Results from Pivotal FOCUS Study in Metastatic Uveal Melanoma with HEPZATO KIT

Delcath Systems, Inc., a company specializing in interventional oncology, has disclosed findings from its pivotal Phase 3 FOCUS study on HEPZATO KIT (melphalan/Hepatic Delivery System), aimed at treating unresectable metastatic Uveal Melanoma (mUM). Published on May 5, 2024, in the Annals of Surgical Oncology, the study has demonstrated significant results.

The Food and Drug Administration (FDA) granted approval for HEPZATO KIT on August 14, 2023, following the outcomes of this pivotal research. The study treated 91 patients with unresectable mUM across 23 medical centers in the United States and Europe. Initial results were previously presented at the American Society of Clinical Oncology Annual Meeting in 2022.

The FOCUS study aimed to rigorously assess the effectiveness and safety of HEPZATO KIT. It included a diverse patient group, featuring both treatment-naïve and previously treated individuals, as well as patients with and without extrahepatic disease, and varying baseline tumor burdens.

Key efficacy measurements included the Overall Response Rate (ORR), which was 36.3%, with 7.7% of patients achieving a Complete Response (CR). Furthermore, 37.4% of patients exhibited Stable Disease (SD). These findings were compared to historical data from 16 published studies involving 476 mUM patients treated with modern immunotherapy. The ORR of 36.3% from the FOCUS study notably surpassed the pooled ORR estimate of 5.5% from the historical group.

Secondary efficacy endpoints such as Duration of Response (DOR), median Progression-free Survival (mPFS), and median Overall Survival (mOS) were also noteworthy, standing at 14, 9, and 20.5 months, respectively.

Safety and tolerability were consistent with prior European clinical experiences with Chemosat. The median number of HEPZATO KIT treatment cycles was four. The most frequent serious treatment-emergent adverse events (SAE) included thrombocytopenia (15.8%) and neutropenia (10.5%), both managed with standard supportive care without ongoing complications. Importantly, no treatment-related deaths were reported.

Dr. Jonathan Zager from Moffitt Cancer Center expressed enthusiasm about the study's publication, highlighting the consistency of its results with previous studies and their own experience with percutaneous hepatic perfusion (PHP). Since becoming available in January, HEPZATO KIT has established itself as the standard liver-directed treatment for qualified mUM patients at Moffitt Cancer Center.

Dr. Vojo Vukovic, Delcath's Chief Medical Officer, remarked on the significance of the FOCUS study results, emphasizing the importance of the PHP procedure, whether using the FDA-approved HEPZATO KIT or the Chemosat device available in Europe, for liver-dominant mUM patients. He also indicated that further results from the FOCUS study would be published later in the year.

The majority of mUM patients develop liver metastases, often resulting in liver failure. The National Comprehensive Cancer Network guidelines recommend liver-directed therapies for treating mUM. HEPZATO stands as the sole FDA-approved liver-directed treatment for unresectable mUM patients.

Delcath Systems, Inc. focuses on treating primary and metastatic liver cancers through its proprietary products, HEPZATO KIT and CHEMOSAT® Hepatic Delivery System. These systems deliver high-dose chemotherapy to the liver while controlling systemic exposure during a PHP procedure. HEPZATO KIT, approved as a combination drug and device product in the U.S., comprises melphalan and Delcath's Hepatic Delivery System (HDS). The HDS isolates the liver and filters hepatic venous blood during melphalan infusion, achieving a high local melphalan dose with minimal systemic exposure. In Europe, the device-only configuration, CHEMOSAT, is used for various liver cancers.

Overall, the FOCUS study affirms HEPZATO KIT's role as a critical treatment option for unresectable mUM, providing promising results in efficacy and safety.