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Praxis Precision Medicines Announces Alignment with FDA on Simplified and Accelerated Registrational Pathway for Elsunersen in Early Onset SCN2A Developmental and Epileptic Encephalopathy
Clear recognition of high unmet need and urgency for the SCN2A community and plausibility of the mechanism of elsunersen FDA agreed to proposed changes to the
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"Clear recognition of high unmet need and urgency for the SCN2A community and plausibility of the mechanism of elsunersen FDA agreed to proposed changes to the EMBRAVE3 trial design to be a single-arm, baseline-controlled study Enrollment in EMBRAVE3 is quickly accelerating and topline results expected in 2026 Topline results from ongoing EMBRAVE study (Part A, n=9) expected in 1H 2026 BOSTON, Dec. 09, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced the completion of a Type C meeting with the U.S. Food and Drug Administration (FDA) and agreement to immediately convert the EMBRAVE3 registrational study of elsunersen in early-onset SCN2A developmental and epileptic encephalopathy (DEE) into a single-arm study where all patients will receive elsunersen for 24 weeks, followed by an open-label extension. Key Changes to EMBRAVE3 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. Update on EMBRAVE Study status 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 2 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 S...