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Published on 10 March 2015

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AOP Orphan Pharmaceuticals announces progress of Phase III Trial PROUD-PV

  • Ropeginterferon alfa 2b is a novel, long-acting, mono-pegylated Interferon, administered only once every 14 days, after achieving therapy response even 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 occured in most patients; moreover, several patients achieved undetectable mutated JAK2 levels and a complete normalisation of their chromosomal make-up.

  • Ropeginterferon alfa 2b is a novel, long-acting, mono-pegylated Interferon, administered only once every 14 days, after achieving therapy response even 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 occured in most patients; moreover, several patients achieved undetectable mutated JAK2 levels and a complete normalisation of their chromosomal make-up.

AOP Orphan Pharmaceuticals AG (AOP Orphan) 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 licenced, 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 commercialisation of P1101 in other major markets, such as North America, Asia, and South America.

Based on very encouraging Phase I/II data (see below), 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 European Medicines Agency EMA and the US FDA, to support global licensure of Ropeginterferon alfa 2b, which also has Orphan Drug status in both Europe and the USA.

Since its commencement in October 2013 over 260 PV patients have been recruited in 50 centres all across Europe. Enrolment 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 haematological 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 enrolment 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.

Haematological responses correlated with molecular responses: JAK2 is the driver mutation of PV and the JAK2 allelic burden was significantly reduced by treatment with Ropeginterferon alfa 2b. Several patients even achieved undetectable mutated JAK2 levels. The clinical relevance of the JAK2 molecular responses was further substantiated by genome wide analysis revealing a complete normalisation of the chromosomal make-up in several patients (Them et al. American J Haematology 2015). These encouraging finding provide the hope for a cure of at least some patients.



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