Praxis Precision Medicines is betting that a new epilepsy medicine can stand out in hard-to-treat patients, then move into broader use if upcoming late-stage studies keep delivering. The big upside depends on those trials staying on track and proving the early results hold up against entrenched competitors and real-world safety and dosing challenges.Read more
Praxis Precision Medicines is pushing several late-stage epilepsy and brain-disorder drug programs toward key trial results, and early data suggest its lead candidate could help hard-to-treat patients who still have frequent seizures. The big question is whether these results hold up in larger studies—and whether the company can win doctors and insurers over in a crowded market before it needs to raise more money.Read more
Praxis Precision Medicines has big hopes riding on a handful of late-stage epilepsy drug programs, but the next round of trial results could quickly change that outlook. It also may need to raise more money to run large, global studies and launch a product, which could weigh on long-term returns even if the science holds up.Read more
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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.; research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.; and research collaboration and license agreement to develop an iontophoretic transdermal patch for the delivery of ulixacaltamide. 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.
