Delcath Systems Hepzato Kit for mUM Evaluated in Subgroup Analysis of FOCUS Trial

September 16, 2024—Delcath Systems, Inc. announced the presentation of new subgroup analysis data from the phase 3 FOCUS trial of Hepzato Kit (melphalan/hepatic delivery system [HDS]) in patients with metastatic uveal melanoma (mUM). The subgroup analysis evaluated patients with and without extrahepatic disease, treatment-naive versus previously treated patients, and patients with low (1%-25%) versus high (26%-50%) liver tumor burden.

The data were presented by Matthew Wheater, MD, at the European Society for Medical Oncology Congress held September 13-17 in Barcelona, Spain. Dr. Wheater is with University Hospital Southampton in Southampton, United Kingdom.

Delcath Systems noted that the FOCUS trial is a pivotal phase 3 study designed to evaluate the efficacy and safety of Hepzato Kit in patients with unresectable hepatic metastases from mUM.

The primary endpoint of FOCUS was objective response rate (ORR), with secondary endpoints including progression-free survival (PFS) and overall survival (OS). The trial enrolled 102 patients, of whom 91 received treatment. The full results of the FOCUS trial were published on May 4, 2024, by Jonathan S. Zager MD, et al in Annals of Surgical Oncology (2024;31:5340-5351).

As summarized in the company’s press release, there were no significant differences in OS, ORR, or PFS in the subgroup analysis between patients with and without extrahepatic lesions or based on previous therapy. ORR and PFS remained consistent regardless of liver tumor burden; however, more extensive liver involvement was associated with worse OS outcomes.

Additionally, objective tumor responses were observed throughout the entire treatment period, with the earliest after completion of the first treatment cycle, and the latest after treatment cycle 6. This result supports the strategy to continue treatment until best response is achieved. Rates of serious adverse events and grade 3/4 adverse events remained consistent, indicating an absence of cumulative toxicity.

These findings underscore the favorable benefit-risk profile of Hepzato Kit in this patient population, offering a meaningful option for patients who typically have limited treatment options, stated the company.

“The lack of significant differences in outcomes between patients with and without extrahepatic disease is encouraging, particularly for a liver-directed therapy like melphalan/HDS,” commented Dr. Wheater in the Delcath System’s press release. “Additionally, the fact that responses were observed through all six treatment cycles supports the strategy of continuing treatment beyond two cycles in patients with stable disease.”

In the United States, Hepzato Kit received FDA approval for the treatment of mUM in August 2023 and launched in January 2024.