A Phase 2a Study of Modified Vaccinia Virus to Treat Sorafenib-naïve Advanced Liver Cancer

A Single-Arm, Open-Label Phase 2 Study of JX 594 (Thymidine Kinase-Deactivated Vaccinia Virus Plus GM-CSF) Administered by Weekly Intravenous (IV) Infusions in Sorafenib-naïve Patients With Advanced Hepatocellular Carcinoma (HCC)

This study is to determine how effectively JX-594 (Pexa-Vec) will prolong life in patients with advanced Hepatocellular Carcinoma (HCC) who have not been previously treated with sorafenib, and the safe administration of JX-594 in five weekly IV infusions.

This was a Phase 2a, two-staged, single-arm, open-label study in sorafenib-naïve patients with advanced HCC.

Patients received 5 weekly IV infusions of Pexa-Vec and could have continued to receive IV infusions of Pexa-Vec every 3 weeks until progressive disease (PD).

KEY Inclusion Criteria:

• Histologic or cytologic confirmation of advanced primary hepatocellular carcinoma (HCC)
• Measurable tumor (at least one tumor with ≥1 cm LD of contrast-enhancement during the arterial phase on CT scanning)
• ECOG performance status 0, 1 or 2
• Child-Pugh Class A; or Child-Pugh Class B7 without clinically significant ascites
• Platelet count ≥50,000 plts/mm3
• WBC count ≥2,000 cells/mm3 and ≤50,000 cells/mm3
• Hemoglobin ≥10 g/dL
• Adequate liver function

KEY Exclusion Criteria:

• Received sorafenib as previous treatment for HCC for more than 14 days
• History of severe exfoliative skin condition (e.g., eczema or atopic dermatitis requiring systemic therapy for > 4 weeks)
• Prior treatment with JX-594
• Known significant immunodeficiency due to underlying illness (e.g. HIV/AIDS) and/or medication
• Severe or unstable cardiac disease
• Viable CNS malignancy associated with clinical symptoms
• Pregnant or nursing an infant
• Significant bleeding event within the last 12 months.