Aop Orphan Pharmaceuticals GmbH

VIENNA--(BUSINESS WIRE)-- AOP Orphan Pharmaceuticals GmbH (AOP Health) announced the publication of final results on the recently developed interferon therapy, ropeginterferon alfa-2b, in patients with polycythemia vera (PV) in the prestigious journal Leukemia1. The full publication expands on results presented at the Annual Meeting of the European Hematology Association. Results of long-term treatment in the CONTINUATION-PV study provide further evidence of the disease modifying capacity of ropeginterferon alfa-2b in PV. This press release features multimedia. View the full release here: Portrait Dr. Rudolf Widmann/ AOP Health Group/ credit: Studio Koekart: Natascha Unkart & Isabelle Köhler (Photo: Business Wire) Link to Leukemia “AOP Health has invested in a comprehensive clinical research program for ropeginterferon alfa-2b in PV, which has led to the approval by the health authorities in several countries. We are very proud that our scientific work has contributed to addressing the questions that are important to patients”, says Dr. Rudolf Widman, Founder and Board Member of the AOP Health Group. Professor Heinz Gisslinger from the Medical University of Vienna, the first author of the paper, adds: “Ropeginterferon alfa-2b is already established as an effective first-line treatment for patients with PV. These new results suggest that its potential should be considered when evaluating treatment options.” 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 following stabilization of hematological parameters. AOP Health’s pivotal clinical development program for ropeginterferon alfa-2b includes 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 high-risk or low-risk PV who previously participated in the randomized, controlled PROUD-PV study. Clinical research conducted by AOP Health in Europe since 2010 has led to marketing authorization of BESREMi® for the treatment of PV, first granted by the European Commission in 2019, and thereafter by Switzerland, Liechtenstein, Israel, Bahrain, Taiwan, South Korea, Japan and the USA. 1 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). About Polycythemia Vera Polycythemia 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. View source version on businesswire.com: Contacts DI Isolde Fally AOP Orphan Pharmaceuticals GmbH Member of the AOP Health Group Isolde.Fally@aop-health.com +43-676-500 4048 aop-health.com Source: AOP Orphan Pharmaceuticals GmbH Smart Multimedia Gallery Photo Portrait Dr. Rudolf Widmann/ AOP Health Group/ credit: Studio Koekart: Natascha Unkart & Isabelle Köhler (Photo: Business Wire) Logo View this news release and multimedia online at:

VIENNA--(BUSINESS WIRE)-- With the publication of the final results from the LOW-PV study in the New England Journal of Medicine Evidence conducted by Fondazione per la Ricerca dell'Ospedale di Bergamo (FROM) under the leadership of Professor Tiziano Barbui, AOP Health announces an important advancement reinforcing its clinical development program for ropeginterferon alpha-2b (BESREMi®) in polycythaemia vera (PV), a rare blood cancer. The academic LOW-PV study supported and funded by AOP Health and public organizations in Italy complements a series of trials performed by AOP Health over more than 10 years to achieve marketing authorization in Europe and the Middle East. With these clinical studies, including PEGINVERA, PROUD-PV, and CONTINUATION-PV, AOP Health opened a new area of treatment options for patients suffering from PV. A further study (PEN-PV) was performed to develop a pen for self-injection allowing ease of self-administration, exact dosing and minimal waste of the medical product. AOP Health’s comprehensive development program in PV is considered by many key opinion leaders as the most significant development in the field of PV treatment in the past 30 years. This press release features multimedia. View the full release here: Dr. Rudolf Widmann, Founder and Board Member of the AOP Health Group/ Copyright: Studio Koekart: Natasche Unkart & Isabella Köhler (Photo: Business Wire) This series of studies has enabled marketing authorizations to be obtained in numerous countries in addition to the AOP Health territories EU, Switzerland, Liechtenstein and Israel and allows patients to have access to BESREMi® in countries including Taiwan, Korea, the US, and Japan. Further studies are ongoing to substantiate the long-term safety and efficacy of BESREMi®. A post-approval safety study (BESREMI-PASS) with a recruitment period of about three years has just completed patient recruitment. “The global marketing authorizations of BESREMi®, all based on AOP Health´s clinical development program conducted in Europe, are proof of the integrated drug development and commercialization expertise of AOP Health. Our success and know-how allow us to further pursue our goal of making treatment options for rare diseases available to patients worldwide” says Dr. Rudolf Widman, Founder and Board Member of the AOP Health Group. About BESREMi® BESREMi® is the first and currently only interferon approved for polycythaemia vera, a myeloproliferative neoplasm (MPN), indicated in the European Union as monotherapy in adults for treatment of polycythaemia vera without symptomatic enlarged spleen. Its overall safety and efficacy were demonstrated in multiple clinical studies. BESREMi® (ropeginterferon alfa-2b) is a long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered with a pen (250 and 500 ug) for self-injection once every 2 weeks initially, or up to every 4 weeks after stabilization of blood values. BESREMi® is designed to be self-administered subcutaneously with a pre-filled pen. For the EMA Summary of Product Characteristics please visit: BESREMi® Link: The drug substance 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 polycythaemia vera 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. The claim “Needs. Science. Trust.” sums up the foundation of the Group’s success: establishing trust through a continually high level of investment in research and development and a highly consistent and pragmatic orientation towards the needs of all stakeholders – especially the patients and their families as well as the healthcare professionals treating them. View source version on businesswire.com: Contacts Further inquiry DI Isolde Fally Isolde.Fally@aop-health.com +43-676-500 4048 From the US: 011-43-676-500 4048 Source: AOP Health Smart Multimedia Gallery Photo Dr. Rudolf Widmann, Founder and Board Member of the AOP Health Group/ Copyright: Studio Koekart: Natasche Unkart & Isabella Köhler (Photo: Business Wire) Logo View this news release and multimedia online at:

VIENNA--( BUSINESS WIRE )-- Fondazione per la Ricerca dell'Ospedale di Bergamo (FROM) announced the publication of the latest results from the LOW-PV trial in the NEJM Evidence. The findings support a paradigm shift in the treatment of patients with Polycythaemia Vera, a rare kind of blood cancer. AOP Orphan Pharmaceuticals GmbH (AOP Health) congratulates Prof. Tiziano Barbui and his team on this success and is pleased to have contributed to FROM’s important work as a reliable research partner. Find out more about the trial and its results here . 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. The claim “Needs. Science. Trust.” sums up the foundation of the Group’s success: establishing trust through a continually high level of investment in research and development and a highly consistent and pragmatic orientation towards the needs of all stakeholders – especially the patients and their families as well as the healthcare professionals treating them.