PharmaEssentia (Taipei, Taiwan) and AOP Orphan (Vienna, Austria) announce the completion of recruitment for the Phase III trial PROUD-PV to support global marketing of P1101 (Ropeginterferon alfa-2b), a novel, long-acting, mono-pegylated interferon for the first line treatment of polycythemia vera.
The pivotal Phase III trial PROUD-PV commenced enrollment in October 2013, with over 260 PV patients now recruited in 14 countries and 50 centers all across Europe. Both naive and treatment-experienced patients have been enrolled and patients are either treated with P1101 or hydroxyurea, the current first line standard of care. The primary endpoint is a composite endpoint of hematocrit, platelets, leukocytes, and spleen size at 12 months.
Design and endpoints of this trial have been discussed and agreed upon with the European Medicines Agency. 12 month final primary endpoint data from the European PROUD-PV trial will also be used to form the basis of submission to US FDA, obviating the need for a US-based registrational trial. This submission to US FDA could potentially be earlier, depending on the data and ongoing discussions with US FDA.
Polycythemia vera is an orphan disease affecting 100,000 individuals in the United States with a similar sized population in the EU, with a potential market size in the billions. There is currently only one other drug approved in the US for the treatment of polycythemia vera -- Incyte's Jakafi, which is approved for second line treatment of PV. P1101 (Ropeginterferon alfa-2b) targets first line treatment of PV. PharmaEssentia estimates that ~65-75% of all PV patients are first line, with 25-35% comprising second line patients.
P1101 (Ropeginterferon alfa-2b) was discovered by scientists at PharmaEssentia and PharmaEssentia exclusively licensed the rights to P1101 (Ropeginterferon alfa-2b) for European, CIS, and Middle Eastern markets in the field of myeloproliferative neoplasms (MPN) to AOP Orphan in 2009, a multinational biotechnology company based in Vienna, Austria. PharmaEssentia retains the rights to P1101 (Ropeginterferon alfa-2b) in other major markets, such as North America, Asia, and South America.
In addition to polycythemia vera, PharmaEssentia is exploring usages for P1101 (Ropeginterferon alfa-2b) as an interferon with an improved side effect profile. These include the fields of essential thrombocythemia, chronic hepatitis B, chronic hepatitis C (genotype 2), and oncology (in combination with PD-1 targeted agents). Essential thrombocythemia is a field very similar to polycythemia vera and there have been no new FDA approvals in this indication for almost 2 decades. Chronic hepatitis B is an area of high unmet need, particularly in Asia, for which nucleoside analogues have several shortcomings. Among these include drug resistance and continuous treatment. Interferon, however, has a direct enhancing effect on the immunologic response to HBV, enabling short courses of therapy and low drug resistance. Pegylated interferon has also demonstrated as high as 90% response rates in Asian patients who carry the genotype 2 chronic hepatitis C virus. A once every two weeks Ropeginterferon will provide a better choice for these patients. Finally, PharmaEssentia is also exploring the potential synergistic effect of Ropeginterferon alfa-2b with immuno-check point inhibitors like PD-1 that it is developing in-house.
"Our collaboration with AOP was a strategic move, as early on we recognized the value of P1101 across several different fields of therapy, based on several publications in medical literature. We expect that if we enhance the compliance for patients when they take a more tolerable interferon, the expanding indications are very achievable to that of Humira, which is now approved in 8 indications with $12.5B of sales. Thus, the completion of recruitment of PROUD-PV marks a very important milestone for this asset. In particular, we are very thankful to AOP Orphan for this fruitful partnership as well as for the support of the US FDA in the process of bringing this important asset to the US market," remarks Ko-Chung Lin, CEO of PharmaEssentia.