Acute GVHD is a life-threatening complication in which donor cells react with host tissue, producing life-threatening inflammation of the liver, the gastrointestinal tract, and the skin. Although approximately half of patients with acute GVHD respond to steroid treatment, steroid-refractory acute GVHD remains the most common cause of morbidity and mortality in patients undergoing allogeneic HSCT and, according to the American Cancer Society, occurs in approximately one-third to one-half of the 8,000 patients undergoing allogeneic HSCT in the United States each year.
Cantex believes that CX-01's potent inhibition of the interaction of HMGB1 with TLR4 may ameliorate acute GVHD, reduce mortality, and prevent the progression to chronic GVHD.
The Company plans support an investigator-initiated Phase IIa clinical trial evaluating the efficacy of CX-01 plus corticosteroids in the treatment of acute GVHD. The primary endpoint of the clinical trial will be complete remission of acute GVHD at day 28 after initiation of therapy. Additional endpoints will include relapse, relapse-related mortality, overall survival (OS), and the incidence of progression to chronic GVHD. Cantex anticipates top-line results of this clinical trial will be available in the second half of 2017.