Study to Determine Safety, Pharmacokinetics and Efficacy of GMI-1271 in Combination With Chemotherapy in AML

Inclusion criteria:

1. AML (including secondary AML) diagnosed as per WHO criteria

2. For relapsed/refractory subjects only:

- Subjects age ≥ 18 years with relapsed or refractory AML after ≤ 2 prior induction
regimens, at least one containing anthracyclines

- Medically eligible to receive MEC

- Absolute blast count (ABC) ≤ 40,000/mm

3. For treatment-naïve subjects only:

- Subjects ≥ 60 years of age with newly diagnosed AML

- Medically eligible to receive "7+3" cytarabine/idarubicin

- ABC count ≤ 40,000/mm

4. ECOG performance status 0-2

5. Hemodynamically stable and adequate organ function

Exclusion criteria:

1. Acute promyelocytic leukemia

2. Acute leukemia of ambiguous lineage (biphenotypic leukemia)

3. Active signs or symptoms of CNS involvement by malignancy

4. No prior G-CSF, GM-CSF or plerixafor within 14 days of study drug dosing

5. Known history or evidence of active hepatitis A, B, or C or HIV

6. Uncontrolled acute life threatening bacterial, viral or fungal infection

7. Active graft versus host disease (GVHD) ≥ Grade 2 or extensive chronic GVHD requiring
immunosuppressive therapy

8. Hematopoietic stem cell transplantation ≤ 4 months of dosing

9. Clinically significant cardiovascular disease