On December 2, 2021 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported positive results from the phase 3 FOCUS study (Press release, Delcath Systems, DEC 2, 2021, View Source [SID1234596392]). The FOCUS study’s intent-to-treat (ITT) population was comprised of a total of 102 subjects, across various lines of therapy. Of the ITT group, 91 evaluable patients were administered at least one study treatment.

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Treatment with HEPZATO in the ITT analysis resulted in an objective-response-rate (ORR) of 31.4% [95% CI: 22.55-41.31], including 6.9% of patients with a complete response (CR). Median duration of response was 14 months [95% CI: 8.54, NC], with over half of responders continuing to be monitored for progression events. Disease control rate (DCR) was 65.7% [95% CI, 55.63, 74.81].

On the primary ORR endpoint, the lower bound 95% Confidence Interval (CI) of 22.55% exceeded the FOCUS trial’s prespecified 8.3% upper bound 95% CI threshold for success. This threshold was derived from a meta-analysis of sixteen checkpoint inhibitor publications documenting the treatment of 476 metastatic ocular melanoma patients.

Supportive, predefined, exploratory analyses were conducted comparing patients in the HEPZATO arm versus a BAC group. The BAC arm was comprised of a total of 42 patients, originally randomized in the FOCUS trial prior to its amendment, in consultation with FDA, to a single-arm pivotal study in 2018. The evaluable BAC subjects were treated predominantly with liver-targeted Transarterial Chemoembolization (TACE).

Among patients who received at least one study treatment, patients in the HEPZATO arm had statistically significant improvements over BAC in the following prespecified endpoints:

ORR of 35.2% versus 12.5% for the BAC arm (p=0.0154).
Disease Control Rate of 73.6% versus 37.5% for patients in the BAC arm (p=0.0002).
Median Progression Free Survival of 9.03 months versus 3.12 months for the BAC arm (HR=0.39; p=0.0002).
Enrollment in the FOCUS trial HEPZATO arm ended in late 2020 with overall survival data continuing to mature. Per the statistical plan, a final predefined exploratory survival analysis, versus BAC, will be conducted at 24-months after last patient last treatment.

As of this analysis, survival at 12-months in the evaluable patients was 75% in the HEPZATO arm versus 47% for BAC [HR=0.37, p=0.01]. Delcath will provide future overall survival analysis updates, as patient follow-up continues, and the Kaplan-Meier analysis matures.

In the HEPZATO safety population, the most commonly reported treatment-emergent serious adverse events were anemia (29.7% of patients), thrombocytopenia (26.4% of patients) and neutropenia (19.8% of patients), which were well-manageable. 5.3% of patients experienced treatment-emergent serious cardiac adverse events. In all cases the events resolved with no ongoing complications. There were no treatment-related deaths in the trial.

"Metastatic ocular melanoma is a disease with a dismal prognosis and new therapies are urgently needed. The FOCUS study results, along with the predefined analyses versus a relevant BAC group, clarify HEPZATO overall clinical benefit in this difficult-to-treat patient population," noted Dr. Jonathan Zager MD FACS, global lead investigator of the FOCUS study, senior member and Director of Regional Therapies at Moffitt Cancer Center. "The overall efficacy, coupled with an improved safety profile versus the first-generation product, suggests that HEPZATO would offer a compelling clinical benefit were it approved by FDA."

"We are thrilled by the HEPZATO response rates and duration of response which far exceed that which has been seen with other agents in this difficult-to-treat patient population. Our data further highlights HEPZATO’s potential superiority to other available liver-targeted therapies, which suggests a broader utility for our platform across multiple liver-metastatic tumor types. In addition to re-filing our NDA by mid-2022, Delcath, along with key opinion leaders, intend to study HEPZATO in additional indications in the near future."

The FOCUS trial results will be presented at a comprehensive Investor Update Meeting taking place today from 10:00am EST – 1:30pm EST. In addition to the FOCUS trial, a distinguished panel of physicians will discuss their personal clinical experience with HEPZATO in both the clinical trial setting and the commercial setting in Europe, as well as the potential for HEPZATO to treat liver metastatic tumor types beyond metastatic ocular melanoma.

Event Details:

Event: Delcath Systems Virtual Investor Update Meeting
Date: Thursday, December 2, 2021
Time: 10:00am – 1:30 p.m. EST

To register for this event, please click here.

The live webcast of the event may be accessed through the Events and Presentation page of Delcath’s website, under the Investors section. The archived webcast and presentation will be available on the Company’s website after the event.