Incyte Corporation (Nasdaq: INCY) today announced that a pivotal Phase III trial of ruxolitinib compared to best available therapy in patients with polycythemia vera who are resistant to or intolerant of hydroxyurea has met its primary endpoint of achieving phlebotomy independence and reducing spleen size by 35 percent or more. The safety profile of ruxolitinib was generally consistent with previous studies based on initial review of the data. Ruxolitinib, marketed as Jakafi® (ruxolitinib) in the United States, is approved to treat people with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF.
"One out of four patients with polycythemia vera remain uncontrolled despite existing standard therapies, and these patients face a profound symptom burden and are at greater risk of cardiovascular complications. These Phase III data give us confidence that ruxolitinib may offer a welcome new treatment option," stated Herve Hoppenot, President and Chief Executive Officer, Incyte.
The global, randomized, open-label Phase III trial, called RESPONSE, was conducted at 109 sites. The trial included 222 patients with polycythemia vera resistant to or intolerant of hydroxyurea. Patients were randomized 1:1 to receive either ruxolitinib (10 mg twice-daily) or best available therapy. The dose was adjusted as needed throughout the trial.
The primary endpoint of the trial is the proportion of patients whose hematocrit level is controlled in absence of phlebotomy and whose spleen volume is reduced by 35 percent or more from baseline as assessed by imaging at 32 weeks. In addition to safety, key secondary endpoints include durable response and complete hematological remission.
Data from RESPONSE are expected to be presented at an upcoming scientific meeting and submitted to the U.S. Food and Drug Administration this year.
Source: Incyte Corporation