More than 80% of events now reached for unblinding of RESMAIN

The occurrence of a well-defined event such as patient mortality or disease progression is often a primary outcome in oncology research. In the RESMAIN study Progression Free Survival (PFS) is defined as the primary endpoint of the study and an event occurs when a patient’s disease progresses. This means that the outcome of interest is not only whether or not an event occurred, but also when that event occurred. The length of follow-up is therefore important to ensure that a sufficient number of events are observed to detect a clinically meaningful and statistically significant outcome of the RESMAIN study.

Susanne Danhauser-Riedl, M.D., Chief Medical Officer of 4SC, commented: "Maintenance therapy is a novel approach with significant potential to benefit patients with CTCL and RESMAIN is the largest clinical study of maintenance therapy in CTCL. It is hoped that resminostat will increase the duration of progression free survival while maintaining or even improving health related quality of life. As we now have seen more than 80% of the 125 events required for unblinding, it is clear the RESMAIN study is moving towards a conclusion and we look forward to data read out."

About resminostat

Resminostat (http://www.4sc.com/product-pipeline/resminostat/) is an orally administered class I, IIb and IV histone deacetylase (HDAC) inhibitor that potentially offers an approach to treating different kinds of cancer. Resminostat demonstrated that it is well tolerated and can inhibit tumor growth and proliferation, cause tumor regression, and strengthen the body’s immune response to cancer.

Resminostat is currently being investigated in a pivotal study in cutaneous T-cell lymphoma (CTCL) as maintenance treatment by 4SC in Europe and by Yakult Honsha in Japan.

About cutaneous T-cell lymphoma (CTCL)

CTCL is a rare disease with approximately 5,000 patients being newly diagnosed in Europe each year. The disease arises from malignant transformation of T-cells, a specialized subgroup of immune cells, primarily affects the skin, but may ultimately involve lymph nodes, blood and visceral organs.

Currently, CTCL is not curable and treatment options for advanced-stage CTCL are limited. Although patients respond to the available treatment options, the duration of response is often short-lived and declines as the severity of the disease increases. The key therapeutic challenge in advanced-stage CTCL is therefore to make remissions more durable by halting disease progression and improving patient’s quality of life.

About the RESMAIN study – resminostat for maintenance treatment of CTCL

The pivotal RESMAIN study (https://clinicaltrials.gov/ct2/show/NCT02953301) is being conducted at more than 50 clinical centers in 11 European countries and Japan. It will include about 190 patients who suffer from advanced-stage cutaneous T-cell lymphoma (CTCL) that have achieved disease control with systemic therapy. The patients are randomized 1:1 to receive either resminostat or placebo. Patients who experience disease progression – while being on placebo – will be offered resminostat in an open label treatment arm.

The primary goal of the study is to determine whether maintenance treatment with resminostat prolongs progression-free survival and the key secondary objective is to prolong the time to symptom worsening (itching). A comprehensive biomarker program is also included in the study to ensure vital knowledge about the biological background of resminostat treatment in CTCL.

About the concept of maintenance therapy

The pivotal RESMAIN study (https://clinicaltrials.gov/ct2/show/NCT02953301) is focused on patients with advanced-stage, incurable cutaneous T-cell lymphoma (CTCL). Such patients suffer from painful and itchy skin lesions resulting in disfigurement and a severely impaired quality of life. Furthermore, lymph nodes, blood or visceral organs can be involved. The current therapeutic options rarely provide long-lasting responses or stabilization of disease for meaningful periods, with most patients progressing within several months.

Resminostat is being evaluated as maintenance treatment – a unique innovative treatment approach in CTCL (Stadler et al., 2021) – intended to prolong the period patients are stable and not progressing. Furthermore, recent preclinical data suggests that resminostat has the potential to alleviate the itching in CTCL patients, thereby additionally improving their quality of life.

Forward-looking information

Information set forth in this press release contains forward-looking statements, which involve risks and uncertainties. The forward-looking statements contained herein represent the judgement of 4SC as of the date of this press release. Such forward-looking statements are neither promises nor guarantees but are subject to a variety of risks and uncertainties, many of which are beyond 4SC’s control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. 4SC expressly disclaims any obligation or undertaking to release any updates or revisions to any such statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based.