Neurogene advances NGN-401 to Phase 3 with FDA Breakthrough designation; NGN-101 discontinued

Filed March 23, 2026 · Period ending December 31, 2025 · Compared to 10-K Mar 23, 2025 · ~2 min read

Key Changes

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  NGN-401 received FDA Breakthrough Therapy designation in Feb 2026 based on Phase 1/2 data showing 4 of 5 patients with 12-month follow-up met registrational trial's responder definition; enables Priority Review and rolling BLA submission.

  Business: Breakthrough Therapy designation verify on EDGAR →
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  Embolden Phase 3 trial fully enrolled (20 patients), >50% dosed, with dosing completion expected Q2 2026; primary endpoint requires 35% responder rate (7 of 20 patients) to reject null hypothesis.

  Business: Embolden trial enrollment verify on EDGAR →
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  Net loss increased 20% to $90.4M in FY2025 (vs $75.1M in FY2024) driven by higher R&D spend on Embolden trial; cash position declined to $269M despite $200M Nov 2024 raise and $30M ATM sales.

  MD&A: Financial performance verify on EDGAR →