• RESMAIN study examines the use of resminostat as maintenance therapy in patients with advanced-stage cutaneous T-cell lymphoma (CTCL) in Europe
  • Topline data expected in 2019

PLANEGG-MARTINSRIED, Germany I December 16, 2016 I 4SC AG (4SC, FSE Prime Standard: VSC) today announced enrollment of the first patient in the RESMAIN study, a European multicenter, double blind, randomized, placebo-controlled, pivotal study to evaluate the epigenetic cancer drug resminostat (RES) for maintenance treatment (MAIN) of patients suffering from advanced-stage cutaneous T-cell lymphoma (CTCL).

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.

The RESMAIN study – Resminostat for maintenance treatment of CTCL

The RESMAIN study is being conducted at more than 50 clinical centers in 10 European countries. It will include 150 patients who suffer from advanced-stage CTCL who have achieved disease control with systemic therapy. The patients will be 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 data to be available in 2019.

Prof Dr Rudolf Stadler, principal investigator of the RESMAIN study and Director of the University Clinic for Dermatology, Venereology, Allergology and Phlebology in Minden, Germany, commented: “New treatment options are desperately needed for patients with advanced-stage CTCL. CTCL inflicts a very high disease burden on patients, who suffer from painful and itchy skin lesions all over the body, resulting in disfigurement and a severely impaired quality of life. The launch of the RESMAIN study is a critical first step toward making new treatments available for these patients. The whole CTCL field is eagerly anticipating the results of this study.”

“Enrolling the first patient in the RESMAIN study is a significant milestone for 4SC,” says Dr Jason Loveridge, CEO of 4SC. “Histone deacetylase (HDAC) inhibitors have been proven to be effective in treating CTCL and two HDAC inhibitors are already on the market in the US. The proven performance of HDAC inhibitors in CTCL should give confidence for the potential of resminostat in the RESMAIN study and, after completion, we intend to immediately submit the data to the relevant regulatory agencies for market approval in Europe. We believe resminostat has the potential to be an important therapeutic option for the patients suffering with CTCL.”

About Resminostat

Resminostat is an orally administered epigenetic anti-cancer compound. As an inhibitor of HDAC (histone deacetylase) that targets class I, IIB, and IV HDACs, resminostat can reactivate e.g. silenced genes in cancer cells or conversely downregulate abnormally expressed genes. In addition to its direct effects, resminostat enhances the body’s own immune response to cancer.

Resminostat is in development both as monotherapy and in combination with other drugs. The compound has been shown to be well tolerated in Phase I studies, and its use in the treatment of cutaneous T-cell lymphoma, Hodgkin’s lymphoma and liver, lung, colon, pancreatic and biliary tract cancers is being investigated in further clinical studies. Initial positive efficacy results for resminostat monotherapy have already been observed in patients with Hodgkin’s lymphoma and in combination with the standard medication sorafenib in selected patients with advanced liver cancer.

About epigenetic cancer therapy

Epigenetic changes modify the activation of certain genes, but not the genetic code of DNA itself, causing activation or silencing of genes. This mechanism enables differentiated cells such as those in the lungs, nerves or skin to serve very different functions despite containing identical genetic code.

Epigenetic alterations are as important as genetic mutations in a cell’s transformation to cancer, and their manipulation holds great promise for cancer therapy. Epigenetic compounds such as 4SC’s resminostat and 4SC-202 may convert these epigenetic alterations back to normal. For example, treatment with epigenetic cancer compounds interrupts or combats the mechanism that is responsible for the onset of cancer, makes cancer cells visible to the body’s own immune system or renders them more responsive to immuno-oncological treatment.

Epigenetics is considered as a future growth market in the field of Oncology based on its significant promise as both a monotherapy and in combined approaches with immuno-oncology drugs and other therapeutic agents. In an October 2015 report by business information publisher Grand View Research, the worldwide epigenetics market was projected to generate revenues of US-$16 billion in 2022, up from US-$ 4 billion in 2014.

About 4SC

4SC (www.4sc.com) is a biotech company dedicated to the research and development of small-molecule drugs focused on epigenetic mechanisms of action for the treatment of cancers with high unmet medical needs. These drugs are intended to provide innovative treatment options for cancer patients that are more tolerable and efficacious than existing therapies, provide a better quality of life and offer increased life expectancy. The Company’s pipeline comprises promising products that are in various stages of clinical development. 4SC’s aim is to generate future growth and enhance its enterprise value by entering into partnerships with pharmaceutical and biotech companies. Founded in 1997, 4SC had 47 employees as of 30 September 2016. 4SC has been listed on the Prime Standard of the Frankfurt Stock Exchange since December 2005.