First Patient Treated With Jakafi in Phase 2 Graft-Versus-Host Disease Study

The first patient has been treated with ruxolitinib (Jakafi), by Incyte Corporation, for steroid-refractory acute graft-versus-host disease (GVHD), as part of the REACH-1 phase 2 trial. The trial is evaluating ruxolitinib in combination with corticosteroids.

GVHD, a significant cause of morbidity and mortality in transplant recipients, can occur after an allogeneic transplant. In GVHD, the donated bone marrow or peripheral blood stem cells attack the body. The condition is very rare, occurring in fewer than 20,000 people in the United States each year.

“We are very pleased to have the first patient treated in the pivotal trial program for ruxolitinib in GVHD, a severe and often life-threatening condition for which there are currently no approved treatments,” Steven Stein, MD, Incyte’s chief medical officer, said in a statement. “We look forward to building on the clinical evidence to-date for ruxolitinib and working with regulators to help address the urgent needs of these patients.”

There are 2 forms of GVHD: acute and chronic. With acute GVHD, the skin, the gastrointestinal tract, and the liver are most commonly affected. Ruxolitinib has been granted Breakthrough Therapy Designation for the treatment of acute GVHD by the FDA. It has also been granted Orphan Drug Designation for the treatment of GVHD.

REACH-1 is a single-cohort phase 2 study that is sponsored by Incyte. It is also expected to include collaborative Novartis-sponsored pivotal studies in steroid-refractory acute GVHD and steroid-refractory chronic GVHD. The Novartis-sponsored studies are expected to begin early this year.

Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor. It is already approved for treatment of people with polycythemia vera and is indicated for treating people with intermediate or high-risk myelofibrosis. In the United States, the drug is marketed as Jakafi by Incyte, but outside of the US, it is marketed as Jakavi by Novartis.

Common side effects of Jakafi are low blood counts, infection, skin cancers, and increases in cholesterol.