AOP Orphan announces three-year results on Ropeginterferon alfa-2b in Polycythemia Vera at ASH

Wien (pts045/03.12.2018/19:15) - * High rates of durable hematological response and symptom control with good tolerability were reconfirmed with Ropeginterferon alfa-2b after 36 months of treatment.
* Disease Modification by Ropeginterferon alfa-2b was illustrated by high molecular response rates, associated with the ability to reduce allelic burden of both mutant JAK2 and importantly also non-JAK2 mutations which are believed to have a role in disease progression.
* In line with molecular findings, disease or treatment related secondary malignancies including 2 cases of acute myeloid leukemia occurred only in patients receiving HU.
* AOP Orphan´s submission for marketing authorization of Ropeginterferon alfa-2b in the EU is in the final stage of EMA review.

AOP Orphan Pharmaceuticals AG (AOP Orphan) announces latest follow-up results on Ropeginterferon alfa-2b in patients with Polycythemia Vera (PV) from CONTINUATION-PV presented at ASH 2018.

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 novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered once every 2 weeks initially, or monthly after stabilization of hematological parameters. It is expected to be the first interferon approved for PV worldwide. AOP Orphan´s submission for marketing authorization in the EU is in the final stage of EMA review.

Clinical evidence:

Previously, at 12 months of treatment, Ropeginterferon alfa-2b was shown to be non-inferior to hydroxyurea (HU) in Complete Hematologic Response (CHR) and to have a significantly better safety and tolerability profile. . At 24 months Ropeginterferon alfa-2b achieved a significantly higher CHR of 70.5% versus 49.3% compared with HU/BAT (p=0.0101).

After 36 months of treatment Ropeginterferon alfa-2b sustained higher CHR response rates compared to HU/BAT (70.5% vs. 51.4%; p=0.0122). Further, the composite endpoint, CHR including disease symptom improvement was higher in patients treated with Ropeginterferon alfa-2b compared to HU/BAT (52.6% vs. 37.8%; p=0.0437).

Disease modification evidence:

66.0% of PV patients treated with Ropeginterferon alfa-2b, but only 27.0% having received HU/BAT showed a mutant JAK2 molecular response (p<0.0001) after 36 months. Importantly, molecular response strongly correlated with CHR, emphasizing the clinical relevance of mutant JAK2 allele burden reduction.