FOCUS Study Outcomes on HEPZATO KIT for Metastatic Uveal Melanoma

Delcath Systems, Inc., a company specializing in interventional oncology for liver cancers, has reported remarkable outcomes from a Phase 3 clinical trial known as FOCUS, which evaluated the HEPZATO KIT in treating patients with unresectable metastatic Uveal Melanoma (mUM). The study, published in the "Annals of Surgical Oncology," demonstrated that HEPZATO KIT, which combines melphalan with a Hepatic Delivery System (HDS), achieved an Overall Response Rate (ORR) of 36.3%, significantly higher than the historical control group's 5.5%. This rate included a 7.7% Complete Response (CR), as verified by an Independent Review Committee, and 37.4% of patients experienced Stable Disease (SD).
The FDA granted approval for HEPZATO KIT on August 14, 2023, following the positive preliminary results presented at the American Society of Clinical Oncology Annual Meeting in 2022. The treatment was administered to 91 patients across 23 centers in the US and Europe, encompassing a diverse group of mUM patients. The study's secondary endpoints showed a Duration of Response (DOR) of 14 months, a median Progression-free Survival (mPFS) of 9 months, and a median Overall Survival (mOS) of 20.5 months.
The safety profile of HEPZATO KIT was found to be in line with the European experience with the Chemosat system. The median number of treatment cycles given was four, with the most frequent serious adverse events being thrombocytopenia (15.8%) and neutropenia (10.5%). These were managed with standard care and did not result in lasting complications. Notably, no treatment-related fatalities were reported.
Dr. Jonathan Zager, MD, Chief Academic Officer and Director of Regional Therapies at Moffitt Cancer Center, expressed enthusiasm for the FOCUS study's findings, which align with their extensive experience with percutaneous hepatic perfusion (PHP). Since HEPZATO KIT's introduction, it has become the standard of care for eligible mUM patients at Moffitt.
Dr. Vojo Vukovic, MD, PhD, Delcath's Chief Medical Officer, highlighted the significance of the PHP procedure, noting that both the FDA-approved HEPZATO KIT and the Chemosat device are crucial treatment options for patients with liver-dominant mUM. Delcath anticipates sharing more FOCUS study results later in the year.
HEPZATO KIT is the sole FDA-approved liver-directed treatment for mUM patients with inoperable liver metastases. Delcath Systems, Inc.'s HEPZATO KIT and CHEMOSAT products are designed to deliver high doses of chemotherapy directly to the liver, minimizing systemic exposure and side effects. In the US, HEPZATO KIT is a drug-device combination approved for adult mUM patients with specific hepatic and extrahepatic disease conditions. The CHEMOSAT system, used in Europe for PHP procedures, is regulated as a Class III medical device.