Newly published subgroup analyses from the Phase 3 FOCUS study provide important clarity on which patients with metastatic uveal melanoma may benefit most from liver-directed therapy. Delcath Systems, Inc. announced the publication of these results in the Journal of Cancer Research and Clinical Oncology, further strengthening the clinical evidence supporting the use of the FDA-approved HEPZATO KIT in unresectable metastatic uveal melanoma (mUM).
The FOCUS study evaluated HEPZATO KIT, a drug-device combination that delivers high-dose melphalan directly to the liver while limiting systemic exposure. Because metastatic uveal melanoma most commonly spreads to the liver, liver-directed approaches remain a critical component of treatment for eligible patients. HEPZATO KIT is currently the only liver-directed therapy approved by the U.S. Food and Drug Administration for patients with unresectable mUM.
This prespecified subgroup analysis included 91 patients and examined outcomes across clinically relevant factors such as age, sex, geographic region, tumor burden, extent of liver involvement, baseline LDH levels, presence of extrahepatic disease, and prior therapies. Importantly, the analysis demonstrated consistent tumor responses across most subgroups, reinforcing the broad applicability of melphalan hepatic delivery in diverse patient populations.
Notably, patients with lower hepatic tumor burden experienced significantly improved outcomes. These patients showed higher objective response rates, longer progression-free survival, and improved overall survival compared with those with higher tumor burden. Overall survival was also significantly longer in patients with smaller liver involvement and in those with low or normal baseline LDH levels—highlighting the importance of early referral and timely intervention.
The study also underscored the value of completing the full treatment course. While many patients responded early, a substantial proportion achieved meaningful tumor responses in later treatment cycles, supporting continued therapy through the maximum of six cycles when clinically appropriate.
Equally important, the safety profile of HEPZATO KIT remained consistent across all subgroups, with no evidence of cumulative toxicity and no treatment-related deaths. Together, these findings validate HEPZATO KIT as a key treatment option for metastatic uveal melanoma and offer practical insights to help clinicians optimize patient selection and timing of therapy.