Neurogene (NasdaqGM:NGNE) FY Conference Transcript

Neurogene FY Conference Summary Company Overview - Company: Neurogene (NasdaqGM:NGNE) - Focus: Development of gene therapies for neurological diseases, specifically targeting Rett syndrome Key Industry Insights - Rett Syndrome: A rare neurological disorder affecting primarily females, characterized by developmental delays and loss of previously acquired skills. Estimated prevalence is 15,000-20,000 patients in major markets with an incidence rate of 1 in 10,000 females worldwide [7][24]. - Market Opportunity: The gene therapy market for Rett syndrome is projected to be multi-billion dollars, with no current disease-modifying treatments available [5][7]. Core Company Insights - Lead Program: NGN401 for Rett syndrome, currently in the Embolden registrational trial aimed at FDA approval [4][5]. - Clinical Progress: - Dosing of patients commenced in Q4 2025, following alignment with the FDA on trial design [4]. - Interim data from phase 1/2 trial shows 100% of participants achieved functional improvements in key domains (hand function, gross motor function, communication) [9][10]. - 35 developmental milestones gained, with improvements durable for up to 24 months [9][10]. Clinical Trial Details - Embolden Trial: - Single-arm, baseline-controlled, open-label trial evaluating NGN401 in 20 females aged three years and older [15]. - Primary endpoint: Composite of Clinician Global Impression of Improvement (CGII) and developmental milestones, measured at 12 months [15]. - Anticipated completion of enrollment and dosing by Q2 2026 [5][29]. Financial and Commercial Readiness - Financial Position: Strong balance sheet expected to fund operations through Q1 2028, covering key milestones including BLA submission [19]. - Commercial Strategy: - Plans to convert clinical trial sites into commercial sites post-approval [18][32]. - Integrated manufacturing capabilities established for commercial product production [18][32]. - Strong reimbursement potential confirmed through payer research [18][26]. Future Outlook - Upcoming Milestones: - Completion of Embolden trial dosing in Q2 2026 [19][29]. - Presentation of new clinical data mid-2026, including 12 months of follow-up data for all participants [19][31]. - Market Positioning: Neurogene aims to establish itself as a leader in the Rett syndrome treatment landscape, with expectations of multiple gene therapy products entering the market [24][27]. Additional Insights - Patient and Caregiver Impact: Emphasis on improving daily living activities and reducing caregiver burden through meaningful functional changes [10][11][36]. - Community Enthusiasm: High levels of interest from physicians and caregivers regarding NGN401 and gene therapy options for Rett syndrome [29][30]. Conclusion - Neurogene is positioned for significant advancements in the treatment of Rett syndrome, with a clear regulatory path, strong clinical data, and a robust commercial strategy aimed at transforming the lives of affected families [20][36].