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Praxis Precision Medicines

Praxis Precision Medicines logo
🇺🇸United States
Ownership
Public
Established
2015-09-22
Employees
82
Market Cap
$950.3M
Website
http://www.praxismedicines.com
Introduction

Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company, which engages in the genetic epilepsies into the development of therapies for central nervous system, or CNS, disorders characterized by neuronal excitation-inhibition imbalance. It focuses on two proprietary platforms: Cerebrum and Solidus. The Cerebrum platform utilizes a deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies. The Solidus platform consists of antisense oligonucleotide, or ASO. It is a targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology. The company was founded by Kiran Reddy, David Goldstein, and Steven Petrou on September 22, 2015 and is headquartered in Boston, MA.

Clinical Trials

24

Active:2
Completed:8

Trial Phases

3 Phases

Phase 1:8
Phase 2:11
Phase 3:3

Drug Approvals

21

FDA:21

Drug Approvals

Famotidine

Approval Date
Jan 1, 2023
FDA

good sense cough dm

Approval Date
Jan 1, 2023
FDA

Acid Controller

Approval Date
Jan 1, 2023
FDA

Cough DM

Approval Date
Jan 1, 2023
FDA

Rugby Famotidine

Approval Date
Jan 1, 2023
FDA

careone acid relief

Approval Date
Jan 1, 2023
FDA

Cough DM

Approval Date
Jan 1, 2023
FDA

dg health heartburn prevention

Approval Date
Jan 1, 2023
FDA

Good Sense Acid Reducer

Approval Date
Jan 1, 2023
FDA

basic care cough dm

Approval Date
Jan 1, 2023
FDA
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Clinical Trials

Distribution across different clinical trial phases (22 trials with phase data)• Click on a phase to view related trials

Phase 2
11 (50.0%)
Phase 1
8 (36.4%)
Phase 3
3 (13.6%)

A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

Phase 3
Not yet recruiting
Conditions
SCN2A Encephalopathy
Epileptic Encephalopathy
Interventions
Drug: 1mg elsunersen
Procedure: sham procedure
Drug: 0.5mg elsunersen
First Posted Date
2025-06-13
Last Posted Date
2025-06-18
Lead Sponsor
Praxis Precision Medicines
Target Recruit Count
50
Registration Number
NCT07019922
Locations
🇺🇸

Praxis Research Site, Chicago, Illinois, United States

A Clinical Trial for Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine

Phase 3
Recruiting
Conditions
Developmental and Epileptic Encephalopathy 1
Interventions
Drug: 1.0mg/kg/day PRAX-562
Drug: Placebo
Drug: 1.5mg/kg/day PRAX-562
First Posted Date
2025-06-08
Last Posted Date
2025-08-05
Lead Sponsor
Praxis Precision Medicines
Target Recruit Count
160
Registration Number
NCT07010471
Locations
🇺🇸

Praxis Research Site, Chevy Chase, Maryland, United States

Double-blind, Randomized Trial of PRAX-628 in Adults With Focal Seizures to Evaluate Efficacy and Safety (POWER1)

Phase 2
Recruiting
Conditions
Focal Seizure
Interventions
Drug: 20mg/day PRAX-628 for 6 weeks then 30mg/day PRAX-628 for remaining 6 weeks
Drug: Placebo
First Posted Date
2025-05-31
Last Posted Date
2025-05-31
Lead Sponsor
Praxis Precision Medicines
Target Recruit Count
230
Registration Number
NCT06999902
Locations
🇪🇸

Praxis Research Site, Valencia, Spain

An Open Label Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adults With Focal Onset or Tonic-Clonic Seizures

Phase 2
Recruiting
Conditions
Focal Onset Seizure
Primary Generalized Epilepsy
Interventions
Drug: 30mg PRAX-628
First Posted Date
2025-04-03
Last Posted Date
2025-04-03
Lead Sponsor
Praxis Precision Medicines
Target Recruit Count
50
Registration Number
NCT06908356
Locations
🇪🇸

Praxis Research Site, Madrid, Spain

A Clinical Trial to Evaluate the Relative Bioavailability of PRAX-628

Phase 1
Not yet recruiting
Conditions
Healthy
Interventions
First Posted Date
2025-02-21
Last Posted Date
2025-02-21
Lead Sponsor
Praxis Precision Medicines
Target Recruit Count
16
Registration Number
NCT06840925
Locations
🇦🇺

Nucleus Network Melbourne, Melbourne, Victoria, Australia

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News

Actio Biosciences Raises $66M Series B to Advance Rare Disease Programs Targeting Ion Channel Disorders

Actio Biosciences secured $66 million in Series B funding co-led by Regeneron Ventures and Deerfield Management to advance two lead programs targeting rare genetic diseases.

Sodium Channel Blockers Pipeline Surges with 20+ Pharmaceutical Companies Developing Novel Therapies

The sodium channel blocker market is expanding rapidly due to increasing prevalence of neuropathic pain and rising demand for selective treatments targeting Nav1.7 and Nav1.8 inhibitors.

Praxis Precision's Essential Tremor Trial Halted After Disappointing Interim Analysis

Data monitoring committee recommends halting Praxis Precision's Essential 3 Study 1 of ulixacaltamide for essential tremor due to futility in meeting primary efficacy endpoints.

Praxis's Phase 3 Essential Tremor Drug Trial Faces Futility Concerns, Shares Plunge 40%

Praxis Precision Medicines faces a major setback as trial monitors recommend stopping Phase 3 study of ulixacaltamide for essential tremor due to likely efficacy endpoint failure.

Praxis Precision Medicines Stock Surges as Analysts Maintain Strong Buy Ratings for CNS Drug Developer

Praxis Precision Medicines (NASDAQ:PRAX) stock reaches $90.77, showing strong market performance with shares up 4.7% and maintaining positive momentum above its 200-day moving average.

Praxis Precision Medicines Announces Strategic Priorities and Pipeline Milestones for 2025

Praxis Precision Medicines anticipates four pivotal clinical trial readouts in 2025, positioning the company for potential commercial launches between 2026 and 2028.

FDA Grants Rare Pediatric Disease Designation to Praxis' Relutrigine for Dravet Syndrome

The FDA has granted Rare Pediatric Disease Designation (RPDD) to Praxis Precision Medicines' relutrigine for the treatment of Dravet syndrome, a severe form of epilepsy.

Praxis Precision Medicines Presents Promising Epilepsy Pipeline Updates at AES 2024

Praxis Precision Medicines is advancing relutrigine for developmental and epileptic encephalopathies (DEEs), showcasing robust Phase 2 data with improvements in motor seizures.

Praxis Precision Medicines Advances CNS Pipeline with Key Clinical Trial Milestones

Praxis Precision Medicines' PRAX-114 Phase 2/3 clinical trial for Major Depressive Disorder (MDD) is set to begin in March 2021 after IND clearance.

GeneDx Launches Innovative Programs to Accelerate Rare Disease Diagnosis and Drug Development

• GeneDx partners with Biogen, Praxis Precision Medicines, and Stoke Therapeutics to expand access to whole exome sequencing for pediatric epilepsy patients, addressing the 5-8 year diagnostic odyssey many children currently face. • The company introduces GeneDx Discover, a first-of-its-kind data visualization tool providing biopharma companies access to deidentified genetic data from over 700,000 clinical exomes and genomes to improve drug development efficiency. • These initiatives aim to transform patient care by increasing diagnostic rates for rare diseases while simultaneously accelerating the development of targeted therapies through genetic insights.

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