PRAX

Praxis Precision Medicines, Inc. operates as a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. Headquartered in Boston, Massachusetts, the company was founded in 2015 and utilizes its proprietary Praxis Cerebral Cortex Platform to identify genetic targets and develop precision medicines for rare epilepsy disorders and other neurological conditions. The company's lead product candidate, ulixacaltamide, is in Phase 3 clinical development for essential tremor and developmental and epileptic encephalopathies (DEEs), while relutrigine is being evaluated for SCN2A-DEE and other genetically defined epilepsies. Praxis also advances elsunersen, an antisense oligonucleotide targeting SCN2A gain-of-function variants. The company operates as a fully integrated research and development organization, leveraging insights from human genetics and electrophysiology to inform drug discovery and clinical development. In 2024, Praxis achieved significant milestones including positive topline data from its Phase 2b study of ulixacaltamide in essential tremor and advancement of multiple clinical programs. The company maintains strategic collaborations and has built a portfolio targeting genetically validated mechanisms in neurological disorders. Praxis employs approximately 150 people and remains focused on addressing high unmet medical needs in patient populations with limited or no approved treatment options.