Pipeline

Autoimmune and Fibrotic Diseases Genetic Diseases
Pre-Clinical Phase 1 Phase 2 Phase 3
KD025 in Chronic Graft-Versus-Host Disease

Chronic graft-versus-host disease (cGVHD) is a potentially life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). cGVHD demonstrates common features of both autoimmune and fibrotic diseases, including aberrant immune system activation with consequent multi-organ fibrosis.

KD025 is an oral small molecule inhibitor of the Rho-associated coiled-coil kinase 2 (ROCK2) signaling pathway and the most advanced candidate in our ROCK inhibitor platform.  KD025 has demonstrated clinical activity and tolerability in a Phase 2 clinical trial (KD025-208) in patients with steroid-dependent or steroid-refractory cGVHD with 1-3 prior lines of treatment for the disease. The FDA has granted Breakthrough Therapy Designation to KD025 for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy.

Click here to view Phase 2 clinical trial data on KD025 in cGVHD.

Pivotal Trial (KD025-213)
KD025-213 is an ongoing open-label, registration clinical trial of KD025 in patients with cGVHD who have received two or more prior lines of systemic therapy. Patients are randomized to receive KD025 200 mg QD or 200 mg BID (63 patients per arm). Either KD025 dose may be considered by the FDA for registration. The primary endpoint is the ORR, defined as the percentage of patients who meet the 2014 National Institutes of Health (NIH) Consensus Conference overall response criteria of Complete Response (CR) or Partial Response (PR).

KD025 in Systemic Sclerosis

Systemic sclerosis is a chronic multi-system disease characterized by skin thickening and internal organ fibrosis. Approximately 75,000 to 100,000 people in the United States are living with systemic sclerosis. There are no FDA-approved targeted therapies for systemic sclerosis.

Phase 2
KD025-209 is a double-blind, placebo-controlled Phase 2 clinical trial of KD025 in systemic sclerosis. The trial will randomize 60 patients to receive KD025 200 mg QD, KD025 200 mg BID or placebo (20 patients per arm) for 24 weeks. The primary endpoint is the change in Combined Response Index for Systemic Sclerosis (CRISS) score, a measure of improvement in systemic sclerosis, at 24 weeks.

KD025 in Idiopathic Pulmonary Fibrosis

Idiopathic pulmonary fibrosis (IPF) is a progressive fibrotic disease of the lungs.  IPF is driven by prolonged activation of wound-healing responses that can lead to permanent scarring, lung malfunction and death.

Phase 2
KD025-207 is an ongoing Phase 2 clinical trial to examine the safety, tolerability and activity of KD025 in adults with IPF who have previously received or been offered pirfenidone and/or nintedanib. The study is enrolling patients randomized 2:1 to receive KD025 400 mg QD or best supportive care excluding pirfenidone and/or nintedanib.

KD034 in Wilson’s Disease

Wilson’s Disease is a genetic liver disease characterized by an inability to excrete copper, leading to severe hepatic, neurologic, psychiatric and/or ophthalmic abnormalities.

KD034 is our generic capsule formulation of trientine hydrochloride for the treatment of Wilson’s Disease patients who are intolerant of penicillamine.  Kadmon has submitted two Abbreviated New Drug Applications (ANDAs) to the U.S. Food and Drug Administration, including for KD034 capsules in blister packaging that offers room temperature stability.