Kadmon Announces Updated Positive Results from Phase 2 Study of KD025 in cGVHD

“KD025 continues to demonstrate a therapeutic benefit in cGVHD, including in the most severe manifestations of the disease characterized by end-organ fibrosis,” said Aleksandr Lazaryan, M.D., MPH, Ph.D., Assistant Professor of Medicine, Division of Hematology, Oncology and Transplantation, University of Minnesota and study investigator. “KD025 was also well tolerated, and many patients have been able to reduce or discontinue steroids and other immunosuppressants, thereby helping to avoid their adverse effects and improve patients’ quality of life.”

Updated data from Cohort 2 of the trial (KD025 200 mg BID; n=16) showed an Overall Response Rate (ORR) of 69%, as of a data cutoff date of January 3, 2018. Responses in Cohort 1 (KD025 200 mg QD; n=17) remained the same, showing an ORR of 65%. Responses were rapid, with approximately 70% of patients across Cohorts 1 and 2 achieving responses by the first assessment (after 8 weeks of treatment). Responses were observed across all affected organs, including in organs with fibrotic manifestations of the disease such as lungs, eyes, skin and joints. In responders with 4 or more organs involved, 6/13 (46%) showed responses in 4 or more organs. In addition, 64% of patients were able to reduce steroid dose, and four patients completely discontinued steroid use. Eighty-three percent (83%) of patients were able to reduce the dose of tacrolimus, another immunosuppressive agent used to treat cGVHD. KD025 was well tolerated, with no drug-related serious adverse events (SAEs) in either cohort.

“These updated results in cGVHD and recent results in idiopathic pulmonary fibrosis further demonstrate the activity and tolerability of KD025 in inflammatory and fibrotic disease settings,” said Harlan W. Waksal, M.D., President and CEO at Kadmon. “We look forward to continuing our study of KD025 in cGVHD and the potential opportunity to offer patients a new therapy for this disease.”

Details of the presentation are as follows:

The BMT Tandem Meetings are the combined annual meetings of the Center for International Blood & Marrow Transplant Research (CIBMTR) and the American Society for Blood and Marrow Transplantation (ASBMT).

About KD025-208

KD025-208 is an ongoing Phase 2 clinical trial of KD025 for the treatment of cGVHD. The trial is being conducted in adults with steroid-dependent or steroid-refractory cGVHD and active disease. The dose-finding trial includes 48 patients divided into three cohorts at different dose levels (KD025 200 mg QD, 200 mg BID and 400 mg QD), enrolled sequentially following a safety assessment of each cohort. We expect to enroll an expansion cohort of approximately 40 patients after the optimal dose has been determined. In October 2017, KD025 received orphan drug designation from the U.S. Food and Drug Administration for cGVHD.

About cGVHD

cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation, a procedure that is often used to treat patients with cancers such as myeloma or leukemia. With cGVHD, transplanted immune cells (graft) attack the patient’s cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and GI tract.

About Kadmon Holdings, Inc.

Kadmon Holdings, Inc. is a fully integrated biopharmaceutical company developing innovative products for significant unmet medical needs. We have a product pipeline focused on inflammatory and fibrotic diseases.

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