Praxis Precision’s relutrigine reduces seizures in rare epileptic disorders in phase 2 clinical trials

1. Relutrigine resulted in a 46% reduction in seizures and seizure freedom in 30% of patients.
2. Patients also experienced meaningful improvements in alertness, behavior, and communication.

The Latest

In a randomized, double-blind, placebo-controlled phase 2 clinical trial researchers from Praxis Precision investigated the efficacy of relutrigine in treating seizures in pediatric patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEE). The study showed that patients taking relutrigine experienced a 46% reduction in motor seizures, with 30% of patients achieving seizure freedom compared to the subjects in the placebo arm. The study also included a long-term extension phase, where patients enrolled went on to experience a 75% reduction in seizures. Additionally, patients achieved meaningful improvements in behavior, communication, and alertness.

Physician’s Perspective

Epilepsy is a chronic neurological disorder defined by a tendency to experience seizures. Seizures occur due to abnormal electrical discharges in the brain which can result in abnormal movements of the body, loss of tone, loss of consciousness, and/or stiffening of the body among other symptoms. SCN2A and SCN8A are genes responsible for the creation of specific sodium channels in the brain. Mutations in these genes lead to a rare group of epileptic disorders which result in seizures and developmental delays. Patients with DEE experience frequent, debilitating, drug-resistant seizures. Praxis Precision’s relutrigine shows promise in significantly changing the treatment landscape of DEE.

Molecular Target of Therapy

SCN2A and SCN8A mutations result in overactivation of specific sodium channels in the brain. Sodium channels in the brain allow for the conduction of neuronal signals. Relutrigine is a sodium channel modulator that achieves its therapeutic effect by binding to and reducing the activation of these mutated sodium channels, thereby reducing seizure frequency and severity.

Company History

Praxis Precision is a biopharmaceutical company specializing in the development of therapies for neurological diseases through its proprietary small molecule platforms (CerebrumTM and SolidusTM). Presently, Praxis has 5 candidates in the preclinical and 4 in the clinical trial stage. Ulixacaltamide, a drug developed to treat essential tremors, has advanced to the registration-enabling stage.

Further reading: https://www.globenewswire.com/news-release/2024/09/03/2939566/0/en/Praxis-Precision-Medicines-announces-positive-topline-results-from-the-EMBOLD-study-in-SCN2A-and-8A-developmental-epilepsies-highlighting-the-disease-modifying-potential-of-relutri.html

©2024 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.