NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the following conferences: Guggenheim Emerging Outlook: Biotech Summit 2026 Format: Management will participate in a fireside chat and investor meetings Date: Thursday, February 12 at 9:00 a.m. ET TD Cowen 46th Annual Health Care Conference F. ...

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].

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]

EVERY BREAKTHROUGH BEGINS WITH BELIEF January 2026 Corporate Presentation Disclaimer Forward Looking Statements This communication contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and ...

Group 1: Market Activity - Several biotech and healthcare companies experienced notable gains in after-hours trading, indicating renewed investor interest in drug development and healthcare services [1] - Zentalis Pharmaceuticals, Inc. (ZNTL) surged 17.65% to close at $4.40, attributed to a recent corporate update that boosted investor confidence in its azenosertib development program [2] - LifeMD, Inc. (LFMD) climbed 12.18% to $4.33 following the announcement of a new senior secured revolving credit facility with Citizens Bank, providing up to $50 million in total availability [3] Group 2: Company-Specific Developments - NanoViricides, Inc. (NNVC) advanced 11.11% to $1.24, driven by speculative interest despite no recent news [4] - Intelligent Bio Solutions Inc. (INBS) posted a 9.10% increase, closing at $19.55, continuing to gain momentum from overall investor enthusiasm in the biotech sector [4] - IO Biotech, Inc. (IOBT) rose 6.49% to $0.60, suggesting bargain-hunting activity at lower price levels [5] Group 3: Financing and Agreements - China SXT Pharmaceuticals, Inc. (SXTC) advanced 2.06% to $18.29 after disclosing a definitive agreement for the purchase of 66,666,666 Class A ordinary shares at $0.15 per share, highlighting efforts to strengthen its capital position [6]

Group 1 - The biotech industry is characterized by lengthy processes where companies incur significant costs while awaiting results from drug candidates, which often leads to binary outcomes [1] - There is a strong inclination towards investing in high-growth companies within sectors that are expected to experience exponential growth [1] - The focus is on understanding and investing in disruptive technologies and innovative enterprises, combining fundamental analysis with future trend predictions [1] Group 2 - The belief in the potential of innovation to generate substantial returns is emphasized, with a commitment to providing insightful analysis on relevant companies [1]

Clinical Trials and Efficacy - NGN-401 for Rett syndrome demonstrated promising interim clinical data, with the first participant dosed in Q4 2025 and enrollment expected to complete in 3-6 months[8]. - The Embolden™ registrational trial for NGN-401 is designed as a single-arm, open-label trial with a sample size of 20 females, requiring a 35% response rate for success[28]. - The primary endpoint of the Embolden trial is a responder-based composite endpoint, with key secondary endpoints focusing on developmental milestones[28]. - The trial's primary analysis will occur at 12 months, assessing the CGI-I score and developmental milestones gained[30]. - 12 of 13 clinical trial sites for the Embolden trial have been initiated, with enrollment expected to complete in 3-6 months[44]. - 5 out of 20 participants (25%) achieved the minimum threshold for success in the Embolden trial, with a CGI-I score of < 3 at 12 months[51]. - Participants dosed in the first half of 2025 show early efficacy, with 3 of 3 demonstrating functional gains at 6 months[54]. - The primary endpoint of the Embolden trial is focused on the gain of any one developmental milestone/skill at 12 months[54]. - NGN-401 demonstrated efficacy and safety in Mecp2 mouse models, with survival rates showing significant improvement[94]. - The company plans to announce additional Phase 1/2 clinical data in 2H 2025 and in 2026[90]. Manufacturing and Operational Capacity - Neurogene's internal manufacturing capabilities are designed to support commercial-grade products, avoiding potential future comparability challenges[14]. - Neurogene's facility in Houston spans 42,000 sq ft, including 6,000 sq ft of cleanrooms, enhancing its manufacturing capabilities[13]. - Cash runway is expected to fund operations through Q1 2028, providing strategic flexibility for ongoing and future projects[8]. - Cash runway is projected to fund operations through Q1 2028[90]. Patient Impact and Caregiver Insights - The U.S. prevalence of Rett syndrome is estimated to be between 6,000 and 9,000 patients, with a worldwide incidence of 1 in 10,000 females, indicating a significant unmet need for new treatment options[21]. - Significant improvements in communication and motor skills have been reported by caregivers, enhancing quality of life[85]. - Caregivers indicated that any skill gain or symptom improvement would provide significant respite and independence for their daughters with Rett syndrome[93]. - The majority of caregivers considered all queried skills in developmental milestones meaningful, highlighting the immense burden of care required[93]. Safety and Tolerability - NGN-401 remains generally well tolerated at the 1E15 vg dose level, with 9 treatment-emergent adverse events related to NGN-401 reported[86]. - All treatment emergent adverse events (TEAEs) related to NGN-401 have been Grade 1 (mild) or Grade 2 (moderate) in severity, with most participants experiencing mild liver enzyme elevations[87]. - Seizures have remained well controlled following the administration of NGN-401[87]. - No evidence of hemophagocytic lymphohistiocytosis (HLH) was found in any participant during the trials[87]. Future Directions - The company is evaluating opportunities for NGN-101 targeting CLN5 Batten Disease, expanding its clinical pipeline[9]. - The trial design for ages over 3 is expected to enable the most efficient path to market for NGN-401[44]. - The company aims to deliver durable, multidomain gains that grow over time with NGN-401[45]. - The Embolden registrational trial will expand the clinical trial footprint, more than doubling the presence in the U.S.[90].

Core Insights - Neurogene reported positive interim data from the NGN-401 Phase 1/2 gene therapy trial for Rett syndrome, showing multidomain, durable gains with 35 developmental milestones acquired across eight participants [1][5] - The first participant has been dosed in the Embolden registrational trial of NGN-401, with complete enrollment expected in three to six months [1][2] - The company maintains a strong cash position, with $265.4 million in cash and equivalents, providing a runway through the first quarter of 2028 [11] Clinical Development Highlights - The NGN-401 gene therapy program for Rett syndrome has advanced significantly, with 12 of 13 trial sites initiated [2][5] - Interim clinical data from the pediatric cohort (ages 4-10) demonstrated functional improvements across all participants, with gains in hand function, language, and ambulation [5] - The therapy has been well-tolerated in both pediatric and adolescent/adult cohorts, with no significant adverse effects reported [5] Financial Results - For the third quarter of 2025, R&D expenses were $17.2 million, up from $16.3 million in the same period in 2024, primarily due to increased clinical trial costs [11] - General and administrative expenses rose to $6.6 million from $5.9 million year-over-year, driven by higher employee-related costs [11] - The net loss for the quarter was $21.0 million, compared to $20.2 million in the prior year [11] Regulatory Designations - NGN-401 has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designation, indicating its potential as a significant treatment for Rett syndrome [8]

Financial Performance - The company reported a net loss of $65.6 million for the nine months ended September 30, 2025, compared to a net loss of $55.6 million for the same period in 2024, indicating an increase in losses year-over-year [129]. - As of September 30, 2025, the company had an accumulated deficit of $327.9 million and cash, cash equivalents, and short-term investments totaling $265.4 million [129]. - The net loss for Q3 2025 was $20.953 million, compared to a net loss of $20.217 million in Q3 2024, indicating a 3.6% increase in losses [148]. - For the nine months ended September 30, 2025, the company used $59.7 million in cash for operating activities, reflecting a net loss of $65.6 million [174]. - For the nine months ended September 30, 2024, the company used $53.1 million in cash for operating activities, reflecting a net loss of $55.6 million [175]. - Interest income for Q3 2025 was $2.739 million, an increase of 48.7% from $1.842 million in Q3 2024 [148]. - Other income increased by $1.1 million for the nine months ended September 30, 2025, mainly from state tax refunds [163]. - The company reported net cash provided by financing activities of $9.8 million for the nine months ended September 30, 2025, primarily from open market sales of common stock [178]. Research and Development - The company is developing NGN-401 for the treatment of Rett syndrome, with a registrational trial named Embolden currently evaluating a 1E15 vg dose in 20 female participants [107]. - The primary endpoint for the Embolden trial is a responder-based composite endpoint, with a minimum success threshold of 35% response rate (7 out of 20 patients) to reject the null hypothesis [109]. - Updated interim data from the Phase 1/2 trial showed that all pediatric participants experienced functional gains, with a total of 35 developmental milestones/skills gained across core clinical domains [111]. - The company completed enrollment in a Phase 1/2 clinical trial for NGN-101 in patients with CLN5 Batten disease but decided not to move forward with the program due to the denial of its RMAT application [125]. - The company is in the early discovery stage for additional potential programs utilizing its EXACT platform for complex neurological diseases [124]. - The company expects significant increases in research and development expenses over the next several years due to expanded clinical trials and personnel costs [139]. - Research and development expenses for Q3 2025 were $17.241 million, an increase of 5.9% from $16.285 million in Q3 2024 [148]. - For the nine months ended September 30, 2025, research and development expenses totaled $54.4 million, up 19.3% from $45.6 million in the same period of 2024 [153]. - The increase in Rett syndrome program expenses included a $2.1 million rise in preclinical costs and a $4.0 million increase in clinical trial costs for NGN-401 [154]. - The company anticipates continued increases in research and development and general and administrative expenses as it advances clinical development [156][159]. Operating Expenses - General and administrative expenses for Q3 2025 were $6.557 million, up 11.2% from $5.895 million in Q3 2024 [148]. - For the nine months ended September 30, 2025, general and administrative expenses reached $21.4 million, up 30.2% from $16.4 million in the same period of 2024 [158]. - Total operating expenses for the nine months ended September 30, 2025, were $75.799 million, compared to $62.018 million for the same period in 2024, reflecting a 22.2% increase [148]. Capital and Funding - The company has raised aggregate net proceeds of approximately $552.3 million since its inception, primarily through the sale of preferred and common stock [129]. - The company expects to require substantial additional capital to support ongoing operations and product development [168]. - The company has federal and state net operating loss carryforwards of $319.8 million and $39.6 million, respectively, which may offset future taxable income [146]. Regulatory and Compliance - The company received FDA clearance for its Investigational New Drug (IND) application for NGN-401 in January 2023 [108]. - The collaboration agreement with the University of Edinburgh was amended and extended through December 2026 to support pipeline development [132]. - The company has no products approved for sale and may never achieve regulatory and marketing approval for its product candidates [130]. - The company has no off-balance sheet arrangements as defined by SEC rules [195]. - The company’s financial statements are prepared in accordance with U.S. GAAP, with no material changes to significant accounting policies during the nine months ended September 30, 2025 [196]. Facilities and Lease Obligations - The company established a fully operational cGMP facility in Houston, Texas, to manufacture products for research and clinical studies, enhancing control over product quality and development timelines [126]. - The company has a lease obligation for its New York headquarters expiring in January 2028, covering approximately 6,000 square feet [181]. - The company has a lease for a manufacturing facility in Houston, Texas, expiring in August 2029, with an option to renew for two additional five-year terms [182]. - The company entered into a License Agreement with the University of Edinburgh, which includes obligations of up to $5.3 million in regulatory-related milestones and up to $25 million in sales-related milestones [191]. Contingent Liabilities - The total contingent value rights (CVR) liability as of September 30, 2025, was $1.1 million, compared to $1.8 million as of December 31, 2024 [188].

Core Insights - Neurogene has presented new interim clinical data for NGN-401, a gene therapy aimed at treating Rett syndrome, indicating strong potential for being both best-in-class and first-in-class [2][3] - The company emphasizes the necessity for gene therapy to achieve multiple developmental milestones and skill gains over time, rather than focusing on a single skill improvement [3] Clinical Data - The updated data from the Phase I/II trial of NGN-401 shows promising results, reinforcing the belief that it sets a new standard for gene therapy in Rett syndrome [3] - Key areas of improvement highlighted include hand function, gross motor function, and communication, with an emphasis on the durability of these skills over time [3]