4SC AG: Significant milestone reached for pivotal RESMAIN study of resminostat as maintenance therapy in CTCL
One third of patients enrolled according to plan
RESMAIN – pivotal study to evaluate resminostat for maintenance treatment of patients with advanced-stage cutaneous T-cell lymphoma (CTCL) who have achieved disease control with prior systemic therapy
Top-line results expected in the first half 2019
Planegg-Martinsried, Germany, 5 December 2017 – 4SC AG (4SC, FSE Prime Standard: VSC) today announced, that one third of patients have been enrolled in the RESMAIN study according to plan by the end of November.
The pivotal RESMAIN study, which is conducted as a multi-center, double blind, randomized, placebo-controlled, 150 patients study, evaluates the histone deacetylase (HDAC) inhibitor resminostat for maintenance treatment of patients with advanced-stage cutaneous T-cell lymphoma (CTCL) who have achieved disease control with prior systemic therapy at more than 50 clinical centers in 11 European countries.
The concept of maintenance therapy
The RESMAIN study is focused on patients with advanced-stage, incurable 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 organ can be involved. None of the current therapeutic options achieve stable disease for long periods, with most patients progressing within six months on average.
Resminostat is being evaluated as maintenance treatment – prolonging the period patients are stable and not progressing. Recent preclinical data further suggests that resminostat has the potential to alleviate the itching in CTCL patients, thereby additionally improving the quality of life for patients.
Susanne Danhauser-Riedl, M.D., Chief Medical Officer of 4SC, commented: “We thank the patients for participating in the RESMAIN study and the study teams and investigators for their efforts and contribution. The study is progressing in line with our expectations with now more than 50 study sites open for enrollment in all participating countries and already 50 out of 150 patients being randomized.
We expect top-line results to be available in the first half of 2019 and, if positive, we plan to submit resminostat for market approval in Europe and the US. If approved, resminostat would be the first HDAC inhibitor approved for CTCL in Europe and the first and only drug approved for maintenance therapy in this indication in Europe and the US.”
Resminostat is orally administered and potentially offers a novel approach to treating a wide variety of cancers, both as monotherapy and in combination therapy with other anti-cancer drugs. Resminostat inhibits tumor growth and proliferation, causes tumor regression, and strengthens the body’s immune response to cancer.
Resminostat has been shown to be well tolerated in several clinical trials. Resminostat is currently being investigated in a Phase II pivotal study in cutaneous T-cell lymphoma (CTCL) by 4SC. A Phase II study in biliary tract cancer is planned by 4SC’s development partner Yakult Honsha in Japan. Amongst others, resminostat has previously been investigated in biliary tract or pancreatic cancer and hepatocellular carcinoma (HCC).
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, halting disease progression, improving quality of life and prolonging progression free and overall survival.
About the RESMAIN study – Resminostat for maintenance treatment of CTCL
The RESMAIN pivotal study is open for recruitment since November 2016 and is being conducted at more than 50 clinical centers in 11 European countries. It will include 150 patients who suffer from advanced-stage cutaneous T-cell lymphoma (CTCL) and 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 and CTCL is acquired.
4SC anticipates top-line results in the first half of 2019. If the results are positive, 4SC will submit the data to the relevant regulatory agencies for market approval of resminostat as maintenance treatment of CTCL.
4SC AG is a clinical-stage biopharmaceutical company developing small-molecule drugs that can target key indications in cancer with high unmet medical needs. Such drugs are intended to provide patients with innovative treatment options that are more tolerable and efficacious than existing therapies and provide a better quality of life. 4SC’s pipeline is protected by a comprehensive portfolio of patents and currently comprises three key drug candidates in various stages of preclinical and clinical development: resminostat, 4SC-202 and 4SC-208.
4SC aims to generate future growth and enhance its enterprise value by entering into partnerships with pharmaceutical and biotech companies and/or the eventual marketing and sales of approved drugs in select territories by 4SC itself. 4SC had 47 employees as of 30 September 2017 and is listed on the Prime Standard of the Frankfurt Stock Exchange (FSE Prime Standard: VSC; ISIN: DE000A14KL72).
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.