Delcath Systems Announces Publication of Subgroup Analyses of the Phase 3 FOCUS Study of Melphalan/Hepatic Delivery System in Patients with Unresectable Metastatic Uveal Melanoma

Key Terms

overall response rate medical

Overall response rate is the percentage of patients in a clinical study whose measurable disease shrinks or disappears after receiving a treatment. Investors watch it like a product’s “hit rate” because higher response rates can signal a drug’s effectiveness, boost chances of regulatory approval and market demand, and affect a company’s future revenue prospects, similar to how a higher batting average suggests a more reliable player.

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Progression-free survival is the length of time during and after a treatment that a patient's disease does not get worse, measured from the start of treatment until the disease shows measurable signs of progression or the patient dies. Investors care because longer progression-free survival in clinical trials often signals that a drug is effective, improving chances of regulatory approval, market adoption, and revenue potential—think of it as a stopwatch showing how long a therapy can keep the illness at bay.

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Overall survival is the average or median length of time patients remain alive after starting a treatment or entering a clinical study, measured regardless of cause of death. Investors care because it is a clear, hard measure of a therapy’s real-world benefit — like timing how long a new battery actually runs — and strong improvements in overall survival can drive regulatory approval, market adoption and revenue potential.

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Serious adverse events are significant problems or negative outcomes that occur during a medical treatment or clinical trial, such as severe side effects, hospitalizations, or life-threatening conditions. They matter to investors because such events can impact a company's reputation, lead to regulatory scrutiny, or delay the development of new products, ultimately affecting the company’s financial performance.

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Grade 3/4 adverse events are the most serious side effects reported during clinical trials or medical use, with grade 3 indicating severe but not immediately life‑threatening harm and grade 4 indicating life‑threatening reactions requiring urgent intervention. For investors, a rise in these high‑grade events is like seeing major roadblocks on a project: they can delay approval, increase development costs, force additional studies, and hurt commercial prospects and company valuation.

hepatic tumor burden medical

Hepatic tumor burden is the amount and extent of cancer present in the liver, typically described by the number, size or percentage of liver tissue affected. For investors, it matters because higher or changing tumor burden can drive patient survival, treatment choices, clinical trial outcomes and potential sales or regulatory decisions—think of it like counting how many and how large the weeds are in a garden to judge how much work and which tools are needed.

extrahepatic lesions medical

Extrahepatic lesions are abnormal growths or damaged tissue located outside the liver, such as in the lungs, bones, lymph nodes, or other organs. For investors, these lesions matter because their presence, number and spread affect how serious a disease is, what treatments are possible, and how well a therapy or drug may perform in trials—think of them as signs that a problem has spread beyond its original room in a house, changing the repair strategy and cost.

ldh medical

LDH stands for lactate dehydrogenase, an enzyme found throughout the body that is released into the blood when cells are damaged or rapidly turn over. Investors watch LDH levels reported in clinical data because rising or falling values can indicate disease severity, treatment effect, or safety concerns—much like an engine temperature gauge, it gives a quick, general signal about patient health that can influence a therapy’s commercial prospects.

 

QUEENSBURY, N.Y.--(BUSINESS WIRE)-- Delcath Systems, Inc. (Nasdaq: DCTH), (“Delcath” or the “Company”) an interventional oncology company focused on the treatment of primary and metastatic liver cancers, today announced the publication of results from subgroup analyses of the phase 3 FOCUS study. The publication, titled “Subgroup Analyses of the Phase 3 FOCUS Study of Melphalan/Hepatic Delivery System in Patients with Unresectable Metastatic Uveal Melanoma” was published in the Journal of Cancer Research and Clinical Oncology. The analysis assessed efficacy and safety in subgroups of patients treated with Delcath’s HEPZATO KIT, a drug/device combination for liver-directed treatment of mUM patients. The HEPZATO KIT is currently the only liver-directed treatment to be approved by the FDA for patients with unresectable mUM. The article provides clinically relevant subgroup analyses of key efficacy endpoints, including overall response rate (ORR), progression-free survival (PFS) and overall survival (OS), and safety categories. Prespecified subgroups included age (<65 or ≥65 years), sex (male or female) geographic region (ex-US or US) extent of tumor liver involvement (1-25% or 26-50%), hepatic tumor burden (above or below median), presence of extrahepatic lesions (yes or no), baseline LDH (low/normal or elevated) and number of prior therapies (0 or 1+).

This prespecified analysis of clinically relevant subgroups included 91 patients treated in the FOCUS study. Key efficacy and safety findings include:

• Consistent tumor response was observed regardless of age, sex, geographic region, presence/absence of extrahepatic lesions, and prior therapy.
• ORR: significantly higher ORR for patients with tumor burden below the median (51.1% vs. 22.2%, p=0.008). No significant differences for other subgroups.
• PFS: significantly longer median PFS for patients with tumor burden below the median (11.3 vs. 5.8 months, p=0.007). No significant differences for other subgroups.
• OS: significantly longer median OS for patients with smaller extent of tumor liver involvement (22.4 vs 16.8 months, p=0.032), tumor burden below the median (26.7 vs 15.4 months, p=0.008) and low/normal LDH (23.4 vs 15.3 months, p=0.019)
• Of the 33 patients achieving an objective response (CR or PR), 57.6% (19 patients) responded within the first or second treatment cycle, while one-third (33.3%; 11 patients) of responses were observed in Cycles 4–6, highlighting the importance of continued treatment up to the maximum of 6 cycles to optimize tumor response.
• The overall safety profile was similar across subgroups, with no evidence of cumulative toxicity with successive treatment cycles. The incidence of serious adverse events and Grade 3/4 adverse events was consistent with the overall study population, and no treatment-related deaths occurred. “These subgroup analyses provide valuable insights into optimizing treatment with Melphalan/HDS for patients with unresectable metastatic uveal melanoma, underscoring the importance of early intervention in patients with lower tumor burden to maximize clinical benefits,” said Vojislav Vukovic, MD, MSc, PhD, Chief Medical Officer of Delcath. “The consistent efficacy and manageable safety profile across diverse patient groups further validate this liver-directed therapy as a key option in managing this challenging disease.”

The FOCUS study utilized HEPZATO KIT (HEPZATO (melphalan) for Injection/Hepatic Delivery System), and results from the FOCUS study lead to the approval of HEPZATO by the U.S. Food and Drug Administration (FDA).

The article is available here.

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 (HDS) 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 considered a combination drug and device product and is regulated and approved for sale as a drug by the FDA. HEPZATO KIT is comprised of the chemotherapeutic drug melphalan and Delcath's proprietary HDS. The HDS is used to isolate the hepatic venous blood from the systemic circulation while simultaneously filtrating hepatic venous blood during melphalan infusion and washout. The use of the HDS results in loco-regional delivery of a relatively high melphalan dose, which can potentially induce a clinically meaningful tumor response with minimal hepatotoxicity and reduce systemic exposure. HEPZATO KIT is approved in the United States 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 the full Prescribing Information, including BOXED WARNING for the HEPZATO KIT.

In Europe, the device-only configuration of the HDS is regulated as a Class Ill medical device and is approved for sale under the trade name CHEMOSAT Hepatic Delivery System for Melphalan, or CHEMOSAT, where it has been used in the conduct of percutaneous hepatic perfusion procedures at major medical centers to treat a wide range of cancers of the liver.

Forward-Looking Statements
The Private Securities Litigation Reform Act of 1995 provides a safe harbor for forward-looking statements made by Delcath or on its behalf. This press release contains forward-looking statements, including statements regarding the potential of CHEMOSAT Hepatic Delivery System and HEPZATO KIT to improve outcomes for patients with metastatic uveal melanoma. All forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially. Factors that may cause such differences include, but are not limited to, those discussed in the Company's filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Delcath undertakes no obligation to publicly update or revise these forward-looking statements except as required by applicable law.

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Investor Relations Contact:

ICR Healthcare

investorrelations@delcath.com

Source: Delcath Systems, Inc.