Praxis Precision Medicines Announces Positive Results from Preliminary Analysis of PRAX-222 Part 1 for SCN2A-Gain-of-Function Pediatric Patients

Praxis Precision Medicines, a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders, has shared positive preliminary results from the analysis of PRAX-222 Part 1. The study showed a promising 44% median reduction in seizures after three doses for pediatric patients with SCN2A-gain-of-function developmental epilepsies and encephalopathies (DEEs).

The PRAX-222 program aims to address the underlying cause of early-seizure-onset SCN2A-DEE by selectively decreasing SCN2A gene expression. The treatment, an antisense oligonucleotide (ASO) called PRAX-222, has demonstrated significant reduction in seizures, improvement in behavioral and locomotor activity, and increased survival in SCN2A mouse models. If successful, PRAX-222 could become the first disease-modifying treatment for SCN2A-DEE.

Praxis is also preparing to initiate its Essential3 program, which focuses on essential tremor, this quarter. The program will evaluate ulixacaltamide as a potential treatment, with the primary endpoint being the modified Essential Tremor Rating Assessment Scale (mADL11). The company has already received significant interest from nearly 600 patients who are eager to participate in the clinical trials.

Marcio Souza, the President and CEO of Praxis Precision Medicines, expressed excitement about the early efficacy data from the PRAX-222 program and the positive response from the community. He emphasized the company’s commitment to translating genetic insights into the development of therapies for CNS disorders.

Praxis Precision Medicines’ R&D Day, where these updates were shared, featured two esteemed key opinion leaders in movement disorders and epilepsy: Alberto J. Espay, M.D., and Jacqueline French, M.D. They provided valuable insights and expertise in their respective fields.

The company expects to announce topline results from Part 1 of the EMBRAVE study, which evaluates the safety and tolerability of PRAX-222 in pediatric patients with SCN2A-DEE, in the fourth quarter of 2023.

For more information about Praxis Precision Medicines and its clinical portfolio, visit their website. A replay of the R&D Day webcast is also available on the company’s website for the next 90 days.