Praxis Precision Medicines, Inc.

Praxis Precision Medicines develops small molecule and antisense oligonucleotide therapies targeting central nervous system disorders characterized by neuronal excitation-inhibition imbalance. The company operates two primary platforms: Cerebrum, which focuses on orally available small molecule precision therapies, and Solidus, dedicated to antisense oligonucleotide discovery and development. Its pipeline includes multiple candidates in late-stage development addressing rare genetic epilepsies and neurological conditions caused by ion channel dysfunction.

The company's most advanced programs include ulixacaltamide, a T-type calcium channel inhibitor in New Drug Application review for essential tremor, and relutrigine, targeting SCN2A- and SCN8A-related developmental and epileptic encephalopathies, also under NDA review while progressing through Phase 3 trials for broader epilepsy indications. Additional clinical candidates include vormatrigine for focal epilepsy and Elsunersen, an antisense oligonucleotide for SCN2A developmental and epileptic encephalopathy. The company maintains early-stage programs addressing KCNT1, PCDH19, SYNGAP1, and SCN2A loss-of-function mutations. Praxis has research and licensing collaborations with Ionis Pharmaceuticals and RogCon Inc.

Based in Boston, Massachusetts, the company operates with 168 full-time employees and was incorporated in Delaware in 2015, having previously operated as EpiPM Therapeutics before adopting its current name in 2016. As a clinical-stage biopharmaceutical company, Praxis does not generate material product revenues.