AOP Orphan has announced completion of recruitment of its pivotal phase III trial PROUD-PV (proud-pv.com) to support global licensure of Ropeginterferon alfa 2b.
Ropeginterferon alfa 2b is a novel, long-acting, mono-pegylated Interferon, administered once every 14 days, after achieving therapy response only monthly.
Based on previous phase I/II trials, Ropeginterferon alfa 2b is expected to be safe and effective in the majority of PV patients.
Haematological and molecular responses occurred in most patients, while several patients achieved undetectable mutated JAK2 levels and a complete normalisation of their chromosomal make-up.
Ultimately, this is expected to result in improved tolerability, convenience and compliance and, as a consequence, better long-term treatment outcomes.
Design and endpoints of this trial have been discussed and agreed with both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA), to support global licensure of Ropeginterferon alfa 2b, which also has Orphan Drug status in both Europe and the USA.
AOP Orphan has exclusively licensed Ropeginterferon alfa 2b for development and commercialisation 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.
Since its commencement in October 2013 over 260 PV patients have been recruited in 50 centres across Europe. Enrolment of patients was successfully completed in February 2015.
Treatment with Ropeginterferon alfa 2b is expected to be safe and effective in the majority of patients and to be superior to hydroxyurea.
Professors Jean-Jacques Kiladjian from Paris and Heinz Gisslinger from Vienna said: “We already know from several smaller studies that interferons work effectively against myeloproliferative diseases.”
Dr. Rudolf Widmann, CEO of AOP Orphan, said: “The unparalleled quick uptake and progress of PROUD-PV, completing enrolment of over 260 patients in around 15 months, proves the eagerness of both patients and physicians for this new treatment paradigm.”