Novartis' Blood Cancer Drug Jakavi Hits Phase III Endpoint
Novartis reported that its potential blockbuster blood cancer drug Jakavi (ruxolitinib) hit its primary endpoint in a pivotal Phase III trial.
Jakavi is a JAK 1and JAK 2 inhibitor licensed by Novartis from Incyte which works by directly targeting underlying mechanisms of disease such as primary myelofibrosis and polycythemia vera. The drug achieved its primary endpoint in the RESPONSE Phase III trial of maintaining hematocrit control without requiring phlebotomy in patients. The drug was also successful in reducing size of enlarged spleens in patients involved in the trial.
RESPONSE is a global, randomized, open-label study conducted at 109 sites involving 222 patients with polycythemia vera resistant to or intolerant of hydroxyurea. Patients were randomized to receive either ruxolitinib at a dosage of 10 mg twice-daily or the best available therapy comprised of an investigator selected monotherapy or observation only. Ruxolitinib was able to meet the trial’s primary endpoint of spleen reduction by 35% from baseline at week 32 along with hematocrit control without phlebotomy. The drug was also able to keep its safety profile consistent with previous studies based on initial review of data.
Alessandro Riva, president of Novartis Oncology ad interim and global head, Oncology Development and Medical Affairs, said “We are encouraged by these pivotal Phase 3 trial results, which show the potential of ruxolitinib to help patients with polycythemia vera. We plan to submit these data to worldwide regulatory agencies this year, as we seek to bring ruxolitinib to patients with polycythemia vera who are no longer responding to or are intolerant of prior therapy.”
Polycythemia vera is an incurable, chronic cancer of the blood linked with uncontrollable production of red blood cells. The disease can lead to serious cardiovascular complications such as heart attack and stroke due to thickening of blood and heightened risk of blood clots. In addition, patients with polycythemia vera usually have enlarged spleens with other debilitating symptoms. A large percentage of patients treated with existing therapies become resistant or intolerant and are associated with increased risks of disease progression.
The company expects Jakavi to get approved next year for the indication of polycythemia vera in addition to its previous approval to treat myelofibrosis thanks to its performance in the Phase III trial.