Medivir AB has tested its skin-directed histone deacetylase (HDAC) inhibitor, remetinostat, in 60 patients with early stage skin cancer CTCL.
The phase II study patients were with the mycosis fungoides (MF) variant of CTCL, who were randomized to receive either 0.5% remetinostat gel BID, 1% remetinostat gel QD or 1% remetinostat gel BID for between 6 and 12 months. Medivir said that across all the dose groups, remetinostat was well-tolerated without signs of systemic adverse effects, including those associated with systemic HDAC inhibitors.
The data has encouraged Medivir to discuss with the regulatory bodies for a phase III study later this year, and to present full phase II trial data at scientific meetings in the second half of 2017.
CTCL is a chronic, orphan hematologic cancer that presents in the skin. Medivir estimates that it can enter about $900 million in the US alone for early-stage CTCL patients. According to the National Cancer Institute, the most common CTCL is the mycosis fungoides (MF) variant of CTCL. MF affects an estimated 15,000 to 20,000 people in the United States, with an estimated 1,500 new cases every year.
Dr Richard Bethell, Medivir’s Chief Scientific Officer. “There are few drugs available for the treatment of the disease, and those currently available have generally poor tolerability. As a result, CTCL patients and their physicians require safe and effective new treatment options. Remetinostat was designed to effectively inhibit HDACs within cutaneous lesions, but to be rapidly broken down in the bloodstream, preventing the side effects associated with systemically administered HDAC inhibitors. Based on the efficacy and safety data from this Phase II study, we believe that remetinostat is capable of meeting a very important unmet need in patients with this chronic and poorly treated orphan disease.”