A nearly completed clinical trial of CPI-613 (Cornerstone Study# CL-CPI-613-009 or Wake Forest Study# CCCWFU 29109, under IND# 107,800) shows that CPI-613 is well tolerated at doses as high as 3,000 mg/m2. Results from this nearly completed trial also suggest that CPI- 613 may be effective against refractory and relapsed AML, as well as against MDS that is relapsed from a hypomethylating agent. Therefore, CPI-613 may be a suitable treatment option for refractory/relapsed AML and MDS relapsed from a hypomethylating agent. The promising preliminary efficacy data from Study# CL-CPI-613-009 (Wake Forest Study# CCCWFU 29109, under IND# 107,800) is the basis on which Cornerstone is conducting the current Phase 2a trial to further assess the efficacy of CPI-613 against these diseases.