FOCUS Trial of HEPZATO KIT Shows Efficacy in Metastatic Uveal Melanoma


A study found that treatment with HEPZATO KIT showed a promising response rate of 36.3% for patients with unresectable metastatic uveal melanoma.

Melanoma: © David A Litman -

Melanoma: © David A Litman -

Treatment with the HEPZATO KIT (melphalan/Hepatic Delivery System) demonstrated a 36.3% (95% CI, 26.4%-47.0%) overall response rate (ORR), including 7.7% of patients with complete response, among those with unresectable metastatic uveal melanoma (mUM), according to results from the pivotal phase 3 FOCUS study.1

Findings were recently published in the journal Annals of Surgical Oncology. In addition to promising findings for the primary efficacy end point of ORR as determined by an independent review committee, 37.4% of patients had stable disease (SD). The partial response rate was 28.6%.

The ORR reached in the FOCUS study was statistically significantly better than the pooled ORR estimate of 5.5% in a historical control group which came from a meta-analysis of data from 16 published clinical studies, consisting of 476 patients with mUM who were given treatment with contemporary immunotherapy agents.

"My team at the Moffitt Cancer Center and I are very excited about the publication of results from the FOCUS study. The study results are consistent with data obtained from previous clinical studies, as well as our own experience with percutaneous hepatic perfusion [PHP] with more than 200 treatments performed at Moffitt over the past 15 years," said Jonathan Zager, MD, chief academic officer and director of regional therapies at Moffitt Cancer Center, in a press release. "Since HEPZATO KIT became available in January, the treatment has become the liver directed standard of care for appropriate patients with mUM at our institution."

For the secondary end points of duration of response, median progression-free survival, and median overall survival, rates were 14 (95% CI, 8.3-17.7), 9, and 20.5 months, respectively.

Additionally, treatment with the HEPZATO KIT had similar safety and efficacy data compared with previously clinical experience with Chemosat in Europe. In the FOCUS study, the median number of HEPZATO KIT treatment cycles given was 4.

The most common serious treatment-emergent adverse events (AEs) included thrombocytopenia (15.8%) and neutropenia (10.5%). These AEs were managed with standard supportive care, and they resolved with no ongoing complications. Additionally, there were no treatment-related deaths observed in the study.

"Results from the pivotal FOCUS study demonstrate that the PHP procedure, whether utilizing the FDA approved HEPZATO KIT or melphalan delivered by the Chemosat device available in Europe, is an important treatment option for patients with liver-dominant mUM," Vojo Vukovic, MD, PhD Delcath's chief medical officer, said in a press release. "Delcath is looking forward to publishing additional results from the FOCUS study later this year."

The HEPZATO KIT is currently the only liver-directed treatment to be approved by the FDA for patients with unresectable mUM. Approval was granted by the FDA in August 2023 to the liver-directed treatment for adult patients with mUM with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.2

Additional findings from the phase 3, single arm, multicenter, open-label FOCUS study showed that the disease control rate was 73.6% (95% CI, 63.3%-82.3%) among those treated with the HEPZATO KIT. Included in the trial were 91 patients who had either hepatic or extrahepatic lesions. Some of the patients were naive to cancer treatment (56.0%) and others were previously treated (44.0%).2,3

1. Publication of results from pivotal FOCUS study in metastatic uveal melanoma patients treated with HEPZATO KIT. News release. Delcath Systems, Inc. May 6, 2024. Accessed May 6, 2024.
2. Delcath Systems, Inc. announces FDA Approval of HEPZATO KIT™ for the treatment of adult patients with unresectable hepatic-dominant metastatic uveal melanoma. News release. Delcath Systems, Inc. August 14, 2023. Accessed May 6, 2024.
3. Percutaneous hepatic perfusion in patients with hepatic-dominant ocular melanoma (FOCUS). Updated August 3, 2023. Accessed May 6, 2024.
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