doctor reviewing MRI


We are a clinical stage pharmaceutical company focused on developing novel, well-tolerated, once-daily oral treatments for cancer, and other serious illnesses.


Our pipeline consists of our azeliragon product candidate, currently in clinical development for the treatment of glioblastoma, brain metastases, pancreatic cancer, breast cancer, and prevention of acute kidney injury in hospitalized pneumonia patients. Azeliragon clinical development targets cancers and other serious illnesses for which innovative and more effective treatments are greatly needed.


Azeliragon is an orally administered small molecule, taken once-daily, that inhibits RAGE interactions with its natural ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Overactivation of RAGE by these ligands stimulates cancer and its progression and metastasis and resistance to cancer treatment. Azeliragon was originally under development for Alzheimer’s disease by the company from which we licensed azeliragon. Clinical safety data from these trials, involving over 2000 individuals dosed for periods up to 18 months, indicates that azeliragon is very well tolerated. There is substantial mechanistic and pre-clinical evidence suggesting Azeliragon could have efficacy in cancer and other serious illnesses.