Praxis Precision Medicines is a clinical-stage biopharmaceutical company developing therapies for CNS disorders driven by excitation-inhibition imbalance. Its pipeline spans small-molecule and antisense platforms targeting indications such as essential tremor and developmental epileptic encephalopathies.
The FDA has accepted Praxis Precision Medicines' NDA for relutrigine with priority review and set a PDUFA target date of September 27, 2026. The therapy targets SCN2A and SCN8A developmental and epileptic encephalopathies and could become the first approved treatment for these rare conditions. The company has bolstered its cash position to support commercial launch preparations.
The FDA has authorized the first human trial of an allogeneic stem cell therapy for epilepsy, developed by Shanghai-based Unixell Biotechnology. The therapy uses donor-derived stem cells to produce GABA and suppress seizures in drug-resistant patients.
Multiple analysts have raised price targets on Praxis Precision Medicines, with Wolfe Research initiating coverage at $500 and Guggenheim increasing its target to $800, citing strong market potential for ulixacaltamide in essential tremor.
Praxis Precision Medicines is preparing to submit two New Drug Applications by mid-February 2026 for ulixacaltamide and relutrigine, both holding FDA Breakthrough Therapy Designation, targeting essential tremor and epilepsy markets.
Praxis Precision Medicines has submitted new drug applications for ulixacaltamide in essential tremor and relutrigine in rare epilepsies, both granted Breakthrough Therapy Designation. The company reported $926 million in cash as of December 31, 2025.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07505004 |
Double-blind, Randomized Clinical Trial Evaluating the Efficacy and Safety of Vormatrigine in Adults With Focal Seizures |
RECRUITING | PHASE3 |
| NCT07287163 |
Clinical Trial of Vormatrigine in Adult Patients With Epilepsy |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT07019922 |
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety |
RECRUITING | PHASE3 |
| NCT07010471 |
A Clinical Trial for Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine |
RECRUITING | PHASE3 |
| NCT06999902 |
Double-blind, Randomized Trial of PRAX-628 in Adults With Focal Seizures to Evaluate Efficacy and Safety (POWER1) |
RECRUITING | PHASE2/PHASE3 |
| NCT06908356 |
An Open Label Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adults With Focal Onset or Tonic-Clonic Seizures |
RECRUITING | PHASE2 |
| NCT06840925 |
A Clinical Trial to Evaluate the Relative Bioavailability of PRAX-628 |
NOT_YET_RECRUITING | PHASE1 |
| NCT06087276 |
Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET) |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT05818553 |
A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE) |
ACTIVE_NOT_RECRUITING | PHASE2/PHASE3 |
| NCT05737784 |
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy |
RECRUITING | PHASE1/PHASE2 |
