A phase 3 trial designed to evaluate ruxolitinib for steroid-refractory acute graft-versus-host disease met its primary endpoint, the agent’s manufacturer announced in a press release.
Results of the REACH2 trial showed ruxolitinib (Jakafi, Incyte) significantly improved overall response rate at 28 days compared with best available therapy.
Researchers observed no new safety signals in the study, and adverse events among ruxolitinib-treated patients appeared consistent with the agent’s known safety profile.
Complete results will be submitted for presentation at a future medical meeting.
Ruxolitinib is an oral inhibitor of the JAK1 and JAK2 tyrosine kinases.
The FDA approved the agent earlier this year for treatment of steroid-refractory acute GVHD for patients aged 12 years and older. The agency based the approval on results of the single-arm, phase 2 REACH1 trial.
Up to half of patients who undergo hematopoietic stem cell transplantation develop acute GVHD, and the 1-year mortality rate associated with this complication ranges from 60% to 80%.
“GVHD is a challenging and serious disease, and physicians around the world need access to therapies that can improve outcomes for patients,” Peter Langmuir, MD, group vice president for targeted therapies at Incyte, said in the release. “This positive result of the REACH2 study is excellent news for patients as it further reinforces the potential of ruxolitinib as a treatment option that can provide meaningful results for patients with steroid-refractory acute GVHD.”
The phase 3 REACH3 study is underway to evaluate ruxolitinib for treatment of steroid-refractory chronic GVHD. Results are expected next year.
Ruxolitinib also is approved in the United States for treatment of certain patients with myelofibrosis and polycythemia vera.