STAT3 Inhibitor for Solid Tumors

The Study Drug:

OPB-31121 is designed to block certain proteins in cancer cells that cause the cancer cells
to reproduce and form more cancer cells.

Study Groups:

If you are found to be eligible to take part in this study, you will be assigned to a dose
level of OPB-31121 based on when you joined this study. The first group of participants
will receive the lowest dose level. Each new group will receive a higher dose than the
group before it, if no intolerable side effects were seen. This will continue until the
highest tolerable dose of OPB-31121 is found.

Study Drug Administration:

On Days 1-21 of each 28-day cycle, you will take the study drug by mouth 2 times a day. You
should take the study drug with at least 1 full glass of room temperature, still
(non-carbonated) water, within 30 minutes after eating a snack or meal. You should either
remain sitting upright or standing for at least 30 minutes after you take the study drug.
You will be given directions by the study doctor, the study nurse, and/or the study
pharmacist on how to take the study drug at home.

Study Visits:

On Day 1 of Cycle 1, the following tests and procedures will be performed:

- You will have a physical exam, including measurement of your vital signs.

- You will have 5 ECGs: 1 before the morning dose of study drug and at 1, 2, 4, and 8
hours after the morning dose.

- Blood (about 3 tablespoons) will be drawn for routine tests.

- Your performance status will be recorded.

- You will be asked about any drugs you may be taking and if you have experienced any
side effects.

- Blood (about 2 teaspoons each time) will be drawn before the morning dose and then 6
more times over the next 12 hours after the dose for pharmacokinetic (PK) testing. PK
testing measures the amount of study drug in the body at different time points.

- Blood (about 2 teaspoons) for pharmacodynamic (PD) testing will be drawn before the
morning dose. PD testing is used to look at how the level of study drug in your body
may affect the disease.

- Blood (about 1 teaspoon) will be drawn for pharmacogenomic (PGx) testing. PGx testing
looks at how differences in people's genes may affect if and how well the study drug
may affect the disease.

- Women who are able to become pregnant will have a urine pregnancy test.

On Day 2 of Cycle 1, before your morning dose of study drug, the following tests and
procedures will be performed:

- Blood (about 1 teaspoon) will be drawn for a troponin test.

- Blood (about 4 teaspoons) will be drawn for PK and PD testing.

- Your performance status will be recorded.

- You will be asked about any drugs you may be taking and if you have experienced any
side effects.

On Days 8, 15, 21, and 28 of Cycle 1, the following tests and procedures will be performed:

- You will have a physical exam, including measurement of your vital signs.

- You will have an ECG.

- Blood (about 3 tablespoons on Days 8 and 15; about 1 tablespoon on Days 21 and 28) and
urine will be collected for routine tests.

- Your performance status will be recorded.

- You will be asked about any drugs you may be taking and if you have experienced any
side effects.

On Day 21 of Cycle 1, blood (about 2 teaspoons each time) will be drawn for PK testing
before the morning dose and then 6 more times over the next 12 hours after the dose. Blood
(about 2 teaspoons each time) will also be drawn for PD testing.

On Day 22 of Cycle 1, before your morning dose of study drug, the following tests and
procedures will be performed:

- Your performance status will be recorded.

- You will be asked about any drugs you may be taking and if you had any side effects
from them.

- Blood (about 2 teaspoons) will be drawn for PK testing before your morning dose of the
study drug.

On Days 1, 15, and 28 of Cycles 2 and beyond, the following tests and procedures will be
performed:

- You will have a physical exam, including measurement of your weight and vital signs.

- Your performance status will be recorded.

- You will be asked about any drugs you may be taking and if you had any side effects
from them.

- You will have an ECG.

- You will have an echocardiogram or MUGA scan.

- Blood (about 3 tablespoons) will be drawn for routine tests.

- You will be asked about any drugs you may be taking and if you have experienced any
side effects.

- Women who are able to become pregnant will have a urine pregnancy test.

About every 8 weeks, you will have an x-ray, CT scan, or MRI scan to check the status of the
disease.

Length of Study:

You may continue taking the study drug for as long as you are benefitting. You will be taken
off study if the disease gets worse, you experience intolerable side effects, or your doctor
thinks that it is no longer in your best interest to receive the study drug.

You may choose to stop taking the study drug at any time. You should tell the study doctor
right away if you are thinking about stopping your participation in this study. The study
doctor will talk to you about how to safely stop taking the study drug.

End-of-Study Visit:

Within 14 days after the last dose of study drug, you will return for a final visit. The
following tests and procedures will be performed at the final visit:

- You will have a physical exam, including measurement of your vital signs.

- You will have an ECG.

- Blood (about 4 tablespoons) and urine will be collected for routine tests.

- You will have either an ECHO or MUGA scan.

- You will have an x-ray, CT scan, or MRI scan to check the status of the disease.

- Your performance status will be recorded.

- You will be asked about any drugs you may be taking and if you have experienced any
side effects.

Follow-Up:

You will be called about 30 days after the last dose of OPB-31121 and asked if you have
experienced any new or ongoing side effects.

This is an investigational study. OPB-31121 is not FDA approved or commercially available.
At this time, OPB-31121 is only being used in research.

Up to 36 patients will take part in this multicenter study. Up to 18 will be enrolled at M.
D. Anderson.