TAS::75 0849::TAS TOPIC 255: A TARGETED DRUG FOR NON-MUSCLE INVASIVE BLADDER CANCER

CEL-031 is being developed as a novel, targeted tre8tment for non-mLiscle invasive bladder cancer (NMIBC). Bladder cancer is the 5th most common cancer in the US, with most cases diagnosed 8t the nonmuscle invasive stage. It is the most expensive cancerto treDt on a per patient basis. There is 8 significant need for more effective and less toxic therapies for NMIBC. CEl-03'1 8Cts through a novelmech()nism of action to selectively kill bladder cancer cells. It does tl1is by targeting certain enzymes that are overexpressed in these cells. In clinical trials, CEl-03'1 WDS well tolemted with preJiminmy evidence of effic8cy 8gainst advanced cancers. However, efficacy was limited by poor ora! bioavailnbi lity. CEl-031 should demonstrate greater efficacy against NI\l1IBC, since it ViiI! be delivered by bladder instillation, thereby minimizing drug metabolism nnd protein binding issues. This project will investigate the effect of CEl-03" on bladder tumor cells and test for potential synergy in combination\Nith oUler drugs. A liquid formulation will be developed, which will be used to test CEl-031's efficacy in 8 mouse bladder C8ncer model. If these studies are sllccessful, a proof-of-concept clinical study of CEl-03'! in NMIBC patients viill be initiatedduring the Phase II SBIR project