[Q3 2024 Financial Results and Corporate Updates](index=1&type=section&id=Neurogene%20Reports%20Third%20Quarter%202024%20Financial%20Results%20and%20Highlights%20Recent%20Updates) Neurogene's Q3 2024 report details financial performance, clinical pipeline advancements, and strategic corporate developments, including a significant capital raise [Financial Performance](index=2&type=section&id=Third%20Quarter%202024%20Financial%20Results) Neurogene's Q3 2024 net loss increased to **$20.2 million**, driven by higher R&D and G&A expenses reflecting increased clinical trial activities and headcount Financial Metric Comparison | Financial Metric | Q3 2024 (in millions) | Q3 2023 (in millions) | Change | | :--- | :--- | :--- | :--- | | R&D Expenses | $16.3 | $11.6 | +$4.7M | | G&A Expenses | $5.9 | $3.6 | +$2.3M | | Net Loss | $20.2 | $14.6 | +$5.6M | - The increase in R&D expenses was primarily due to higher costs for the NGN-401 clinical trial, preclinical discovery programs, and increased compensation expenses from a larger R&D team[10](index=10&type=chunk) - The rise in G&A expenses was mainly driven by increased employee-related costs, professional fees, rent, and other corporate expenses[10](index=10&type=chunk) [Clinical Pipeline Updates](index=1&type=section&id=Third%20Quarter%202024%20and%20Recent%20Highlights%2C%20and%20Anticipated%20Milestones) Neurogene advances NGN-401 for Rett syndrome at the 1E15 vg dose after positive data, while pausing the 3E15 vg dose and discontinuing the NGN-101 program for CLN5 Batten disease following FDA's RMAT denial [NGN-401 for Rett Syndrome](index=1&type=section&id=Phase%201%2F2%20Trial%20of%20NGN-401%20Gene%20Therapy%20for%20Treatment%20of%20Rett%20Syndrome) NGN-401 for Rett syndrome shows positive interim data and a favorable safety profile at the 1E15 vg dose, with the 3E15 vg dose paused due to a serious adverse event - Positive interim data from the 1E15 vg dose showed consistent improvements, with all four participants rated as **"much improved"** on the CGI-I scale and showing a **28% to 52% improvement** on the RSBQ[2](index=2&type=chunk)[5](index=5&type=chunk) - The 1E15 vg dose was well-tolerated, with all treatment-related adverse events being **mild (Grade 1)**[5](index=5&type=chunk) - The company paused the 3E15 vg dose following a treatment-related serious adverse event (SAE) and does not plan to enroll more participants at this dose[3](index=3&type=chunk) - The FDA has reviewed safety data and allowed the trial to proceed with the 1E15 vg dose; an update on the registrational trial design is expected in the first half of 2025[3](index=3&type=chunk)[5](index=5&type=chunk) [NGN-101 for CLN5 Batten Disease](index=2&type=section&id=CLN5%20Batten%20Disease%20Update) Neurogene will not proceed with the NGN-101 program for CLN5 Batten disease after the FDA denied its RMAT application - Neurogene announced it does not expect to move forward with the NGN-101 gene therapy program for CLN5 Batten disease at this time[6](index=6&type=chunk) - The decision was made after the FDA denied the company's Regenerative Medicine Advance Therapy (RMAT) application, which was considered necessary for a streamlined registrational pathway due to the rarity of the disease[6](index=6&type=chunk) [Corporate and Strategic Developments](index=1&type=section&id=Additional%20Corporate%20Updates) Neurogene significantly strengthened its financial position by executing an oversubscribed private placement of approximately **$200 million**, extending its cash runway into the second half of 2027 and supporting future clinical advancements - Executed an oversubscribed private placement, raising approximately **$200 million** in gross proceeds[9](index=9&type=chunk) - The company's cash, cash equivalents, and investments totaled **$139.0 million** as of September 30, 2024; combined with private placement proceeds, the cash runway is expected to last into the **second half of 2027**[1](index=1&type=chunk)[8](index=8&type=chunk)[10](index=10&type=chunk) - Neurogene plans to advance an additional product candidate into clinical trials in 2025[9](index=9&type=chunk) [Financial Statements](index=5&type=section&id=Financial%20Tables%20Follow) The condensed consolidated financial statements reflect a decrease in total assets to **$164.1 million** as of September 30, 2024, and a net loss of **$20.2 million** for Q3 2024 [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheets detail assets, liabilities, and stockholders' equity, showing a decrease in total assets from year-end 2023 to Q3 2024 | (In thousands of U.S. dollars) | September 30, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $66,633 | $148,210 | | Total assets | $164,120 | $222,573 | | **Liabilities and Stockholders' Equity** | | | | Total liabilities | $26,706 | $36,549 | | Stockholders' equity | $137,414 | $186,024 | | Total liabilities and stockholders' equity | $164,120 | $222,573 | [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) The statements of operations present the company's operating expenses, loss from operations, and net loss for the three and nine months ended September 30, 2024 | (In thousands of U.S. dollars) | Three Months Ended Sep 30, 2024 | Three Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2024 | Nine Months Ended Sep 30, 2023 | | :--- | :--- | :--- | :--- | :--- | | Total operating expenses | $22,180 | $15,219 | $62,018 | $40,850 | | Loss from operations | $(22,180) | $(15,219) | $(61,093) | $(40,850) | | Net loss | $(20,217) | $(14,577) | $(55,630) | $(38,700) | | Net loss per share, basic and diluted | $(1.19) | $(32.67) | $(3.29) | $(87.66) |

Core Viewpoint - Neurogene's stock experienced a significant decline of 44% in a single day due to adverse news regarding its clinical trials and regulatory challenges, despite a strong year-to-date performance of 269% prior to the drop [1][5]. Group 1: Company Overview - Neurogene is focused on developing gene therapy medicines targeting specific genes that cause harmful effects in the body [2]. - The company primarily targets Rett Syndrome and CLN5 Batten Disease, both of which currently lack FDA-approved treatments that can reverse their progression [3]. Group 2: Recent Developments - Neurogene faced a setback when results from the Phase 1/2 trial of its Rett Syndrome treatment, NGN-401, indicated a serious adverse event (SAE) associated with the high dose, leading to a 33% incidence rate among the three patients tested [5][6]. - The company announced it would not pursue its treatment for CLN5 Batten disease after the FDA denied its application for a streamlined process, deeming it uneconomical to continue [7]. Group 3: Clinical Trial Results - Despite the high-dose concerns, the low-dose NGN-401 treatment showed promising results, with no SAEs reported among five patients, and all participants rated "much improved" on the Clinical Global Impression Scale [8][9]. - Caregivers reported behavior improvements in patients ranging from 28% to 52% from baseline, and all patients achieved developmental milestones rarely regained in Rett Syndrome [10]. Group 4: Market Potential - The addressable market for Rett Syndrome is estimated to be around $2.5 billion, highlighting significant revenue potential for Neurogene if its treatment is approved [4]. - Analysts suggest that the stock's recent drop may be an overreaction, given the encouraging data from the low-dose treatment and the potential for future success [12].

On Monday, Neurogene Inc. NGNE released interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase 1/2 open-label trial of NGN-401 gene therapy for female pediatric patients with Rett syndrome, a rare genetic neurological and developmental disorder.Low-dose (1E15 vg) and high-dose (3E15 vg) NGN-401 have been well-tolerated with a favorable safety profile in the first seven pediatric participants (N=5 low-dose; N=2 high-dose):No treatment-related serious adverse events ( ...

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 June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ACT OF 1934 For the tra ...

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 ________ ...