Praxis Precision Medicines has released the latest topline data from its Phase 2 EMBOLD study, focusing on the effectiveness of relutrigine in patients with SCN2A and SCN8A developmental and epileptic encephalopathy. This study included participants aged between 2 and 18 who were diagnosed with early-onset developmental or epileptic encephalopathy.
In the trial, sixteen patients were randomized to receive the drug, and another thirteen opted to continue into an open-label extension. The results showed promising outcomes, with patients on the drug experiencing a 46% reduction in countable motor seizures. Additionally, caregivers and clinicians reported improvements in various behavioral and physiological aspects such as communication, alertness, and the severity and intensity of seizures.
The open-label extension arm of the study included eight patients who completed at least 28 days of the treatment. These participants showed a median reduction in seizures of 75%. Notably, five of these patients went through a 28-day period without experiencing any seizures, in contrast to none in the placebo group.
In terms of safety, the most frequent adverse events reported were infections, vomiting, and constipation. However, none of the patients discontinued treatment due to these adverse events, suggesting that the drug was generally well-tolerated.
During an investor call, Praxis president and CEO Marcio Souza highlighted the significance of these results. He stated that the outcomes have encouraged the company to initiate an expanded registration cohort. This step aims to further validate the benefits of the drug and support its path toward regulatory approval.
Souza also discussed the financial implications of relutrigine, projecting that the drug could generate more than $500 million in peak revenue potential in the United States alone, given that there are approximately 5,000 potential patients and currently no approved treatments. The company plans to meet with regulatory authorities by the start of next year to discuss the next steps.
Earlier in the year, Praxis had announced positive topline data from a Phase 2a study involving photo-sensitive epilepsy patients. Building on this success, the company has outlined plans to initiate a study on the efficacy of PRAX-628 for focal epilepsy in the latter half of this year. Additionally, Praxis is advancing another epilepsy treatment candidate, PRAX-222, which is currently in Phase 1 of its development.
These developments underscore Praxis Precision Medicines' commitment to advancing innovative treatments for epilepsy and related neurological disorders, driving them closer to providing effective therapeutic options for patients in need.
How to obtain the latest research advancements in the field of biopharmaceuticals?
In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!