Phase 1 Study of PLX7486 as Single Agent in Patients With Advanced Solid Tumors

Part 1. Open-label, sequential PLX7486 TsOH single-agent dose escalation in approximately 60
patients with solid tumors.

Inclusion Criteria

- Male or female ≥18 years old

- Patients with histologically confirmed solid tumors who:

o Part 1: have tumor progression following standard therapy, have treatment-refractory
disease, or for whom there is no effective standard of therapy

- Women of child-bearing potential must have a negative pregnancy test within 7 days of
initiation of dosing and must agree to use an acceptable method of birth control.
Women of non-childbearing potential may be included if they are either surgically
sterile or have been postmenopausal for ≥1 year. Fertile men must also agree to use an
acceptable method of birth control while on study drug and up to 3 months after the
last dose of study drug.

- All associated toxicity from previous or concurrent cancer therapy must be resolved
(to ≤Grade 1 or Baseline) prior to study treatment administration

- Patients with stable, treated brain metastases are eligible for this trial. However,
patients must not have required steroid treatment for their brain metastases within 30
days of Screening.

- Willing and able to provide written informed consent prior to any study related
procedures and to comply with all study requirements

- Karnofsky performance status ≥70%

- Life expectancy ≥3 months

- Adequate hematologic, hepatic, and renal function

Exclusion Criteria

- Other than the primary malignancy, active cancer (either concurrent or within the last
3 years) that requires non-surgical therapy (e.g., chemotherapy or radiation therapy),
with the exception of surgically treated basal or squamous cell carcinoma of the skin,
melanoma in situ, or carcinoma in-situ of the cervix

- Chemotherapy within 28 days prior to C1D1

- Biological therapy within 5 half-lives prior to C1D1

- Radiation therapy within 28 days or 5 half-lives prior to C1D1, whichever is longer

- Investigational drug use within 28 days or 5 half-lives, whichever is longer, prior to
C1D1

- Part 1 only: (a) Patients with active or a history of glucose intolerance or diabetes
mellitus and (b) Hemoglobin A1c ≥7%

- ≥Grade 2 sensory neuropathy at baseline

- Uncontrolled intercurrent illness (i.e., active infection) or concurrent condition
that, in the opinion of the Investigator, would interfere with the study endpoints or
the patient's ability to participate

- Refractory nausea and vomiting, malabsorption, small bowel resection that, in the
opinion of the Investigator, would preclude adequate absorption

- Mean QTcF ≥450 msec (for males) or ≥470 msec (for females) at Screening

- The presence of a medical or psychiatric condition that, in the opinion of the
Principal Investigator, makes the patient inappropriate for inclusion in this study