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 serious, sometimes life-threatening complication following stem cell transplantation from blood or bone marrow.  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 (ROCK) signaling pathway and the lead candidate in our ROCK inhibitor platform.  Preclinical research has indicated that KD025 targets the immunologic and fibrotic aspects of cGVHD, demonstrating improvement across multiple organs affected by the disease.

Click here to view scientific publications on the role of ROCK inhibition in autoimmunity and fibrosis.

Phase 2
KD025-208 is an ongoing Phase 2 clinical trial to examine the safety, tolerability and activity of KD025 in adults with steroid-dependent or steroid-refractory cGVHD and active disease. This dose-finding study is enrolling patients 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.  In results presented at the 59th American Society of Hematology (ASH) Annual Meeting in December 2017, KD025 was reported to be well tolerated, with clinically meaningful responses in patients enrolled in the first two cohorts of the trial.

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.

KD025 is an oral small molecule inhibitor of the Rho-associated coiled-coil kinase (ROCK) signaling pathway and the lead candidate in our ROCK inhibitor platform.  In preclinical studies, KD025 attenuated pulmonary fibrosis, significantly reducing inflammation and fibrosis in the lung in a dose-dependent manner.

Click here to view scientific publications on the role of ROCK inhibition in autoimmunity and fibrosis.

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.

KD025 in Moderate to Severe Psoriasis

Psoriasis is a chronic, immune mediated, inflammatory skin condition affecting as many as 7.5 million people in the United States. Most psoriasis patients (approximately 80% to 90%) have chronic plaque psoriasis, characterized by recurrent exacerbations of thickened, erythematous, scaly patches of skin that can occur anywhere on the body.

KD025 is an oral small molecule inhibitor of the Rho-associated coiled-coil kinase (ROCK) signaling pathway and the lead candidate in our ROCK inhibitor platform. In preclinical studies, KD025 down-regulated pro-inflammatory responses and increased T regulatory (Treg) cell function, helping to rebalance the immune response instead of suppressing the entire immune system. In a completed Phase 2 clinical trial in moderate to severe psoriasis, KD025 was well tolerated and demonstrated Psoriasis Area and Severity Index (PASI) score reductions in 85% of patients completing the study.

Click here to view scientific publications on the role of ROCK inhibition in autoimmunity and fibrosis.

Phase 2
KD025-211 is an ongoing randomized, double-blind, placebo-controlled Phase 2 clinical trial to examine the safety, tolerability and activity of KD025 in adults with moderate to severe psoriasis.  The study is enrolling patients across five cohorts: KD025 200 mg QD, 200 mg BID, 400 mg QD, 600 mg (400 mg a.m., 200 mg p.m.) and placebo.

Tesevatinib in Autosomal Dominant Polycystic Kidney Disease

Autosomal dominant polycystic kidney disease (ADPKD) is an inherited disorder affecting adults and is characterized by the formation of fluid-filled spherical cysts, primarily in kidneys.  ADPKD leads to loss of kidney function and rapid progression to end-stage renal disease.

Tesevatinib is an oral, reversible inhibitor of epidermal growth factor receptor (EGFR), a central driver of PKD progression.

Phase 2a
KD019-101 is a Phase 2a dose-escalation clinical trial to examine the safety and pharmacokinetics of tesevatinib in adults with ADPKD.  In a preliminary data analysis, tesevatinib 50 mg QD demonstrated safety and tolerability, with patients showing no significant increase in kidney size and no deterioration in kidney function.

Phase 2 (Enrolling)
KD019-103 is a randomized, double-blind, placebo-controlled, Phase 2 clinical trial to examine the safety and activity of tesevatinib 50 mg QD in adults with ADPKD.

Tesevatinib in Autosomal Recessive Polycystic Kidney Disease

Autosomal recessive polycystic kidney disease (ARPKD) is a rare inherited disorder affecting infants.  ARPKD causes cyst formation in multiple organs, leading to significant morbidity and mortality in childhood, with those surviving typically requiring dialysis by the age of 10.

Tesevatinib is an oral, reversible inhibitor of epidermal growth factor receptor (EGFR), a central driver of PKD progression.

Phase 1
KD019-103 is an ascending, single-dose, Phase 1 clinical trial to examine the safety of tesevatinib in pediatric patients with ARPKD.  The study consists of three cohorts: tesevatinib 0.25 mg/kg, 0.50 mg/kg and 1.0 mg/kg. In order to accommodate the pediatric population, we developed a taste-masking liquid formulation of tesevatinib.

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.