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 ________ ...

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 _____________ ...