Neurogene Inc. announced details of Embolden?, the Company's registrational clinical trial designed to evaluate NGN-401 gene therapy for the treatment of females with Rett syndrome in participants ages 3 years and older. The Company has written agreement from the U.S. Food and Drug Administration (FDA) on key aspects of the registrational trial's design. Ob obtained Written Agreement from FDA on the Following Key Elements of Embolden Registrational Trial Design After Discussions Under the START Program: Study Design: Open-label, single arm, baseline control (i.e., participantsserving as their own control); N=18 proposed, subject to feedback from the FDA.
Developmental milestones/skills were informed by a Rett syndrome caregiver survey, provided to the FDA, demonstrating that the acquisition of any single skill within the selected set is considered clinically meaningful. The primary endpoint will be evaluated at 12 months following NGN-401 administration, with feedback pending from the FDA to consider the addition of a 6-month endpoint. Efficacy data from the first four participants; as of data cut-off date of October 17, 2024.
Neurogene previously shared that five additional participants had been dosed in the Phase 1/2 portion of the NGN-401 clinical trial during the first half of 2025. NGN-401 was selected by the U.S. Food & Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA. Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology.
These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: trial designs, clinical development plans and timing for NGN-401 administration, With feedback pending from the FDA to considered the addition of a 6- month endpoint. Efficacy datafrom the first four participants; as the first four participants; asof data cut-off date of September 17, 2024. Neurog gene therapy is expected to be evaluated in the Phase 1/2 trial.
