mycosis fungoides
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Results of a phase 2 trial suggest a topical, skin-directed histone deacetylase (HDAC) inhibitor can elicit responses in patients with early stage mycosis fungoides (MF).
The drug, remetinostat, was designed to be active in the skin but rapidly broken down and inactivated in blood in order to limit the adverse effects associated with systemic exposure to HDAC inhibitors.
The trial included 60 MF patients who were randomized to receive 0.5% remetinostat gel twice daily, 1% remetinostat gel once daily, or 1% remetinostat gel twice daily for between 6 and 12 months.
Results from this trial were recently released by Medivir AB, the company developing remetinostat.
The primary endpoint of the study was the proportion of patients with either a complete or partial confirmed response to therapy, assessed using the Composite Assessment of Index Lesion Severity.
Based on an intent-to-treat analysis, patients receiving the 1% remetinostat gel twice daily arm had the highest proportion of confirmed responses. Eight of 20 patients (40%) responded, which included 1 complete response.
Five of 20 patients (25%) receiving 0.5% remetinostat gel twice daily responded, as did 4 of 20 (20%) patients receiving 1% remetinostat gel once daily. None of these responses were complete responses.
Remetinostat was well-tolerated across all the dose groups, according to Medivir. There were no signs of systemic adverse effects, including those associated with systemic HDAC inhibitors.
Based on these data, Medivir expects to initiate discussions with regulatory authorities with the aim of initiating a phase 3 study later this year, and to present full phase 2 trial data at scientific meetings in the second half of 2017.
“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,” said Richard Bethell, Medivir’s chief scientific officer.
“Based on the efficacy and safety data from this phase 2 study, we believe that remetinostat is capable of meeting a very important unmet need in patients with this chronic and poorly treated orphan disease.”
