Drug Discovery

Through our drug discovery platforms, we identify and develop new product candidates for significant unmet medical needs

Our biologics and small molecule chemistry drug discovery platforms augment our pipeline of clinical-stage product candidates. We leverage our multi-disciplinary team of scientific experts and our advanced understanding of the molecular mechanisms of disease to establish development paths for disease areas where significant unmet medical needs exist.

In April 2015, we transferred our gene therapy platform to MeiraGTx Limited, an independent clinical-stage company in which Kadmon retains a meaningful interest.

Small Molecules

ROCK Inhibitors

Using innovative medicinal chemistry, computational and structure-based design approaches, Kadmon has developed a portfolio of proprietary, next-generation inhibitors of Rho-associated coiled-coil kinase (ROCK), an “on” switch in cells that regulates cell movement, shape and differentiation.  Two ROCK isoforms exist: ROCK1 and ROCK2, and dysregulation of ROCK-driven cellular functions is implicated in many chronic diseases.   Kadmon research has demonstrated that inhibition of ROCK signaling can regulate aberrant immune responses and fibrotic processes.

Kadmon’s ROCK inhibitor platform includes a distinct series of oral ROCK2 and pan-ROCK (ROCK1/2) inhibitors in preclinical and clinical development.  Our portfolio includes pan-ROCK and ROCK2-selective inhibitors for autoimmune and fibrotic diseases and blood-brain barrier-penetrant pan-ROCK inhibitors for neurodegenerative diseases.

GLUT Inhibitors

The emerging field of immuno-metabolism has identified the regulation of glucose transports (GLUT) as a requisite component of immune cell growth and activation. Kadmon is developing oral, potent small molecule GLUT inhibitors that are designed to block aberrant immune cell activity. In preclinical studies, our GLUT inhibitors were well tolerated and reduced inflammation at therapeutically relevant doses.


Kadmon’s fully human monoclonal antibody platform is run by an experienced team of scientists who have developed commercially successful biologics, including Erbitux® (cetuximab) and Cyramza® (ramucirumab). We are developing monoclonal antibodies as well as fusion proteins and bi-specific antibodies that we believe represent the next generation of cancer therapeutics.

Our most advanced candidate from our biologics platform, KD033, is a novel anti PD-L1/IL-15 fusion protein that combines the effects of two complementary immuno-oncology approaches to potentially achieve greater efficacy than single-agent therapy.

We are also developing KD035, an antibody targeting vascular endothelial growth factor receptor 2 (VEGFR2), which inhibits the formation of new blood vessels (angiogenesis), thus blocking blood supply to tumors.