Delcath Systems Gets FDA Clearance for Phase 2 Trial of HEPZATO™ in Liver-Metastatic Colorectal Cancer

Delcath Systems, Inc., a company specializing in interventional oncology and listed on NASDAQ under DCTH, revealed that the FDA has finished its 30-day review of the company's Investigational New Drug (IND) application. This review pertains to a Phase 2 clinical trial that aims to evaluate HEPZATO™ in combination with standard care treatments for liver-dominant metastatic colorectal cancer (mCRC). Consequently, Delcath is now permitted to begin enrolling patients for this trial.

The Phase 2 clinical trial is designed to assess the safety and effectiveness of HEPZATO when used alongside trifluridine-tipiracil and bevacizumab, as opposed to using only trifluridine-tipiracil and bevacizumab. This research will focus on patients who have liver-dominant mCRC and are receiving third-line treatment. The trial will involve approximately 90 participants and will be conducted at more than 20 locations across the United States and Europe. Patient enrollment is projected to commence in the latter half of 2025. The primary endpoint, hepatic progression-free survival (hPFS), is anticipated by the end of 2027, with overall survival (OS) data expected in 2028.

Delcath Systems has estimated that the market for treatments targeting liver-dominant mCRC in third-line patients ranges between 6,000 to 10,000 individuals annually in the U.S. This group includes patients exhibiting significant liver disease, with liver-dominant status determined through radiological and clinical assessments. Delcath aims to deliver a new treatment option to this patient segment, which currently has limited therapeutic alternatives.

Gerard Michel, CEO of Delcath Systems, Inc., emphasized the importance of this Phase 2 trial, noting, “This trial marks a significant advancement in exploring HEPZATO as a treatment option for liver-dominant metastatic colorectal cancer. It underscores our dedication to broadening HEPZATO's applications beyond metastatic uveal melanoma, thereby providing hope to another subgroup of patients with liver-dominant cancers.”

Delcath Systems, Inc. is focused on developing treatments for primary and metastatic liver cancers. The company’s flagship products include the HEPZATO KIT (a combination of HEPZATO (melphalan) for Injection and Hepatic Delivery System) and the CHEMOSAT® Hepatic Delivery System for Melphalan percutaneous hepatic perfusion (PHP). These products are crafted to deliver high-dose chemotherapy to the liver while managing systemic exposure and related side effects.

In the United States, the HEPZATO KIT is considered a combination of drug and device and is regulated and approved for sale as a pharmaceutical product by the FDA. The HEPZATO KIT consists of melphalan and Delcath's proprietary Hepatic Delivery System (HDS). This system isolates hepatic venous blood from systemic circulation while simultaneously filtering hepatic venous blood during melphalan infusion and washout. This method allows for a high dose of melphalan to be delivered directly to the liver, potentially inducing a significant tumor response with minimal liver toxicity and reducing systemic exposure. The HEPZATO KIT is approved for use in the U.S. as a liver-directed treatment for adults with metastatic uveal melanoma (mUM) with unresectable liver metastases affecting less than 50% of the liver and no extrahepatic disease, or where extrahepatic disease is confined to areas treatable by resection or radiation.

In Europe, the HDS is regulated as a Class III medical device and is marketed under the name CHEMOSAT Hepatic Delivery System for Melphalan. It has been utilized in percutaneous hepatic perfusion procedures at major medical centers to treat various liver cancers.