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.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240610730599/en/
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.
References
- Kiladjian J-J, Klade C, Georgiev P, et al. JAK2V617F MOLECULAR RESPONSE CORRELATES WITH EVENT-FREE SURVIVAL IN AN EARLY POLYCYTHEMIA VERA POPULATION. 2024. https://library.ehaweb.org/eha/2024/eha2024-congress/422323 Last accessed 27 May 2024.
- Kiladjian J-J, Ferrer Marín F, Al-Ali FK 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. 2024. https://library.ehaweb.org/eha/2024/eha2024-congress/421616. Last accessed 27 May 2024.
- 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.
- 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®
Link: https://www.ema.europa.eu/en/documents/product-information/besremi-epar-product-information_en.pdf
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
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240610730599/en/
Contact information
DI Isolde Fally
Isolde.Fally@aop-health.com
+43-676-500 4048
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
EIG Acquires a 49.87% Stake in Transportadora de Gas del Perú (TgP)19.12.2025 19:42:00 EET | Press release
EIG, through its managed investment vehicles, acquired a 49.87% equity stake in Transportadora de Gas del Perú S.A. (“TgP”) from Canada Pension Plan Investment Board today. TgP operates Peru’s principal natural gas and natural gas liquids pipelines under a long-term concession, supplying approximately 40% of the country’s power generation. “We are delighted to complete this transaction and embark on the next chapter of our partnership with TgP,” said Matt Hartman, EIG’s Global Head of Infrastructure. “Our priority is to support TgP’s operational excellence and long-term stability, delivering value for customers and stakeholders throughout Peru.” About EIG EIG is a leading institutional investor in the global energy and infrastructure sectors with $24.3 billion assets under management as of September 30, 2025. EIG specializes in private investments in energy and energy-related infrastructure on a global basis. During its 43-year history, EIG has committed over $51.7 billion to the energ
CyberArk Named a Leader in IDC MarketScape: Worldwide Integrated Solutions for Identity Security 202519.12.2025 18:00:00 EET | Press release
CyberArk (NASDAQ: CYBR), the global leader in identity security, today announced that it has been recognized as a Leader in the IDC MarketScape: Worldwide Integrated Solutions for Identity Security 2025 Vendor Assessment. CyberArk extends dynamic privilege controls across all identity types with its unified platform, enabling organizations to improve efficiencies and streamline security operations. This IDC MarketScape report notes, “More change has occurred in the identity security marketplace in the past two years than in almost a decade. Vendors are entering a new phase defined by the emergence of intelligence technologies, none of which are specifically defined by any industry standards. Though different by design, the new adjacent IAM offerings are largely focused on improved vulnerability and threat management visibility and automated and predictive attack detection capabilities.” It also notes, “By addressing these evolving identity types within a unified framework, CyberArk enh
New York Liberty and Ant International’s Alipay+ Announce Multiyear Partnership Focused on Empowerment, Sustainability and Youth Development19.12.2025 15:30:00 EET | Press release
The New York Liberty and Ant International’s Alipay+, a leading cross-border fintech services platform based in Singapore, today announced a multiyear partnership, making Alipay+ an Official Sponsor and Innovation Partner for Sustainability of the New York Liberty. Through this partnership, Alipay+ and the Liberty will jointly support community programs designed to advance community empowerment, environmental sustainability and youth development across New York City. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251219678825/en/ Peng Yang, CEO, Ant International and Clara Wu Tsai, Vice Chair, Brooklyn Sports and Entertainment; Governor, New York Liberty “Our partnership with Alipay+ goes beyond the game,” said Keia Clarke, Chief Executive Officer, New York Liberty. “Together, we are investing in the future of New York—its people, its environment, and its youth. Ant International’s commitment to community empowerment, sustai
Parse Biosciences and Codebreaker Labs Partner to Apply Whole Transcriptome Single Cell Profiling and Causal Genomics at Scale19.12.2025 15:00:00 EET | Press release
Parse Biosciences, the leading provider of scalable and accessible single cell sequencing solutions, today announced a collaboration with Codebreaker Labs to develop and validate a breakthrough platform capable of testing thousands of genetic variants in parallel and measuring their effects at single cell resolution. By combining Codebreaker’s synthetic biology platform and variant engineering capabilities with the scale and accessibility of Parse’s Evercode™ technology, the collaboration aims to generate the causal data increasingly sought by AI developers, drug discovery teams, and clinical researchers. Today’s genomic studies rely heavily on observational data, or variants that appear in large populations. But rare and private variants, often only seen in one individual or family, are nearly impossible to study this way because too few carriers of the variant exist to draw statistically meaningful conclusions. As a result, even the most advanced models trained on observational datas
Cyviz Awarded Two Classified NATO Defense Contracts19.12.2025 09:00:00 EET | Press release
Norwegian technology and platform company Cyviz AS has been awarded two classified defense contracts by major European NATO-partner nations to deliver a fully mobile, containerised Command & Control (C2) capability, strengthening Europe’s operational readiness in high-threat and expeditionary environments. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251218625485/en/ Image from a previous military exercise where Cyviz demonstrated a mobile command center solution. The contracts cover the design, integration, and delivery of secure, deployable operations centres engineered to NATO security standards, enabling military and security forces to establish full theatre-class command capability wherever operations require. Due to the sensitive nature of the missions, contract value and customer identities remain undisclosed. From Demonstrated Readiness to Operational Deployment The awards follow growing interest among European def
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom