Vienna, Austria/Atlanta, USA (pts001/10.12.2017/14:00) - * High rates and durable Clinical and Hematological response were achieved with Ropeginterferon alfa-2b at 24 months of treatment
* The disease modifying capability of Ropeginterferon alfa-2b was further supported by 24 month clinical and molecular data
* The favorable safety and tolerability profile of Ropeginterferon alfa-2b was confirmed beyond 24 months
* AOP Orphan´s submission for marketing authorization of Ropeginterferon alfa-2b in the EU is currently under review by EMA
Vienna, 10 December 2017: AOP Orphan Pharmaceuticals AG (AOP Orphan) announces that the latest follow-up results on Ropeginterferon alfa-2b from AOP Orphan´s trial CONTINUATION-PV for patients with Polycythemia Vera (PV) were presented at ASH 2017.
CONTINUATION-PV is an open-label, multicenter, phase IIIb study assessing the long-term efficacy and safety of Ropeginterferon alfa-2b versus hydroxyurea (HU) or best available treatment (BAT) in patients with polycythemia vera who previously participated in the pivotal PROUD-PV study.
Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered once every 2 weeks, or monthly during long-term maintenance, and is expected to be the first interferon approved for PV worldwide. AOP Orphan´s submission for marketing authorization in the EU is currently under EMA review.
At 12 months (PROUD-PV study), Ropeginterferon alfa-2b was shown to be non-inferior in Complete Hematologic Response (CHR) and to have a significantly better safety and tolerability profile compared to hydroxyurea (HU).
At 24 months, treatment with Ropeginterferon alfa-2b achieved a high CHR of 70.5%, significantly higher than a CHR of 49.3% with HU/BAT (p=0.0101).
Importantly, response rates increased steadily in the Ropeginterferon alfa-2b-treated patients over the two-year treatment period in contrast to HU/BAT. Also, the composite endpoint, CHR including disease symptom improvement, was higher in patients treated with Ropeginterferon alfa-2b versus HU/BAT at 24 months (49.5% versus 36.6%, p=0.1183).
A pronounced treatment effect of Ropeginterferon alfa-2b was also observed on the mutant JAK2 allele burden at 24 months: 69.6% of patients treated with Ropeginterferon alfa-2b compared to only 28.6% on HU/BAT achieved partial molecular response (p=0.0046).
A comparable number of patients experienced treatment-related adverse events (70.1% for Ropeginterferon alfa-2b and 77.2% for HU). Events of special interest for the interferon alfa-class, in particular thyroid disorders and depression were below 5% in the Ropeginterferon alfa-2b arm.
Notably, disease- or treatment-related secondary malignancies including two leukemias occurred only in the HU cohort.
Professor Heinz Gisslinger from the Medical University of Vienna, presenting the results at ASH stated, "the observed superior efficacy of Ropeginterferon alfa-2b over hydroxyurea/best-available-therapy after 24 months, is a clear proof of the long-term value of this treatment modality. Thus, Ropeginterferon alfa-2b will provide a valuable and safe new first line therapy for PV patients".
Professor Jean-Jacques Kiladjian from the Saint-Louis Hospital & Paris Diderot University in France, concluded, "the disease modification capability of Ropeginterferon alfa-2b suggested by a significant reduction of mutant JAK2 allelic burden and the malignant clone, holds promise for improvement of progression-free survival and long-term patient benefit."
About Ropeginterferon alfa-2b
Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon with improved pharmacokinetic properties offering improved tolerability and convenience. Ropeginterferon alfa-2b was discovered by PharmaEssentia (http://www.pharmaessentia.com ). Ropeginterferon alfa-2b has Orphan Drug designation in the European Union and the United States of America. PharmaEssentia (http://www.pharmaessentia.com ) has exclusively licensed the rights for 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 Europe, the Middle East & Asia. Currently AOP Orphan is concentrating on orphan and complex diseases in Hematology & Oncology, Cardiology & Pulmonology, and CNS & Gastroenterology.
AOP Orphan Pharmaceuticals AG
Wilhelminenstrasse 91/IIf, 1160 Vienna, Austria
Dr. Christoph Klade, Chief Scientific Officer