Praxis Precision Medicines, Inc.
Drugmaker with rare disease work · based in Boston, United States · ticker PRAX · praxismedicines.com
Praxis Precision Medicines, Inc. works on SCN2A Encephalopathy and SCN8A Encephalopathy, 2 of the rare diseases we track. It has 5 active clinical trials, and 2 are looking for volunteers now.
Praxis Precision Medicines is a Boston company developing medicines for brain disorders, including several rare genetic epilepsies as well as more common conditions such as essential tremor. Its drug relutrigine is under FDA review for SCN2A and SCN8A developmental and epileptic encephalopathies, with a decision due December 27, 2026, and the company is testing elsunersen in children with early-onset SCN2A disease.
Praxis Precision Medicines, Inc. pipeline
Each row lists the diseases Praxis Precision Medicines, Inc. is studying at that stage, from the ones closest to approval down to the earliest work.
SCN2A Encephalopathy 2 trials recruiting · Essential Tremor recruiting · Focal Epilepsy
Praxis Precision Medicines, Inc. clinical trials
These are Praxis Precision Medicines, Inc. trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials looking for volunteers now
Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET)
Tests 60 mg ulixacaltamide, Placebo
For Essential Tremor
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Tests 1mg elsunersen, 0.5mg elsunersen
For Epileptic Encephalopathy and SCN2A Encephalopathy
3 trials underway, not taking new volunteers
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
Tests PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222 - Optional Ascending Doses, PRAX-222 - Fixed Doses, Placebo
For SCN2A-DEE and Epilepsy
Randomized Clinical Trial Evaluating the Efficacy and Safety of Vormatrigine in Adults With Focal Seizures
Tests 40 mg/day vormatrogine for 12 weeks, 30 mg/day vormatrogine for 12 weeks, 20 mg/day vormatrogine for 12 weeks, Placebo
For Focal Epilepsy
A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)
Tests PRAX-562
For SCN2A Encephalopathy and SCN8A Encephalopathy
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More about Praxis Precision Medicines, Inc.
Within its broader pharmaceutical portfolio, Praxis Precision Medicines, Inc. has active clinical trial programs and drug development efforts across 2 rare diseases, including SCN2A Encephalopathy, SCN8A Encephalopathy. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows active and recruiting rare disease studies sponsored by Praxis Precision Medicines, Inc., sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites.
The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Praxis Precision Medicines, Inc. is headquartered in Boston, United States, publicly traded under the ticker symbol PRAX. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Where Praxis Precision Medicines, Inc. trials take place
The trials above run at 29 study sites in 7 countries. Tap a country to see its states, cities and hospitals.
Rare diseases Praxis Precision Medicines, Inc. works on
Each disease page lists every open trial, not only Praxis Precision Medicines, Inc.'s, plus approved treatments.
SCN2A encephalopathy is a rare genetic epilepsy caused by a change in the SCN2A gene, which builds a sodium channel that brain cells use to fire.
How common: No reliable population count exists; the FamilieSCN2A Foundation describes SCN2A as a leading single-gene cause of infantile seizures, autism and intellectual disability, and estimates that about 75% of cases are still undiagnosed
SCN8A encephalopathy is a rare genetic epilepsy caused by a change in the SCN8A gene, which builds the Nav1.6 sodium channel that brain cells use to fire.
How common: Unknown; estimated to account for about 1% of epilepsies with encephalopathy (MedlinePlus Genetics), and GeneReviews states the true prevalence has not been established
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Praxis Precision Medicines, Inc.'s own site is praxismedicines.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- MedlinePlus Genetics (NIH) - SCN2A geneSCN2A Encephalopathy
- FamilieSCN2A Foundation - The SCN2A spectrum of autism and epilepsySCN2A Encephalopathy
- MedlinePlus Genetics (NIH) - SCN8A-related epilepsy with encephalopathySCN8A Encephalopathy
- GeneReviews - SCN8A-Related Epilepsy and/or Neurodevelopmental DisordersSCN8A Encephalopathy