Updated results from phase III studies of P 1101 (ropeginterferon alfa-2b) for patients with polycythemia vera- AOP Orphan Pharmaceuticals and PharmaEssentia

AOP Orphan Pharmaceuticals and PharmaEssentia Corporation announced latest results from 3 phase III clinical studies on P 1101 (ropeginterferon alfa-2b) for patients with Polycythemia Vera (PV) presented at EHA 2017. Abstract EHA-3520: An update from the PEGINVERA study (NCT: 2010-018768-18) on long-term maintenance treatment of up to 6 years (median of 4 years) with Ropeginterferon alfa-2b was provided. Patients were successfully switched from the once every 2 weeks to the more convenient once every 4 weeks' long-term maintenance dosing schedule after a median of approximately 2 years. All 29 patients could be maintained on this schedule for another 2 years, representing 100% treatment adherence. The high rates of hematological (over 80% of the patients achieved partial or complete hematologic response) and molecular responses (up to 80% of the patients achieved partial or complete molecular response) were maintained after the switch to the 4 weeks' schedule and remain stable (trial still ongoing). After 4 years on Ropeginterferon alfa-2b, the majority of patients showed a sustained reduction of the mutant JAK2V617F allelic burden to below 10%, demonstrating the disease modifying capability of Ropeginterferon alfa-2b treatment. No disease progression was reported.

Abstract EHA-3556: To alleviate the known burden of frequent health care office visits for product administration, home self-administration and patient individual dosing of Ropeginterferon alfa-2b were performed in the PEN-PV study (NCT: 2014-001356-31). Based on investigator assessments, none of the 36 patients exhibited any visible pain or physical discomfort, nor did any patient report pain arising from the use of the pen. The Ropeginterferon alfa-2b pen was well accepted by patients and health care professionals, hematological parameters and spleen size remained stable throughout the study and there were no safety concerns. The pen allows for individual dosing and patient-convenient self-administration at home. It is expected to support adherence to Ropeginterferon alfa-2b in the long-term treatment of PV-patients.

Abstract EHA-1564: To assess the disease modifying capability of Ropeginterferon alfa-2b compared to hydroxyurea (HU), the effect of treatment on hematopoietic bone marrow progenitor cells was investigated in 10 patients of the French PROUD-PV study population (NCT01949805). Although at 12 months, both drugs led to a decrease of the mutant JAK2V617F allelic burden in peripheral blood, only Ropeginterferon alfa-2b induced a meaningful decrease of mutated hematopoietic bone marrow progenitor cells (median decrease 64% compared to only 25% under HU).