Neurogene (NasdaqGM:NGNE) FY Earnings Call Presentation

NGN-401 Clinical Development and Trial Design - NGN-401 is being developed as a potential one-time, best-in-class gene therapy for Rett syndrome, showing durable multidomain improvements across the disease spectrum[8] - The Embolden registrational trial, aligned with the FDA, is a single trial for ages ≥ 3 years, with multiple participants dosed in 4Q'25[8] - The Embolden trial is a single-arm, baseline-controlled, open-label trial of NGN-401 in females with Rett Syndrome, using a one-time 1E15 vg dose in patients ≥ 3 years[30, 31] - The primary endpoint for the Embolden trial at 12 months is a responder-based composite endpoint, requiring a 35% response rate (7 of 20 participants) for success[32] Market Opportunity and Commercial Readiness - Rett syndrome presents a multi-billion-dollar market opportunity for disease-modifying gene therapy, with an estimated prevalence of ~15,000 – 20,000 patients in major markets (US, EU, and UK)[9, 13] - Payors are receptive to reimbursement for Rett syndrome treatments, valuing functional changes that are clinically meaningful and show improvements in activities of daily living[13] - Early commercial-readiness activities are underway, including positioning Embolden clinical trial sites for rapid conversion to commercial sites at launch[9, 42] Phase 1/2 Clinical Trial Data - Interim Phase 1/2 data showed 100% of participants demonstrated functional improvements across core disease domains (fine motor/hand function, gross motor/ambulation, and communication)[24] - A total of 35 developmental milestones were gained in the Phase 1/2 trial, with no plateau observed out to 24 months[24] - In the Phase 1/2 trial, 88% of participants achieved an improved CGI-I score[24] Upcoming Milestones and Financial Position - Complete dosing of the Embolden trial is expected in 2Q'26, with presentation of 12+ months Phase 1/2 data for all 10 participants planned for mid-2026[10] - The company has a strong cash balance expected to fund operations through Embolden data readout, BLA submission, and key pre-launch activities (as of 1Q'28)[49]