AG-221: Interim Phase I data

Interim data from 45 evaluable patients in an open-label, dose-escalation U.S. and French Phase I trial showed that once-daily doses of oral AG-221 up to 200 mg and twice-daily doses of oral AG-221 up to 150 mg led to an overall response rate (ORR) of 56%, including 6 complete responses, 4 complete responses with incomplete platelet recovery (CRp), 4 marrow complete responses (mCR), 1 complete response with incomplete hematologic recovery (CRi) and 10 partial responses. There were 17 cases of stable disease. AG-221 was well tolerated with 1 dose-limiting toxicity (DLT) of hypoxia reported. The MTD has not been reached and dose escalation is ongoing. The most common adverse events reported were nausea, pyrexia, diarrhea and fatigue. The most common serious adverse events reported were leukocytosis, disseminated intravascular coagulation and tumor lysis syndrome. Data will be presented at American Society of Hematology meeting in San Francisco in December. ...