Study of FF-10101-01 in Patients With Relapsed or Refractory Acute Myeloid Leukemia

Subjects will receive FF-10101-01 orally once a day repeated every 28 days =1 cycle Frequent
blood draws will be collected to measure pharmacodynamic parameters and pharmacodynamic
activity.

Disease assessments, including bone marrow aspirates, will be performed at the beginning of
cycles 1-3, and every 3 months thereafter. Subjects who demonstrate objective response or
stable disease will be allowed to continue therapy with FF-10101-01 until , observation of
unacceptable adverse events, or until the subject is no longer deriving benefit based on the
opinion of the investigator.

For Phase 2a long term phone follow-up for assessment of overall survival will also occur.

Subjects who are able and willing to give written informed consent

- Documented primary or secondary AML, as defined by the WHO criteria (2008), by
histopathology refractory to previous induction chemotherapy and/or relapsed after
achieving remission with a prior chemotherapy and who are not candidates for other
available therapy likely to confer clinical benefit.

- For Phase 2a only: in addition to inclusion criteria 2 above, patients must have a
FLT3 mutation of any type

- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2

- In the absence of rapidly progressing disease, the interval from prior treatment to
time of FF-10101-01 administration should be at least 14 days for cytotoxic agents
other than hydroxyurea, at least 5 half-lives for non-cytotoxic agents, and 14 days
for monoclonal antibody therapies. Hydroxyurea may be continued for a maximum of 14
days from the start of FF-10101-01 dosing, through Cycle 1 Day 14, with a maximal dose
of 5 grams/day

- Persistent chronic clinically significant toxicities from prior chemotherapy or
surgery must be ≤Grade 2

- If subject has had a hematopoietic stem cell transplant, subject must be ≥60 days
post-transplant with no clinically significant GVHD requiring systemic therapy

- Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≤3 times the
upper limit of normal and total bilirubin of ≤1.5x the upper limit of normal. If total
bilirubin is equal to or exceeds 1.5x the upper limit of normal, the subject can still
be included if direct bilirubin is ≤1.5x the upper limit of normal

- Calculated creatinine clearance of ≥60 mL/min

- Female subjects of childbearing potential and sexually mature male subjects must agree
to use a medically accepted method of contraception other than an oral contraceptive
for the duration of the study.

Exclusion Criteria:

- Subjects diagnosed with acute promyelocytic leukemia

- Subjects with Bcr-Abl positive leukemia (chronic myelogenous leukemia in blast crisis)

- Subjects with clinically active CNS leukemia

- Subjects with major surgery within 28 days prior to the first administration of
FF-10101-01

- Subjects with radiation therapy within 28 days prior to the first administration of
FF-10101-01

- Subjects with active malignant disease requiring therapy other than AML or
myelodysplastic syndrome with transformation into AML

- Subjects with an active uncontrolled infection

- Subjects with a medical condition, serious intercurrent illness, or other circumstance
that, in the Investigator's judgment, could jeopardize the subject's safety as a study
subject, or that could interfere with the study objectives

- Subjects known to have human immunodeficiency virus infection, or who have active
hepatitis B or C infection as determined by serological testing

- Subjects with congestive heart failure, New York Heart Association (NYHA) Class 3 or
4, or subjects with a past history of congestive heart failure NYHA Class 3 or 4 and
in whom echocardiogram or multiple gate acquisition (MUGA) scan performed within 3
months prior to screening or at screening showed a LVEF <40%

- Female subjects who are pregnant or breast feeding

- Subjects on 3-hydroxy-3-methylglutaryl-coenzyme A reductase inhibitors (statins) or
other drugs known to have muscle toxicity

- Subjects taking strong inhibitors of CYP3A4 will be excluded from the study unless
therapeutic substitution is possible

- Subjects taking strong inducers of CYP3A4 will be excluded from the study unless
therapeutic substitution is possible

- Use of systemic immunosuppressive agents within 14 days prior to first dose of
FF-10101

- Subjects taking drugs known to cause Torsades de Pointes will be excluded from the
study unless therapeutic substitution is possible

- Subjects known to have long QT syndrome

- Subjects with mean QTcF values following 3 ECGs conducted 5 minutes apart of >470 msec