Phase III trial of P 1101 demonstrates superiority in Polycythemia Vera.- AOP Orphan Pharmaceuticals + PharmaEssentia.

A 36 month analysis of data with P 1101 (ropeginterferon alfa-2b), from AOP Orphan Pharmaceuticals and PharmaEssentia, in phase III clinical trials for Polycythemia Vera (PV) demonstrates superiority across three key aspects of disease control, including complete hematological response (CHR), disease burden, and molecular response. After 36 months of treatment, maintenance of higher responder rates (full analysis set) was shown in the ropeginterferon alfa-2b arm compared to hydroxyurea/best available therapy (HU/BAT) for CHR (70.5% vs. 51.4%; p=0.0122) and for CHR plus symptom improvement (52.6% vs. 37.8%; p=0.0437). In contrast to HU/BAT, response rates were steadily increasing in the ropeginterferon arm throughout 24 months of treatment and remained constant after 36 months. Strongly significant molecular response in the JAK2 mutant allele reduction and the ability to reduce the non-JAK2 mutant clone burden suggest potential disease modification capability. Importantly, molecular response strongly correlated with CHR, emphasizing the clinical relevance of mutant JAK2 allele burden reduction. Safety profile was similar to previous reports and no new safety signals emerged. Rate of adverse events (89.8% vs 90.6%) and treatment-related adverse events (74.8% vs 78.7%) were comparable between ropeginterferon alfa-2b and HU/BAT arms. No new safety signals appeared in the third year of treatment.

A long-term development partner of Ropeg, AOP Orphan's submission for marketing authorization of ropeginterferon alfa-2b in the EU is in the final stage of European Medicines Agency (EMA) regulatory review. PharmaEssentia is continuing to discuss with the US Food and Drug Administration (FDA) the best path forward to make ropeginterferon alfa-2b available to PV patients in the US. PharmaEssentia is also planning to initiate a global clinical development program of ropeginterferon alfa-2b in Essential Thrombocythemia (ET).