Praxis Precision Medicines Inc.

04/06/2026 | Press release | Distributed by Public on 04/06/2026 14:24

Material Event (Form 8-K)

Item 8.01. Other Events.
Praxis Precision Medicines, Inc. (the "Company") is providing the following business updates:
Elsunersen
The Company announced positive topline results from the EMBRAVE Part A trial of elsunersen in pediatric patients with early-seizure onset SCN2A development and epileptic encephalopathy ("DEE"). EMBRAVE Part A is a randomized, placebo-controlled Phase 1/2 trial evaluating the safety and efficacy of ascending doses of elsunersen in patients with SCN2A DEE. Nine patients, aged 2-12 years old, were randomized 3:1 to receive either elsunersen or sham procedure every 4 weeks for 24 weeks, followed by an open-label extension ("OLE"); all 9 patients continued to the OLE. Patients received a starting dose of 1 mg with optional dose escalation based on observed seizure reduction and individual tolerability.
Key results from EMBRAVE Part A
Efficacy
•Elsunersen treatment led to a 77% placebo-adjusted seizure reduction from baseline (p=0.015, 95 CI [33,92]).
•57% of patients had at least a 28-day period of seizure freedom.
•Efficacy was sustained in the OLE for up to one year.
•100% of elsunersen patients improved across sleep, motor function, muscle tone, attention or neuropsychomotor development compared to no improvements in placebo group.
Safety
•Elsunersen was well-tolerated, with no drug-related serious adverse events, no discontinuations and no neuroinflammation signals at doses up to 8 mg.
•Most treatment-emergent adverse events ("TEAEs") were mild to moderate. TEAEs were consistent with the EMBRAVE Part 1 study.
Relutrigine
The U.S. Food and Drug Administration (the "FDA") has accepted for priority review the Company's New Drug Application for relutrigine for the treatment of SCN2A and SCN8A DEEs. The FDA has set a target action date under the Prescription Drug User Fee Act of September 27, 2026.
Praxis Precision Medicines Inc. published this content on April 06, 2026, and is solely responsible for the information contained herein. Distributed via EDGAR on April 06, 2026 at 20:24 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]