Delcath Systems Stock

Delcath Systems, Inc. operates as an interventional oncology company. The company focuses on the treatment of primary and metastatic liver cancers.

The company's lead product candidate, the HEPZATO KIT (melphalan hydrochloride for injection/hepatic delivery system), or HEPZATO, is a drug/device combination product designed to administer high-dose chemotherapy to the liver while controlling systemic exposure and associated side effects. In Europe, the hepatic delivery system is a stand-alone medical device having the same device components as the HEPZATO but without the melphalan hydrochloride and is approved for sale under the trade name CHEMOSAT Hepatic Delivery System for Melphalan, or CHEMOSAT, where it has been used at major medical centers to treat a wide range of cancers of the liver. Approximately consisting primarily of metastatic ocular melanoma, or mOM. Approximately 80% of the estimated 800 patients annually with mOM will be eligible for would be candidates for HEPZATO.

In the United States, HEPZATO is considered a combination drug and device product and is regulated as a drug by the United States Food and Drug Administration, or the FDA. Primary jurisdiction for regulation of HEPZATO has been assigned to the FDA's Center for Drug Evaluation and Research. The FDA has granted Delcath six orphan drug designations (five for melphalan the treatment of patients with ocular (uveal) melanoma cutaneous melanoma, intrahepatic cholangiocarcinoma, hepatocellular carcinoma, and neuroendocrine tumor indications and one for doxorubicin in the treatment of patients with hepatocellular carcinoma). HEPZATO has not been approved for sale in the United States.

The company's clinical development program for HEPZATO consisted of the FOCUS Clinical Trial for Patients with Hepatic Dominant Ocular Melanoma (the FOCUS Trial), a global registration clinical trial that is investigating objective response rate in metastatic ocular melanoma, or mOM, a type of primary liver cancer. The company's most advanced development program is the treatment of mOM. It is reviewing the incidence, unmet need, available efficacy data and development requirements for a broad set of liver cancers in order to select a portfolio of follow-on indications that will maximize the value of the HEPZATO platform. In addition to HEPZATO's use to treat mOM, HEPZATO has the potential to treat other liver dominant cancers, such as Metastatic Colorectal Cancer and Cholangiocarcinoma, and plan to begin the study of HEPZATO to treat such conditions in the near future. The disease states the company is investigating and intend to investigate are unmet medical needs that represent significant market opportunities. In December 2021, the company announced that the FOCUS Trial for HEPZATO met its pre-specified endpoint.

On February 14, 2023, the company completed a NDA (new drug application) resubmission to the FDA for the HEPZATO Kit (melphalan hydrochloride for Injection/Hepatic Delivery System) seeking approval of the HEPZATO Kit in the treatment of patients with unresectable hepatic-dominant mOM. The resubmission is in response to a September 12, 2013 Complete Response Letter, or CRL, from the FDA for the company's NDA in December 2010 seeking approval of its first generation melphalan hydrochloride for injection/hepatic delivery system. The NDA resubmission contains comprehensive data and information on Generation Two HEPZATO Kit relating to the matters identified in the CRL. On March 20, 2023, the FDA determined the resubmission constituted a complete response and set a Prescription Drug User Fee Act target action date of August 14, 2023. The company continues to have early access programs in the United States to make HEPZATO available to mOM patients. The company is focused on continuing to treat these patients with mOM as regulatory approval is sought in the United States.

On February 28, 2022, CHEMOSAT received Medical Device Regulation (MDR) certification under the European Medical Devices Regulation [2017/745/EU], which may be considered by jurisdictions when evaluating reimbursement. As of March 1, 2022, the company had assumed direct responsibility for sales, marketing and distribution of CHEMOSAT in Europe.

CHEMOSAT and HEPZATO

The company's product candidate, the HEPZATO Kit, is a drug/device combination product designed to administer concentrated regional chemotherapy to the liver while controlling systemic exposure and associated side effects. This whole organ therapy is performed by isolating the circulatory system of the liver, infusing the liver with a chemotherapeutic agent, and then filtering the blood prior to returning it to the patient's circulatory system. During the procedure, known as percutaneous hepatic perfusion, PHP, or PHP therapy, three catheters are placed percutaneously through standard interventional radiology techniques. The catheters temporarily isolate the liver from the body's circulatory system, allow administration of the chemotherapeutic agent melphalan hydrochloride directly to the liver, and collect blood exiting the liver for filtration by the company's proprietary filters.

PHP therapy is performed in an interventional radiology suite in approximately two to three hours. Patients remain in an intensive care or step-down unit overnight for observation following the procedure. Treatment with CHEMOSAT and HEPZATO is repeatable, and a new disposable system is used for each treatment. In the company's clinical trials, melphalan hydrochloride for injection is provided to both European and the United States clinical trial sites.

Clinical Development Program

The focus of the company's clinical development program is to generate clinical data for CHEMOSAT and HEPZATO in various disease states to demonstrate efficacy and validate the safety profile of the version of the product and treatment procedure. The improvements the company has made to CHEMOSAT, HEPZATO and the PHP therapy have addressed the adverse event profile and procedure-related risks that led to the issuance of the CRL. The company's clinical development program is also designed to support clinical adoption of and reimbursement for CHEMOSAT in Europe, and to support regulatory filings and reimbursement in various jurisdictions, including the United States.

The FOCUS Trial evaluated the safety and efficacy of treatment with the HEPZATO Kit for patients with mOM. This is consistent with CHEMOSAT, the HDS device component of the HEPZATO Kit, approved in Europe under a CE mark to deliver melphalan to the liver. The safety data submitted in the NDA is consistent with the CHEMOSAT safety data documented in numerous European single-center and multi-center publications.

The Leiden University Medical Center completed a Phase 1b trial (CHOPIN trial) on the use of the Delcath CHEMOSAT Hepatic Delivery System with Melphalan (CHEMOSAT) in combination with the immune checkpoint inhibitors (ICI) ipilimumab and nivolumab to treat patients with metastatic uveal melanoma with liver metastases. The determination of a safe and effective dose was a primary goal of the Phase 1b portion of the CHOPIN trial. The phase 2 part of the CHOPIN study will provide more information on both hepatic and systemic toxicity associated with the combination therapy.

Market Access and Commercial Clinical Adoption

Since launching CHEMOSAT in Europe, there has been over 1,343 commercial treatments and CHEMOSAT is available in over 23 European cancer centers. Physicians in Europe have used CHEMOSAT to treat patients with a variety of cancers in the liver, primarily ocular melanoma liver metastases, and other tumor types, including cutaneous melanoma, hepatocellular carcinoma, cholangiocarcinoma, and liver metastases from colorectal cancer, breast, pancreatic and neuroendocrine. As of March 1, 2022, the company had assumed direct responsibility for sales, marketing and distribution of CHEMOSAT in Europe.

Research and Development

For the year ended December 31, 2022, the company's research and development expenses were $18.6 million.

Intellectual Property

The company holds rights in ten U.S. utility patents, one U.S. design patent, three pending U.S. utility patent applications, six issued foreign counterpart utility patents (including the validations of European Patents with claims directed to its filter and frame apparatus in 19 European countries, a European patent with claims directed to its filter apparatus and media in nine countries, and a European patent with claims to a kit of parts, directed to CHEMOSAT, in 18 countries), five issued foreign counterpart design patents, and two pending foreign counterpart patent applications. Patents directed to the company's chemotherapy filtration system 'Apparatus for Removing Chemotherapy Compounds from Blood' were issued by the United States Patent and Trademark Office in July 2017, October 2018, August 2019, February 2020, and February 2022. The patent issued in August 2019 has claims to a kit of parts capable of being assembled for delivering a small molecule chemotherapeutic agent to a subject. These claims are directed to HEPZATO KIT. The patent that issued in February 2020 has claims directed to the company's methods of treatment. In February 2019, a patent was issued by the United States Patent and Trademark Office with claims directed to a method of using the company's filter and frame apparatus and in August 2021 a patent was issued with claims directed to its filter and frame apparatus. A Hong Kong patent directed to the company's Filter and Frame Apparatus was issued in March 2018. A European patent was granted by the European Patent Office for the company's chemotherapy filtration apparatus in December 2018 and in July 2019 a European patent was granted by the European Patent Office with claims to a kit of parts, directed to CHEMOSAT. A European patent directed to a method of using the company's filter and frame apparatus was granted in April 2019 by the European Patent Office. In August 2019, a European patent was granted by the European Patent Office with claims directed to the company's filter and frame apparatus and validated in eleven countries to provide additional European patent coverage for the company's filter and frame apparatus to the European patent directed to the frame apparatus that was granted in April 2017. When appropriate, the company actively pursue protection of its proprietary products, technologies, processes, and methods by filing United States and international patent and trademark applications. The company seeks to pursue additional patent protection for technology invented through research and development, manufacturing, and clinical use of CHEMOSAT and HEPZATO that will enable the company to expand its patent portfolio around advances to its systems, technology, and methods for its applications, as well as beyond the treatment of cancers in the liver. In March 2022, a European patent was granted by the European Patent Office and validated in eight countries with claims to a kit of parts, directed to CHEMOSAT.

Government Regulation

The company's products are subject to extensive and rigorous government regulation by foreign regulatory agencies and the FDA. In the EU, the advertising and promotion of its products is also subject to EU Member States laws implementing the EU Medical Device Directive, Directive 2006/114/EC concerning misleading and comparative advertising and Directive 2005/29/EC on unfair commercial practices, as well as other EU Member State legislation governing the advertising and promotion of medical devices.

On February 28, 2022, CHEMOSAT received Medical Device Regulation certification under the European Medical Devices Regulation [2017/745/EU], which may be considered by jurisdictions when evaluating reimbursement.

The company is required to comply with the FDA's current good manufacturing practice (cGMP) regulations and international quality system regulations, including those established by the International Standards Organization (ISO), with respect to products sold in the EU.

History

Delcath Systems, Inc., a Delaware corporation, was founded in 1988. The company was incorporated in 1988.