Patients receiving Ropeginterferon alfa-2b experienced clear advantages compared to the control treatment (mostly hydroxyurea) in this long-term analysis in the randomized-controlled setting.

• Majority of patients treated with Ropeginterferon alfa-2b achieved deep molecular responses (mutant JAK2 allele burden below 10%): allele burden decreased from median 37.3% at baseline to 7.3%, while it increased from 38.1% to 42.6% in the control group (p<0.0001).
• Achieving deep molecular response correlated statistically significant with lower age and lower allele burden at the start of treatment.
• Patients achieving deep molecular responses had the benefit of statistically significant more and longer clinical responses (Complete Haematological Response).
• BESREMi® is the first interferon having gained marketing authorization in any of the Myeloproliferative Neoplasms. It is approved as first-line monotherapy in adults for the treatment of Polycythaemia vera (PV) without symptomatic splenomegaly throughout Europe and Israel. AOP Orphan continues to develop Ropeginterferon alfa-2b in the field of Myeloproliferative Neoplasms including ET, MF and CML.

Vienna, July 1st, 2021: AOP Orphan Pharmaceuticals (AOP Orphan) announced its latest analysis on Ropeginterferon alfa-2b in patients with Polycythaemia vera (PV) from its CONTINUATION-PV study presented at EHA 2021 by Professor Jean-Jacques Kiladjian from the Saint-Louis Hospital & Université de Paris in France.

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 PV who previously participated in the PROUD-PV study. 

Ropeginterferon alfa-2b is a long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered once every 2 weeks initially, or monthly after stabilization of hematological parameters. Based on data from AOP Orphan clinical trials the European Commission (EC) has granted Marketing Authorization for BESREMi® (Ropeginterferon alfa-2b) as a first line monotherapy in adults for the treatment of Polycythaemia vera (PV) without symptomatic splenomegaly. BESREMi® is now approved for use in the 30 European countries covered by the European Medicines Agency (EMA) and in addition in Switzerland, Liechtenstein, United Kingdom and Israel.

This latest analysis focused on the disease modifying capability of Ropeginterferon alfa-2b:

The majority of patients treated with Ropeginterferon alfa-2b achieved deep molecular responses (mutant JAK2 allele burden below 10%). Mutant JAK2 is believed to be responsible for and driving PV. In patients receiving Ropeginterferon alfa-2b for up to five years, mutant JAK2 allele burden decreased from 37.3% at baseline to 7.3%, while it increased at the same time from 38.1% to 42.6% in the control group (p<0.0001).

Patients achieving deep molecular responses had the benefit of statistically significant more and longer clinical responses (Complete Hematological Response).

Achieving deep molecular response correlated statistically significant with lower age and lower allele burden at the start of treatment, underscoring the value of starting Ropeginterferon alfa-2b early.

Professor Jean-Jacques Kiladjian stated, “Ropeginterferon alfa-2b is a valuable new first line therapy for PV patients. The disease modification ability of Ropeginterferon alfa-2b treatment, is suggested by the striking JAK2 molecular response, and we hope that this may translate into slowing disease progression and thus improving progression-free survival and long-term patient benefit.”

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 self-administered at home subcutaneously with a pen once every two weeks, or 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®  Link  

Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Orphan. In 2009, AOP Orphan in-licensed the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in PV and other MPNs 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.

AOP Orphan Pharmaceuticals AG is an international pharmaceutical company with its registered office in Vienna and a focus on special diseases with a complex management. Over the past 25 years, the company has become an established provider of holistic therapy solutions from its headquarters in Vienna. 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 our stakeholders on the other - especially the patients and their families but also the doctors and care professionals treating them. In the third quarter of 2020, AOP Orphan took over Amomed and SciPharm, two European health care companies, continuing its consistent path of growth into a pan-European health care group specializing in special diseases with a complex management.

Contact
AOP Orphan Pharmaceuticals AG
Wilhelminenstrasse 91/IIf, 1160 Vienna, Austria
Dr. Christoph Klade, Chief Scientific Officer
christoph.klade@aoporphan.com