On December 31, 2025 Delcath Systems, Inc. (Nasdaq: DCTH), ("Delcath" or the "Company") an interventional oncology company focused on the treatment of primary and metastatic liver cancers, reported the publication of results from subgroup analyses of the phase 3 FOCUS study. The publication, titled "Subgroup Analyses of the Phase 3 FOCUS Study of Melphalan/Hepatic Delivery System in Patients with Unresectable Metastatic Uveal Melanoma" was published in the Journal of Cancer Research and Clinical Oncology. The analysis assessed efficacy and safety in subgroups of patients treated with Delcath’s HEPZATO KIT, a drug/device combination for liver-directed treatment of mUM patients. The HEPZATO KIT is currently the only liver-directed treatment to be approved by the FDA for patients with unresectable mUM. The article provides clinically relevant subgroup analyses of key efficacy endpoints, including overall response rate (ORR), progression-free survival (PFS) and overall survival (OS), and safety categories. Prespecified subgroups included age (<65 or ≥65 years), sex (male or female) geographic region (ex-US or US) extent of tumor liver involvement (1-25% or 26-50%), hepatic tumor burden (above or below median), presence of extrahepatic lesions (yes or no), baseline LDH (low/normal or elevated) and number of prior therapies (0 or 1+).
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This prespecified analysis of clinically relevant subgroups included 91 patients treated in the FOCUS study. Key efficacy and safety findings include:
Consistent tumor response was observed regardless of age, sex, geographic region, presence/absence of extrahepatic lesions, and prior therapy.
ORR: significantly higher ORR for patients with tumor burden below the median (51.1% vs. 22.2%, p=0.008). No significant differences for other subgroups.
PFS: significantly longer median PFS for patients with tumor burden below the median (11.3 vs. 5.8 months, p=0.007). No significant differences for other subgroups.
OS: significantly longer median OS for patients with smaller extent of tumor liver involvement (22.4 vs 16.8 months, p=0.032), tumor burden below the median (26.7 vs 15.4 months, p=0.008) and low/normal LDH (23.4 vs 15.3 months, p=0.019)
Of the 33 patients achieving an objective response (CR or PR), 57.6% (19 patients) responded within the first or second treatment cycle, while one-third (33.3%; 11 patients) of responses were observed in Cycles 4–6, highlighting the importance of continued treatment up to the maximum of 6 cycles to optimize tumor response.
The overall safety profile was similar across subgroups, with no evidence of cumulative toxicity with successive treatment cycles. The incidence of serious adverse events and Grade 3/4 adverse events was consistent with the overall study population, and no treatment-related deaths occurred. "These subgroup analyses provide valuable insights into optimizing treatment with Melphalan/HDS for patients with unresectable metastatic uveal melanoma, underscoring the importance of early intervention in patients with lower tumor burden to maximize clinical benefits," said Vojislav Vukovic, MD, MSc, PhD, Chief Medical Officer of Delcath. "The consistent efficacy and manageable safety profile across diverse patient groups further validate this liver-directed therapy as a key option in managing this challenging disease."
The FOCUS study utilized HEPZATO KIT (HEPZATO (melphalan) for Injection/Hepatic Delivery System), and results from the FOCUS study lead to the approval of HEPZATO by the U.S. Food and Drug Administration (FDA).
(Press release, Delcath Systems, DEC 31, 2025, View Source [SID1234661670])