Neurogene advances Rett therapy to 90% enrollment in pivotal trial, secures FDA Breakthrough

Filed May 12, 2026 · Period ending March 31, 2026 · Compared to 10-Q May 9, 2025 · ~2 min read

Key Changes

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  NGN-401 pivotal trial (Embolden) reached 90% enrollment with dosing on track to complete Q2 2026; FDA Breakthrough designation received February 2026, enabling Priority Review and rolling BLA submission.

  MD&A: Clinical Development verify on EDGAR →
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  Phase 1/2 data showed 80% of patients (4 of 5) met pivotal trial's primary endpoint at 12 months, with 35 developmental milestones gained across all pediatric participants regardless of baseline severity.

  MD&A: Clinical Results verify on EDGAR →
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  Quarterly net loss rose 37% to $30.9M (Q1 2026 vs Q1 2025) driven by $5.1M higher clinical trial costs and $2.4M higher manufacturing expenses; cash runway extended to Q1 2028 despite $49M cash decline.

  MD&A: Financial Results verify on EDGAR →