Financial Performance - Neurogene reported a net loss of $36.3 million for the full year 2023, an improvement from a net loss of $55.2 million in 2022[9]. - General and Administrative (G&A) expenses increased to $11.2 million in 2023 from $9.0 million in 2022, driven by higher personnel and consulting costs[9]. Research and Development - Research and Development (R&D) expenses for 2023 were $44.4 million, a decrease from $47.5 million in 2022, primarily due to reduced manufacturing and pre-clinical costs[9]. - The Phase 1/2 trial of NGN-401 for Rett syndrome is expanding to include more patients, with interim clinical data expected in Q4 2024[3]. - The third patient in the NGN-401 trial was dosed in early Q1 2024, with plans to complete enrollment in Cohort 1 by the second half of 2024[4]. - The NGN-401 trial has been well-tolerated with no serious adverse events reported[4]. - Neurogene is on track to share data from both the NGN-401 and NGN-101 programs later in 2024[2]. - The company aims to advance an additional product candidate using its EXACT technology into the clinic by 2025[9]. Cash Position and Funding - Cash and cash equivalents as of December 31, 2023, were $197.2 million, providing a runway into the second half of 2026[9]. - The company completed a reverse merger and private placement of $95 million, beginning trading on Nasdaq under the ticker "NGNE" in December 2023[9].

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 ________ ...

Workforce Reduction - 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[84][98]. Financial Performance - Research and development expenses for Q2 2023 were $(0.4) million, a decrease of 104% compared to $11.0 million in Q2 2022[90]. - General and administrative expenses for Q2 2023 were $3.5 million, down 29% from $4.9 million in Q2 2022[93]. - Total operating expenses for the six months ended June 30, 2023, were $18.2 million, a decrease of 42% compared to $31.2 million for the same period in 2022[88]. - The company recorded an impairment charge of $3.4 million on property and equipment for the six months ended June 30, 2023[95]. - The net loss for the six months ended June 30, 2023, was $16.3 million, adjusted for non-cash items, compared to a net loss of $31.0 million in the same period of 2022[105]. Restructuring and Future Plans - The company expects to incur additional restructuring charges of $1.8 million related to the March 2023 restructuring plan, with all charges expected to be paid by the end of Q1 2024[99]. - The company has suspended all research and development activities to conserve capital and focus on strategic alternatives[77]. - Future capital requirements will depend on various factors, including the completion of a merger and the development of new product candidates[109]. Cash Flow and Funding - Operating activities used $17.1 million in cash flows for the six months ended June 30, 2023, compared to $25.4 million in the same period of 2022, indicating a reduction in cash outflow[102]. - Cash provided by investing activities for the six months ended June 30, 2023, was $10.3 million, primarily from the maturities of available-for-sale securities, compared to cash used of $39.8 million in the same period of 2022[104][106]. - The company anticipates that existing cash and short-term investments will be sufficient to fund operations for at least 12 months following the filing date of the report[87]. - The company expects its existing cash resources to fund operations for at least 12 months following the filing date, but may need additional funds sooner than planned[108]. Merger and Stock Sales - The merger agreement with Neurogene Inc. will result in pre-Merger Neurogene stockholders owning approximately 84% of the combined company[80]. - As of March 20, 2023, the company is subject to limitations on raising funds through the sale of common stock due to SEC regulations[103]. - The company has not made any sales of common stock under the ATM facility established in November 2021, which allows for an aggregate offering price of up to $40.0 million[103]. Revenue Generation - The company has not generated product revenue or achieved profitability since inception and anticipates continued net losses[108]. - The company plans to finance operations through equity sales, debt financings, or collaborations, as it currently does not generate significant revenue from product sales[86]. Interest Income - Interest income for the three months ended June 30, 2023, was $1.0 million, up from $0.2 million in the same period of 2022, reflecting a significant increase due to higher interest rates and U.S. treasury securities purchases[101].

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 March 31, 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 ____________ ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _________________________ FORM 10-K _________________________ (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 OR ☐ 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 _________________________ Neoleukin Therapeutics, Inc. ( ...

UNITED STATES For the transition period from ________ to ________ ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ACT OF 1934 SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 __________________________________________________ FORM 10-Q __________________________________________________ (Mark One) 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, 2022 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, 2022, research and development expenses increased to $10.7 million, a 10% rise from $9.7 million in the same period of 2021[68] - General and administrative expenses decreased to $4.7 million for the three months ended March 31, 2022, down 11% from $5.2 million in the prior year[72] - The total operating expenses for the three months ended March 31, 2022, were $15.4 million, reflecting a 3% increase compared to $14.9 million for the same period in 2021[68] - The company reported a net cash used in operating activities of $14.1 million for the three months ended March 31, 2022, compared to $13.4 million in the same period of 2021[74] - Net cash used in operating activities for Q1 2022 was $15.4 million, compared to $14.9 million in Q1 2021, with non-cash items totaling $3.1 million in 2022 and $2.9 million in 2021[77] - The company has not generated product revenue or achieved profitability since inception and anticipates continued net losses in the foreseeable future[81] Cash Position - As of March 31, 2022, the company had cash and cash equivalents of $128.1 million and an accumulated deficit of $408.8 million[74] - As of March 31, 2022, the company had approximately $128.1 million in cash and cash equivalents, expected to fund operations through 2023[81] Capital Requirements - The company anticipates needing to raise substantial additional capital to support ongoing operations and growth strategies[63] - Future capital requirements may vary significantly, and the company may need to seek additional funds sooner than planned[81] - Factors influencing future capital requirements include product development costs, regulatory approval expenses, and commercialization activities[83] Clinical Development - The company plans to enroll up to 252 patients in a Phase 1 clinical trial for NL-201, assessing safety and antitumor activity[59] - The company has initiated a clinical collaboration with Merck to evaluate NL-201 in combination with KEYTRUDA®[58] - The company is focused on developing de novo protein therapeutics to address unmet medical needs in immuno-oncology, inflammation, and autoimmunity[61] - The company faces potential delays in development programs due to the ongoing COVID-19 pandemic and related economic pressures[83] Investing and Financing Activities - Cash used in investing activities for Q1 2022 primarily consisted of purchases of laboratory equipment, similar to Q1 2021[78] - Net cash provided by financing activities for both Q1 2022 and Q1 2021 was mainly from proceeds from stock option exercises[79] - The company has entered into an ATM Equity Offering Sales Agreement with BofA Securities, allowing for the potential sale of up to $40 million in common stock[75] Accounting and Reporting - There have been no material changes to significant accounting policies during Q1 2022[84] - As a smaller reporting company, the company is not required to provide quantitative and qualitative disclosures about market risk[86]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _________________________ FORM 10-K _________________________ (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 OR ☐ 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 _________________________ Neoleukin Therapeutics, Inc. ( ...