[Part I - Financial Information](index=4&type=section&id=Part%20I%20Financial%20Information) [Financial Statements](index=4&type=section&id=Item%201.%20Financial%20Statements) Neurogene Inc.'s unaudited condensed consolidated financial statements as of March 31, 2024, report a **$16.9 million net loss** and **$169.5 million in cash and investments**, detailing the December 2023 reverse merger Condensed Consolidated Balance Sheet Highlights (Unaudited) | Account | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $150,140 | $148,210 | | Short-term investments | $19,393 | $48,947 | | Total current assets | $174,141 | $200,348 | | Total assets | $195,350 | $222,573 | | **Liabilities & Equity** | | | | Total current liabilities | $11,818 | $22,973 | | Total liabilities | $24,573 | $36,549 | | Accumulated deficit | $(204,075) | $(187,154) | | Total stockholders' equity | $170,777 | $186,024 | Condensed Consolidated Statement of Operations (Unaudited) | Account | Three Months Ended March 31, 2024 (in thousands) | Three Months Ended March 31, 2023 (in thousands) | | :--- | :--- | :--- | | Research and development expenses | $13,541 | $10,283 | | General and administrative expenses | $5,238 | $2,752 | | **Loss from operations** | **$(18,779)** | **$(13,035)** | | Interest income | $2,320 | $777 | | **Net loss** | **$(16,921)** | **$(12,263)** | | Net loss per share, basic and diluted | $(1.00) | $(28.28) | Condensed Consolidated Statement of Cash Flows Highlights (Unaudited) | Activity | Three Months Ended March 31, 2024 (in thousands) | Three Months Ended March 31, 2023 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | $(21,650) | $(13,555) | | Net cash provided by (used in) investing activities | $29,935 | $(29) | | Net cash (used in) provided by financing activities | $(6,524) | $107 | | **Net increase (decrease) in cash** | **$1,761** | **$(13,477)** | - On December 18, 2023, the company completed a reverse merger with Neoleukin Therapeutics, Inc., becoming Neurogene Inc., and raised approximately **$95.0 million** through a Pre-Closing Financing[22](index=22&type=chunk)[23](index=23&type=chunk) - As of March 31, 2024, the company held **$169.5 million** in cash, cash equivalents, and investments, expected to fund operations for at least one year[27](index=27&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses Neurogene's focus on developing genetic medicines for neurological diseases, reporting a **Q1 2024 net loss of $16.9 million** due to increased R&D and G&A expenses, with **$169.5 million cash** expected to fund operations into H2 2026 - The company is a clinical-stage biotechnology firm focused on neurological diseases, with lead programs **NGN-401** for Rett syndrome and **NGN-101** for CLN5 Batten disease, utilizing proprietary EXACT gene regulation technology[87](index=87&type=chunk)[89](index=89&type=chunk)[93](index=93&type=chunk) - Preliminary clinical data for **NGN-401 Phase 1/2 trial** is expected in **Q4 2024**, with interim data for **NGN-101 Phase 1/2 trial** in **H2 2024**[91](index=91&type=chunk)[93](index=93&type=chunk) Results of Operations Comparison (in thousands) | | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | Change | | :--- | :--- | :--- | :--- | | Research and development expenses | $13,541 | $10,283 | $3,258 | | General and administrative expenses | $5,238 | $2,752 | $2,486 | | **Total operating expenses** | **$18,779** | **$13,035** | **$5,744** | | **Net loss** | **$(16,921)** | **$(12,263)** | **$(4,658)** | - R&D expenses increased primarily due to a **$1.3 million** rise in clinical trial costs for NGN-401 and a **$0.9 million** increase in preclinical costs[118](index=118&type=chunk) - G&A expenses increased mainly due to higher professional fees (**$0.9 million**), rent (**$0.5 million**), and compensation costs (**$0.8 million**) post-reverse merger[121](index=121&type=chunk) - As of March 31, 2024, the company held **$169.5 million** in cash, cash equivalents, and short-term investments, projected to fund operations into the **second half of 2026**[126](index=126&type=chunk)[171](index=171&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=35&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a "smaller reporting company," Neurogene is exempt from providing quantitative and qualitative disclosures about market risk - As a **"smaller reporting company,"** Neurogene is exempt from detailed market risk disclosures[157](index=157&type=chunk) [Controls and Procedures](index=35&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective as of March 31, 2024, with no material changes to internal control over financial reporting during the quarter - Management concluded the company's disclosure controls and procedures were **effective** as of the report period end[160](index=160&type=chunk) - No material changes in internal control over financial reporting occurred during the quarter ended March 31, 2024[161](index=161&type=chunk) [Part II - Other Information](index=36&type=section&id=Part%20II%20Other%20Information) [Legal Proceedings](index=36&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any legal proceedings expected to have a material adverse effect on its business or financial condition - Neurogene is not currently involved in any legal proceedings expected to have a **material adverse effect** on the company[164](index=164&type=chunk) [Risk Factors](index=36&type=section&id=Item%201A.%20Risk%20Factors) This section outlines significant risks including Neurogene's limited operating history, substantial capital needs, development and commercialization challenges for **NGN-401** and **NGN-101**, intellectual property, third-party reliance, and stock price volatility [Risks Related to Our Limited Operating History, Financial Position and Capital Requirements](index=37&type=section&id=Risks%20Related%20to%20Our%20Limited%20Operating%20History%2C%20Financial%20Position%20and%20Capital%20Requirements) - The company has a limited operating history, no approved products, and an accumulated deficit of **$204.1 million** as of March 31, 2024[168](index=168&type=chunk)[170](index=170&type=chunk)[176](index=176&type=chunk) - Neurogene requires substantial additional capital, with existing cash expected to fund operations into the **second half of 2026**, based on potentially inaccurate assumptions[170](index=170&type=chunk)[171](index=171&type=chunk) - The company has not generated product revenue and may never achieve profitability, with speculative biotechnology investment posing risks to capital raising and operations[175](index=175&type=chunk)[182](index=182&type=chunk) [Risks Related to Discovery, Development and Commercialization](index=39&type=section&id=Risks%20Related%20to%20Discovery%2C%20Development%20and%20Commercialization) - Lead product candidates **NGN-401** and **NGN-101** are in early clinical development and face risks of failure or delays, materially harming the business[185](index=185&type=chunk)[199](index=199&type=chunk) - The company faces significant competition from multinational biopharmaceutical and specialized biotechnology firms with greater resources[183](index=183&type=chunk) - Gene therapy development is novel and complex, with high failure risk; preclinical results may not predict clinical success, and adverse events could halt development[192](index=192&type=chunk)[206](index=206&type=chunk)[213](index=213&type=chunk) - Low prevalence of targeted rare diseases may hinder patient enrollment in clinical trials, potentially delaying or preventing regulatory approvals[195](index=195&type=chunk) [Risks Related to Manufacturing](index=46&type=section&id=Risks%20Related%20to%20Manufacturing) - Gene therapy manufacturing is complex and subject to strict cGMP regulations; production difficulties, contamination, or non-compliance could cause delays and harm the business[222](index=222&type=chunk)[223](index=223&type=chunk)[236](index=236&type=chunk) - Reliance on a limited number of third-party suppliers for critical raw materials poses risks of supply interruption, materially harming product candidate manufacturing[227](index=227&type=chunk)[233](index=233&type=chunk) - Despite having its own GMP manufacturing facility, the company may face challenges in scaling up production, potentially delaying development and commercialization[234](index=234&type=chunk)[237](index=237&type=chunk) [Risks Related to Our Reliance on Third Parties](index=49&type=section&id=Risks%20Related%20to%20Our%20Reliance%20on%20Third%20Parties) - Heavy reliance on academic collaboration with the University of Edinburgh for preclinical R&D means any failure, delay, or termination would materially harm the business[240](index=240&type=chunk)[241](index=241&type=chunk) - Dependence on third-party CROs for preclinical studies and clinical trials means poor performance or termination could delay or end clinical programs[245](index=245&type=chunk)[246](index=246&type=chunk)[247](index=247&type=chunk) [Risks Related to Our Business and Operations](index=51&type=section&id=Risks%20Related%20to%20Our%20Business%20and%20Operations) - High dependence on key personnel means inability to attract and retain qualified staff could impede business strategy implementation[252](index=252&type=chunk) - Vulnerability to security breaches, exemplified by an August 2023 business email compromise attack resulting in **$0.9 million** misappropriation, poses risks of significant liabilities and operational disruption[255](index=255&type=chunk) - Stringent and changing privacy and data protection laws mean non-compliance could lead to government enforcement actions, fines, and reputational harm[263](index=263&type=chunk) - The company's ability to use **$277.9 million** federal and **$35.1 million** state net operating loss carryforwards may be limited by Section 382 ownership changes[265](index=265&type=chunk) [Risks Related to Intellectual Property](index=55&type=section&id=Risks%20Related%20to%20Intellectual%20Property) - Protection of patents and proprietary rights is uncertain, with an early-stage IP portfolio, no issued patents as of March 31, 2024, and pending applications that may not result in issuance[271](index=271&type=chunk)[272](index=272&type=chunk) - Reliance on in-licensed IP from third parties like the University of Edinburgh and UNC means termination or breach of agreements could lead to loss of product candidate development rights[276](index=276&type=chunk)[280](index=280&type=chunk)[283](index=283&type=chunk) - The company may face patent infringement claims or need to file claims to protect its IP, incurring substantial costs, liability, and resource diversion[286](index=286&type=chunk) [Risks Related to Government Regulation](index=61&type=section&id=Risks%20Related%20to%20Government%20Regulation) - The gene therapy regulatory approval process is lengthy, complex, and unpredictable; delays or failure to obtain FDA and other authority approvals would prevent commercialization[310](index=310&type=chunk)[313](index=313&type=chunk) - Approved products will face extensive ongoing regulatory obligations, including expensive and restrictive post-marketing studies and surveillance[328](index=328&type=chunk) - Orphan Drug and Rare Pediatric Disease designations for **NGN-401** and **NGN-101** do not guarantee faster development, regulatory approval, or market exclusivity[344](index=344&type=chunk)[348](index=348&type=chunk) - Unfavorable pricing regulations and third-party coverage/reimbursement policies could hinder competitive product pricing, harming the business[334](index=334&type=chunk) [General Risk Factors](index=68&type=section&id=General%20Risk%20Factors) - Market opportunity estimates and growth forecasts may be inaccurate, and the addressable patient population might be less significant than anticipated[349](index=349&type=chunk)[350](index=350&type=chunk) - Macroeconomic conditions, including economic downturns, inflation, interest rate increases, and geopolitical events, could adversely affect the business[353](index=353&type=chunk) - Exposure to potential product liability claims from clinical trials and commercial sales means insurance may not sufficiently cover all damages[351](index=351&type=chunk) [Risks Related to Owning Our Stock](index=69&type=section&id=Risks%20Related%20to%20Owning%20Our%20Stock) - The common stock market price has been and may remain volatile due to clinical trial results, regulatory actions, and general market conditions[355](index=355&type=chunk) - Executive officers, directors, and principal stockholders beneficially own a significant percentage of outstanding stock, influencing matters submitted for stockholder approval[371](index=371&type=chunk) - Future sales of substantial common stock by existing stockholders, especially post-lock-up expiration, could cause stock price decline[370](index=370&type=chunk) - A material weakness in internal control over financial reporting, related to a 2023 business email compromise attack, has been remediated, but future weaknesses could lead to inaccurate reporting and share price decline[363](index=363&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=73&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities occurred during the reporting period - The company reports no unregistered sales of equity securities for the period[376](index=376&type=chunk) [Defaults Upon Senior Securities](index=73&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) No defaults upon senior securities occurred during the reporting period - The company reports no defaults upon senior securities[377](index=377&type=chunk) [Mine Safety Disclosures](index=73&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company's operations - Mine safety disclosures are not applicable to Neurogene[378](index=378&type=chunk) [Other Information](index=73&type=section&id=Item%205.%20Other%20Information) No directors or Section 16 officers adopted or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during the quarter - No directors or Section 16 officers adopted or terminated any Rule 10b5-1 or non-Rule 10b5-1 trading plans during the quarter[379](index=379&type=chunk) [Exhibits](index=74&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with Form 10-Q, including employment agreements, a consulting agreement, officer certifications, and XBRL data - Exhibits filed include new executive employment agreements, an amended consulting agreement, and required CEO/CFO certifications[381](index=381&type=chunk)

[CEO Statement and Corporate Strategy](index=1&type=section&id=CEO%20Statement%20and%20Corporate%20Strategy) CEO highlights NGN-401 program progress, trial expansion, strong financial position with cash into H2 2026, and new clinical candidate plans for 2025 - Significant progress in **NGN-401 Rett syndrome program**, with trial expanding to Australia and a high-dose cohort[2](index=2&type=chunk) - The company plans to advance an additional product candidate into clinical trials in **2025**, leveraging its transgene regulation platform[2](index=2&type=chunk) - Neurogene confirms a **strong financial position**, with sufficient cash runway to fund operations into the **second half of 2026**[2](index=2&type=chunk) [Clinical Program Updates](index=1&type=section&id=Clinical%20Program%20Updates) Neurogene provides updates on key clinical trials, including NGN-401 expansion, NGN-101 enrollment, and plans for a new candidate in 2025 [NGN-401 for Rett Syndrome (Phase 1/2 Trial)](index=1&type=section&id=Phase%201%2F2%20Trial%20of%20NGN-401%20Gene%20Therapy%20for%20Treatment%20of%20Rett%20Syndrome) NGN-401 trial shows favorable safety, no MeCP2 overexpression toxicity, expands to Australia, with interim efficacy data expected in Q4 2024 - Favorable safety data showed NGN-401 was generally **well-tolerated** in the first three patients, with all treatment-related adverse events being **mild (Grade 1)**[3](index=3&type=chunk) - **No signs of MeCP2 overexpression toxicity** were reported, addressing a key safety concern[3](index=3&type=chunk) - The trial has been cleared to proceed in **Australia**, the third region for the study[3](index=3&type=chunk) - Key upcoming milestones include interim efficacy data from Cohort 1 in **Q4 2024** and additional data from Cohort 2 in **H2 2025**[3](index=3&type=chunk) [NGN-101 for CLN5 Batten Disease (Phase 1/2 Trial)](index=2&type=section&id=Phase%201%2F2%20Trial%20of%20NGN-101%20Gene%20Therapy%20for%20Treatment%20of%20CLN5%20Batten%20Disease) NGN-101 trial enrollment continues for Cohort 3, with an H2 2024 update, contingent on FDA alignment for a streamlined registrational pathway - Enrollment continues for the **high-dose Cohort 3**, with an interim clinical and regulatory update planned for **H2 2024**[4](index=4&type=chunk) - Future investment in the NGN-101 program is **contingent upon FDA agreement** on a streamlined registrational pathway[4](index=4&type=chunk) [Early-Stage Portfolio Development](index=2&type=section&id=Additional%20Corporate%20Updates) Neurogene actively advances its early-stage portfolio, planning to move an additional product candidate into clinical trials in 2025 - The company plans to advance an additional product candidate into the clinic in **2025**[5](index=5&type=chunk) [First Quarter 2024 Financial Results](index=2&type=section&id=First%20Quarter%202024%20Financial%20Results) Neurogene reported a Q1 2024 net loss of **$16.9 million**, with **$169.5 million** cash extending runway into H2 2026, driven by increased operating expenses Summary Financial Metrics | Financial Metric | Q1 2024 | Q1 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | R&D Expenses | $13.5M | $10.3M | +$3.2M | | G&A Expenses | $5.2M | $2.8M | +$2.4M | | Net Loss | $16.9M | $12.3M | +$4.6M | - Cash, cash equivalents, and investments totaled **$169.5 million** as of March 31, 2024[8](index=8&type=chunk) - The current cash position is expected to fund operations into the **second half of 2026**[8](index=8&type=chunk) - The increase in R&D expenses was driven by **NGN-401 clinical trial costs** and preclinical discovery programs, while G&A expenses rose due to **higher headcount** and public company costs[8](index=8&type=chunk) [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of March 31, 2024, total assets were **$195.4 million**, liabilities **$24.6 million**, and stockholders' equity **$170.8 million**, compared to year-end 2023 figures Balance Sheet Summary | (In Thousands of U.S. dollars) | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $150,140 | $148,210 | | Total assets | $195,350 | $222,573 | | **Liabilities & Equity** | | | | Total liabilities | $24,573 | $36,549 | | Stockholders' equity | $170,777 | $186,024 | | Total liabilities and stockholders' equity | $195,350 | $222,573 | [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Q1 2024 net loss increased to **$16.9 million** from **$12.3 million** in Q1 2023, driven by higher total operating expenses of **$18.8 million** Statements of Operations Summary | (In thousands of U.S. dollars) | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Research and development | $13,541 | $10,283 | | General and administrative | $5,238 | $2,752 | | **Total operating expenses** | **$18,779** | **$13,035** | | Loss from operations | ($18,779) | ($13,035) | | **Net loss** | **($16,921)** | **($12,263)** | | Net loss per share, basic and diluted | ($1.00) | ($28.28) |

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 ________ ...

Financial Performance - Research and development expenses for Q2 2022 were $11.0 million, an increase of 12% from $9.8 million in Q2 2021[81]. - Total operating expenses for the first half of 2022 were $31.2 million, a 4% increase from $30.1 million in the same period of 2021[79]. - General and administrative expenses for Q2 2022 were $4.9 million, a decrease of 7% from $5.3 million in Q2 2021[83]. - Interest income increased significantly to $194,000 for the three months ended June 30, 2022, compared to $5,000 for the same period in 2021, representing a 3780% increase[84]. - The company incurred net cash used in operating activities of $25.4 million for the six months ended June 30, 2022, compared to $26.6 million for the same period in 2021[85]. - The net loss for the six months ended June 30, 2022, was $31.0 million, adjusted for non-cash items including stock-based compensation of $4.8 million[88]. - Cash used in investing activities for the six months ended June 30, 2022, was $39.8 million, primarily for purchases of available-for-sale securities and laboratory equipment[89]. - The net change in cash, cash equivalents, and restricted cash for the six months ended June 30, 2022, was a decrease of $64.9 million[87]. - The company has not generated product revenue or achieved profitability since inception and expects to continue incurring net losses for the foreseeable future[92]. - The company believes its existing cash and investments will be sufficient to fund operations through 2023, but may need to seek additional funds sooner than planned[92]. - The company entered into an ATM Equity Offering Sales Agreement with BofA Securities for an aggregate offering price of up to $40.0 million, but no sales have been made to date[86]. Clinical Trials and Research - The Phase 1 clinical trial of NL-201 is planned to enroll up to 252 patients, with interim data expected to be disclosed in 2023[67][68]. - NL-201 is designed to enhance binding to the beta and gamma subunits of the IL-2 receptor while eliminating binding to CD25, aiming to reduce toxicity[62][65]. - The company plans to initiate a Phase 1 clinical trial in hematological malignancies, dependent on data from the ongoing solid tumor trial[69]. - The company is exploring new therapeutic candidates using de novo protein design, including targeted IL-2/IL-15 mimetics and T-Reg agonists[72]. Company Overview - The merger with Aquinox was completed on August 8, 2019, resulting in the renaming of the company to Neoleukin Therapeutics, Inc.[61]. - Cash-on-hand is expected to be sufficient to fund operations through 2023[75]. - The company is focused on maintaining research continuity during the COVID-19 pandemic while ensuring employee safety[76]. Future Outlook - The company’s future capital requirements will depend on various factors, including the development of product candidates and the costs of regulatory approvals[94].