The pivotal RESMAIN study, a multi-center, double-blind, randomized, placebo-controlled trial, is evaluating resminostat for maintenance treatment in patients with advanced-stage cutaneous T-cell lymphoma (CTCL). The study, conducted across more than 50 clinical centers in 11 European countries and 5 centers in Japan, is expected to unblind in Q1 2023, with top-line data available shortly thereafter.
The Data Safety Monitoring Board (DSMB), an independent committee of clinical and drug safety experts, has recommended the continuation of the RESMAIN study without modification following its final pre-planned review before unblinding. This positive outcome suggests a favorable safety profile for resminostat in this patient population.
RESMAIN Study Details
The RESMAIN study has completed recruitment, enrolling over 200 patients with advanced-stage CTCL who have achieved disease control with prior systemic therapy. Patients were randomized 1:1 to receive either resminostat or placebo. Those on placebo who experience disease progression will be offered resminostat in an open-label treatment arm.
The primary endpoint of the study is progression-free survival (PFS), while a key secondary objective is to prolong the time to symptom worsening, specifically itching. The study also includes a comprehensive biomarker program to enhance understanding of resminostat's biological effects in CTCL.
Resminostat: An HDAC Inhibitor for CTCL
Resminostat is an orally administered class I, IIb and IV histone deacetylase (HDAC) inhibitor. Preclinical data suggest that resminostat can inhibit tumor growth and proliferation, induce tumor regression, and enhance the body’s immune response to cancer. It is currently being investigated by 4SC in Europe and by Yakult Honsha in Japan as a maintenance treatment for CTCL.
Cutaneous T-Cell Lymphoma: An Unmet Need
CTCL is a rare disease, with approximately 5,000 new cases diagnosed in Europe each year. It arises from the malignant transformation of T-cells, primarily affecting the skin but potentially involving lymph nodes, blood, and visceral organs. Current treatment options for advanced-stage CTCL are limited, and while patients may respond to available therapies, the duration of response is often short-lived. The key therapeutic challenge is to prolong remissions by halting disease progression and improving patients' quality of life.
The RESMAIN study is focused on patients with advanced-stage, incurable CTCL who suffer from painful and itchy skin lesions, leading to disfigurement and a severely impaired quality of life. Resminostat is being evaluated as a maintenance treatment to prolong the period patients are stable and not progressing. Recent preclinical data also suggests that resminostat may alleviate itching in CTCL patients, further improving their quality of life.
Susanne Danhauser-Riedl, M.D., Chief Medical Officer of 4SC, stated, "We are encouraged by the positive outcomes of the repeated and regular independent DSMB safety reviews and are very happy that, despite the challenges caused by the pandemic, we were able to successfully complete enrolment in the RESMAIN study – the largest prospective clinical trial evaluating maintenance therapy in advanced-stage CTCL."
