Neurogene (NGNE) Stifel 2026 Virtual CNS Forum summary

Company and program overview

• Focuses on genetic medicines for severe neurological diseases using a biology-first approach.

• Lead program NGN-401 targets Rett syndrome, a disorder with high unmet need and a multi-billion dollar market opportunity.

• Embolden, the phase 3 registrational trial, is enrolling females 3+ years post-regression, with dosing completion expected in Q2 this year.

Clinical data and milestones

• NGN-401 is considered de-risked due to strong phase 1/2 data and recent FDA Breakthrough Therapy designation.

• Additional phase 1/2 data with at least 12 months follow-up will be presented mid-year.

• All phase 1/2 participants showed functional improvement, with an average of 4 developmental milestones gained per patient.

• No cases of HLH or severe inflammatory reactions have been observed at the current dose.

Regulatory and FDA engagement

• Embolden trial design was closely aligned with FDA through multiple meetings and administrations.

• The study uses a traditional FDA endpoint based on gain in function, not surrogate markers.

• Ongoing, high-level engagement with FDA and participation in the START Pilot program provide regulatory advantages.

• The review team has remained consistent across FDA administrations, supporting regulatory continuity.