Delcath Systems, Inc. recently announced the publication of important subgroup analyses from its Phase 3 FOCUS study evaluating melphalan delivered via a Hepatic Delivery System in patients with unresectable metastatic uveal melanoma (mUM). Published in the Journal of Cancer Research and Clinical Oncology, the article provides deeper insight into which patient populations may benefit most from this FDA-approved, liver-directed treatment.
Metastatic uveal melanoma is a rare and aggressive cancer, with the liver being the most common site of metastasis. Treatment options have historically been limited, making the FOCUS study particularly meaningful. The subgroup analyses assessed efficacy and safety outcomes—including overall response rate (ORR), progression-free survival (PFS), and overall survival (OS)—across clinically relevant patient characteristics such as age, sex, tumor burden, liver involvement, LDH levels, and prior therapies.
Results demonstrated consistent tumor response across most subgroups, reinforcing the broad applicability of Delcath’s therapy. Notably, patients with lower hepatic tumor burden experienced significantly improved outcomes, including higher ORR (51.1%), longer median PFS (11.3 months), and extended OS (26.7 months). Patients with smaller extents of liver involvement and normal LDH levels also showed meaningful survival advantages, underscoring the importance of earlier intervention.
Equally important, the safety profile remained stable across all subgroups, with no evidence of cumulative toxicity and no treatment-related deaths. Over half of responders achieved tumor reduction within the first two treatment cycles, while additional responses occurred later, emphasizing the value of completing the full treatment course.
These findings further validate HEPZATO KIT as a critical option for managing unresectable mUM and support its approval by the U.S. Food and Drug Administration. For patients and clinicians navigating metastatic uveal melanoma, this analysis represents meaningful progress toward more effective, targeted care.