Praxis Precision achieves alignment on elsunersen pathway following FDA meeting

Praxis Precision (PRAX) announced the completion of a Type C meeting with the FDA and agreement to immediately convert the EMBRAVE3 registrational study of elsunersen in early-onset SCN2A developmental and epileptic encephalopathy into a single-arm study where all patients will receive elsunersen for 24 weeks, followed by an open-label extension. The current study has been immediately converted from a double-blind, sham-controlled study to a single-arm, baseline-controlled study, enrolling 30 patients reduced from 40 patients. All patients currently in screening will be assigned to receive elsunersen. The primary analysis will be the change from baseline in countable motor seizures. The EMBRAVE Study Part A enrolled 9 patients randomized 3:1 to elsunersen or placebo/sham for 20 weeks, followed by a blinded transition to elsunersen for up to two years in an open-label extension. Praxis expects to complete Part A and disclose the topline results in the first half of 2026. “This alignment with the FDA represents a meaningful step forward for patients and families living with SCN2A-DEE. The Agency’s recognition of both the urgency of the unmet need and the strong mechanistic rationale for elsunersen enables us to move with greater clarity and speed. Converting EMBRAVE3 to a single-arm, baseline-controlled study ensures that every child entering the trial will receive active treatment from day one, while preserving a rigorous and approvable pathway. Momentum in enrollment continues to build, and we remain focused on generating the evidence needed to bring the first targeted therapy for SCN2A gain-of-function disease to patients as quickly as possible,” said Marcio Souza, president and chief executive officer.