Neurogene (NGNE) investor relations material

Key updates and clinical progress

• Completed FDA discussions and will begin dosing in the embolden registrational study this quarter.

• Head-to-head non-human primate data show ICV administration delivers 10–100x more drug to the brain than IT lumbar, with no liver-sparing benefit for IT lumbar.

• U.S. clinical trial presence more than doubled to 13 sites, supporting rapid conversion to commercial sites.

• RMAT designation and SAR pilot program participation provide regulatory pathways to expedite BLA submission.

• Ability to enroll patients as young as 3 years old, potentially capturing a broader patient population.

Competitive landscape and differentiation

• Emphasis on best-in-class efficacy and durability over speed to market, as caregivers prioritize meaningful, lasting improvements.

• Overlap exists between trial sites with competitors, but focus remains on U.S. sites for faster commercialization.

• Composite primary endpoint combines milestone gains and CGII, enhancing interpretability in a heterogeneous disease.

• Product differentiation built into trial design, including early-age enrollment and composite endpoints.

Data interpretation and regulatory perspectives

• Durability and deepening of effect over time are key for families, not just initial skill acquisition.

• Most milestones are identical between studies; depth and duration of response will drive product differentiation.

• FDA encouraged a 12-month primary endpoint for clinical meaningfulness; six-month interim analyses unlikely to confer significant approval timing advantage.

• Structured scales like R-MBA and ORCA not used; focus is on broader, clinically meaningful assessments.