Positive comparison study of Jakavi and best available therapy for polycythemia vera.- Novartis.

Novartis announced results from a new comparison study showing that Jakavi (ruxolitinib)-treated patients with polycythemia vera (PV), who were resistant or intolerant to hydroxyurea (HU), had a significantly reduced risk of thrombosis (blood clots) and death compared to PV patients who received best available therapy The study findings are based on a comparison of patients in the Phase III RESPONSE Jakavi clinical trial and the real-world Spanish GEMFINI patient registry. PV is a rare and incurable blood cancer associated with an overproduction of blood cells that can cause serious cardiovascular complications, such as blood clots, stroke and heart attack[ The new findings were presented at the 23rd Congress of the European Hematology Association (EHA) in Stockholm, Sweden.

About the PV Real-World Comparison Study - The new data presented at EHA compares overall survival and thrombosis (blood clots) rates using data from patients treated in the Jakavi arm of the RESPONSE trial and patients treated in a real-world setting with best available therapy (BAT) from the Grupo Español de Enfermedades Mieloproliferativas Crónicas Filadelfia Negativas (GEMFIN) registry. In the previously reported Phase III RESPONSE trial, the high rate of crossover from BAT to Jakavi precluded the comparison of overall survival and thrombosis rates.

RESPONSE was a global, open-label study that included patients with PV resistant to or intolerant of hydroxyurea, who were randomized 1:1 to receive either Jakavi (starting dose of 10 mg twice daily) or BAT, which was defined as investigator-selected monotherapy or observation only. The GEMFIN registry patients in the real-world BAT group had resistance or intolerance to hydroxyurea according to the modified European Leukemia Net criteria and received hydroxyurea (44%), busulfan (10%), radioactive phosphorus (2%), interferon (6%), anagrelide (12%), other therapy (11%) or no cytoreductive therapy (26%). Some patients were also treated with multiple therapies. In the GEMFIN study, patients treated with Jakavi had a significantly prolonged overall survival (HR=0.28 [0.11-0.72]) and a lower risk of blood clots (HR=0.21 [0.06-0.76]) when compared to real-world patients treated with BAT.

See- Alvarez-Larran, et al. Comparison of Ruxolitinib Real-World Best Available Therapy in Terms Of overall Survival and Thrombosis in Patients with Polycythemia Vera who are Resistant or Intolerant to Hydroxyurea. EHA 2018. Abstract PF628..