Neurogene Announces Successful Completion of Dosing in Embolden Registrational Trial of NGN-401 for Rett Syndrome

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the successful completion of dosing in the Embolden registrational trial of NGN-401, an investigational gene therapy designed to be a potential best-in-class, one-time treatment for Rett syndrome. While the initial enrollment target was 20 participants, the statistical analysis plan pre-specified an intent-to-treat (ITT) population of up to 24 participants, providing flexibility to support robust trial execution. Due to strong demand from the Rett syndrome community and to ensure timely completion of dosing, the Company elected to overenroll the trial and dose all eligible participants already in screening for a total of 25.

“We are pleased to have completed dosing in the Embolden trial of NGN-401 within our original timeline while exceeding our enrollment target, reflecting both the significant unmet need and strong demand from the Rett syndrome community,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “This milestone brings us one step closer to delivering a potential best-in-class, one-time treatment for Rett syndrome. We are deeply grateful to the participants, their families and the investigators for their trust, partnership and ongoing support of the development of NGN-401. We look forward to sharing topline results from Embolden in the second half of 2027 and advancing NGN-401 towards a planned BLA submission.”

Embolden is a multi-center, single-arm, open-label, baseline-controlled registrational trial designed to evaluate the efficacy, safety and tolerability of a one-time dose of NGN-401 (1E15 vg) in females with Rett syndrome ages three and older. The primary endpoint is a composite of a Clinical Global Impression-Improvement (CGI-I) score of ≤ 3 and a gain from baseline of any one developmental milestone from a pre-specified list.

The primary analysis to support the planned Biologics License Application (BLA) submission is expected to occur after the first 24 participants (pre-specified ITT) have completed 12 months of follow-up, with topline data expected in the second half of 2027. The threshold for success required for the registrational trial is a 33% (or 8 of 24 participants) minimum response rate. Data from the additional participant is expected to supplement the overall safety and durability dataset.

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