Novartis blood cancer drug ruxolitinib meets Ph III endpoint

Swiss drug major Novartis (NOVN: VX) revealed yesterday that it expects to file for approval in the second quarter of this year for its investigational Janus kinase (JAK) inhibitor INC424 (ruxolitinib), after the drug met its primary endpoint of significantly reducing spleen size in patients with myelofibrosis (MF), a life-threatening blood cancer, in a second Phase III study.

The European study, called COMFORT-II (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Therapy), showed treatment with INC424 provided a statistically-significant reduction in spleen size in patients with primary MF, post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF), when compared with best available therapy, administered at doses and schedules determined by the investigator. The safety profile of INC424 was consistent with previous studies. Complete study data will be submitted to an upcoming medical meeting.

These results support findings from another large Phase III clinical trial (COMFORT-I) conducted by the collaboration partner, Incyte Corp (Nasdaq: INCY), in the USA, Canada and Australia comparing treatment with INC424 to placebo in patients with MF at 24 weeks. In addition, a Phase I/II study published in the September 16, 2010 issue of The New England Journal of Medicine showed that treatment with INC424 resulted in marked, fast and durable clinical benefits in patients with MF. These benefits included alleviation of debilitating symptoms and reduction of spleen size, an accepted measurement of clinical improvement in MF.