Praxis Precision Medicines, Inc. PRAX R2K

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance in the United States. The company's platforms include Cerebrum, a small molecule platform for CNS small molecule therapies to develop orally available precision therapies; and Solidus to discover and develop antisense oligonucleotide. It also develops ulixacaltamide, a small molecule inhibitor of T-type calcium channels that in NDA for the treatment of essential tremor; relutrigine, a small molecule, which is in NDA for the treatment of SCN2A- and SCN8A-developmental and epileptic encephalopathies (DEE), as well as in Phase 3 trial for broad DEEs; vormatrigine, a small molecule targeting the hyperexcitable state of sodium-channels in the brain that is in Phase 3 trial for the treatment of adjunctive focal epilepsy, as well as in Phase 2 trial for monotherapy focal epilepsy; and Elsunersen, a clinical-stage ASO designed to selectively decrease SCN2A gene expression, which is in Phase 3 trial for the treatment of SCN2A-DEE. In addition, the company is developing product candidates in pre-clinical trial comprising PRAX-020 to treat KCNT1; PRAX-080 that targets PCDH19 mosaic expression disorders; PRAX-090 for targeting SYNGAP1 loss-of-function; and PRAX-100 that targets SCN2A LoF mutations. It has a cooperation and license agreement with RogCon Inc.; and a research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc. The company was formerly known as EpiPM Therapeutics, Inc. and changed its name to Praxis Precision Medicines, Inc. in October 2016. Praxis Precision Medicines, Inc. was incorporated in 2015 and is headquartered in Boston, Massachusetts.