Epilepsy affects as many as 50 million people worldwide, with a broad range of disease subtypes with limited treatment options. Fortunately, a new epilepsy drug under development by Praxis Precision Medicines could reduce seizures in large numbers of epilepsy patients — particularly children with two specific types of this neurological disorder. The drug, which was tested in a midphase trial known as the EMBOLD study, is a sodium channel inhibitor. While this drug class is not new, the study is an exciting new breakthrough for a class of medications that have been explored for various indications over the last 50 years. Below, we’ll outline how this exciting new study could improve the treatment of epilepsy in one of the most vulnerable patient populations: children.
New Epilepsy Drug Is Latest Win for Pharma Company
The exciting development comes via Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company working to translate genetic insights into the development of therapies for central nervous system (CNS) disorders — particularly those disorders characterized by neuronal excitation-inhibition imbalance. Praxis has already reached significant milestones in the epilepsy space, achieving another midphase epilepsy win in March of this year. At that time, Praxis announced that a high dose of its next-generation NaV blocker PRAX-628 was linked to a complete response rate in epilepsy patients with a form of photosensitivity known as photoparoxysmal response. The EMBOLD study is just the latest in key epilepsy developments specifically targeting forms of epilepsy with limited treatment options.
A Significant Reduction in Seizures
The EMBOLD study enrolled 16 participants. All participants were between the ages of 2 and 18, and all had been diagnosed with either early-onset SCN2A-DEE or SCN8A-DEE. These are both forms of epilepsy for which there are no approved treatments at this time. Non-placebo patients received relutrigine, a sodium channel inhibitor. Inhibitors like relutrigine work by holding back sodium current, which is known to be a major driver of seizure symptoms in both SCN2A-DEE and SCN8A-DEE.
The results were stunning: Participants who received relutrigine saw an average 46 percent reduction in their seizures during the double-blind part of the study, per Praxis. The participants also saw significant improvements in “disrupted movement” and communication. Additionally, seizure severity and intensity were reduced by an average of 62 percent. In fact, five of the patients went 28 days without a single seizure.
A Continued Focus on Safety
The EMBOLD study is a Phase 2 study that evaluated safety, tolerability, efficacy, and pharmacokinetics of PRAX-562. Fortunately, Praxis reported that no patients discontinued their treatment due to an adverse event. The company noted that relutrigine was “generally safe and well tolerated,” with relatively mild adverse events, including vomiting and constipation.
_____
At this point, the drug appears to be a viable option for juvenile patients with limited epilepsy treatment options. “Seizure freedom is the ultimate goal for patients, and we were humbled by the progress made with relutrigine during the EMBOLD study with over 30 percent of patients achieving this life-altering milestone,” noted Marcio Souza, president and chief executive officer of Praxis, in a press release.
Did you enjoy this blog post? Check out our other blog posts as well as related topics on our Webinar page.
QPS is a GLP- and GCP-compliant contract research organization (CRO) delivering the highest grade of discovery, preclinical and clinical drug research development services. Since 1995, it has grown from a tiny bioanalysis shop to a full-service CRO with 1,100+ employees in the US, Europe and Asia. Today, QPS offers expanded pharmaceutical contract R&D services with special expertise in pharmacology, DMPK, toxicology, bioanalysis, translational medicine, cell therapy (including PBMCs, leukopaks and cell therapy products) and clinical development. An award-winning leader focused on bioanalytics and clinical trials, QPS is known for proven quality standards, technical expertise, a flexible approach to research, client satisfaction and turnkey laboratories and facilities. Through continual enhancements in capacities and resources, QPS stands tall in its commitment to delivering superior quality, skilled performance and trusted service to its valued customers. For more information, visit www.qps.com or email info@qps.com.
