Praxis Precision Medicines(PRAX.US)

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.