SMT-D001

SMT-D001 is a second-generation small molecule utrophin modulator that was under development by Summit Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). The drug was designed to address the underlying cause of DMD—the absence of the protein dystrophin—by upregulating the production of utrophin, a functional paralog of dystrophin. Utrophin is naturally expressed during fetal development and in regenerating muscle fibers; by sustaining its expression in mature muscle, SMT-D001 aimed to stabilize the sarcolemma and prevent the progressive muscle wasting characteristic of the disease. SMT-D001 was intended to offer improved pharmacological properties over Summit's first-generation candidate, ezutromid (SMT-C1100). However, following the clinical failure of ezutromid in the Phase 2 PhaseOut DMD trial in 2018, Summit Therapeutics discontinued its entire utrophin modulation pipeline, including SMT-D001, to focus on its anti-infectives and oncology portfolios.