AOP Orphan Pharmaceuticals Announces Progress of Pivotal Phase III Trial PROUD-PV

AOP Orphan Pharmaceuticals (AOP Orphan) has reported the completion of recruitment for its phase III trial PROUD-PV to support global licensure of Ropeginterferon alfa 2b (AOP2014/P1101), a novel, long-acting, mono-pegylated Interferon for the treatment of Polycythemia Vera (PV). Importantly, Ropeginterferon alfa 2b is administered only every other week. After achieving therapy response, administration frequency may be further extended to monthly intervals. Ultimately, this is expected to result in improved tolerability, convenience and compliance and, as a consequence, better long-term treatment outcomes.

AOP Orphan has exclusively licensed, Ropeginterferon alfa 2b for development and commercialization in the field of Myeloproliferative Neoplasms (MPNs) from Pharmaessentia Corp. a biotech company based in Taiwan for European, CIS, and Middle Eastern markets. Pharmaessentia retains the rights for development and commercialization of P1101 in other major markets, such as North America, Asia, and South America.

Based on very encouraging Phase I/II data, AOP Orphan has set up the pivotal phase III trial PROUD-PV. Design and endpoints of this trial have been discussed and agreed with both the EMA and the FDA, to support global licensure of Ropeginterferon alfa 2b, which also has Orphan Drug status in both Europe and the US.

Since its commencement in October 2013 more than 260 PV patients have been recruited in 50 centers all across Europe. Enrollment of patients has been successfully completed in February 2015. Patients are either treated with Ropeginterferon alfa 2b, or hydroxyurea, which is a registered treatment for PV for one year. Throughout the trial, a number of clinical and hematological parameters are assessed. Treatment with Ropeginterferon alfa 2b is expected to be safe and effective in the majority of patients and to be superior to hydroxyurea.

“We already know from several smaller studies that interferons work effectively against myeloproliferative diseases”, remarked both Professors Jean-Jacques Kiladjian from Paris and Heinz Gisslinger from Vienna.

“The unparalleled quick uptake and progress of PROUD-PV, completing enrollment of over 260 patients in around 15 months, proves the eagerness of both patients and physicians for this new treatment paradigm”, said Dr Rudolf Widmann, CEO of AOP Orphan.

Results from a phase I/II trial sponsored and conducted by AOP Orphan presented at ASH (American Society of Hematology) in 2012, 2013 and 2014 appear very encouraging: the overall clinical response rate including reduction of red and white blood cells and platelets was around 90% and after 6–12 months of treatment, 45–50% of patients even showed complete response. Importantly, after one year all patients were completely independent from phlebotomies. In addition, all patients remaining in this trial could be switched to an even more convenient monthly treatment schedule.