Incyte Corporation (Nasdaq:INCY) announced today positive top-line results from COMFORT-I, the pivotal Phase III clinical trial of INCB18424 (also known as INC424) in patients with myelofibrosis (MF) being conducted under a Food and Drug Administration (FDA) Special Protocol Assessment (SPA) Agreement. COMFORT-I (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Treatment) is a double-blind, placebo-controlled Phase III trial involving 309 patients. The primary endpoint was the response rate defined as the percentage of patients achieving a 35% or greater reduction in spleen volume at 24 weeks as measured by magnetic resonance imaging, or computerized tomography, comparing the rates in patients receiving INCB18424 or placebo. The response rate was 42% in patients randomized to INCB18424 versus less than 1% of patients randomized to placebo; thus a high level of statistical significance (p < 0.0001) was achieved.
“In addition to meeting the efficacy endpoints in this controlled trial, the safety and tolerability profile suggests that INCB18424 could become the first widely available medication to relieve the debilitating symptoms in patients with myelofibrosis”
High levels of statistical significance were also achieved for the key secondary endpoints based on symptomatic improvement as measured by the modified Myelofibrosis Symptom Assessment Form Diary. Response rates among patients receiving INCB18424 were similar to those previously reported with INCB18424 in the Phase II trial while much lower response rates were reported for patients receiving placebo in COMFORT-I.
The safety profile of INCB18424 was consistent with previous studies, which included reversible thrombocytopenia and anemia.
Incyte intends to submit the data from COMFORT-I for presentation at the 2011 American Society of Clinical Oncology Annual Meeting.
Paul Friedman, M.D., President and CEO of Incyte stated, "These highly significant results from COMFORT-I are similar to what was seen in the Phase II trial recently published in the New England Journal of Medicine. We look forward to continuing our work with the FDA and our investigators as we strive to make this new medicine available to patients with myelofibrosis as rapidly as possible. In this regard, we are proceeding with the preparation of a New Drug Application and believe INCB18424 has the potential to become the first FDA-approved treatment for this debilitating, life-threatening disease."
"In addition to meeting the efficacy endpoints in this controlled trial, the safety and tolerability profile suggests that INCB18424 could become the first widely available medication to relieve the debilitating symptoms in patients with myelofibrosis," added Richard Levy, M.D., Executive Vice President and Chief Drug Development and Medical Officer at Incyte.
Based on the SPA agreement, COMFORT-I is the only pivotal trial required for approval in the U.S. Data from a second Phase III trial, COMFORT-II, which is the pivotal trial for European Marketing Authorization, comparing INCB18424 to best available therapy, is being conducted by Novartis in Europe and is also expected to provide supportive data for approval and labeling in the U.S. Results of COMFORT-II are expected early next year. Incyte and Novartis announced a collaboration and license agreement in November 2009 in which Incyte retained exclusive rights to develop and commercialize INCB18424 in the U.S. and Novartis received exclusive rights to develop and commercialize INCB18424 for territories outside the U.S. Both the FDA and the European Medicines Agency have granted INCB18424 orphan drug status for MF.