Vienna, December 14, 2021:  AOP Orphan Pharmaceuticals GmbH (AOP Orphan) announced its latest analysis on Ropeginterferon alfa-2b in patients with Polycythaemia vera (PV) from its CONTINUATION-PV study presented at the ASH 2021 Annual Meeting by Professor Heinz Gisslinger from the Medical University of Vienna, Austria.

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.

AOP Orphan has been conducting a clinical development program since 2010, including the studies PEGINVERA,  PROUD-PV and CONTINUATION-PV. 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.

AOP Orphan’s clinical development program was the basis for the marketing authorizations of BESREMi® for the treatment of Polycythaemia vera in the European Union in February 2019, in Switzerland and in Taiwan in 2020, and in Israel and Korea in 2021. In November 2021, based on AOP Orphan's clinical development program, the FDA approved BESREMi® for the treatment of Polycythaemia vera in the USA.

The presentation during the ASH 2021 Annual Meeting focused on the treatment response to Ropeginterferon alfa-2b in PV patients who were either treated naïve or pre-treated with HU:

High rates of hematological and molecular responses were sustained at 5 years in both HU-naïve and HU pre-treated patients in the Ropeginterferon alfa-2b arm, even in patients with a high baseline JAK2V617F burden of more than 50%. In contrast, in the control arm, response rates were lower in HU pre-treated patients. Although early initiation of Ropeginterferon alfa-2b treatment is thought to have the greatest disease modifying benefit in patients with PV¹, these data suggest that Ropeginterferon alfa-2b is also a suitable treatment option for patients switching from HU therapy, and for those with more active disease.

“This latest presentation at the American Society of Hematology Annual Meeting is yet another example of the high scientific knowledge and development capabilities of our company.” says Dr. Rudolf Widmann, Member of the Governing Board of AOP Orphan International AG.

Professor Jean-Jacques Kiladjian, from the Saint-Louis Hospital & Université de Paris in France stated earlier this year, “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.”

¹ Kiladjian JJ, Klade C, Georgiev P, et al. Towards a potential operational cure in patients with polycythaemia vera? Results from five years’ ropeginterferon alpha-2b therapy in a randomized setting. In: HemaSphere, 2021;5:(S2). Abstract Book. Abstract nr EP1076.