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AUSSENDER



AOP Orphan Pharmaceuticals Aktiengesellschaft
Ansprechpartner: AOP
Tel.: +43 503 72 44 24
E-Mail: nina.roth@aoporphan.com

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pts20201208021 Medizin/Wellness, Forschung/Entwicklung

AOP Orphan announces 5-year results on BESREMi® (Ropeginterferon alfa-2b) in PV at the ASH Annual Meeting 2020


Vienna (pts021/08.12.2020/20:15) - Ropeginterferon alfa-2b showed clear advantages in efficacy and safety/tolerability versus best-available-treatment (mostly hydroxyurea) in this long-term treatment analysis in the randomized-controlled setting.

Freedom of phlebotomy with hematocrit below 45 % was achieved in 81.8 % of patients receiving Ropeginterferon alfa-2b vs. only 63.2 % in the control group (p=0.01).

Mutant JAK2 allele burden decreased from 37.3 % at baseline to 7.3 % in patients receiving Ropeginterferon alfa-2b indicating disease modification, while it increased from 38.1 % to 42.6 % in the control group (p<0.0001).

Disease progression was reduced to 0.2%-per-patient year (one case of myelofibrosis) in patients receiving Ropeginterferon alfa-2b vs. 1.0%-per-patient year observed in the control group (two cases of transformation to acute leukemia and two cases of myelofibrosis).
Safety and tolerability of Ropeginterferon alfa-2b were generally good, with no new safety signals during the 5th year of treatment. Major thromboembolic events were rare occurring in only 4 patients receiving Ropeginterferon alfa-2b versus 5 patients in the control group during the entire 5-year treatment period.

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. AOP Orphan AG continues to develop Ropeginterferon alfa-2b in the field of Myeloproliferative Neoplasms including ET, MF and CML.

Vienna, December 7, 2020: AOP Orphan Pharmaceuticals AG (AOP Orphan) announced latest follow-up results on Ropeginterferon alfa-2b in patients with Polycythaemia Vera (PV) from its CONTINUATION-PV study presented at ASH 2020 by Univ. Prof. Dr. Professor Heinz Gisslinger, Medical University of Vienna, Austria.

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.

BESREMi® approved for use in 31 European countries

Ropeginterferon alfa-2b is a novel, 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 AOPs clinical trials the European Commission (EC) has granted Marketing Authorization for BESREMi® (Ropeginterferon alfa-2b) as first line monotherapy in adults for the treatment of Polycythaemia vera (PV) without symptomatic splenomegaly. BESREMi® is now approved for use in the 31 European countries covered by the European Medicines Agency (EMA) and Switzerland and Liechtenstein.

Professor Jean-Jacques Kiladjian from the Saint-Louis Hospital & Université de Paris in France, stated, "Ropeginterferon alfa-2b is a valuable and safe 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"

5-year treatment update confirmed positive results

The positive results for Ropeginterferon alfa-2b reported previously, were confirmed in the current 5-year treatment update: 70 patients in the Ropeginterferon alfa-2b arm and 57 in the control arm remained on study at the time of analysis. Discontinuation rates were balanced between the treatment arms (Ropeginterferon alfa-2b: 26.3%; control: 25.0% during the entire CONTINUATION-PV trial). Ropeginterferon alfa-2b achieved higher and sustained rates of hematological responses: the key patient relevant parameter "freedom of phlebotomy with hematocrit below 45 %" was achieved in 81.8 % of patients receiving Ropeginterferon alfa-2b vs. only 63.2% in the control group (p=0.01).

Even more strikingly, mutant JAK2 allele burden, which is believed to be the causative oncogene in PV, decreased from 37.3% at baseline to 7.3 % in patients receiving Ropeginterferon alfa-2b, while it increased from 38.1 % to 42.6 % in the control group (p<0.0001).

In line with the observed disease modification (reduction of mutant JAK2 allele burden) the natural disease progression was reduced to 0.2%-per-patient year (one case of myelofibrosis) in patients receiving Ropeginterferon alfa-2b vs. 1.0%-per-patient year observed in the control group( two cases of transformation to acute leukemia and two cases of myelofibrosis).

Safety and tolerability of Ropeginterferon alfa-2b were generally good, and no new safety signals appeared in the 5th year of treatment. Major thromboembolic events were rare occurring in only 4 (Ropeginterferon alfa-2b arm) versus 5 patients (BAT arm) during the entire 5-year treatment period. A specific assessment of skin toxicity revealed that skin cancers (in 3.2% of patients) and skin ulcers (in 5.5% of patients) were exclusively reported in the control group.

About BESREMi®
BESREMi® is a novel, 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 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: 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 Orphan. In 2009, AOP Orphan in-licensed the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in PV and other MPNs 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
AOP Orphan 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

Ms. Nina Roth, MAS, Corporate Communications Manager
nina.roth@aoporphan.com

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Aussender: AOP Orphan Pharmaceuticals Aktiengesellschaft
Ansprechpartner: AOP
Tel.: +43 503 72 44 24
E-Mail: nina.roth@aoporphan.com
Website: www.aoporphan.com
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