Clinical Data Safety Results

• The most common Grade 3/4 adverse events (AEs) regardless of cause were thrombocytopenia (61%), anemia (44%), febrile neutropenia (44%) and neutropenia (30%).
• Investigator reported IDH differentiation syndrome was reported in four patients, of which three were serious AEs. All four cases resolved, among whom two achieved a complete response (CR), one stable disease and one was not evaluable for response.
• Mean neutrophil and platelet counts were maintained near or above thresholds for CR with partial hematologic recovery (CRh) while on study treatment with TIBSOVO® and azacitidine. CRh is defined as <5% of blasts in the bone marrow, no evidence of disease and partial recovery of peripheral blood counts (ANC >500/microliter and platelets >50,000/microliter).

Efficacy Results

• Overall, 78% (18/23) of patients had a response.
• 70% (16/23) of patients had a CR or CRh.
• 61% (14/23) of patients had a CR.
• The median duration of CR (95% CI 9.3, NE) as well as CR+CRh (95% CI 12.2, NE) had not been reached.
• The median time to response was 1.8 months (range 0.7-3.8 months) and the median time to CR was 3.7 months (range 0.8-15.7 months).
• The 12-month survival rate was 82% (95% CI 58.8, 92.8).
• The median duration of follow-up was 16.1 months (range 1.3-31.7 months).
• For patients who achieved a CR, IDH1 mutation clearance was observed in 9 of 14 (64%) patients with available bone marrow mononuclear cells (BMMCs) and 11 of 14 patients (79%) with available peripheral blood mononuclear cells (PBMCs) as quantified by a digital PCR assay with lower limit of sensitivity for mutant IDH1 of 0.02-0.04% (or 10-4).