Incyte Corporation announced that the first patient has been treated in the REACH-1 pivotal phase 2 trial evaluating ruxolitinib (Jakafi) in combination with corticosteroids for the treatment of patients with steroid-refractory acute graft-versus-host disease (GVHD).

“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,” said Steven Stein, M.D. Incyte’s Chief Medical Officer. “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.”

GVHD is a condition that might occur after an allogeneic transplant (the transfer of genetically dissimilar blood stem cells). In GVHD, the donated bone marrow or peripheral blood stem cells view the recipient’s body as foreign and attack the body. There are two forms of GVHD: acute and chronic. GVHD is a significant cause of morbidity and mortality in transplant recipients. The skin, gastrointestinal (digestive) tract, and liver are the most commonly affected organs in patients with acute GVHD.

The US Food and Drug Administration (FDA) has granted ruxolitinib Breakthrough Therapy designation for the treatment of acute GVHD, designed to expedite the development and review of drugs for serious or life-threatening conditions, as well as Orphan Drug designation for the treatment of GVHD, granted to investigational compounds intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people.

The REACH clinical trial program for ruxolitinib in steroid-refractory acute GVHD includes the Incyte-sponsored REACH-1 study—a single-cohort, pivotal phase 2 study (NCT02953678) evaluating ruxolitinib in combination with corticosteroids in patients with steroid-refractory acute GVHD—and is also expected to include collaborative Novartis-sponsored randomized pivotal studies in steroid-refractory acute GVHD and steroid-refractory chronic GVHD. The Novartis-sponsored pivotal studies are expected to begin in early 2017.

The primary endpoint of the REACH-1 study is overall response rate at day 28. Key secondary endpoints include duration of response, overall response rate at day 14, 56, and 100, non-relapse mortality and safety.

Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor approved by the US Food and Drug Administration, as Jakafi® (ruxolitinib), for treatment of people with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.

Jakafi is also indicated for treatment of people with intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF, and post–essential thrombocythemia MF.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States.