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January 13, 2020
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Phase 3 study of itacitinib for acute GVHD misses key endpoints

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A randomized phase 3 study designed to evaluate itacitinib for patients with treatment-naive acute graft-versus-host disease failed to meet its primary and key secondary endpoints, according to the agent’s manufacturer.

Itacitinib (INCB039110, Incyte) is a novel JAK1 inhibitor under investigation for chronic and acute GVHD.

The double-blind, placebo-controlled GRAVITAS-301 study compared the addition of first-line itacitinib or placebo to corticosteroids.

Overall response rate at day 28 served as the primary endpoint.

Results showed a higher ORR among patients who received itacitinib (74% vs. 66.4%), but the difference did not reach statistical significance.

Results also showed no significant difference between treatment groups with regard to nonrelapse mortality at 6 months, the study's key secondary endpoint.

The safety profile of itacitinib in GRAVITAS-301 appeared consistent with that observed in prior studies of the agent combined with corticosteroids. The most common adverse events included thrombocytopenia (34.9% with itacitinib vs. 34.7% with placebo) and anemia (29.8% vs. 25%).

Complete results of GRAVITAS-301 will be submitted for presentation at a future scientific meeting. Healio will provide updates when those results are available.

“The result of this study is disappointing. ... We will continue to study the role of JAK inhibition in chronic GVHD and in the prophylactic setting, as we seek to develop treatments for patients with this debilitating and often fatal disease,” Steven Stein, MD, Incyte's chief medical officer, said in a company-issued press release.