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Hematology/Oncology: AOP Health’s Expanding Clinical Research Program Delivers New Results


AOP Orphan Pharmaceuticals GmbH (AOP Health) continues a successful clinical research program in hematology/oncology with two abstracts accepted for presentation at the European Hematology Association (EHA) 2024 hybrid congress in Madrid, Spain. One results in an oral presentation of the latest results from the PROUD-PV and CONTINUATION-PV-trials. The outcomes show a correlation between the changes at genetic level (molecular response) and event-free survival (EFS) among patients with a rare blood cancer (polycythemia vera /PV) who received a certain interferon (ropeginterferon alfa-2b/BESREMi®) or best available treatment.1 A second accepted abstract reports on AOP Health’s recently initiated phase III clinical study extending investigation of ropeginterferon alfa-2b to patients with another rare blood cancer, essential thrombocythemia (ET).2 In a furthersignal of AOP Health’s commitment to innovative research, early development of a first-in-class investigative oncology drug is progressing towards clinical trials.

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Professor Jean-Jacques Kiladjian, University of Paris, France Photo credit: AOP Health/Lorenz Paulus

Reduction of mutated cells improves event free survival in patients with PV and is effectively achieved by ropeginterferon alfa-2b

The latest findings of the large randomized controlled PROUD-PV trial and its extension CONTINUATION-PV3 showed that ropeginterferon alfa-2b reduced the amount of cells that carried a mutation of a certain gene (JAK2V617F, a gene, which regulates cellular growth) in the patient population studied.3 Analysis of all patients included in the trial over at least 6 years showed significantly prolonged event free survival (period in which patients remain free from events that the treatment was intended to prevent or delay) among patients in which the amount of mutated cells were reduced at the last available assessment.

Professor Jean-Jacques Kiladjian from the University of Paris, France and first author of the paper emphasizes: “This new evidence supports the clinical relevance of reducing the load resulting of JAK2V617F mutations to improve long-term outcomes in patients with PV, which should be considered as an aim of treatment. Ropeginterferon alfa-2b targets mutated blood-forming stem cells and is highly effective in decreasing the burden resulting from the mutation of JAK2V617F gene.”

Phase III clinical study of ropeginterferon alfa-2b in ET underway in Europe

With the aim of examining the potential utility of ropeginterferon alfa-2b in ET patients with significant unmet need, AOP Health has rolled out the ROP-ET trial, a prospective, multicenter, single-arm phase III study to assess the long-term safety and efficacy of ropeginterferon alfa-2b in ET patients unable to receive available cytoreductive therapies.4 The trial is being conducted at 36 sites in 10 countries in Europe. Almost 50 % of the required number of patients have already been enrolled in the study. This fast recruitment underlines the importance of the clinical trial.

New drug candidate: official start of clinical development imminent

Building on proven success in the development of cancer stem cell targeting therapies, AOP Health is expanding into a new area of research with a first-in-class investigational oncology drug candidate. This orally available, highly selective serotonin receptor 1B antagonist was discovered and initially developed by Leukos Biotech based on research performed at the Josep-Carreras Leukemia Research Institute and licensed by AOP Health for further development and commercial rights. SERONCO-1, a phase I, first-in-human trial in patients with solid tumors and lymphomas will be conducted in collaboration with Leukos Biotech at the Vall d’Hebron Institute of Oncology (VHIO) with partial funding from the Spanish Ministry of Science and Innovation through the Public-Private Partnership Program (CPP2021-008715). A subsequent trial in acute myeloid leukemia is expected to commence in 2026.


  3. Gisslinger H, Klade C, Georgiev P, et al. Event-free survival in patients with polycythemia vera treated with ropeginterferon alfa-2b versus best available treatment. Leukemia. 2023 Oct;37(10):2129-32.
  4. Kiladjian JJ, Marin FF, Al-Ali HK, et al. ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options. Annals of Hematology. 2024 Mar 4:1-2.

About BESREMi®

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.

Approximately 7800 patients are currently treated or have been treated with BESREMi®.

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 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.

Needs. Science. Trust.
AOP Orphan Pharmaceuticals GmbH
Member of the AOP Health Group

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