Delcath Systems, Inc., a specialty pharmaceutical and medical device company, announces the initiation of a phase 3 clinical trial of Melphalan Hydrochloride for injection for use with the Delcath Hepatic Delivery System (Melphalan/HDS) for the treatment of patients with hepatic dominant ocular melanoma (OM).

The FOCUS Clinical Trial for Patients with Hepatic Dominant Ocular Melanoma (the FOCUS Trial) will evaluate the safety, efficacy and pharmacokinetic profile of Melphalan/HDS versus best alternative care in 240 patients with OM. The primary endpoint is a comparison of overall survival between the two study arms; secondary and exploratory endpoints include progression-free survival, overall response rate and quality of life measures. The FOCUS Trial is being conducted under a Special Protocol Assessment (SPA) with the US Food and Drug Administration (FDA), and per the SPA is the only phase 3 trial required for submission of a New Drug Application.

In the trial's treatment phase, patients randomized to the Melphalan/HDS arm will receive up to six treatments at intervals of six to eight weeks for up to 12 months. Tumor response will be assessed in both study arms every 12 weeks until evidence of hepatic disease progression. For patients progressing to the follow-up phase, disease assessment scans will continue every 12 weeks for up to two years. The FOCUS Trial will be conducted at leading cancer centers in the United States and Europe.

Under the terms of the SPA, at least 50% of patients will be treated in the US.  The Moffitt Cancer Center in Tampa, Fla. has been activated as a participating center and Jonathan Zager, M.D., FACS, Professor of Surgery in the Cutaneous Oncology and Sarcoma Departments and a Senior Member at Moffitt Cancer Center, is serving as the trial's principal investigator.

"I am particularly pleased to serve as principal investigator in this very promising study as I have treated patients with Melphalan/HDS through both formal clinical research and compassionate use since 2007," said Dr. Zager. "Our experience at Moffitt with Melphalan/HDS in patients with hepatic dominant ocular melanoma has shown significant potential. We are pleased to be taking a leadership role in the FOCUS Trial, and look forward to verifying the potential for Melphalan/HDS in this life-threatening cancer with no effective treatment options."

"We believe the FOCUS Trial puts us on the fastest path to a regulatory submission in the U.S. and initiation of this trial is a landmark event for Delcath," said Jennifer K. Simpson, Ph.D., MSN, CRNP, president and chief executive officer of Delcath. "We are delighted to be working with the Moffitt Cancer Center and look forward to activating a number of other premier cancer centers as clinical sites in the coming months.  Our goal is to have an interim analysis, which we expect to occur in the second half of 2017.  We look forward to bringing this potentially life-saving therapy to patients suffering with hepatic ocular melanoma."

"The FOCUS Trial will utilize an improved Melphalan/HDS product/procedure that addresses safety issues raised in our previous Phase 3 study. Based on our commercial experience in Europe, and the bolus of clinical data recently presented and published, we are optimistic that the FOCUS Trial will demonstrate a compelling benefit/risk profile and that the study's objectives will be met," added Dr Simpson.

Located in Tampa, Moffitt is one of only 45 National Cancer Institute-designated Comprehensive Cancer Centers, a distinction that recognizes Moffitt's excellence in research, its contributions to clinical trials, prevention and cancer control. Moffitt is the top-ranked cancer hospital in Florida and has been listed in US News & World Report as one of the "Best Hospitals" for cancer care since 1999.

Delcath Systems, Inc. is a specialty pharmaceutical and medical device company focused on oncology with a principal focus on the treatment of primary and metastatic liver cancers.