Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 __________________________________ FORM 10-K (Mark One) (State or other jurisdiction of incorporation or organization) 535 W 24th St. 5th Floor New York, NY x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2025 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commis ...

NEUROGENE® every Breakthrough BEGINS WITH BELIEF Corporate Presentation March 2026 This communiciation contains forward-Colonial container of the Printed Christian (Collection Renoments of the Printed States Libration Reform Art of 1985. There statestilio or francial condition, or otherevise, based on current experations and deles of the management of Neuropene, as wells assumptions made by and ritritmation corently and driv regardnig: the therapestic polenial and dility, efficacy and clinical aerentis of i ...

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Summary of Neurogene Conference Call Company Overview - **Company**: Neurogene - **Focus**: Development of genetic medicines for neurological diseases, particularly Rett syndrome [2][3] Core Points and Arguments - **Lead Program**: NGN-401 for Rett syndrome, a severe disorder primarily affecting girls, with a market opportunity estimated in the billions, targeting 15,000-20,000 patients in the U.S. and Europe [3][4] - **Clinical Trials**: The Phase 3 trial, named Embolden, is actively enrolling patients aged 3 and older who have completed the regression phase of the disease, with dosing expected to complete in Q2 of the current year [3][4] - **Regulatory Interaction**: Neurogene has regular interactions with the FDA, participating in the START Pilot program, which provides significant access and feedback on trial design [7][8] - **Trial Design**: The Embolden trial was designed in close collaboration with the FDA, incorporating feedback from multiple meetings to ensure alignment on key elements [8][9] - **Efficacy Data**: In the Phase 1/2 trial, 88% of participants showed improvement on the CGI-I score, with an average of 4 developmental milestones gained per participant [23][24] - **Safety Profile**: A hyper-inflammatory reaction was observed at a higher dose, leading to a reduction in dosage. Robust monitoring procedures are in place to manage potential adverse events [31][32] Important but Overlooked Content - **Statistical Success Threshold**: A 35% threshold for success has been established based on natural history data, requiring 7 out of 20 participants to be considered responders for statistical success [35] - **Market Dynamics**: The market for Rett syndrome treatments is large enough to accommodate multiple players, with Neurogene positioning NGN-401 as a best-in-class gene therapy product [38][39] - **Financial Position**: Neurogene has sufficient capital to fund key clinical and regulatory milestones through Q1 2028, ensuring a runway for upcoming catalysts [41]

Neurogene (NasdaqGM:NGNE) 2026 Conference March 09, 2026 01:40 PM ET Company ParticipantsRachel McMinn - Founder and CEOConference Call ParticipantsMarc Frahm - Managing Director and Senior Equity AnalystMarc FrahmWelcome back to our afternoon session of the first day of the Leerink Partners Global Healthcare Conference here in Miami. I'm still Marc Frahm, Senior Analyst at TD Cowen, and I have with me Rachel McMinn, CEO of Neurogene. Rachel, how are you doing?Rachel McMinnI'm good. I'm good. Thank you for ...

Neurogene FY Conference Summary Company Overview - **Company**: Neurogene (NasdaqGM:NGNE) - **Focus**: Development of genetic medicines for neurological diseases, specifically targeting Rett syndrome with their lead program NGN-401 [1][2] Core Points and Arguments Rett Syndrome and Market Opportunity - Rett syndrome is a severe neurological disorder primarily affecting girls, with an estimated 15,000-20,000 patients in the U.S. and Europe, representing a multi-billion dollar market opportunity for genetic treatments [3] - The company is actively progressing towards the commercialization of NGN-401, with ongoing Phase 3 trial (Embolden) enrollment expected to complete in Q2 2026 [3][4] EXACT Platform - The EXACT platform is designed to control gene expression levels, specifically the MECP2 transgene, which is crucial for preventing Rett syndrome [5][6] - The platform includes a microRNA-based safety valve to regulate transgene expression, avoiding toxic levels [6] Breakthrough Therapy Designation - Neurogene received Breakthrough Therapy designation from the FDA based on encouraging Phase 1-2 trial results, including new patient-level data and video evidence demonstrating skill gains [4][8][9] Clinical Trial Insights - The primary endpoint of the Embolden trial includes video-based documentation of skill gains and developmental milestones [16] - The Phase 1-2 trial showed a time-dependent improvement in function, with patients gaining skills over time rather than just stabilizing [18][27] - The trial has reported an increase in developmental milestones from 23 to 35 across 8 patients, indicating a deepening effect over time [61][172] Safety and Monitoring - The company has implemented safety protocols, including monitoring for HLH (Hemophagocytic Lymphohistiocytosis), which was previously observed at higher doses but has not occurred at current dosing levels [180][185] - No cases of HLH have been reported in the ongoing trials, indicating effective safety measures [185] Regulatory Considerations - Neurogene emphasizes the importance of a 12-month primary endpoint for the Embolden study, arguing that the FDA and caregivers prefer more data for assessing long-term benefits [192] - The company believes that a 12-month assessment is necessary for understanding the benefit-risk profile of gene therapies for Rett syndrome [192] Other Important Content - The company is focused on establishing a commercialization pathway and engaging with payers to support the launch of NGN-401 [4] - The commitment of families involved in the trial is highlighted, as they are already engaged in physical and occupational therapies to maximize outcomes for their children [95] - The company is optimistic about the long-term data from the trial, which will be valuable for various stakeholders, including caregivers, payers, and regulators [112][188] This summary encapsulates the key points discussed during the Neurogene FY Conference, focusing on the company's advancements in gene therapy for Rett syndrome, the significance of their EXACT platform, and the ongoing clinical trials.

Core Insights - Premarket trading is showing notable activity with significant price movements indicating potential investment opportunities before the market opens [1] Premarket Gainers - Neurogene Inc. (NGNE) is up 34% at $26.42 [3] - Cardio Diagnostics Holdings, Inc. (CDIO) is up 20% at $6.29 [3] - Applied Optoelectronics, Inc. (AAOI) is up 19% at $64.00 [3] - Caris Life Sciences, Inc. (CAI) is up 19% at $22.93 [3] - Block, Inc. (XYZ) is up 18% at $64.59 [3] - MARA Holdings, Inc. (MARA) is up 16% at $9.84 [3] - NCR Atleos Corporation (NATL) is up 14% at $48.13 [3] - Birchtech Corp. (BCHT) is up 12% at $2.70 [3] - The RealReal, Inc. (REAL) is up 11% at $13.80 [3] - Candel Therapeutics, Inc. (CADL) is up 10% at $5.68 [3] Premarket Losers - Duolingo, Inc. (DUOL) is down 24% at $88.30 [4] - Xponential Fitness, Inc. (XPOF) is down 23% at $6.16 [4] - Emergent BioSolutions Inc. (EBS) is down 22% at $8.58 [4] - Flutter Entertainment plc (FLUT) is down 14% at $105.85 [4] - Ginkgo Bioworks Holdings, Inc. (DNA) is down 14% at $8.27 [4] - Willdan Group, Inc. (WLDN) is down 12% at $104.63 [4] - Duos Technologies Group, Inc. (DUOT) is down 12% at $7.67 [4] - CoreWeave, Inc. (CRWV) is down 11% at $85.96 [4] - Zscaler, Inc. (ZS) is down 9% at $152.22 [4] - Progyny, Inc. (PGNY) is down 8% at $20.40 [4]

Neurogene (NasdaqGM:NGNE) 2026 Conference February 12, 2026 09:00 AM ET Company ParticipantsChristine Mikail - President and CFOConference Call ParticipantsDebjit - Therapeutic AnalystDebjitMorning. Thank you for joining Guggenheim's 2026 Emerging Outlook Biotech Summit. I'm Debjit, one of the therapeutic analysts, and my next presenting company is Neurogene. From Neurogene, we have Christine Mikail, President and CFO.Christine MikailGood morning.DebjitThank you for joining us.Christine MikailGood morning, ...

Core Viewpoint - Neurogene Inc. is focused on developing genetic medicines for patients with rare neurological diseases and is actively engaging with investors through upcoming conferences [1] Group 1: Company Participation - Company management will participate in the Guggenheim Emerging Outlook: Biotech Summit, which includes a fireside chat and investor meetings [1] - The date for the Guggenheim conference is set for Thursday, February 12 at 9:00 a.m. ET [1] - Additionally, the company will be present at the TD Cowen 46th Annual Health Care Conference [1]

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].