KayoThera specializes in pioneering a novel class of safe oral antagonists that are the first of their kind to target retinoid signaling. Previously considered “undruggable” the retinoid pathway has posed challenges to drug development for nearly three decades. Past clinical experience with retinoid pathway activators has revealed two consistent findings among thousands of patients: adverse outcomes in solid cancers and a range of cardiometabolic effects, from hyperlipidemias to insulin resistance.

In response, KayoThera has developed a comprehensive pipeline of drugs focused on inhibiting retinoid signaling based on new biological insights to the pathway. The lead program in oncology aims to target retinoid synthesis in immune cells, while the cardiometabolic program concentrates on inhibiting retinoids in the pancreas, muscle, and fat-storing tissues. By pursuing this innovative approach, KayoThera aims to address critical medical needs and potentially revolutionize the treatment landscape in both oncology and cardiometabolic diseases.


Credit: Judy Rakin


KayoThera’s retinoid pathway inhibitors exhibit remarkable potency, as evidenced by cellular activity below 10 nM in in vitro studies. Moreover, in vivo experiments demonstrate excellent safety and drug-likeness. Notably, preclinical investigations in Type 2 diabetes reveal the ability to modify the disease by regenerating diseased pancreatic beta cells, ultimately restoring insulin balance in affected animals. Based on these findings, we believe that our retinoid inhibitors could serve as a potent and enduring solution to combat the global epidemic of Type 2 diabetes.


KayoThera’s lead compound in the field of oncology exhibits potent immune activation leading to tumor stasis when administered alone and achieves an impressive nearly 50% complete response (CR) rates when combined with anti-PD-1 antibodies in syngeneic models. We anticipate that this treatment will have clinical benefit for patients with specific types of genetically-defined cancers that have amplified the retinoid pathway components.


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