Ruxolitinib Five Year Safety and Efficacy

Below is a press release from Incyte Corporation with data that was presented at ASCO early June. 

- COMFORT-I data demonstrate that treatment with Jakafi resulted in a 31 percent reduction in the risk of death and sustained durable spleen volume reduction in patients with MF
- These five-year data support previously published findings for Jakafi

WILMINGTON, Del.--(BUSINESS WIRE)--Jun. 6, 2016-- Incyte Corporation (Nasdaq:INCY) today announces five-year data from the Phase 3 COMFORT-I study evaluating the long-term safety and efficacy of Jakafi^® (ruxolitinib) in patients with intermediate-2 or high-risk myelofibrosis (MF). These follow-up results showed an overall survival (OS) benefit among patients treated with ruxolitinib with a 31 percent reduction in the risk of death (HR=0.69; 95% CI: 0.50, 0.96; P=0.025) compared to patients randomized to placebo. Because the majority of patients on the placebo arm crossed over to receive ruxolitinib therapy (median 41.1 weeks after randomization), these data suggest that earlier treatment with ruxolitinib may be associated with improved long-term survival in patients with intermediate-2 or high-risk MF.
Additionally, over the five-year period, 59 percent (92/155) of patients who continued on treatment with ruxolitinib achieved at least a 35 percent reduction in spleen volume at any given time. The median duration of spleen response was 168.3 weeks. The mean spleen volume reduction from baseline at five years was 37.6 percent for patients who continued on treatment with ruxolitinib. After week 24, hemoglobin and platelet count remained stable through 5 years.

“Nearly, five years after the launch of Jakafi for the treatment of intermediate and high-risk MF, these findings provide important insight into the treatment’s long-term clinical benefits,” said Steven Stein, M.D., Incyte’s Chief Medical Officer. “The overall survival benefit observed in the COMFORT-I study, along with sustained reductions in spleen volume, are meaningful for patients with this rare disease who often experience significant, debilitating symptoms, and even mortality, as a result of their disease.”

These data are scheduled for presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting 2016 taking place June 3–7, 2016 in Chicago, Illinois.

“Previous findings from this study and overall survival trends reinforce the benefits of long-term treatment with ruxolitinib in patients with intermediate and high-risk MF. Achieving outcomes such as reduction in spleen volume is critical to the successful management of patients with this chronic and debilitating disease," said Ruben A. Mesa, M.D., FACP, Chair, Division of Hematology & Medical Oncology, Deputy Director, Mayo Clinic Cancer Center, Chair, Arizona Cancer Coalition, and Professor of Medicine.

Results from the COMFORT-I Study

COMFORT-I, a randomized (1:1), double-blind, placebo-controlled Phase 3 study, compared the efficacy and safety of ruxolitinib to placebo in 309 patients with intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

Of the 155 patients randomized to ruxolitinib, 28 percent of patients were still on treatment at the time of this analysis (median follow-up 268 weeks). Of the 111 patients originally randomized to the placebo arm who crossed over to ruxolitinib (median 41.1 weeks after randomization), 25 percent remained on treatment at the time of this analysis (median follow-up 268 weeks).

Overall, 69 (45%) and 82 (53%) deaths were reported in the ruxolitinib and placebo arms, respectively. Median OS has not been reached for patients randomized to receive ruxolitinib.

Adverse events (AEs) were consistent with those reported in previous studies of ruxolitinib and there was no increase in the incidence of AEs with longer exposure to treatment.

COMFORT-I (Abstract #7012) is scheduled for presentation by Dr. Mesa on Monday, June 6, 2016, 8:00–11:30 a.m., E354b Hall A.

Bottom Line:  The safety information cited in this press release is important, because Concert will be advancing d-ruxolitinib (CTP-543), for Alopecia Areata potentially on a long-term therapy basis.  Thank you for reading.