A Study of APX3330 in Patients With Advanced Solid Tumors

Apurinic/apyrimidinic endonuclease 1/redox factor-1 (APE1/Ref-1) is a protein that regulates
multiple transcription factors involved in cancer cell signaling and APX3330 is a highly
selective inhibitor of APE1/Ref-1 redox function.

The anti-tumor effect of APX330 has been demonstrated in a variety of preclinical models and
the human safety profile of APX3330 was established in prior clinical studies. Apexian
Pharmaceuticals is developing APX3330 as an orally administered anti-cancer agent targeting
the APE1/Ref-1 protein.

APX_CLN_0011 is a Phase 1, multi-center, open-label, dose-escalation oncology study in
patients with advanced solid tumors. The study primary objective is to determine the
recommended Phase 2 study dose of APX3330. Secondary objectives include assessment of APX3330
safety, anti-tumor activity, pharmacokinetic and pharmacodynamic profile.

Inclusion Criteria:

1. Written informed consent must be obtained from the patient.

2. Patient must be > 18 years of age.

3. Patient must have recurrent or advanced cancer (i.e., solid tumors) for whom standard
therapy offers no curative potential.

4. Evaluable disease by RECIST v1.1.

5. Performance status (PS) of 0-2 on the Eastern Cooperative Oncology Group (ECOG) scale.
Note: PS 2 patients can only participate if, in the assessment of the clinical
investigator, and with the consent of the medical monitor, the patient has the ability
to participate in the clinical study for a minimum of at least 2 cycles.

6. > 21 days from therapeutic radiation or chemotherapy (>6 weeks from nitrosoureas and
mitomycin C) and recovery to (NCI CTCAE v4.03) Grade ≤ 1 from all clinically
significant toxicities related to prior therapies.

7. Must have adequate organ function defined as:

1. Absolute neutrophil count (ANC) ≥ 1.5 x 10^9/L.

2. Platelet ≥ 100 x 10^9/L.

3. Hemoglobin ≥ 9 g/dL.

4. Activated partial thromboplastin time/ partial thromboplastin time (aPTT/PTT) ≤
1.5 x ULN

5. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤ 2.5 × upper
limit of normal (ULN). In the case of known (i.e., radiological or biopsy
documented) liver metastasis, serum transaminase levels must be < 5 x ULN.

6. Total serum bilirubin ≤ 1.5 x ULN, (except for patients with known Gilbert's
Syndrome ≤ 3 x ULN is permitted)

7. Renal: Serum creatinine < 2.0 x ULN or creatinine clearance ≥ 50 mL/min/1.73m^2
for patients with serum creatinine levels above 2 x ULN.

8. Agreement to use acceptable methods of contraception during the study and for at least
120 days after the last dose of APX3330 if sexually active and able to bear or beget
children.

Exclusion Criteria:

1. Diagnosed with another malignancy within the past 2 years (excluding a history of
carcinoma in situ of the cervix, superficial non-melanoma skin cancer, superficial
bladder cancer, or endometrial cancer that has been adequately treated, or stage 1
prostate cancer that does not require treatment).

2. History of a major surgical procedure or a significant traumatic injury within 14 days
prior to commencing treatment, or the anticipation of the need for a major surgical
procedure during the course of the study.

3. Patients who have been treated with an investigational agent within 21 days prior to
the first dose of study drug.

4. Concurrent serious (as determined by the Principal Investigator) medical conditions,
including, but not limited to, New York Heart Association (NYHA) class III or IV
congestive heart failure, history of congenital prolonged QT syndrome, uncontrolled
infection, active hepatitis B, C or HIV, or other significant co-morbid conditions
that in the opinion of the investigator would impair study participation or
cooperation.

5. Impaired liver function Child-Pugh class B or C (score 7-15).

6. Women who are pregnant or lactating.

7. Patients with evidence of symptomatic brain metastases. Patients with treated
(surgically excised or irradiated) and stable brain metastases are eligible assuming
the patient has adequately recovered from treatment, the treatment was at least 28
days prior to initiation of study drug, and baseline brain computed tomography (CT)
with contrast, or magnetic resonance imaging (MRI) within 14 days of initiation of
study drug, is negative for new or worsening brain metastases

8. Other concurrent chemotherapy, immunotherapy, radiotherapy or investigational therapy
except for hormonal therapy (e.g., tamoxifen, etc.).

9. Patients requiring palliative radiotherapy to lesions that are defined as target
lesions by RECIST criteria at the time of study entry.