MT1110

MT1110 is a non-brain-penetrant small molecule developed by Montara Therapeutics as a "universal" peripheral blocker to enable brain-restricted drug activity. It is the cornerstone of the company's BrainOnly platform, designed to saturate FKBP12 binding sites in peripheral tissues. By doing so, MT1110 prevents FKBP12-dependent therapeutic agents from binding to their targets outside of the central nervous system, thereby mitigating systemic toxicities and expanding the therapeutic window for potent drugs. MT1110 is being developed as a component of two-drug combination therapies, including MTX-E1 (MT1110 paired with the mTOR inhibitor everolimus) for the treatment of Tuberous Sclerosis Complex (TSC)-related epilepsy and other mTOR-driven CNS diseases. It is also being paired with a novel LRRK2-targeting BrainTAC for the treatment of Parkinson's disease. As of early 2024, MT1110 is in preclinical development, with clinical entry for the MTX-E1 combination anticipated in the second half of 2026.