
REC-4539 is the first LSD1 inhibitor designed to be both CNS-penetrant and reversible, and is being developed for multiple hematology and solid tumor indications, including small-cell lung cancer (SCLC) and acute myeloid leukemia (AML). There are currently no LSD1 inhibitors approved by the FDA. REC-4539’s combined properties distinguish it from other LSD1 inhibitors in development, with the potential to reduce adverse events seen from on-target platelet effects.
Lysine-specific demethylase 1 (LSD1) is a promising epigenetic target in aggressive cancers like small cell lung cancer (SCLC) and acute myeloid leukemia (AML). Successfully drugging LSD1 could provide a new option for the ~45,000 extensive stage SCLC patients in the US and EU5 who have progressed following frontline therapy and currently face limited options, as well as patients with certain blood cancers. Previous clinical attempts have been stalled by two critical challenges: