NOVOMEDIX TRANSITION TO PHASE II - THERAPEUTICS SBIR CONTRACTSBIR PHASE II - ENHANCED OVERALL SURVIVAL IN HIGH-RISK PEDIATRIC SOLID TUMORS USING A NOVEL ORAL SMALL MOLECULE

The overall goal of this project is the commercialization of a novel, first-in-class, small molecule to safely andeffectively treat rare, high-risk, pediatric tumors; and to increase survival rates without the long-term sideeffects which arise from current treatments. The phase 1 effort focused on rhabdomyosarcoma, anextremely rare pediatric cancer (- 250 patients/year). The goals of this effort are to expand the patient poolfor a pediatric clinical trial to include other high-risk sarcomas and blastomas to decrease accrual times; toidentify which drugs can safely and effectively be used in combination with the drug candidate as pediatriconcologists are reluctant to give new drugs as single agents; and to complete IND-enabling studies and filean IND. Therefore, the aims of this study are: 1) confirm efficacy in other rare pediatric tumor types anddetermine synergy with commonly used chemotherapy drugs in vitro, 2) determine safety of chemotherapycombinations in vivo, 3) conduct animal studies alone and in combination with chemotherapy in 5 pediatriccancer models to demonstrate in vivo efficacy, 4) complete IND-enabling studies, 5) optimize the drugproduct, and 6) prepare and submit an IND.