ARQ 501 in Combination With Gemcitabine in Subjects With Pancreatic Cancer

• Phase II: Document progression free survival after treatment with ARQ 501 and gemcitabine
• Phase Ib: Define a maximum tolerated dose of ARQ 501 in combination with gemcitabine

• Phase Ib: Safety and tolerability of ARQ 501 in combination with gemcitabine
• Phase II: Document safety and efficacy of ARQ 501 in combination with gemcitabine

The study will document the safety and efficacy of the combination of ARQ 501 and gemcitabine in patients with treatment-naïve, unresectable, metastatic pancreatic adenocarcinoma.

This is a single-arm, non-randomized study of ARQ 501 in combination with gemcitabine in adult patients with treatment-naïve, unresectable, metastatic pancreatic adenocarcinoma. The study objectives are:

Primary Objective:

• Assess the overall response rate (ORR) of patients treated with ARQ 501 in combination with gemcitabine.

Secondary Objectives:

• Determine time to tumor progression (TTP) of patients treated with ARQ 501 in combination with gemcitabine
• Further characterize the safety of ARQ 501 in combination with gemcitabine

• Pancreatic Cancer
• Adenocarcinoma

Inclusion Criteria:

• Have a pathologically confirmed diagnosis of unresectable, metastatic pancreatic adenocarcinoma
• Be treatment-naïve.
• Have measurable disease per RECIST Criteria.
• Be ≥18 years old.
• Have a Karnofsky Performance Status (KPS) of ≥70%.
• Have an estimated life expectancy of ≥12 weeks.
• Be male or a non-pregnant, non-lactating female patient. Patients who are fertile agree to use an effective barrier method of birth control (e.g., latex condom, diaphragm, or cervical cap) to avoid pregnancy.
• Have a negative serum or urine pregnancy test within 7 days prior to the first dose of study drug (if patient is a female of childbearing potential).
• Sign a written informed consent form.
• Have adequate organ function as indicated by acceptable laboratory values obtained within 7 days prior to the first dose of study drug.

Exclusion Criteria:

• Have received any prior therapy for the treatment of their pancreatic malignancy (including chemotherapy, immunotherapy, vaccines, monoclonal antibodies, major surgery, or irradiation, whether conventional or investigational).
• Have an active, uncontrolled systemic infection considered opportunistic, life threatening or clinically significant at the time of treatment.
• Are pregnant or lactating.
• Have a psychiatric disorder(s) that would interfere with consent, study participation, or follow-up.
• Have any other severe concurrent disease, which, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
• Have symptomatic or untreated central nervous system (CNS) metastases.
• Have a known hypersensitivity to gemcitabine.