ARQ 501 in Combination With Gemcitabine in Subjects With Pancreatic Cancer

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

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.