4SC is focused primarily on the development of resminostat (Kinselby), and, in particular, preparation of the marketing authorisation for the commercialization of resminostat (Kinselby), a unique maintenance treatment for CTCL.

Key highlights of Q3 2023

Data from the Company’s RESMAIN study was published in September 2023 at the EORTC Cutaneous Lymphoma Tumour Group Annual Meeting. In this study, resminostat (Kinselby) showed*:
A statistically significant improvement in progression free survival (median PFS: 8.3 vs. 4.2 months; p=0.015; HR: 0.623)
A median time to next treatment versus placebo showed a significant improvement of 8.8 months compared to 4.2 months
A clinically meaningful improvement in median "total" PFS of 24.3 months, compared to 14.9 months for those in the placebo group
Side effects of resminostat were mainly mild to moderate, manageable and reversible
In Q3, 4SC has been actively progressing the preparation of its Marketing Authorization Application in the European Union, Switzerland and the United Kingdom
In addition, a significant effort has been made in preparing for discussions with the United States (US) Food and Drug Administration regarding requirements for marketing resminostat (Kinselby) for CTCL in the US
On 27 September 2023, the Company announced that the US FDA had granted resminostat (Kinselby) orphan drug designation, which gives a number of benefits, most importantly seven years’ market exclusivity in the US
4SC’s partner for Japan, Yakult Honsha Co. Ltd (Yakult Honsha) is continuing with its preparation and filing of a marketing application for resminostat (Kinselby) in Japan.
Cash balance development in Q3 2023 and financial forecast

As at 30 September 2023, 4SC held cash balance/funds of €10.44 million compared to €12.16 million as of 30 June 2023. The decrease of cash funds in the first nine months of 2023 is mainly a result of the expenditure on the ongoing RESMAIN clinical study.

The monthly use of cash from operations amounted to €487 thousand in Q3 2023. The average monthly use of cash for 2023 is now expected to be between €500 – €700 thousand, a significant reduction from the previous forecast of between €800 thousand – €1.1 million.

Based on current financial and operating activities, the Manage­ment Board estimates that current funds should now be sufficient to finance 4SC into the third quarter of 2024, longer than previously expected.

Post Period Event

In October 2023, the European Commission adopted the European Medicines Agency’s decision to grant orphan drug designation to resminostat (Kinselby) in cutaneous T-cell lymphoma (CTCL). Orphan drug designation (ODD) provides privileged status to drugs that show promise for the treatment of rare diseases in the European Union. ODD qualifies 4SC for benefits including protocol assistance, market exclusivity and fee reductions.

Jason Loveridge, Ph.D., CEO of 4SC, commented: "The last quarter has been a very busy and productive period for 4SC. Not only has the RESMAIN study demonstrated the significant benefit of resminostat (Kinselby) as a maintenance treatment for patients suffering from CTCL, a debilitating and incurable condition, but we have also secured orphan drug designation in both the US and, post period, in Europe. This designation comes with meaningful benefits, including market exclusivity for seven and ten years respectively. We remain focused on securing marketing authorization for resminostat (Kinselby) and are actively engaged in finding the right partner to take Kinselby forward to patients."

About Kinselby (resminostat)  

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, and primarily affects the skin, but may ultimately involve lymph nodes, blood and visceral organs.  

Currently, CTCL is incurable 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 was conducted at more than 50 clinical centers in 11 European countries and Japan. It included 201 patients who suffer from advanced-stage cutaneous T-cell lymphoma (CTCL) that have achieved disease control with systemic therapy. The patients were randomized 1:1 to receive either resminostat or placebo. Patients who experienced disease progression – while being on placebo – were offered resminostat in an open label treatment arm.  

The primary goal of the study was to determine whether maintenance treatment with resminostat prolongs progression-free survival and other secondary objectives. Data demonstrating that resminostat met the primary endpoint of the RESMAIN study was published in May 2023.

About the concept of maintenance therapy  

The pivotal RESMAIN study 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 a few 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.

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.