Pipeline

PIPELINE CANDIDATES

Novel Targeted Agents for Pancreatic cancers: Pancreatic cancer is one of the most devastating diseases amongst all cancers. Only 15 to 20% of patients with pancreatic cancer are eligible for surgical resection and all patients are treated with chemo and radiation therapies as standard of care. Many of these agents have serious side effects including neutropenia and immunosuppression, neurotoxicity, nephrotoxicity, and many others. Life threatening side effects and cancer recurrence in treated patients prompted us to undertake this project with the intention to rapidly translate novel, well-tolerated, and effective treatment protocols for an improved therapeutic outcome for these patients. Our work in this area utilizes an innovative drug delivery of highly potent cytotoxic agents selectively to tumor sites. These selective agents do not use any antibodies and do not have the need to release the payloads inside the tumor.

Novel drug candidates for non-alcoholic steatohepatitis: Nonalcoholic fatty liver disease (NAFLD) has emerged in the recent past as the leading cause of chronic liver disease, affecting ~20% to 30% of the population in the United States alone. NAFLD has a complex pathophysiology ranging from relatively benign fatty liver to the aggressive non-alcoholic steatohepatitis (NASH). Untreated NASH can progress to liver fibrosis, cirrhosis, hepatocellular carcinoma, and end-stage liver disease. Hence novel drugs are urgently required for patients with advanced stages of NASH disease. Our drug candidates are highly potent targeting a novel mechanism involved in NASH pathophysiology. These candidates are expected to decrease metabolite flux and mitigate increased ROS induced tissue injury. Furthermore, the associated inhibition of critical metabolite oxidation should also lead to increased mitochondrial fatty acid oxidation, further promoting the recovery of lipid-mediated inflammation in hepatic fibrosis.

RBB-1009SV: Development of targeted therapies by exploiting the genetic differences in cancers. Our work in this area has identified candidate compounds which achieve synthetic lethality in BRCA mutated cancers through new molecular mechanisms of action. These agents also overcome resistance to clinically used PARP inhibitors.

Oral drug candidates that selectively inhibit Clostridium difficile infections: C. difficile is the leading cause of nosocomial diarrhea and colitis in developed countries. This pathogen secrets different protein toxins that can drive disease pathogenesis. One of the leading causes of recurrent C. difficile infections is the long-term use of broad-spectrum antibiotics which disturb gut microbiota. In this regard, development of drug candidates that selectively inhibit C. difficile virulence without affecting gut microbiota is critically needed. Our work in this regard has identified a candidate compound with the ability to prevent primary and recurrent C. difficile infections.