A phase 2 trial of ruxolitinib met its primary endpoint of overall response rate for the treatment of patients with steroid-refractory graft-versus-host disease, according to the agent’s manufacturer.
The FDA previously granted ruxolitinib (Jakafi, Incyte) — a first-in-class JAK1/JAK2 inhibitor — breakthrough therapy designation and orphan drug designation for GVHD.
In the phase 2 single arm REACH1 trial, researchers assessed ruxolitinib in combination with corticosteroids among patients with steroid-refractory acute GVHD
Researchers observed an ORR of 55% at day 28. The best ORR was 73%.
The most common treatment-emergent adverse events of any grade included anemia (61%), thrombocytopenia (61%) and neutropenia (56%).
“The results of the REACH1 study demonstrate the potential of ruxolitinib to meaningfully improve the outcomes of allogeneic transplant patients who develop steroid-refractory acute GVHD and further underscore the promise of JAK inhibition to advance the treatment of this potentially devastating condition,” Steven Stein, MD, chief medical officer at Incyte, said in a press release. “We look forward to sharing additional results from this study with the medical community, and to working with U.S. regulatory authorities to submit our supplementary new drug application seeking approval of ruxolitinib in this indication later this year.”
Ruxolitinib is indicated for the treatment of people with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea, as well as for those with intermediate or high-risk myelofibrosis.