and in the United States (U.S.).
Ropeginterferon alfa-2b, a novel, long-acting, mono-pegylated proline interferon, uniquely administered once every two weeks is expected to be the first interferon approved for PV worldwide, and the only approved first-line treatment for PV in the U.S.
In PROUD-PV, a study sponsored and conducted by AOP Orphan, 254 PV patients from 48 centers across Europe were treated either once every two weeks with ropeginterferon alfa-2b or daily with the cytoreductive therapy hydroxyurea (HU). The study included both treatment-naïve and HU-pretreated patients with characteristics of an ‘early, first-line’ PV population.
At 12 months, Complete Hematologic Response1 (CHR) was achieved in a high proportion of patients and non-inferiority was demonstrated (43.1% for ropeginterferon alfa-2b versus 45.6% for HU in the intent-to-treat-population, p=0.0028).
The pre-specified primary endpoint, which was a composite of CHR and spleen length normality, was confounded by the fact that the median spleen length was almost normal at baseline and the observed change was not clinically relevant (21.3% for ropeginterferon alfa-2b versus 27.6% for HU in the intent-to-treat-population, p=0.2233).
Ropeginterferon alfa-2b showed significantly better tolerability than HU. Overall, 59.6% of the patients in the ropeginterferon alfa-2b arm experienced treatment related adverse events compared to 75.6 % of the patients treated with HU (p<0.05). There was no difference in adverse events of special interest concerning interferons (auto-immune, psychiatric), or concerning PV (cardiovascular disorders).
Most importantly, throughout the phase III program (PROUD-PV and CONTINUATION-PV) five related secondary malignancies were observed, all in the HU cohort (two acute leukemias, two basal carcinomas and one melanoma).
Professor Heinz Gisslinger from the Medical University of Vienna, presenting the results at ASH said, “We did already know from several smaller studies that interferons can be a valuable treatment option for myeloproliferative diseases, however this is the first and largest prospective controlled trial. This trial confirms the expected efficacy, while the observed safety and tolerability appears superior to previously reported data.”
Professor Jean-Jacques Kiladjian from the Saint-Louis Hospital & Paris Diderot University in France, added, “The potential to improve progression-free survival holds promise for long-term patient benefit, in line with the unique disease modification capabilities of interferon.”
Ko-Chung Lin, Ph.D., founder and CEO of PharmaEssentia, added, “The founding cornerstone of PharmaEssentia was to solve the discontinuity of long acting interferon beyond the weekly dosing regimen. We have been able to do this with ropeginterferon alfa-2b. The advantages provided by ropeginterferon alfa-2b as shown by this promising Phase III trial study and in prior studies, along with our state-of-the-art manufacturing facility in Taiwan, collectively bring us closer to making our treatment available to PV patients in the United States. We are proud to discover, develop, manufacture and eventually market ropeginterferon alfa-2b. This is fully in line with our mission to offer efficacious, safe and cost effective therapies for the treatment of myeloproliferative neoplasms such as PV, myelofibrosis, chronic myeloid leukemia, as well as hepatitis and other diseases.”
“AOP Orphan has continuously invested over many years into development of treatments in the field of MPNs. Our clinical development of ropeginterferon alfa-2b and today’s release of the PROUD-PV data mark another milestone in our mission to provide innovative solutions for patients with rare diseases,” said Rudolf Widmann, Ph.D. founder and CEO of AOP Orphan.

About Ropeginterferon alfa-2b
Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon with improved pharmacokinetic properties allowing once every two weeks administration offering improved tolerability and convenience. Ropeginterferon alfa-2b was discovered by PharmaEssentia. Ropeginterferon alfa-2b has Orphan Drug designation in the United States of America and the European Union. PharmaEssentia plans to commercialize ropeginterferon alfa-2b in North and South America, as well as Asia. PharmaEssentia has exclusively licensed the rights to ropeginterferon alfa-2b to AOP Orphan for European, Commonwealth of Independent States (CIS), and Middle Eastern markets for the development and commercialization in the field of Myeloproliferative Neoplasms (MPNs).

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 is a multinational company with headquarters in Vienna, Austria focusing on clinical research, development and distribution of medicines for rare and complex diseases. The company also provides individualized and customized services to meet and accommodate the needs of physicians and patients across Central Europe, the Middle East & Asia. Currently AOP Orphan is concentrating on orphan and complex diseases in Hematology & Oncology, Cardiology & Pulmonology, and CNS & Gastroenterology.

About PharmaEssentia
PharmaEssentia Corporation (Taipei Exchange:6446) is a global biopharmaceutical company delivering efficacious, safe and cost-effective therapeutic products for the treatment of human diseases while aiming to bring long lasting value to stakeholders. PharmaEssentia was founded in 2003 by a group of Taiwanese-American executives and high-ranking scientists from leading U.S. biotechnology and pharmaceutical companies in order to develop treatments for myeloproliferative neoplasms, hepatitis and other diseases. The company is committed to the improvement of health and quality of life for patients suffering from these diseases. The Company’s world-class cGMP biologics facility in Taichung is certified by the Taiwan Food and Drug Administration (TFDA) and is designed and operated to be compliant with all U.S. FDA and EMA requirements.

Contact
AOP Orphan Pharmaceuticals AG
Wilhelminenstrasse 91/IIf
1160 Wien
christoph.klade(at)aoporphan.com

PharmaEssentia Corporation
13F, No.3, YuanQu St., NanKang Dist.,Taipei 115, Taiwan
Dr. Ching-Leou Teng, Chairperson of the Board
chingleou_teng(at)pharmaessentia.com

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