Praxis Precision Medicines, Inc. (PRAX)
Praxis Precision Medicines, Inc. is a biopharmaceutical firm operating at the clinical stage, dedicated to creating innovative treatments for central nervous system (CNS) conditions that stem from an imbalance in neuronal activity. The company's primary investigational compounds currently undergoing evaluation include: PRAX-114, an extrasynaptic-preferring positive allosteric modulator of the GABAA receptor, which is currently in Phase IIa clinical trials for addressing both major depressive disorder and perimenopausal depression. Additionally, PRAX-944, a small molecule designed to selectively inhibit T-type calcium channels, is also progressing through Phase IIa clinical trials to manage essential tremor. Beyond these, Praxis is also advancing a pipeline of other promising drug candidates, such as: PRAX-562, a persistent sodium current blocker, undergoing Phase I clinical evaluation for treating severe pediatric epilepsy and adult cephalgia (headaches). PRAX-222, an antisense oligonucleotide (ASO) aimed at individuals suffering from gain-of-function (GOF) SCN2A epilepsy. And a program targeting KCNT1 for the treatment of KCNT1 GOF epilepsy. To further its research and development efforts, Praxis maintains several strategic alliances. These include a cooperation and licensing arrangement with RogCon Inc.; a licensing deal with Purdue Neuroscience Company; a comprehensive research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.; and a collaborative project with The Florey Institute focused on the creation of three new antisense oligonucleotides (ASOs). Established in 2015, the company's headquarters are situated in Boston, Massachusetts.
Sector: Pharmaceutical Preparations
Industry: Biotechnology
Praxis Precision Medicines, Inc.
Trials need funding. See this company's dilution risk and cash position.
- Phase 3Recruiting
Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET)
60 mg ulixacaltamide, Placebo
Essential Tremor
NCT06087276Enrollment1,000StartNov 2023Primary CompletionOct 2025CompletionMar 2027est. - Phase 3Active, not recruiting
A Clinical Trial for Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine
1.0mg/kg/day PRAX-562, 1.5mg/kg/day PRAX-562
Developmental and Epileptic Encephalopathy 1
NCT07010471Enrollment160StartJul 2025Primary CompletionDec 2026est.CompletionNov 2027est. - Phase 3Recruiting
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
1mg elsunersen, 0.5mg elsunersen
Epileptic Encephalopathy, SCN2A Encephalopathy
NCT07019922Enrollment40StartAug 2025Primary CompletionDec 2026est.CompletionDec 2028est. - Phase 3Enrolling by invitation
Clinical Trial of Vormatrigine in Adult Patients With Epilepsy
vormatrogine
Epilepsy, Tonic-Clonic, Focal Seizure +1
NCT07287163Enrollment700StartOct 2025Primary CompletionMay 2028est.CompletionDec 2028est. - Phase 3Active, not recruiting
Randomized Clinical Trial Evaluating the Efficacy and Safety of Vormatrigine in Adults With Focal Seizures
40 mg/day vormatrogine for 12 weeks, 30 mg/day vormatrogine for 12 weeks
Focal Epilepsy
NCT07505004Enrollment300StartJan 2026Primary CompletionJan 2027est.CompletionMay 2027est. - Phase 2/3Completed
A Clinical Trial of PRAX-114 in Participants With Major Depressive Disorder
PRAX-114, Placebo
Major Depressive Disorder
NCT04832425Enrollment216StartMar 2021Primary CompletionMay 2022CompletionMay 2022 - Phase 2/3Active, not recruiting
A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)
PRAX-562
SCN2A Encephalopathy, SCN8A Encephalopathy
NCT05818553Enrollment77StartAug 2023Primary CompletionNov 2025CompletionMar 2027est. - Phase 2/3Completed
Double-blind, Randomized Trial of PRAX-628 in Adults With Focal Seizures to Evaluate Efficacy and Safety (POWER1)
20mg/day PRAX-628 for 6 weeks then 30mg/day PRAX-628 for remaining 6 weeks, Placebo
Focal Seizure
NCT06999902Enrollment255StartDec 2024Primary CompletionMay 2026CompletionMay 2026 - Phase 2Completed
A Clinical Trial of Adjunctive and Monotherapy PRAX-114 in Participants With Major Depressive Disorder
10 mg PRAX-114, 20 mg PRAX-114
Major Depressive Disorder
NCT04969510Enrollment110StartJun 2021Primary CompletionJul 2022CompletionAug 2022 - Phase 2Completed
A Clinical Trial of PRAX-944 in Participants With Essential Tremor
Part A: 20 and 40 mg PRAX-944, Part B: 120 mg PRAX-944
Essential Tremor
NCT05021978Enrollment24StartMar 2020Primary CompletionMar 2022CompletionMar 2022 - Phase 2Completed
A Clinical Trial of 2 Doses of PRAX-944 in Participants With Essential Tremor
100 mg PRAX-944, 60 mg PRAX-944
Essential Tremor
NCT05021991Enrollment133StartOct 2021Primary CompletionFeb 2023CompletionFeb 2024 - Phase 2Terminated
A Clinical Trial of PRAX-114 in Participants With Post-Traumatic Stress Disorder
60 mg PRAX-114 or 40 mg PRAX-114, Placebo
Post-traumatic Stress Disorder, Post Traumatic Stress Disorder +4
NCT05260541Enrollment11StartJan 2022Primary CompletionAug 2022CompletionSep 2022 - Phase 2Withdrawn
A Clinical Trial of PRAX-114 in Participants With Essential Tremor
10 mg PRAX-114, 20 mg PRAX-114, and Placebo, 10 mg PRAX-114 or 20 mg PRAX-114
Essential Tremor
NCT05387642Enrollment0StartApr 2022Primary CompletionJun 2022CompletionJun 2022 - Phase 2Recruiting
An Open Label Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adults With Focal Onset or Tonic-Clonic Seizures
30mg PRAX-628
Focal Onset Seizure, Primary Generalized Epilepsy
NCT06908356Enrollment50StartJan 2025Primary CompletionJul 2025est.CompletionJul 2025est. - Phase 1/2Active, not recruiting
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses
SCN2A-DEE, Epilepsy
NCT05737784Enrollment60StartApr 2023Primary CompletionApr 2026CompletionFeb 2028est. - Phase 1Completed
A Clinical Trial to Evaluate the Relative Bioavailability of PRAX-628
PRAX-628
Healthy
NCT06840925Enrollment16StartApr 2025Primary CompletionJun 2025CompletionJun 2025 - ObservationalCompleted
A Prospective, Remote Observational Study in Pediatric Participants With Early-Onset SCN2A-Developmental and Epileptic Encephalopathy
-
SCN2A-DEE, Epilepsy
NCT05407727Enrollment5StartApr 2022Primary CompletionMar 2023CompletionMar 2023
| Drug / Therapy | Link | ||
|---|---|---|---|
| Phase 3 | Recruiting | 60 mg ulixacaltamide, Placebo | NCT06087276 |
| Phase 3 | Active, not recruiting | 1.0mg/kg/day PRAX-562, 1.5mg/kg/day PRAX-562 | NCT07010471 |
| Phase 3 | Recruiting | 1mg elsunersen, 0.5mg elsunersen | NCT07019922 |
| Phase 3 | Enrolling by invitation | vormatrogine | NCT07287163 |
| Phase 3 | Active, not recruiting | 40 mg/day vormatrogine for 12 weeks, 30 mg/day vormatrogine for 12 weeks | NCT07505004 |
| Phase 2/3 | Completed | PRAX-114, Placebo | NCT04832425 |
| Phase 2/3 | Active, not recruiting | PRAX-562 | NCT05818553 |
| Phase 2/3 | Completed | 20mg/day PRAX-628 for 6 weeks then 30mg/day PRAX-628 for remaining 6 weeks, Placebo | NCT06999902 |
| Phase 2 | Completed | 10 mg PRAX-114, 20 mg PRAX-114 | NCT04969510 |
| Phase 2 | Completed | Part A: 20 and 40 mg PRAX-944, Part B: 120 mg PRAX-944 | NCT05021978 |
| Phase 2 | Completed | 100 mg PRAX-944, 60 mg PRAX-944 | NCT05021991 |
| Phase 2 | Terminated | 60 mg PRAX-114 or 40 mg PRAX-114, Placebo | NCT05260541 |
| Phase 2 | Withdrawn | 10 mg PRAX-114, 20 mg PRAX-114, and Placebo, 10 mg PRAX-114 or 20 mg PRAX-114 | NCT05387642 |
| Phase 2 | Recruiting | 30mg PRAX-628 | NCT06908356 |
| Phase 1/2 | Active, not recruiting | PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses | NCT05737784 |
| Phase 1 | Completed | PRAX-628 | NCT06840925 |
| Observational | Completed | - | NCT05407727 |