RMX-5002

RMX-5002 is an orally bioavailable small molecule designed to modulate RNA processing, specifically targeting the alternative splicing of XBP1 (X-box binding protein 1). Developed by Remix Therapeutics using their proprietary RMx platform, the drug aims to inhibit the production of the spliced, active form of XBP1 (XBP1s), a key transcription factor in the unfolded protein response (UPR) that is frequently co-opted by cancer cells to survive metabolic stress and evade the immune system. By reducing XBP1s levels, RMX-5002 is intended to disrupt tumor growth and enhance anti-tumor immune responses. It is currently being evaluated in Phase 1 clinical trials for the treatment of advanced solid tumors.