Resources

Clinical Trials

Essential3 study is an at-home research study evaluating an investigational drug, to see if it may improve your ability to complete everyday tasks, such as eating, drinking and dressing. The Essential3 study has been designed so that you may participate from the comfort of your own home. For more information, visit www.essential3study.com

To learn more about the EMBOLD study for children diagnosed with SCN2A and SCN8A DEEs, please visit www.emboldstudy.com

To learn more about the EMBRAVE study for children diagnosed with early-onset SCN2A DEEs, please visit www.embravestudy.org

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Videos

Praxis DEEP Dive Day 2024

Scientific Publications

Ulixacaltamide
MDS 2024 Congress Essential3: An Innovative Multi-Study Phase 3 Program to Evaluate the Efficacy and Safety of Ulixacaltamide
2024 AAN Annual Meeting An Innovative Multi-Study Phase 3 Program to Evaluate the Efficacy and Safety of Ulixacaltamide: The Future of Clinical Trial Design in Essential Tremor Assessing Clinically Meaningful Improvement Associated with Ulixacaltamide Treatment in Adults with Essential Tremor on Concomitant Propranolol: Findings from Essential1
2023 IAPRD Meeting Essential1: Results from a Phase 2 Trial Evaluating the Tolerability, Safety, and Efficacy of Ulixacaltamide in Adults with Essential Tremor
2023 MDS Congress Patient-Focused, Clinically Meaningful Endpoints as Evidence of Improved Outcomes and Durability of Effect Following Ulixacaltamide Treatment in Adults with Essential Tremor: Findings from Essential1
2022 AAN Annual Meeting Translational Pharmacology of PRAX-944, a Novel T-type Calcium Channel Blocker for the Treatment of Essential Tremor
2022 MDS Congress A Phase 2 Clinical Trial Evaluating the Efficacy, Safety, Tolerabitlity, and Pharmacokinetics of PRAX-944 in Adults with Essential Tremor A Phase 2 Randomized, Double-Blind, Placebo-Controlled Trial of PRAX-944 for the Treatment of Essential Tremor The Hidden Disease Burden and Treatment Experience of Patients with Essential Tremor: A Retrospective US Claims Analysis
Ulixacaltamide Translational Findings in Movement Disorders Translational Pharmacology of PRAX-944, a Novel T-Type Calcium Channel Blocker in Development for the Treatment of Essential Tremor
ET Claims Analysis in Advances in Therapy The Hidden Burden of Disease and Treatment Experiences of Patients with Essential Tremor: A Retrospective Claims Data Analysis
TTCC Mechanistic Review in Annals of Clinical and Translational Neurology T-type Calcium Channels as Therapeutic Targets in Essential Tremor and Parkinson's Disease

Relutrigine (PRAX-562)
2024 AES Annual Meeting Relutrigine Demonstrates Robust Seizure Reduction and Seizure Freedom in DEEs: Results from the EMBOLD Study Emergency Use Case of Relutrigine, a Next-Generation Sodium Channel Functional State Modulator, in an Infant with SCN2A-DEE and Refractory Seizures and Recurrent Status Epilepticus
2024 EEC Meeting EMBOLD: A Clinical Trial of PRAX-562 in Subjects with Developmental and Epileptic Encephalopathies
2023 AES Behind the Seizure Scientific Exhibit; Partners Against Mortality in Epilepsy Conference EMBOLD: A Clinical Trial of PRAX-562 in Subjects with Developmental and Epileptic Encephalopathies Followed by an Open-Label Extension
2023 IEC Meeting PRAX-562 is a Well-Tolerated, Next Generation Anti-Seizure Small Molecule with Broad Anticonvulsant Activity in Multiple DEE Mouse Models PRAX-562-101: A Phase1 Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Food Effect of PRAX-562 in Healthy Volunteers PRAX-562-102: A Phase1 Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PRAX-562 in Healthy Volunteers
2022 Epilepsy Foundation Pipeline Conference PRAX-562 and Praxis Pipeline Updates
2022 Familes of SCN2A and SCN8A Advocacy Talk PRAX-562-221 Synopsis Review
2022 AES Annual Meeting and Behind the Seizure Scientific Exhibit A Novel Approach to Assess the Impact of Disease in Patients with SCN8A-Related Developmental and Epileptic Encephalopathy
2021 AES Annual Meeting Patient-guided Approaches to Drug Development in SCN8A-related Epilepsy: Insights from a Caregiver Survey of Feeding & Medication Formulation Priorities
PRAX-562 Pharmacology in Epilepsia The Novel Persistent Sodium Current Inhibitor PRAX-562 has Potent Anticonvulsant Activity with Improved Protective Index Relative to Standard of Care Sodium Channel Blockers

Elsunersen (PRAX-222)
2024 AES Annual Meeting Clinical Updates from the Elsunersen Emergency Use Program: A Novel ASO for Treatment of Early Onset SCN2A Developmental and Epileptic Encephalopathy
2024 EEC Meeting A Novel Antisense Oligonucleotide for the Treatment of Early Onset SCN2A Developmental and Epileptic Encephalopathy: A First-in-Patient Report in a Preterm Infant with Refractory Status Epilepticus Significant Seizure Reduction in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy following Treatment with Elsunersen, a Novel Antisense Oligonucleotide: Findings from the EMBRAVE Study
2023 AES Annual Meeting and Behind the Seizure Scientific Exhibit; Partners Against Mortality in Epilepsy Conference EMBRAVE: A Clinical Trial of PRAX-222, a Novel Antisense Oligonucleotide, in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy
2023 Oligonucleotides for CNS Meeting Leveraging Preclinical Models To Inform Clinical Trial Strategy in SCN2A Developmental and Epileptic Encephalopathy
2022 AES Annual Meeting and Behind the Seizure Scientific Exhibit Disease Impact and Burden in Patients with SCN2A-Related Developmental and Epileptic Encephalopathy
2022 Drug Discovery for Ion Channels XXII Meeting Using Dynamic Action Potential Clamp Modeling of NaV1.2 Variants to Support the Prediction of Clinical Phenotype in DEE

Vormatrigine (PRAX-628)
2024 AES Annual Meeting Vormatrigine Demonstrates Potent Antiseizure Activity Across Three Acute Models with Highest Predictive Validity for Focal Onset Seizures
2024 EEC Meeting Pharmacokinetics, Tolerability and Cardiac Safety for PRAX-628, a Precision Medicine Therapeutic Set to Initiate Registration Enabling Studies for Focal Onset Seizures
2023 AES Annual Meeting and Behind the Seizure Scientific Exhibit PRAX-628 is a Next Generation, Functionally Selective Small Molecule with Potent Anti-Seizure Activity and Potential as Best-in-Class Treatment for Focal Epilepsy Link to Platform Talk
2023 AAN Annual Meeting PRAX-628: A Novel Sodium Channel Blocker with Greater Potency and Activity Dependence Compared to Standard of Care
2023 IEC Meeting PRAX-628: A Next Generation Functionally Selective Small Molecule with Potent Anticonvulsant Activity Safety, Tolerability and Pharmacokinetic Findings from A First-In-Human, Randomized, Double-blind, Placebo Controlled Trial of Single and Multiple Ascending Doses of PRAX-628 in Healthy Participants

Other
2024 AES Annual Meeting Establishing the Predictive Validity of Preclinical Seizure Models in Generalized Epilepsies: An Extension of the Praxis Analysis of Concordance Framework
2024 EEC Meeting A Novel Translational Concordance Framework Identifies Preclinical Seizure Models with Highest Predictive Validity in Focal Onset Seizures A Novel Method to Define an EEG Composite for the Detection of Drug Effects of Next Generation Small Molecules for Epilepsy
2023 SYNGAP1 Conference Progress Toward the Discovery of an ASO for Therapeutic Upregulation of SYNGAP1
2021 AES Annual Meeting PRX-2904 is a Small Molecule Selective Inhibitor of KNa1.1 (KCNT1) that Reverses the ECoG Phenotype of a KCNT1 Gain-of-Function Mouse Model
PRX-2904 Chemistry in ACS Medicinal Chemistry Letters Discovery of the First Orally Available, Selective KNa1.1 Inhibitor: In Vitro and In Vivo Activity of an Oxadiazole Series