Develops therapies for central nervous system disorders, focusing on precision medicine and targeted treatments.
Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company headquartered in Boston, Massachusetts, focuses on developing innovative therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. The company's primary candidate, ulixacaltamide, is a small molecule inhibitor targeting T-type calcium channels. Currently, ulixacaltamide is undergoing Phase IIb clinical trials for essential tremor and Phase II trials for Parkinson's disease, demonstrating its potential in addressing these debilitating conditions.
In addition to ulixacaltamide, Praxis Precision Medicines is advancing PRAX-562 for pediatric patients with developmental and epileptic encephalopathies (DEE), as well as PRAX-628 for focal epilepsy. The company's pipeline also includes PRAX-222 for early-onset SCN2A-DEE, PRAX-020 for KCNT1-related epilepsies, PRAX-080 for PCDH19, and PRAX-090 and PRAX-100 for SYNGAP1 and SCN2A-LoF syndromes. These efforts underscore Praxis' commitment to addressing a spectrum of neurological disorders with significant unmet medical needs.
Established in 2015, Praxis Precision Medicines collaborates extensively within the biopharmaceutical industry and academia to bolster its research and development initiatives. Collaborative efforts include partnerships with RogCon Inc., Ionis Pharmaceuticals, Inc., UCB Biopharma SRL, and The Florey Institute, focusing on advancing therapeutic solutions through innovative approaches such as antisense oligonucleotides (ASOs). With a dedicated team and strategic alliances, Praxis Precision Medicines is poised to make meaningful advancements in the treatment landscape for CNS disorders.