Delcath Systems Announces Publication of Comparative Analysis from Randomized Portion of FOCUS Study in Metastatic Uveal Melanoma

Rhea-AI Summary

Delcath Systems (NASDAQ: DCTH) has published a comparative analysis from the randomized portion of their Phase 3 FOCUS study in the Annals of Surgical Oncology. The study evaluated Melphalan/Hepatic Delivery System (Melphalan/HDS) against best alternative care (BAC) in patients with unresectable metastatic uveal melanoma.

Key findings from the 72 treated patients (40 Melphalan/HDS; 32 BAC) showed significant improvements with Melphalan/HDS:

• Median progression-free survival: 9.1 months vs 3.3 months for BAC
• Median overall survival: 18.5 months vs 14.5 months for BAC
• Objective response rate: 27.5% vs 9.4% for BAC
• Disease control rate: 80.0% vs 46.9% for BAC
• Median hepatic progression-free survival: 11.4 months vs 3.3 months for BAC

The safety profile was consistent with prior reports, primarily showing hematologic effects, with no treatment-related deaths reported.

Positive

• Significantly improved progression-free survival (9.1 vs 3.3 months)
• Higher overall survival rate (18.5 vs 14.5 months)
• Superior objective response rate (27.5% vs 9.4%)
• Strong disease control rate (80.0% vs 46.9%)
• Tripled hepatic progression-free survival (11.4 vs 3.3 months)
• No treatment-related deaths reported

Negative

• Study was amended from randomized to single-arm design
• Comparative efficacy analyses were only exploratory in nature

Insights

Oncology Clinical Trial Specialist

Delcath's publication of comparative data from their FOCUS trial represents a meaningful clinical advancement for metastatic uveal melanoma (mUM), a rare cancer with historically poor outcomes. The data demonstrates Melphalan/HDS outperforming best alternative care across multiple critical endpoints.

The progression-free survival of 9.1 months versus 3.3 months with standard care represents a 175% improvement - particularly significant in this aggressive disease. Similarly, the overall survival advantage of 18.5 versus 14.5 months represents a 28% survival benefit. The objective response rate of 27.5% versus 9.4% and disease control rate of 80% versus 46.9% further validate clinical efficacy.

While these results are designated as exploratory due to the trial design modification from randomized to single-arm, the consistent improvement across all endpoints provides compelling evidence of clinical benefit. The safety profile aligns with previous findings, primarily showing manageable hematologic effects.

This represents significant progress for mUM patients with liver metastases who currently face treatment options. The hepatic-directed approach addresses a critical challenge in this disease, where liver progression often drives mortality. The ability for repeated administration further enhances its clinical utility for managing this challenging malignancy.

Biotech Investment Analyst

This publication marks a strategic milestone for Delcath by demonstrating substantial clinical advantages for their proprietary Melphalan/Hepatic Delivery System. The data shows meaningful improvements across all five efficacy endpoints compared to physician's choice alternatives - a comprehensive efficacy signal rarely seen in oncology trials.

The consistency of benefit across multiple endpoints significantly strengthens Delcath's position in the metastatic uveal melanoma treatment landscape. The triple improvement in progression-free survival and response rates addresses a critical unmet need in this orphan indication with effective therapies.

From a commercial perspective, liver-directed therapies represent a focused approach in oncology, with specialized delivery systems potentially commanding premium pricing. While the FOCUS study shifted from randomized to single-arm design (limiting the comparative claims in regulatory filings), these exploratory analyses still provide valuable differentiation data for market positioning and physician education.

Delcath's focus on hepatic-directed therapy aligns with the clinical understanding that controlling liver metastases is crucial in mUM patient outcomes. With a favorable safety profile and no treatment-related deaths, the risk-benefit profile appears compelling. The company's targeted approach in a rare cancer with established regulatory pathways and orphan drug incentives represents a focused commercial strategy that may help maximize the return on their specialized technology platform.

QUEENSBURY, N.Y.--(BUSINESS WIRE)-- Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, today announced the publication of a comparative analysis from the randomized cohort of the Company’s Phase 3 FOCUS study in Annals of Surgical Oncology. The study, titled “An Open-label, Randomized Study of Melphalan/Hepatic Delivery System Versus Best Alternative Care in Patients with Unresectable Metastatic Uveal Melanoma,” indicates that treatment with the Melphalan/Hepatic Delivery System (Melphalan/HDS) shows a trend toward favorable clinical outcomes compared to best alternative care (BAC), which included the physician’s choice of dacarbazine, transarterial chemoembolization (TACE), ipilimumab or pembrolizumab in patients with unresectable metastatic uveal melanoma (mUM) with liver involvement.

The FOCUS study was initially designed and conducted as a randomized controlled trial but was amended to a single-arm design. As a result, comparative efficacy analyses were designated as exploratory. A total of 85 patients were enrolled during the randomized portion of the trial, with 72 receiving study treatment (40 Melphalan/HDS; 32 BAC).

Key Findings from the Randomized Portion of the FOCUS Study:

• All efficacy endpoints of the trial demonstrated substantial and consistent improvements in patients treated with Melphalan/HDS over BAC, including:
  • Median progression-free survival in patients treated with Melphalan/HDS was 9.1 months, nearly three times longer than the 3.3 months observed in patients treated with BAC
  • Median overall survival was 18.5 months for Melphalan/HDS compared to 14.5 months with BAC
  • Objective response rate was 27.5% with Melphalan/HDS, nearly three times higher than 9.4% with BAC
  • Disease control rate was 80.0% with Melphalan/HDS, substantially higher than the 46.9% observed with BAC
  • Median hepatic progression-free survival was 11.4 months for Melphalan/HDS – more than three times longer than the 3.3 months for BAC
• The safety profile of patients treated with Melphalan/HDS was consistent with prior reports and primarily hematologic in nature. No treatment-related deaths were observed.

“These results further support the use of our hepatic delivery system as a liver-directed treatment option for patients with metastatic uveal melanoma,” said Dr. Vojislav Vukovic, Chief Medical Officer at Delcath. “Melphalan/HDS delivered meaningful improvements across all clinical endpoints, including progression-free survival, overall survival, disease control, and hepatic outcomes. The findings, while exploratory in nature, reinforce our belief that effective management of liver disease is key to improving outcomes in this patient population, and we are proud to contribute a therapy with both clinical impact and the ability to be administered repeatedly.”

The publication is available online at: https://doi.org/10.1245/s10434-025-17231-x

About Delcath Systems, Inc., HEPZATO KIT™ and CHEMOSAT^®

Delcath Systems, Inc. is an interventional oncology company focused on the treatment of primary and metastatic liver cancers. The company’s proprietary products, HEPZATO KIT™ (HEPZATO (melphalan) for Injection/Hepatic Delivery System) and CHEMOSAT^® Hepatic Delivery System for Melphalan percutaneous hepatic perfusion (PHP), are designed to administer high-dose chemotherapy to the liver while controlling systemic exposure and associated side effects during a PHP procedure.

In the United States, HEPZATO KIT is regulated and approved as a combination drug and device product by the FDA. HEPZATO KIT is approved as a liver-directed treatment for adult patients with metastatic uveal melanoma (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. Please see full Prescribing Information, including BOXED WARNING, at www.hepzatokit.com.

In Europe, the device-only configuration of the HDS is regulated as a Class III medical device and is marketed under the trade name CHEMOSAT^®, where it has been used in major cancer centers to treat a wide range of cancers of the liver.

Safe Harbor / Forward-Looking Statements

The Private Securities Litigation Reform Act of 1995 provides a safe harbor for forward-looking statements made by the Company or on its behalf. This press release contains forward-looking statements, which are subject to certain risks and uncertainties, that can cause actual results to differ materially from those described. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Factors that may cause such differences include, but are not limited to, uncertainties relating to: the Company’s commercialization plans and its ability to successfully commercialize the HEPZATO KIT; contributions to adjusted EBITDA; the Company’s successful management of the HEPZATO KIT supply chain, including securing adequate supply of critical components necessary to manufacture and assemble the HEPZATO KIT and the managing of any trade restrictions; successful FDA inspections of the facilities of the Company and those of its third-party suppliers/manufacturers; the Company’s successful implementation and management of the HEPZATO KIT Risk Evaluation and Mitigation Strategy; the potential benefits of the HEPZATO KIT as a treatment for patients with primary and metastatic disease in the liver; the Company’s ability to obtain reimbursement for the HEPZATO KIT; and the Company’s ability to successfully enter into any necessary purchase and sale agreements with users of the HEPZATO KIT. For additional information about these factors, and others that may impact the Company, please see the Company’s filings with the Securities and Exchange Commission, including those on Forms 10-K, 10-Q, and 8-K. However, new risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. Accordingly, you should not place undue reliance on these forward-looking statements, which speak only as of the date they are made. We undertake no obligation to publicly update or revise these forward-looking statements to reflect events or circumstances after the date they are made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250409746540/en/

Investor Relations:

ICR Westwicke

investorrelations@delcath.com

Source: Delcath Systems, Inc.

FAQ

What are the survival benefits of Melphalan/HDS compared to best alternative care in the FOCUS study?

Melphalan/HDS showed median overall survival of 18.5 months versus 14.5 months for BAC, and progression-free survival of 9.1 months versus 3.3 months for BAC.

What is the disease control rate achieved by DCTH's Melphalan/HDS in metastatic uveal melanoma?

Melphalan/HDS achieved an 80.0% disease control rate, compared to 46.9% with best alternative care.

How many patients were enrolled in the randomized portion of DCTH's FOCUS trial?

85 patients were enrolled, with 72 receiving treatment (40 Melphalan/HDS; 32 best alternative care).

What was the hepatic progression-free survival benefit shown in DCTH's Phase 3 FOCUS study?

Median hepatic progression-free survival was 11.4 months for Melphalan/HDS, more than three times longer than the 3.3 months for best alternative care.

What were the safety outcomes of Melphalan/HDS in DCTH's FOCUS trial?

The safety profile was primarily hematologic in nature, consistent with prior reports, and no treatment-related deaths were observed.