On May 6, 2024 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported the publication of results from the pivotal Phase 3 FOCUS study of HEPZATO KIT (melphalan/Hepatic Delivery System) in patients with unresectable metastatic Uveal Melanoma (mUM) on May 5, 2024 in the journal Annals of Surgical Oncology (Press release, Delcath Systems, MAY 6, 2024, View Source [SID1234642675]).
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The Food and Drug Administration (FDA) approved HEPZATO KIT on August 14, 2023 based on the results from the pivotal FOCUS study. A total of 91 patients with unresectable mUM were treated with HEPZATO KIT at 23 treatment centers in the US and Europe. Preliminary results from the FOCUS study were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in 2022.
The FOCUS study was designed to provide a robust evaluation of efficacy and safety of HEPZATO KIT treatment, and enrolled a heterogeneous mUM patient population, including treatment-naïve and pretreated patients, patients with and without extrahepatic disease and patients with a range of baseline tumor burden.
The primary efficacy endpoint of the FOCUS study was Overall Response Rate (ORR), which was 36.3%, including 7.7% of patients with Complete Response (CR), as determined by an Independent Review Committee. 37.4% of patients had Stable Disease (SD). ORR achieved in the FOCUS study was compared to a Meta-analysis of historic data, encompassing 16 published clinical studies with a total of 476 mUM patients treated with contemporary immunotherapy drugs. ORR of 36.3% in the FOCUS study was statistically significantly better than the pooled ORR estimate (a weighted mean of the observed ORR) of 5.5% in the historical control group.
Secondary efficacy endpoints included Duration of Response (DOR), median Progression-free Survival (mPFS) and median Overall Survival (mOS), which were 14, 9 and 20.5 months, respectively.
Safety and tolerability of HEPZATO KIT treatment reported in the FOCUS study was comparable with published clinical experience with Chemosat in Europe. Median number of administered HEPZATO KIT treatment cycles in the FOCUS study was 4. The most common serious treatment-emergent adverse events (SAE) were thrombocytopenia (15.8%) and neutropenia (10.5%), managed with standard supportive care and resolved with no ongoing complications. No treatment-related deaths were observed.
"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 Dr. Jonathan Zager, MD Chief Academic Officer and Director of Regional Therapies at Moffitt Cancer Center. "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."
"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," said Dr. Vojo Vukovic, MD, PhD Delcath’s Chief Medical Officer. "Delcath is looking forward to publishing additional results from the FOCUS study later this year."
The vast majority of patients with mUM will have liver metastases, often leading to liver failure, and the National Comprehensive Cancer Network guidelines recommend liver-directed therapies be considered for the treatment of mUM. HEPZATO is the only liver-directed treatment for unresectable mUM patients approved by the FDA.