AOP Health presents final results from its CONTINUATION-PV study -up to 7.5 years treatment with BESREMi® (Ropeginterferon alfa-2b) of Polycythaemia Vera patients - at EHA 2022 Annual Meeting
Vienna, June 13, 2022. AOP Health announced the final results on Ropeginterferon alfa-2b in patients with Polycythaemia Vera (PV) from its CONTINUATION-PV study in an oral presentation at the prestigious EHA (European Hematology Association) 2022 Annual Meeting by Professor Heinz Gisslinger, Medical University of Vienna, Austria1.
Ropeginterferon alfa-2b is a long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered once every 2 weeks initially, or up to monthly after stabilization of hematological parameters.
AOP Health has been conducting a pivotal clinical development program, including the studies PEGINVERA, PROUD-PV and CONTINUATION-PV. The latter 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 PV who previously participated in the PROUD-PV study.
The clinical development program conducted by AOP Health in Europe since 2010, led to marketing authorizations of BESREMi® for the treatment of PV, first granted by the European Commission in 2019, thereafter by Switzerland, Liechtenstein, Israel, Taiwan, Korea and most recently by the US FDA in November 2021.
The presentation during the EHA 2022 Annual Meeting focused on patient-relevant outcomes after this long-term (up to 7.5 years) treatment:
Symptoms/Quality of Life: only a minority of patients (15.7%) treated with Ropeginterferon alfa-2b experienced disease related symptoms, and less than 2 out of 10 patients had to undergo phlebotomy to maintain hematocrit levels during their last year of treatment.
Disease modification: Besides that, treatment with Ropeginterferon alfa-2b led to reduction of the disease-causing mutated JAK2 allele burden below 1% in a sizeable proportion of patients (>20%). Most importantly, event-free survival, with events defined as death, thrombosis, or disease progression over the entire 7.5 years treatment period, was significantly better in the Ropeginterferon alfa-2b group with only 5 of 95 patients experiencing an event, whereas in the control group such an event was observed in 12 of 74 patients. (p=0.04).
“The results of CONTI-PV after 7.5 years of treatment provide further evidence of the value of BESREMi® for patients suffering from PV. We are very proud that our work not only delivered the first interferon with regulatory approval for any of the Myeloproliferative Neoplasms, but also contributed to a change of treatment paradigm in PV”, says Dr. Christoph Klade, Chief Scientific Officer of AOP Health.
Professor Jean-Jacques Kiladjian from Paris, senior author of the paper adds: “Since its first approval in Europe in 2019, Ropeginterferon alfa-2b has become the first line therapy of choice for many PV patients. The striking JAK2 molecular response and the extremely low rate of disease progression and thrombosis suggest that Ropeginterferon alfa-2b might be the best available treatment option for disease modification, which may translate into improved survival and even the chance to stop treatment for some patients.”
BESREMi® is a long-acting, mono-pegylated proline interferon (ATC L03AB15). Its unique pharmacokinetic properties offer a new level of tolerability. BESREMi® is designed to be conveniently self-administered subcutaneously with a pen once every two weeks, or up to monthly after stabilization of hematological parameters. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons.
For the EMA Summary of Product Characteristics please visit: BESREMi®
Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Health. In 2009, AOP Health in-licensed the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in PV and other MPNs such as chronic myelogenous leukemia (CML) for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.
About Polycythaemia Vera
Polycythaemia Vera (PV) is a rare cancer of the blood-building stem cells in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. This condition increases the risk for circulatory disorders such as thrombosis and embolism, its symptoms lead to a reduced quality of life and on the long run may progress to myelofibrosis or transform to leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to blood-building stem cells in the bone marrow with a set of acquired mutations, the most important being a mutant form of JAK2 that make up the malignant clone.
Important PV treatment goals are to achieve healthy blood counts (hematocrit below 45%), improve quality of life and to slow or delay the progression of disease.
About AOP Health
The AOP Health Group incorporates several companies including AOP Orphan Pharmaceuticals GmbH with its seat in Vienna, Austria (“AOP Health”). The AOP Health Group is the European pioneer for integrated therapies for rare diseases and in critical care. Over the past 25 years, the Group has become an established provider of integrated therapy solutions operating from its headquarters in Vienna, its subsidiaries and representative offices throughout Europe and the Middle East, as well as through partners worldwide. This development has been made possible by a continually high level of investment in research and development on the one hand and a highly consistent and pragmatic orientation towards the needs of all its stakeholders on the other – especially the patients and their families as well as also the healthcare professionals treating them.
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1 Ropeginterferon alpha-2b achieves patient-specific treatment goals in Polycythaemia Vera: final results from the PROUD-PV/CONTINUATION-PV studies. Heinz Gisslinger, Christoph Klade, Pencho Georgiev, Dorota Krochmalczyk, Liana Gercheva-Kyuchukova, Miklos Egyed, Petr Dulicek, Arpad Illes, Halyna Pylypenko, Lylia Sivcheva, Jiří Mayer, Vera Yablokova, Kurt Krejcy, Victoria Empson, Hans C. Hasselbalch, Robert Kralovics, and Jean-Jacques Kiladjian for the PROUD-PV Study Group, European Hematology Association EHA, 27th Annual Meeting June 2022