It has a primary endpoint defined as overall response rate at day 28 of the single-cohort clinical trial.
Some of its key secondary endpoints include response duration, overall response rate at the 14th, 56th and the 100th days besides non-relapse mortality and safety.
Ruxolitinib was granted a breakthrough therapy designation by the US Food and Drug Administration (FDA) for the treatment of acute GVHD in June 2016.
GVHD is a condition that might occur following an allogeneic transplant. It affects skin, liver and the gastrointestinal tract in its acute form. The condition also causes morbidity and mortality in transplant recipients.
A JAK1/JAK2 inhibitor, ruxolitinib was approved by the FDA for the treatment of polycythemia vera (PV) patients with inadequate response to or are hydroxyurea intolerant.
It is also indicated for treating intermediate or high-risk myelofibrosis (MF). It is marketed by Incyte in the US while Novartis has the marketing rights outside the US.
The REACH clinical study of the drug in steroid-refractory acute GVHD which includes the REACH-1 study sponsored by Incyte is also likely to feature randomized pivotal studies in steroid-refractory acute GVHD and steroid-refractory chronic GVHD which will be carried out in collaboration with Novartis.
Novartis will be sponsoring the pivotal studies which are likely to commence early this year.
Photo: Incyte’s phase 2 study of ruxolitinib has commenced. Photo: courtesy of jk1991 at FreeDigitalPhotos.net.