Kadmon Holdings Inc. said updated efficacy and safety results from its ongoing phase 2 trial of KD025 graft-versus-host disease remain positive.
The study, called KD025-208, is evaluating KD025 as a treatment for chronic graft-versus-host disease, or cGVHD, which occurs when transplanted blood or bone marrow stem cells attack the recipient's body.
Corticosteroid therapy is a mainstay approach for the disease. Kadmon's trial enrolled patients with cGVHD that is dependent on or unresponsive to steroids. The company gave 17 patients 200 milligrams of KD025 once a day, and 16 patients 200 mg of KD025 twice a day.
Kadmon said 11 patients from each group responded to treatment — 65% and 69% in the once-daily and twice-daily groups, respectively. The responses remained for at least 20 weeks in eight patients in the once-daily group, and six patients in the twice-daily group, according to initial durability data.
Meanwhile, 66% or 22 of the 33 patients enrolled were able to reduce their corticosteroid doses after receiving KD025, and 15%, or 5 patients, completely discontinued corticosteroid use. Additionally, 55%, or 18 patients, saw a clinically meaningful improvement in cGVHD symptoms.
KD025 remained well-tolerated, causing no serious adverse events or apparent increase in risk of infection, according to Kadmon, which presented the data at the European Hematology Association's 23rd Congress in Stockholm.
"These study results continue to show accumulation of clinical benefit and underscore the significant potential of KD025 in cGVHD, particularly in patients with multiple organs involved," President and CEO Harlan Waksal said in a news release.
New York-based Kadmon said it plans to start a pivotal study of KD025 in the third quarter of 2018, based on previous guidance from the U.S. Food and Drug Administration.