Neurogene Reports Second Quarter 2024 Financial Results and Highlights Recent Updates NGN-401 gene therapy for Rett syndrome received RMAT designation from FDA based on preliminary clinical evidence indicating the potential to address unmet medical needs NGN-401 selected for FDA START Program, also designed to accelerate development Interim NGN-401 efficacy data from Cohort 1 remains on track for 4Q:24 NEW YORK – August 9, 2024 – Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life- ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 __________________________________________________ FORM 10-Q __________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ACT OF 1934 For the transition period from ________ to ________ Commission file number: 001-36327 __________ ...

Financial Performance - Neurogene reported a net loss of $16.9 million for Q1 2024, compared to a net loss of $12.3 million for Q1 2023, reflecting an increase of 37% year-over-year[8]. - Research and Development (R&D) expenses increased to $13.5 million in Q1 2024 from $10.3 million in Q1 2023, driven by higher clinical trial costs for NGN-401[8]. - General and Administrative (G&A) expenses rose to $5.2 million in Q1 2024, up from $2.8 million in Q1 2023, primarily due to increased compensation and professional fees[8]. - Total assets decreased to $195.4 million as of March 31, 2024, from $222.6 million as of December 31, 2023[13]. - Total liabilities decreased to $24.6 million as of March 31, 2024, from $36.5 million as of December 31, 2023[13]. Cash Position - Cash, cash equivalents, and investments as of March 31, 2024, were $169.5 million, with a cash runway projected into the second half of 2026[8]. Clinical Trials and Product Development - Neurogene presented favorable safety data from the NGN-401 gene therapy trial for Rett syndrome, indicating it has been well-tolerated in all three pediatric patients[3]. - The company remains on track to provide interim efficacy data from the NGN-401 trial in Q4 2024, with additional data expected in the second half of 2025[3]. - Neurogene has received approval from the Australian Human Research Ethics Committee to conduct the NGN-401 trial in Australia, expanding the trial to a third region[3]. - The company plans to advance an additional product candidate into the clinic in 2025, utilizing transgene regulation technology[5].

Exhibit 99.1 Neurogene Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Updates Expanded Phase 1/2 gene therapy trial for Rett syndrome to inform future registrational study design; Company remains on track to share interim clinical data in 4Q:24 Strong financial position with runway into 2H:26 following reverse merger and private financing in December 2023 Additional Corporate Updates Fourth Quarter and Full Year 2023 Financial Results Upcoming Events NEW YORK – March 18, 2 ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K __________________________________ (Mark One) x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-36327 __________________________________ Neurogene Inc. ________________________ ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 __________________________________________________ FORM 10-Q __________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ACT OF 1934 For the transition period from ________ to ________ Commission file number: 001-36327 ________ ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 __________________________________________________ FORM 10-Q __________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ACT OF 1934 For the transition period from ________ to ________ Commission file number: 001-36327 _____________ ...

Financial Performance - For the three months ended March 31, 2023, research and development expenses were $7.7 million, a decrease of 28% from $10.7 million in the same period of 2022[84]. - General and administrative expenses for the same period were $4.0 million, down 14% from $4.7 million in the prior year[86]. - Total operating expenses for the three months ended March 31, 2023, were $15.1 million, a slight decrease of 1% from $15.4 million in the same period of 2022[82]. - The net loss for the three months ended March 31, 2023, was $14.2 million, adjusted for non-cash items including impairment of $3.4 million and stock-based compensation of $1.2 million[96]. - For the three months ended March 31, 2023, the net cash used in operating activities was $(13,033) thousand, compared to $(14,091) thousand for the same period in 2022, indicating a 7.5% improvement[95]. Restructuring and Workforce Changes - The company reduced its workforce by approximately 40% in November 2022 and by an additional 70% in March 2023 as part of its restructuring plans[72][89]. - The restructuring plans are expected to incur aggregate charges of $1.7 million and $1.8 million for the November 2022 and March 2023 reductions, respectively[88][90]. - The company has suspended its research and development activities following the decision to discontinue the development of NL-201[78]. Cash and Financing - The company had an accumulated deficit of $465.3 million and cash, cash equivalents, and short-term investments of $83.4 million as of March 31, 2023[93]. - As of March 31, 2023, the company had approximately $83.4 million in cash, cash equivalents, and short-term investments, expected to fund operations for at least the next 12 months[99]. - The company plans to finance operations through equity sales, debt financing, or strategic transactions, with no assurance of success in obtaining adequate financing[79]. - Future capital requirements may vary significantly, and the company may need to seek additional funding sooner than planned[99]. - The company may face challenges in obtaining additional equity or debt financing on acceptable terms, which could negatively impact its business and financial condition[99]. Investment Activities - Cash provided by investing activities for the three months ended March 31, 2023, was $10,706 thousand, primarily from proceeds of available-for-sale securities[97]. - The company’s cash used in financing activities for the three months ended March 31, 2023, was $(66) thousand, primarily for finance lease payments[98]. - The company experienced a net decrease of $3.7 million in operating assets and liabilities for the three months ended March 31, 2023[96]. Future Outlook - The company has not generated product revenue or achieved profitability since inception and anticipates continued net losses in the foreseeable future[99]. - The company’s future capital requirements will depend on various factors, including the development and commercialization costs of future product candidates[100]. - Interest income increased to $0.9 million for the three months ended March 31, 2023, compared to $13 thousand in the same period of 2022, due to higher interest rates[92].

Financial Performance - The company incurred a net loss of $57.6 million for the year ended December 31, 2022, compared to a net loss of $60.7 million in 2021[333]. - Cash used in operating activities was $45.6 million in 2022, slightly improved from $47.6 million in 2021[330]. - The company has an accumulated deficit of $451.1 million as of December 31, 2022[330]. Expenses - Research and development expenses for the year ended December 31, 2022, were $41.1 million, an increase of 5% from $39.2 million in 2021[324]. - General and administrative expenses decreased by 17% to $18.0 million in 2022 from $21.5 million in 2021[326]. - Total operating expenses for 2022 were $59.1 million, a decrease of 3% compared to $60.7 million in 2021[322]. Restructuring and Workforce - The company announced a workforce reduction of approximately 40% as part of a strategic restructuring plan[327]. - The company expects to incur total restructuring charges of $1.8 million, with $1.4 million already incurred in 2022[328]. Cash and Investments - As of December 31, 2022, the company had cash, cash equivalents, and short-term investments totaling $96.4 million[330]. - Interest income increased significantly to $1.6 million in 2022 from $19.0 thousand in 2021 due to higher interest rates[329]. Research and Development Accounting - Research and development costs are charged to expense as incurred, including employee-related expenses, clinical trial costs, and technology licenses[345]. - No product development expenditures have been deferred to date, and costs are recorded based on the evaluation of specific tasks[346]. - Stock-based compensation is recognized over the vesting period, with all share-based payments accounted for based on grant-date fair values[347]. - The fair value of options granted is estimated using the Black-Scholes option pricing model, which involves significant estimates and judgments[348]. - There is inherent uncertainty in forecasts and projections, which could materially affect stock-based compensation expense and net loss amounts[349]. Market Risk Disclosures - As a smaller reporting company, the company is not required to provide quantitative and qualitative disclosures about market risk[351].

Financial Performance - For the three months ended September 30, 2022, total operating expenses decreased by 12% to $13.6 million from $15.5 million in the same period of 2021[82]. - Operating expenses for the nine months ended September 30, 2022, were $44.8 million, a slight decrease of 1% from $45.5 million in the same period of 2021[82]. - The net loss for the nine months ended September 30, 2022, was $44.1 million, adjusted for non-cash items including stock-based compensation expense of $8.8 million[95]. - The company has not generated product revenue or achieved profitability since inception and anticipates continued net losses for the foreseeable future[98]. Research and Development - Research and development expenses for the three months ended September 30, 2022, were $9.5 million, a decrease of 4% compared to $9.9 million for the same period in 2021[82]. - Research and development expenses for the nine months ended September 30, 2022, were $31.1 million, an increase from $29.4 million in the same period of 2021, primarily due to costs related to the Phase 1 clinical trial of NL-201[85]. - The decision to discontinue NL-201 was based on a review of preliminary data and the expected benefit-to-risk ratio for patients[66]. - The company is exploring additional therapeutic candidates beyond NL-201, including a targeted activator of T-regulatory cells for inflammation and autoimmune diseases[73]. - The company is focusing on the next generation of immunotherapies using de novo protein design and advanced machine learning[65]. Cost Management - General and administrative expenses decreased by 26% to $4.1 million for the three months ended September 30, 2022, compared to $5.6 million in the same period of 2021[82]. - General and administrative expenses for the three months ended September 30, 2022, were $4.1 million, down from $5.6 million in the same period of 2021, mainly due to a decrease in personnel-related costs[87]. - For the nine months ended September 30, 2022, general and administrative expenses totaled $13.7 million, compared to $16.1 million for the same period in 2021, reflecting decreases in personnel-related and facility-related costs[88]. - The company expects cost savings from a 40% reduction in workforce and the discontinuation of NL-201 development to extend its cash runway into the second half of 2025[67]. Cash Flow and Investments - Net cash used in operating activities for the nine months ended September 30, 2022, was $34.7 million, slightly lower than $35.4 million in the same period of 2021[92]. - Cash used in investing activities for the nine months ended September 30, 2022, was $67.6 million, significantly higher than $2.9 million in the same period of 2021[94]. - As of September 30, 2022, the company had an accumulated deficit of $437.6 million and cash, cash equivalents, and short-term investments of $106.9 million[92]. - The company expects its existing cash resources to fund operations into the second half of 2025, but may need to seek additional funds sooner than planned[98]. Capital Raising - The company plans to raise substantial additional capital to support operations and growth strategy, relying on equity sales, debt financing, or collaborations[77]. Interest Income - Interest income for the three months ended September 30, 2022, was $559,000, an increase from $6,000 in the same period of 2021, attributed to higher interest rates and U.S. treasury securities purchases[91]. Work Environment - The company has transitioned to a work-from-home policy during the COVID-19 pandemic but has returned to in-office work with hybrid schedules as of the end of Q3 2022[80].