Delcath Systems has been granted FDA clearance to commence a Phase 2 clinical trial assessing its Hepzato Kit as an adjunctive therapy for patients with liver-dominant metastatic colorectal cancer (mCRC). This open-label, randomized study (NCT06607458) is set to enroll 90 participants across 20 sites in the United States and Europe, with patient enrollment slated to begin in the latter half of 2025. The trial aims to evaluate the efficacy and safety of the Hepzato Kit in combination with standard-of-care trifluridine-tipiracil chemotherapy plus bevacizumab, compared against the standard-of-care regimen alone.
Trial Design and Endpoints
The Phase 2 trial's primary endpoint is hepatic progression-free survival (hPFS), with results anticipated by the end of 2027. Secondary endpoints include overall survival (OS), progression-free survival (PFS), overall response rate (ORR), and hepatic ORR, with readouts expected in 2028. The Hepzato Kit combines melphalan, a chemotherapy drug, with Delcath’s proprietary hepatic delivery system (HDS). This system isolates and filters hepatic venous blood from systemic circulation during melphalan infusion, designed to induce a clinically meaningful tumor response while minimizing hepatotoxicity and reducing systemic exposure.
Hepzato Kit and its Applications
The Hepzato Kit is already FDA-approved for treating extra-ocular metastases of uveal melanoma, specifically when affecting up to 50% of the liver tissue without extrahepatic disease, or with metastases limited to bone, lymph nodes, subcutaneous tissue, or lung. It has been approved in Europe since 2012 as the Chemosat hepatic delivery system for liver cancers. Delcath intends to broaden the Hepzato Kit's indications to include cholangiocarcinoma and other primary and metastatic liver cancers.
Market and Future Directions
Gerard Michel, CEO of Delcath Systems, Inc., stated, "This Phase 2 trial represents an exciting step forward in evaluating HEPZATO as a treatment for patients with liver-dominant metastatic colorectal cancer. The study reflects our commitment to expand the potential applications of HEPZATO beyond metastatic uveal melanoma, offering new hope to an additional group of patients with liver dominant cancers."
Delcath estimates the total addressable market for liver-dominant mCRC receiving third-line treatment to be between 6,000 and 10,000 patients annually in the United States. This encompasses patients with significant liver disease burden, determined through radiological and clinical criteria, for whom Delcath aims to provide a novel therapeutic option.
