JAKAFI
JAKAFI (ruxolitinib) is an oral small-molecule inhibitor developed by Incyte that blocks Janus kinase 1 and 2 (JAK1/JAK2), enzymes whose overactivity drives abnormal blood cell proliferation and inflammatory signaling in myeloproliferative diseases. The drug is approved for myelofibrosis, polycythemia vera, and steroid-refractory graft-versus-host disease; in myelofibrosis, JAK inhibition reduces the enlarged spleen and debilitating constitutional symptoms caused by progressive marrow fibrosis. Incyte is studying ruxolitinib combined with the PI3Kδ inhibitor parsaclisib in patients who have an inadequate response to JAKAFI monotherapy.
Upcoming catalysts
Programs
Myelofibrosis
Myelofibrosis is a clonal bone marrow malignancy driven by aberrant JAK-STAT signaling that causes progressive marrow fibrosis, massive splenomegaly, severe constitutional symptoms, and a high risk of AML transformation; JAKAFI is first-line standard of care. This Phase 3 program adds parsaclisib (PI3Kδ inhibitor) to JAKAFI in patients with sub-optimal spleen or symptom responses to ruxolitinib monotherapy, measuring spleen volume reduction (SVR35) and total symptom score (TSS50) at week 24.