PART I—FINANCIAL INFORMATION Presents Gain Therapeutics' unaudited condensed consolidated financial statements and detailed notes for Q1 2023 and 2022 [Item 1. Condensed Consolidated Financial Statements](index=5&type=section&id=Item%201%20Condensed%20Consolidated%20Financial%20Statements) Presents unaudited consolidated financial statements and notes for Q1 2023 and 2022 [Condensed Consolidated Balance Sheets (Unaudited)](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets%20%28Unaudited%29) Details the company's financial position (assets, liabilities, equity) as of March 31, 2023, and December 31, 2022 Condensed Consolidated Balance Sheets (Unaudited) Table | Metric | Dec 31, 2022 ($) | Mar 31, 2023 ($) | | :--------------------------------- | :--------------- | :--------------- | | Cash and cash equivalents | 7,311,611 | 5,988,202 | | Marketable securities - current | 12,826,954 | 11,827,528 | | Total current assets | 21,091,296 | 19,321,984 | | Total assets | 24,099,387 | 21,322,575 | | Accounts payable | 1,626,100 | 2,213,489 | | Total current liabilities | 4,125,251 | 5,154,959 | | Total liabilities | 5,219,873 | 6,183,745 | | Total stockholders' equity | 18,879,514 | 15,138,830 | [Condensed Consolidated Statements of Operations (Unaudited)](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20%28Unaudited%29) Details the company's revenues, expenses, and net loss for the three months ended March 31, 2023, and 2022 Condensed Consolidated Statements of Operations (Unaudited) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Total revenues | 55,180 | 45,006 | | Research and development expenses | (2,791,205) | (1,556,440) | | General and administrative expenses | (2,493,759) | (1,777,043) | | Total operating expenses | (5,284,964) | (3,333,483) | | Loss from operations | (5,229,784) | (3,288,477) | | Net loss | (5,137,319) | (3,272,643) | | Net loss per share - basic and diluted | (0.43) | (0.28) | [Condensed Consolidated Statements of Comprehensive (Loss) (Unaudited)](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20%28Loss%29%20%28Unaudited%29) Reports net loss and other comprehensive income/loss for Q1 2023 and 2022 Condensed Consolidated Statements of Comprehensive (Loss) (Unaudited) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :---------------------------------- | :------------------------------ | :------------------------------ | | Net loss | (5,137,319) | (3,272,643) | | Unrealized gain on available-for-sale securities | 43,262 | — | | Foreign currency translation | 18,091 | (27,806) | | Other comprehensive income/(loss) | 60,683 | (23,657) | | Comprehensive loss | (5,076,636) | (3,296,300) | [Condensed Consolidated Statement of Stockholders' Equity (Unaudited)](index=11&type=section&id=Condensed%20Consolidated%20Statement%20of%20Stockholders%27%20Equity%20%28Unaudited%29) Outlines changes in stockholders' equity, including net loss and stock-based compensation Condensed Consolidated Statement of Stockholders' Equity (Unaudited) Table | Metric | Dec 31, 2022 ($) | Mar 31, 2023 ($) | | :--------------------------------- | :--------------- | :--------------- | | Balance as of period end | 18,879,514 | 15,138,830 | | Stock-based compensation | — | 565,432 | | Issuance of shares in ATM offering | — | 770,520 | | Net loss | (5,137,319) | (5,137,319) | [Condensed Consolidated Statements of Cash Flows (Unaudited)](index=13&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20%28Unaudited%29) Summarizes cash flows from operating, investing, and financing activities for Q1 2023 and 2022 Condensed Consolidated Statements of Cash Flows (Unaudited) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Cash used in operating activities | (4,217,415) | (2,447,391) | | Cash provided by/(used in) investing activities | 2,106,980 | (7,825) | | Cash provided by/(used in) financing activities | 748,908 | (37,974) | | Net (decrease)/increase in cash, cash equivalents and restricted cash | (1,323,105) | (2,542,507) | | Cash, cash equivalents and restricted cash at end of period | 6,019,324 | 34,369,445 | [Notes to Condensed Consolidated Financial Statements (Unaudited)](index=14&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements%20%28Unaudited%29) Provides detailed explanations of financial statement figures, business nature, and significant accounting policies [1. Nature of the Business and Basis of Presentation](index=14&type=section&id=1.%20Nature%20of%20the%20Business%20and%20Basis%20of%20Presentation) Describes Gain Therapeutics' biotechnology focus, drug discovery platform, and ongoing financial challenges - Gain Therapeutics is a biotechnology company developing novel small molecule therapeutics for CNS disorders, LSDs, metabolic disorders, and oncology[36](index=36&type=chunk) - The company uses its exclusively in-licensed computational target and drug discovery platform, Site-Directed Enzyme Enhancement Therapy ("SEE-Tx®"), to discover novel allosteric binding sites and identify proprietary small molecules[36](index=36&type=chunk) - The company has incurred recurring losses and negative cash flows from operations since inception and anticipates needing additional capital to fund operations into the **third quarter of 2024**[43](index=43&type=chunk)[46](index=46&type=chunk) [2. Summary of Significant Accounting Policies](index=18&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) Outlines the key accounting principles and methods used in preparing the financial statements - The company's functional currency is USD, with foreign operations in Switzerland (Swiss Franc) and Spain (Euro), and translation adjustments are recorded in accumulated other comprehensive income/(loss)[51](index=51&type=chunk) - Marketable securities are classified as available-for-sale and carried at fair value, with unrealized gains/losses excluded from net income and accounted for in other comprehensive loss[55](index=55&type=chunk) - All costs incurred in performing research and development activities are expensed as incurred[88](index=88&type=chunk) [3. Cash, cash equivalents and restricted cash](index=29&type=section&id=3.%20Cash%2C%20cash%20equivalents%20and%20restricted%20cash) Details the composition of cash, cash equivalents, and restricted cash balances 3. Cash, cash equivalents and restricted cash Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :----------------------------- | :------------------- | :-------------------- | | Cash | 2,911,404 | 2,910,446 | | Money market | 3,076,798 | 4,401,165 | | Total cash and cash equivalents | 5,988,202 | 7,311,611 | | Restricted cash | 31,122 | 30,818 | - Restricted cash is held as a guarantee for expenses under the company's office lease agreement in Lugano[98](index=98&type=chunk) [4. Marketable Securities](index=30&type=section&id=4.%20Marketable%20Securities) Provides a breakdown of marketable securities, primarily U.S. Treasury Securities, and their fair values 4. Marketable Securities Table | Category | Amortized Cost ($) | Estimated Fair Value ($) | Gross Unrealized Gains ($) | Gross Unrealized Losses ($) | | :--------------------------------------- | :----------------- | :----------------------- | :----------------------- | :------------------------ | | Debt Securities - U.S. government treasury securities, current | 11,879,563 | 11,827,528 | — | (52,035) | | Debt Securities - U.S. government treasury securities, non-current | 987,370 | 988,388 | 1,018 | — | | Totals | 12,866,933 | 12,815,916 | 1,018 | (52,035) | - Marketable securities are primarily U.S. Treasury Securities with maturities from **April 2023 to April 2024**, classified as available-for-sale[99](index=99&type=chunk) [5. Prepaid Expenses and Other Current Assets](index=30&type=section&id=5.%20Prepaid%20Expenses%20and%20Other%20Current%20Assets) Itemizes the company's prepaid expenses and other current assets 5. Prepaid Expenses and Other Current Assets Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :----------------------------- | :------------------- | :-------------------- | | Tax credits | 137,383 | 103,877 | | Prepaid and deferred expenses | 730,108 | 552,882 | | Other receivables | 33,261 | 87,430 | | Prepaid D&O insurance costs | 605,502 | 208,542 | | Total | 1,368,871 | 848,854 | [6. Property and Equipment, net](index=32&type=section&id=6.%20Property%20and%20Equipment%2C%20net) Presents the net value of property and equipment after accumulated depreciation 6. Property and Equipment, net Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :----------------------- | :------------------- | :-------------------- | | Total property and equipment | 206,899 | 197,708 | | Less: accumulated depreciation | (62,263) | (53,329) | | Property and equipment, net | 144,636 | 144,379 | - Depreciation expense for the three months ended March 31, 2023, was **$8,300**, up from **$5,300** in the same period of 2022[103](index=103&type=chunk) [7. Operating Lease; Right of Use ("ROU") Assets](index=32&type=section&id=7.%20Operating%20Lease%3B%20Right%20of%20Use%20%28%22ROU%22%29%20Assets) Details the company's operating lease assets and liabilities, including lease terms and discount rates 7. Operating Lease; Right of Use ("ROU") Assets Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :------------------------------ | :------------------- | :-------------------- | | Operating lease- right of use assets | 609,877 | 659,933 | | Operating lease liability - current | 232,507 | 229,080 | | Operating lease liability - non-current | 385,922 | 441,784 | | Weighted average remaining lease term - years | 2.90 | 3.05 | | Weighted average discount rate | 1.52 | 1.53 | - Operating lease costs for the three months ended March 31, 2023, were **$60,601**, compared to **$58,170** in the prior year[106](index=106&type=chunk) [8. Accounts Payable](index=33&type=section&id=8.%20Accounts%20Payable) Reports the company's accounts payable balance as of the reporting dates - Accounts payable increased to **$2.2 million** as of **March 31, 2023**, from **$1.6 million** as of December 31, 2022[108](index=108&type=chunk) [9. Other Current Liabilities and Deferred Income](index=33&type=section&id=9.%20Other%20Current%20Liabilities%20and%20Deferred%20Income) Itemizes other current liabilities and deferred income balances 9. Other Current Liabilities and Deferred Income Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :------------------------------------ | :------------------- | :-------------------- | | Payable for social security and withholding taxes | 225,772 | 256,798 | | Accrued payroll | 990,025 | 660,556 | | Accrued expenses | 1,263,183 | 1,082,091 | | Tax provision | 120,783 | 107,311 | | Total other current liabilities | 2,599,763 | 2,106,756 | | Deferred income | — | 55,180 | [10. Pension Obligations](index=34&type=section&id=10.%20Pension%20Obligations) Details the company's funded status and costs related to pension obligations 10. Pension Obligations Table | Metric | March 31, 2023 ($) | December 31, 2022 ($) | | :--------------------------------- | :------------------- | :-------------------- | | Funded status at end of period | (164,568) | (157,580) | | Service cost (3 months ended Mar 31) | 35,078 | 169,709 | | Interest cost (3 months ended Mar 31) | 4,675 | 3,376 | [11. Loans](index=34&type=section&id=11.%20Loans) Describes the company's zero-interest loan and its future payment schedule - The company has a **CHF 638,000** (**$700,221** historical FX rate) **nine-year**, zero-interest loan obtained in August 2020, with quarterly payments of **CHF 20,000** starting December 31, 2021, and ending September 30, 2029[113](index=113&type=chunk) 11. Loans Table | Year | Loan Payments ($) | | :--------- | :---------------- | | Total | 587,496 | | 2024 | 109,200 | | 2025 | 87,360 | | 2026 | 87,360 | | 2027 | 87,360 | | 2028 | 87,360 | | Thereafter | 128,856 | [12. Fair Value Measurement](index=34&type=section&id=12.%20Fair%20Value%20Measurement) Presents financial assets measured at fair value, categorized by input levels 12. Fair Value Measurement Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :--------------------------------------- | :------------------- | :-------------------- | | Marketable securities available for sale | 12,815,916 | 14,768,442 | | Cash equivalents: Money market funds | 3,076,798 | 4,401,165 | | Total financial assets | 15,892,714 | 19,169,607 | - All financial assets measured at fair value are classified as **Level 1** inputs, based on quoted prices in active markets[116](index=116&type=chunk)[119](index=119&type=chunk) [13. Common and Preferred Stock](index=36&type=section&id=13.%20Common%20and%20Preferred%20Stock) Details the number of common shares issued and outstanding, and recent ATM program activities - As of March 31, 2023, **12,087,142 shares** of common stock were issued and outstanding, compared to **11,883,368 shares** as of December 31, 2022[120](index=120&type=chunk) - During the three months ended March 31, 2023, the company sold **203,774 shares** of common stock under its ATM Program at an average price of **$4.48 per share**, generating aggregate net proceeds of **$0.8 million**[121](index=121&type=chunk)[122](index=122&type=chunk) [14. Equity Incentive Plans](index=38&type=section&id=14.%20Equity%20Incentive%20Plans) Outlines stock option, restricted stock unit, and performance restricted stock unit plans [Stock Option Grants](index=38&type=section&id=Stock%20Option%20Grants) Provides details on stock options outstanding, their fair value, and exercise prices Stock Option Grants Table | Metric | Dec 31, 2022 | Mar 31, 2023 | | :--------------------------------- | :----------- | :----------- | | Options outstanding | 1,879,662 | 2,438,512 | | Weighted Average Grant Date Fair Value | $2.94 | $3.05 | | Weighted Average Exercise Price | $4.42 | $4.50 | - **558,850 options** were granted during the three months ended March 31, 2023, with a weighted average grant date fair value of **$3.46**[128](index=128&type=chunk)[131](index=131&type=chunk) [Restricted Stock Units and Performance Restricted Stock Units](index=39&type=section&id=Restricted%20Stock%20Units%20and%20Performance%20Restricted%20Stock%20Units) Details outstanding restricted stock units, their fair value, and vesting periods Restricted Stock Units and Performance Restricted Stock Units Table | Metric | Dec 31, 2022 | Mar 31, 2023 | | :--------------------------------- | :----------- | :----------- | | RSUs outstanding | 103,050 | 295,550 | | Weighted average Grant Date Fair Value | $3.48 | $3.97 | | Weighted average Duration of Vesting Period (years) | 2.40 | 3.44 | - **192,500 RSUs** were granted during the three months ended March 31, 2023, with a weighted-average grant date fair value of **$4.24**[133](index=133&type=chunk) - Total unrecognized compensation cost related to non-vested stock options and RSUs was **$5.7 million** as of March 31, 2023, expected to be recognized over **4 years**[135](index=135&type=chunk) [15. Warrants](index=40&type=section&id=15.%20Warrants) Describes the outstanding equity-classified warrants, including exercise prices and expiration dates - **237,249** equity-classified warrants are outstanding with an exercise price of **$5.07 per share**, exercisable until **July 20, 2025**[136](index=136&type=chunk) - An additional **200,000** equity-classified warrants are outstanding with an exercise price of **$13.75 per share**, exercisable for **four years** from May 6, 2021[137](index=137&type=chunk) [16. Collaboration Agreement](index=40&type=section&id=16.%20Collaboration%20Agreement) Discusses the concluded multi-target collaboration agreement with Zentalis for cancer treatment - The multi-target collaboration agreement with Zentalis to discover new product candidates for cancer treatment has concluded[138](index=138&type=chunk)[141](index=141&type=chunk) [17. Net loss per common share](index=42&type=section&id=17.%20Net%20loss%20per%20common%20share) Presents basic and diluted net loss per common share and weighted-average shares outstanding 17. Net loss per common share Table | Metric | 3 Months Ended Mar 31, 2023 | 3 Months Ended Mar 31, 2022 | | :--------------------------------------- | :-------------------------- | :-------------------------- | | Net loss per share - basic and diluted | (0.43) | (0.28) | | Weighted-average common shares - basic and diluted | 11,935,081 | 11,883,368 | - Potentially dilutive securities (stock options, RSUs, PRSUs, and warrants) were excluded from diluted EPS calculation as their effect would be anti-dilutive due to the company's net losses[94](index=94&type=chunk)[142](index=142&type=chunk)[143](index=143&type=chunk) [18. Related Parties](index=42&type=section&id=18.%20Related%20Parties) Identifies related parties and outlines the exclusive license agreement with Minoryx Therapeutics SL - Minoryx Therapeutics SL is a related party due to shared board chairmanship[144](index=144&type=chunk) - The company has an exclusive worldwide license agreement with Minoryx for intellectual property, with royalty payments of **8%** on net revenues from products infringing composition of matter claims or Minoryx molecules, and **3%** on net revenues from products infringing method claims or Minoryx know-how[144](index=144&type=chunk)[147](index=147&type=chunk) [19. Commitments](index=42&type=section&id=19.%20Commitments) Details the company's research commitments for the current fiscal year - As of March 31, 2023, the company had **$3.2 million** in research commitments for activities to be performed in **fiscal year 2023**[146](index=146&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations.](index=44&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations.) Analyzes Gain Therapeutics' business, financial condition, and operating results for Q1 2023 vs Q1 2022 [Overview](index=44&type=section&id=Overview) Provides a high-level description of Gain Therapeutics' biotechnology focus and strategic operational plans - Gain Therapeutics is a biotechnology company developing novel small molecule therapeutics for CNS disorders, lysosomal storage disorders (LSDs), metabolic disorders, and oncology using its SEE-Tx® platform[150](index=150&type=chunk) - The lead product candidate, GT-02287 for GBA1 Parkinson's disease, has completed IND-enabling toxicology studies, with a **Phase 1 clinical trial** expected to begin in Australia in the **second half of 2023**[151](index=151&type=chunk) - The company is streamlining operational plans to become more capital efficient, focusing on Parkinson's and Gaucher disease programs, and seeking non-dilutive funding for other research programs, including Krabbe disease and alpha-1 antitrypsin deficiency, and new oncology programs[153](index=153&type=chunk)[154](index=154&type=chunk) [Recent Developments](index=46&type=section&id=Recent%20Developments) Highlights recent grants awarded and key personnel appointments - Awarded a **€1.2 million** (approx. **$1.3 million**) Eurostars and Innosuisse grant for the alpha-1 antitrypsin deficiency program, with research starting in **Q2 2023**[156](index=156&type=chunk) - Awarded a **CHF 2.5 million** (approx. **$2.8 million**) Innosuisse grant for the GBA1 Parkinson's disease program, with research starting in **Q2 2023**[157](index=157&type=chunk) - Appointed C. Evan Ballantyne as Chief Financial Officer on **May 3, 2023**[157](index=157&type=chunk) [Financial Condition](index=46&type=section&id=Financial%20Condition) Assesses the company's financial health, including accumulated deficit and capital runway - The company has incurred recurring losses and negative cash flows from operations since inception, with an accumulated deficit of **$43.6 million** as of **March 31, 2023** (vs. **$38.5 million** at Dec 31, 2022)[160](index=160&type=chunk) - As of **March 31, 2023**, cash, cash equivalents, and marketable securities totaled **$18.8 million**[160](index=160&type=chunk) - Management believes existing capital will fund operating expenses and capital expenditure requirements into the **third quarter of 2024**, but substantial additional financing is needed for product development and potential commercialization[161](index=161&type=chunk) [Financing Requirements; Current Financing Environment](index=48&type=section&id=Financing%20Requirements%3B%20Current%20Financing%20Environment) Discusses future capital needs and macroeconomic impacts on financing - Future cash needs are expected to be financed through public/private equity offerings (including ATM Program), debt financings, government/private grants, collaborations, strategic alliances, and licensing arrangements[162](index=162&type=chunk) - Worsening global macroeconomic conditions (war in Ukraine, geopolitical tension, inflation, rising interest rates, bank liquidity concerns, capital market volatility) may impact the ability to obtain additional financing on favorable terms[163](index=163&type=chunk) [Strategic Transactions; Collaboration and Licensing Agreement](index=48&type=section&id=Strategic%20Transactions%3B%20Collaboration%20and%20Licensing%20Agreement) Explains the company's approach to collaborations and licensing for its SEE-Tx® platform - The company enters into collaboration and licensing arrangements to use its SEE-Tx® computational platform technology to discover novel allosteric sites and identify proprietary small molecules[164](index=164&type=chunk) [Components of Our Consolidated Results of Operations](index=48&type=section&id=Components%20of%20Our%20Consolidated%20Results%20of%20Operations) Details primary components of consolidated results: limited revenue and significant R&D and G&A expenses [Revenue](index=48&type=section&id=Revenue) Discusses the company's historical and expected revenue sources - The company has not generated any revenue from product sales and does not expect to in the foreseeable future[165](index=165&type=chunk) - Historically, limited collaboration revenue was derived from the Zentalis collaboration agreement[165](index=165&type=chunk) [Total Operating Expenses](index=48&type=section&id=Total%20Operating%20Expenses) Explains the composition of the company's operating expenses - Operating expenses since inception have consisted solely of research and development and general and administrative costs[166](index=166&type=chunk) [Research and Development Expenses](index=48&type=section&id=Research%20and%20Development%20Expenses) Analyzes the trends and drivers of research and development expenditures Research and Development Expenses Table | Category | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Preclinical activities and outside services | 1,676,704 | 794,296 | | Personnel expenses | 980,576 | 726,954 | | Other | 133,925 | 69,899 | | Research grants | — | (34,709) | | Total research and development expenses | 2,791,205 | 1,556,440 | - The increase in R&D expenses was primarily due to increased third-party services related to Parkinson's and Gaucher disease programs[179](index=179&type=chunk) - R&D expenses are anticipated to increase substantially in future periods due to increased headcount, expanded infrastructure, increased insurance costs, and the commencement of clinical trials[169](index=169&type=chunk)[171](index=171&type=chunk) [General and Administrative Expenses](index=50&type=section&id=General%20and%20Administrative%20Expenses) Examines the changes and factors influencing general and administrative costs - General and administrative expenses increased by **$0.7 million** to **$2.5 million** for the three months ended March 31, 2023, compared to **$1.8 million** for the same period in 2022[180](index=180&type=chunk) - The increase was primarily due to higher legal fees (intellectual property and corporate matters), directors and officers insurance, professional accounting fees, and increased personnel-related costs (headcount and stock-based compensation)[180](index=180&type=chunk) [Other Financial Income (Expense)](index=52&type=section&id=Other%20Financial%20Income%20%28Expense%29) Presents non-operating financial income and expenses, including interest and foreign exchange Other Financial Income (Expense) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Interest income, net | 152,035 | (1,651) | | Foreign exchange gain/(loss), net | (42,842) | 19,162 | [Consolidated Results of Operations](index=52&type=section&id=Consolidated%20Results%20of%20Operations) Summarizes net loss and key drivers for Q1 2023 compared to 2022 [Comparison of the Three Months ended March 31, 2023 and 2022](index=52&type=section&id=Comparison%20of%20the%20Three%20Months%20ended%20March%2031%2C%202023%20and%202022) Provides a detailed comparative analysis of financial performance between the two periods [Revenues](index=52&type=section&id=Revenues) Compares total revenues for the three months ended March 31, 2023, and 2022 Revenues Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :----------------------- | :------------------------------ | :------------------------------ | | Total revenues | 55,180 | 45,006 | [Research and Development Expenses](index=54&type=section&id=Research%20and%20Development%20Expenses) Compares research and development expenses for the three months ended March 31, 2023, and 2022 Research and Development Expenses Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Research and development expenses | 2,791,205 | 1,556,440 | [General and Administrative Expenses](index=54&type=section&id=General%20and%20Administrative%20Expenses) Compares general and administrative expenses for the three months ended March 31, 2023, and 2022 General and Administrative Expenses Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | General and administrative expenses | 2,493,759 | 1,777,043 | [Liquidity and Capital Resources](index=54&type=section&id=Liquidity%20and%20Capital%20Resources) Assesses the company's ability to meet short-term and long-term financial obligations - As of **March 31, 2023**, cash, cash equivalents, and marketable securities totaled **$18.8 million** (down from **$22.1 million** at Dec 31, 2022)[182](index=182&type=chunk) - The company had an accumulated deficit of **$43.6 million** as of **March 31, 2023**[182](index=182&type=chunk) - Management believes existing capital will fund operating expenses and capital expenditure requirements into the **third quarter of 2024**[182](index=182&type=chunk) - The company has approximately **$15 million** of remaining capacity under its ATM Program as of **March 31, 2023**[184](index=184&type=chunk) [Cash Flows](index=56&type=section&id=Cash%20Flows) Analyzes cash flows from operating, investing, and financing activities for Q1 2023 and 2022 [Cash Flows from Operating Activities](index=56&type=section&id=Cash%20Flows%20from%20Operating%20Activities) Details cash used in operating activities for the three months ended March 31, 2023, and 2022 Cash Flows from Operating Activities Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Cash used in operating activities | (4,217,415) | (2,447,391) | [Cash Flows from Investing Activities](index=56&type=section&id=Cash%20Flows%20from%20Investing%20Activities) Details cash from investing activities for Q1 2023 and 2022 Cash Flows from Investing Activities Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Cash provided by/(used in) investing activities | 2,106,980 | (7,825) | - Investing activities were positively impacted by **$4.1 million** from marketable securities maturities, partially offset by **$2.0 million** in purchases[190](index=190&type=chunk) [Cash Flows from Financing Activities](index=56&type=section&id=Cash%20Flows%20from%20Financing%20Activities) Details cash from financing activities for Q1 2023 and 2022 Cash Flows from Financing Activities Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Cash provided by/(used in) financing activities | 748,908 | (37,974) | | Net (decrease)/increase in cash, cash equivalents and restricted cash | (1,323,105) | (2,542,507) | | Cash, cash equivalents and restricted cash at end of period | 6,019,324 | 34,369,445 | - Cash provided by financing activities was mainly related to the ATM Program[191](index=191&type=chunk) [Funding Requirements](index=57&type=section&id=Funding%20Requirements) Discusses the factors influencing future funding needs and potential financing strategies - Future funding requirements depend on the scope, timing, progress, and results of R&D, preclinical studies, and clinical trials for product candidates[193](index=193&type=chunk)[194](index=194&type=chunk) - The company expects to finance operations through equity offerings, debt financings, government/private grants, collaborations, strategic alliances, and licensing arrangements[194](index=194&type=chunk) - Inability to obtain additional funds on acceptable terms could lead to delays, reductions, or elimination of R&D programs, commercialization efforts, or curtailment of operations[194](index=194&type=chunk) [Critical Accounting Policies and Use of Estimates](index=58&type=section&id=Critical%20Accounting%20Policies%20and%20Use%20of%20Estimates) Highlights key accounting policies and estimates requiring significant management judgment - The preparation of financial statements requires estimates and judgments related to accrued expenses, defined benefit pension liability, share-based compensation, and research grants[195](index=195&type=chunk) - No material changes to critical accounting policies occurred during the three months ended March 31, 2023[195](index=195&type=chunk) [JOBS Act](index=58&type=section&id=JOBS%20Act) Explains the company's status as an "emerging growth company" and associated reporting exemptions - The company qualifies as an **"emerging growth company"** under the JOBS Act, allowing reliance on reduced reporting requirements[196](index=196&type=chunk)[419](index=419&type=chunk) - Exemptions include reporting only **two years** of audited financial statements, exemption from auditor attestation for internal control over financial reporting, and reduced executive compensation disclosures[196](index=196&type=chunk)[419](index=419&type=chunk) - The **"emerging growth company"** status will continue until the earliest of **December 31, 2026**, or if annual gross revenue exceeds **$1.07 billion**, or if **$1.0 billion** in non-convertible debt is issued, or if market value of non-affiliate held common stock exceeds **$700 million**[197](index=197&type=chunk)[420](index=420&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=58&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) States that as a "smaller reporting company," Gain Therapeutics is exempt from market risk disclosures - As a **"smaller reporting company,"** Gain Therapeutics is not required to provide quantitative and qualitative disclosures about market risk[198](index=198&type=chunk) [Item 4. Controls and Procedures](index=58&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective, with no material changes in internal control [Evaluation of Disclosure Controls and Procedures](index=58&type=section&id=Evaluation%20of%20Disclosure%20Controls%20and%20Procedures) Management's assessment of the effectiveness of disclosure controls and procedures - Disclosure controls and procedures were evaluated by management, including the CEO and CFO, and concluded to be effective at the reasonable assurance level as of **March 31, 2023**[201](index=201&type=chunk) [Changes in Internal Control over Financial Reporting](index=60&type=section&id=Changes%20in%20Internal%20Control%20over%20Financial%20Reporting) Reports on any material changes in internal control over financial reporting during the period - No material changes in internal control over financial reporting occurred during the period covered by this Quarterly Report[202](index=202&type=chunk) [Inherent Limitations on Effectiveness of Controls](index=60&type=section&id=Inherent%20Limitations%20on%20Effectiveness%20of%20Controls) Acknowledges the inherent limitations of control systems in providing absolute assurance - Control systems provide reasonable assurance but cannot guarantee absolute assurance that objectives will be met or that all misstatements/fraud will be detected[203](index=203&type=chunk) PART II—OTHER INFORMATION Presents additional information not covered in financial statements, including legal, risk, and equity details [Item 1. Legal Proceedings.](index=60&type=section&id=Item%201.%20Legal%20Proceedings.) The company is not involved in material legal proceedings, nor aware of any threatened actions - The company is not currently a party to any material legal proceedings[205](index=205&type=chunk) - No pending or threatened legal proceedings are known that could adversely affect the business, operating results, or financial condition[205](index=205&type=chunk) [Item 1A. Risk Factors.](index=60&type=page&id=Item%201A.%20Risk%20Factors.) Details significant risks impacting operations, financial condition, and stock price [Risk Factor Summary](index=60&type=section&id=Risk%20Factor%20Summary) Provides a concise overview of the company's most significant operational and financial risks - The company has a history of operating losses and expects to incur losses for the foreseeable future, with no guarantee of generating revenues or achieving profitability[208](index=208&type=chunk)[211](index=211&type=chunk) - Significant risks include the inability to initiate or complete preclinical/clinical trials, challenges in patient enrollment for low-prevalence disorders, and the uncertainty of developing novel product candidates[213](index=213&type=chunk) - The company will need to raise additional capital, which may dilute stockholders, restrict operations, or require relinquishing rights to technologies/product candidates[213](index=213&type=chunk) - Other key risks include extensive government regulation, potential lack of market acceptance for approved products, reliance on licensed intellectual property (SEE-Tx® platform), and the adverse effects of global macroeconomic conditions and data privacy/security laws[213](index=213&type=chunk) [Risks Related to Our Business](index=62&type=section&id=Risks%20Related%20to%20Our%20Business) Discusses risks inherent to the business model, including operating losses and future success uncertainty - The company has a history of operating losses and expects to incur losses for the foreseeable future, with no significant revenues generated to date[211](index=211&type=chunk) - Future success is uncertain due to dependence on product candidate development, ability to scale operations, establish brand, expand to meet commercial demand, and minimize security risks[212](index=212&type=chunk) - Operating results are expected to fluctuate due to factors such as funding, preclinical/clinical trial progress, regulatory approvals, market acceptance, competition, and global macroeconomic conditions[217](index=217&type=chunk)[222](index=222&type=chunk) [Risks Related to Product Development, Regulatory Approval, Manufacturing and Commercialization](index=66&type=section&id=Risks%20Related%20to%20Product%20Development%2C%20Regulatory%20Approval%2C%20Manufacturing%20and%20Commercialization) Outlines risks in developing, approving, manufacturing, and commercializing product candidates - Conducting clinical trials outside the U.S. (e.g., Australia for Parkinson's program) carries risks, as the FDA may not accept foreign data, potentially delaying or halting development[221](index=221&type=chunk)[224](index=224&type=chunk) - Low prevalence of target disorders may lead to difficulties and delays in patient identification and enrollment for clinical trials, increasing development costs[233](index=233&type=chunk)[234](index=234&type=chunk) - Product candidates are novel and in early development, requiring extensive testing; success in early preclinical studies is not indicative of later clinical trial results, and there's a high failure rate in late-stage trials[239](index=239&type=chunk)[241](index=241&type=chunk)[243](index=243&type=chunk) - Product candidates may cause serious adverse events (SAEs) or undesirable side effects, which could delay or prevent marketing approval, or lead to market withdrawal or sales limitations[269](index=269&type=chunk)[270](index=270&type=chunk) [Risks Related to Our Financial Condition and Capital Requirements; Competition](index=83&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Requirements%3B%20Competition) Addresses risks concerning financial stability, capital needs, and industry competition - Substantial additional capital is required for R&D, preclinical/clinical trials, and marketing, which may dilute stockholders, restrict operations, or require relinquishing rights to technologies/product candidates[278](index=278&type=chunk)[280](index=280&type=chunk) - The company faces intense competition from existing and future drugs marketed by large companies with substantially greater resources[283](index=283&type=chunk)[286](index=286&type=chunk) - Competition and technological change may render product candidates and technologies less attractive or obsolete, as other companies may develop products earlier, obtain approval faster, or develop more effective treatments[289](index=289&type=chunk)[290](index=290&type=chunk) [Risks Related to Our Intellectual Property](index=89&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Details risks concerning intellectual property, including licensing, patents, and trade secrets - The company is significantly dependent on its exclusive license with Minoryx Therapeutics S.L. for the SEE-Tx® platform; termination of this license would materially adversely affect the business[298](index=298&type=chunk)[299](index=299&type=chunk) - Ability to obtain and enforce patents is uncertain; patents may be challenged, invalidated, or circumvented, and may not provide significant protection against competitors[300](index=300&type=chunk)[304](index=304&type=chunk) - Reliance on trade secrets, confidentiality, and security measures may not adequately protect proprietary information, and others may independently develop similar technologies[308](index=308&type=chunk) - Involvement in intellectual property lawsuits (infringement, validity challenges) could be expensive, time-consuming, divert management attention, and result in significant liabilities or restrictions on product sales[310](index=310&type=chunk)[311](index=311&type=chunk)[316](index=316&type=chunk) [Risks Related to Third Parties and Collaborators](index=95&type=section&id=Risks%20Related%20to%20Third%20Parties%20and%20Collaborators) Explains risks associated with reliance on third-party contractors and collaboration agreements - Reliance on third parties (CROs, consultants) for preclinical studies and clinical trials means less control over timing and execution, with potential for delays, increased costs, or denial of regulatory approval if performance is unsatisfactory or non-compliant with GCPs[226](index=226&type=chunk)[321](index=321&type=chunk)[322](index=322&type=chunk) - Dependence on third-party CMOs for manufacturing product candidates for clinical trials and commercial sale poses risks of insufficient quantities, unacceptable costs, supply chain disruptions (e.g., COVID-19, geopolitical conflicts), and non-compliance with cGMPs, which could delay or prevent development and commercialization[327](index=327&type=chunk)[329](index=329&type=chunk)[332](index=332&type=chunk)[334](index=334&type=chunk) - Collaborations are crucial but carry risks of termination, partners changing focus, insufficient resources, or disputes over proprietary rights, potentially delaying or terminating development and commercialization programs[336](index=336&type=chunk)[343](index=343&type=chunk)[346](index=346&type=chunk) - Failure to establish internal marketing, sales, and distribution capabilities or successful third-party arrangements could limit market creation and product sales, impacting revenues[338](index=338&type=chunk)[341](index=341&type=chunk)[342](index=342&type=chunk) [General Risk Factors](index=103&type=section&id=General%20Risk%20Factors) Covers broad risks such as internal controls, macroeconomic conditions, personnel, and regulatory compliance - The company previously identified and remediated material weaknesses in internal control over financial reporting, but future failures could lead to misstatements and harm business[347](index=347&type=chunk)[348](index=348&type=chunk)[349](index=349&type=chunk) - Global and macroeconomic conditions (credit market volatility, bank failures, inflation, geopolitical conflicts) could adversely affect revenue, financial condition, and operating goals, potentially delaying or abandoning development plans[351](index=351&type=chunk)[352](index=352&type=chunk) - Future growth requires expanding operations, increasing headcount, and improving controls, which may be difficult to manage effectively[353](index=353&type=chunk)[354](index=354&type=chunk) - Dependence on key personnel and the ability to attract/retain qualified employees is critical; loss of senior management or inability to hire specialized staff could harm business[355](index=355&type=chunk)[356](index=356&type=chunk)[357](index=357&type=chunk) - Relationships with healthcare providers and payors are subject to federal and state healthcare fraud and abuse laws, false claims laws, and privacy laws (e.g., Anti-Kickback Statute, False Claims Act, HIPAA), with potential for substantial penalties for non-compliance[362](index=362&type=chunk)[364](index=364&type=chunk)[367](index=367&type=chunk) - Market acceptance and sales depend on adequate reimbursement from third-party payors; cost containment measures and healthcare legislative reforms (e.g., ACA, IRA) could limit coverage and reduce drug prices[369](index=369&type=chunk)[370](index=370&type=chunk)[371](index=371&type=chunk)[375](index=375&type=chunk) - International operations expose the company to risks like different regulatory requirements, reduced intellectual property protection, economic/political instability, and compliance with anti-corruption laws (e.g., FCPA)[379](index=379&type=chunk)[380](index=380&type=chunk)[384](index=384&type=chunk) - Product liability lawsuits related to clinical trials or commercialized products could result in substantial liabilities, even with insurance, and harm the business[385](index=385&type=chunk)[386](index=386&type=chunk) - Stringent and evolving data privacy and security laws (e.g., HIPAA, CCPA, GDPR) and cyber threats (ransomware, supply-chain attacks) pose risks of regulatory actions, litigation, fines, business disruptions, and reputational harm[387](index=387&type=chunk)[390](index=390&type=chunk)[391](index=391&type=chunk)[392](index=392&type=chunk)[397](index=397&type=chunk)[398](index=398&type=chunk)[399](index=399&type=chunk)[400](index=400&type=chunk)[405](index=405&type=chunk) [Risks Related to Ownership of Our Common Stock](index=124&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Common%20Stock) Discusses risks pertinent to common stock ownership, including price volatility and dilution - The market price for common stock has been and is likely to remain highly volatile, influenced by R&D results, financial estimates, competition, regulatory actions, and macroeconomic conditions[410](index=410&type=chunk)[413](index=413&type=chunk)[415](index=415&type=chunk) - Securities class action litigation is a risk for volatile stocks, potentially leading to substantial costs and diversion of management attention[413](index=413&type=chunk) - Failure to meet Nasdaq listing requirements could result in delisting, leading to limited market quotations, reduced trading activity, and decreased ability to raise future financing[414](index=414&type=chunk)[416](index=416&type=chunk) - Operating as a public company incurs increased costs and requires substantial management time for compliance initiatives and corporate governance[414](index=414&type=chunk) - The company does not anticipate paying dividends, so stockholders must rely on stock appreciation for returns, which is uncertain[424](index=424&type=chunk) - Sales of a substantial number of shares by existing stockholders or future equity raises could cause the stock price to fall and dilute existing ownership[425](index=425&type=chunk)[426](index=426&type=chunk) - Anti-takeover provisions in organizational documents and Delaware law might discourage or delay acquisition attempts[429](index=429&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=134&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) Reports no unregistered equity sales and details the use of proceeds from the March 2021 IPO [Sales of Unregistered Securities](index=134&type=section&id=Sales%20of%20Unregistered%20Securities) Confirms that no unregistered sales of equity securities occurred during the period - No unregistered sales of equity securities occurred[435](index=435&type=chunk) [Use of Proceeds from Initial Public Offering of Common Stock](index=134&type=section&id=Use%20of%20Proceeds%20from%20Initial%20Public%20Offering%20of%20Common%20Stock) Details the net proceeds from the IPO and confirms no material change in their planned use - The IPO, closed on **March 22, 2021**, generated net proceeds of **$40.5 million** from **4,181,818 common shares** at **$11.00 per share**[436](index=436&type=chunk) - There has been no material change in the planned use of proceeds from the IPO[437](index=437&type=chunk) [Item 3. Defaults Upon Senior Securities.](index=134&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities.) States that this item is not applicable to the company - Not applicable[438](index=438&type=chunk) [Item 4. Mine Safety Disclosures](index=134&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) States that this item is not applicable to the company - Not applicable[439](index=439&type=chunk) [Item 5. Other Information](index=134&type=section&id=Item%205.%20Other%20Information) States that this item is not applicable to the company - Not applicable[440](index=440&type=chunk) [Item 6. Exhibits](index=136&type=section&id=Item%206.%20Exhibits) Lists all exhibits filed with the Form 10-Q, including certifications and XBRL documents - The report includes certifications from the Principal Executive Officer and Principal Financial Officer (**Exhibits 31.1, 31.2, 32.1**)[442](index=442&type=chunk)[443](index=443&type=chunk) - Inline XBRL Instance Document and Taxonomy Extension Documents are filed as **Exhibits 101

Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant's executive officers during the relevant recovery period pursuant to § 240.10D-1(b). ☐ Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Act). Yes ☐ No ☒ The aggregate market value of the common equity held by non-affiliates of the Registrant on June 30, 2022 (the last business day of ...

[PART I FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20FINANCIAL%20INFORMATION) Presents the unaudited condensed consolidated financial statements and management's discussion for the period ended September 30, 2022, along with disclosures on market risk and controls [Item 1. Condensed Consolidated Financial Statements](index=5&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements) Presents the unaudited condensed consolidated financial statements for Gain Therapeutics, Inc., including balance sheets, statements of operations, and cash flows, reporting a **$12.9 million net loss** for the nine months ended September 30, 2022 [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Details the company's financial position, including assets, liabilities, and stockholders' equity as of September 30, 2022, and December 31, 2021 Condensed Consolidated Balance Sheet Highlights (Unaudited) | Balance Sheet Item | Sep 30, 2022 | Dec 31, 2021 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $10,879,884 | $36,880,673 | | Marketable securities (current & non-current) | $14,766,944 | $0 | | Total current assets | $24,349,323 | $37,722,044 | | Total assets | $28,325,619 | $38,985,071 | | **Liabilities & Stockholder's Equity** | | | | Total current liabilities | $4,332,293 | $2,552,546 | | Total liabilities | $5,662,858 | $4,167,525 | | Accumulated deficit | ($33,841,731) | ($20,925,459) | | Total stockholders' equity | $22,662,761 | $34,817,546 | [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Presents the company's revenues, expenses, and net loss for the three and nine months ended September 30, 2022 and 2021 Condensed Consolidated Statements of Operations (Unaudited) | Metric | Three Months Ended Sep 30, 2022 | Three Months Ended Sep 30, 2021 | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | | :--- | :--- | :--- | :--- | :--- | | Total revenues | $0 | $27,345 | $140,108 | $121,786 | | Research and development | ($1,964,784) | ($2,476,739) | ($6,103,448) | ($5,691,756) | | General and administrative | ($2,786,200) | ($2,186,531) | ($7,252,506) | ($5,047,434) | | Loss from operations | ($4,750,984) | ($4,635,925) | ($13,215,846) | ($10,617,404) | | Net loss | ($4,558,209) | ($4,663,533) | ($12,916,272) | ($10,670,673) | | Net loss per share (basic and diluted) | ($0.38) | ($0.39) | ($1.09) | ($1.11) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Outlines the cash inflows and outflows from operating, investing, and financing activities for the nine months ended September 30, 2022 and 2021 Condensed Consolidated Statements of Cash Flows (Unaudited) | Cash Flow Activity | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | | :--- | :--- | :--- | | Cash used in operating activities | ($10,787,526) | ($8,219,305) | | Cash used in investing activities | ($14,908,643) | ($83,114) | | Cash (used in)/provided by financing activities | ($57,192) | $41,776,510 | | Net (decrease)/increase in cash | ($26,003,209) | $33,510,361 | | Cash at beginning of period | $36,911,952 | $7,504,281 | | Cash at end of period | $10,908,743 | $41,014,642 | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed explanations and disclosures supporting the condensed consolidated financial statements - The company is a biotechnology firm developing small molecule therapeutics for CNS disorders, LSDs, and oncology using its in-licensed SEE-Tx® platform to discover novel allosteric binding sites[37](index=37&type=chunk) - Operations to date have focused on organization, financing, R&D, and preclinical studies, with **no approved products** and **no revenue from product sales**[39](index=39&type=chunk) - In March 2021, the company completed its IPO, raising aggregate net proceeds of **$40.56 million** after deducting underwriting discounts and offering expenses[43](index=43&type=chunk) - The company expects its cash, cash equivalents, and marketable securities as of September 30, 2022, to be sufficient to fund operations for at least the **next 12 months**[46](index=46&type=chunk)[59](index=59&type=chunk) - Under a collaboration agreement with Zentalis Pharmaceuticals, the company recognized **$133 thousand in revenues** for the nine months ended September 30, 2022[162](index=162&type=chunk)[165](index=165&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=31&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's therapeutic development, financial condition, and operating results, highlighting a **$12.9 million net loss** and extended cash runway into Q2 2024 [Overview and Recent Developments](index=31&type=section&id=Overview%20and%20Recent%20Developments) Provides an overview of the company's therapeutic development strategy and recent operational milestones, including pipeline advancements and cash runway extension - The company is developing small molecule therapeutics for CNS disorders, LSDs, and oncology using its SEE-Tx® platform to identify novel allosteric binding sites[172](index=172&type=chunk) - The lead compound, GT-02287 for GBA1 Parkinson's disease, is expected to complete IND-enabling studies in Q1 2023, with a Phase 1 first-in-human study planned to start in **mid-2023 in Australia**[174](index=174&type=chunk) - Due to the financing environment, the company has streamlined operations to focus on Parkinson's and Gaucher disease programs, extending its cash runway into **Q2 2024**[177](index=177&type=chunk)[225](index=225&type=chunk) [Results of Operations](index=35&type=section&id=Results%20of%20Operations) Analyzes the company's revenues, research and development, and general and administrative expenses, explaining changes in operating results for the period Comparison of Operating Results (Nine Months Ended Sep 30) | Item | 2022 | 2021 | Change | | :--- | :--- | :--- | :--- | | Total Revenues | $140,108 | $121,786 | $18,322 | | R&D Expenses | $6,103,448 | $5,691,756 | $411,692 | | G&A Expenses | $7,252,506 | $5,047,434 | $2,205,072 | | Net Loss | $12,916,272 | $10,670,673 | $2,245,599 | - The increase in R&D expenses for the nine months ended Sep 30, 2022, was primarily due to higher personnel-related costs from increased headcount and stock-based compensation, offset by a decrease in outside services[207](index=207&type=chunk) - The increase in G&A expenses for the nine months ended Sep 30, 2022, was mainly due to higher costs for corporate governance, legal fees, D&O insurance, professional services, and increased personnel costs as a public company[208](index=208&type=chunk) [Liquidity and Capital Resources](index=37&type=section&id=Liquidity%20and%20Capital%20Resources) Examines the company's cash position, capital requirements, and financing activities, detailing its ability to fund future operations - As of September 30, 2022, the company had **$25.6 million** in cash, cash equivalents, and marketable securities, with an accumulated deficit of **$33.8 million**[183](index=183&type=chunk)[210](index=210&type=chunk) - The company believes its existing cash, cash equivalents, and marketable securities will fund operating expenses and capital expenditure requirements into **Q2 2024**[210](index=210&type=chunk)[225](index=225&type=chunk) - On May 18, 2022, the company filed a shelf registration statement on Form S-3 for up to **$100 million** and a sales agreement for up to **$16 million** in an at-the-market offering, with **no shares sold** under these agreements as of September 30, 2022[186](index=186&type=chunk)[212](index=212&type=chunk) Cash Flow Summary (Nine Months Ended Sep 30) | Cash Flow Activity | 2022 | 2021 | | :--- | :--- | :--- | | Cash used in operating activities | ($10,787,526) | ($8,219,305) | | Cash used in investing activities | ($14,908,643) | ($83,114) | | Cash (used in)/provided by financing activities | ($57,192) | $41,776,510 | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=41&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, Gain Therapeutics, Inc. is not required to provide the information requested under this item - Not required for smaller reporting companies[233](index=233&type=chunk) [Item 4. Controls and Procedures](index=42&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were ineffective as of September 30, 2022, due to an un-remediated material weakness in financial reporting controls - Management concluded that disclosure controls and procedures were **not effective** as of September 30, 2022, due to a material weakness in internal control over financial reporting[235](index=235&type=chunk) - The material weakness relates to a lack of adequate procedures and controls to ensure accurate financial statements can be prepared and reviewed on a timely basis[237](index=237&type=chunk)[239](index=239&type=chunk) - The company is implementing a remediation plan, including a new ERP system which went live as of March 31, 2022, but the material weakness is not yet considered fully remediated[238](index=238&type=chunk)[239](index=239&type=chunk) [PART II OTHER INFORMATION](index=43&type=section&id=PART%20II%20OTHER%20INFORMATION) Presents additional information including legal proceedings, risk factors, equity sales, and other disclosures not covered in the financial statements [Item 1. Legal Proceedings](index=43&type=section&id=Item%201.%20Legal%20Proceedings) The company states that it is not currently a party to any material legal proceedings and is not aware of any pending or threatened legal actions that could have a material adverse effect on its business - The company is **not currently a party to any material legal proceedings**[244](index=244&type=chunk) [Item 1A. Risk Factors](index=43&type=section&id=Item%201A.%20Risk%20Factors) Outlines significant risks including a history of operating losses, dependence on novel product development, need for additional capital, reliance on third-party licenses, and uncertainties of clinical trials - The company has a history of operating losses and expects to incur losses for the foreseeable future, with **no guarantee of ever achieving profitability**[246](index=246&type=chunk)[249](index=249&type=chunk) - The company's business is highly dependent on a license from Minoryx Therapeutics for its core SEE-Tx® technology platform; termination of this license would have a **material adverse effect**[252](index=252&type=chunk)[334](index=334&type=chunk) - The company will need to raise additional capital, which may not be available on favorable terms, if at all, potentially forcing delays or elimination of R&D programs[252](index=252&type=chunk)[313](index=313&type=chunk) - The company's product candidates are novel and in early development; success in preclinical studies may not be indicative of results in later clinical trials, which are expensive, time-consuming, and have uncertain outcomes[246](index=246&type=chunk)[275](index=275&type=chunk)[277](index=277&type=chunk) - The business is subject to risks from global and macroeconomic conditions, including economic instability, pandemics (like COVID-19), and geopolitical conflicts, which could adversely affect operations and financing[252](index=252&type=chunk)[319](index=319&type=chunk)[391](index=391&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=81&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports no unregistered sales of equity securities during the period and confirms no material change in the planned use of **$40.5 million** net proceeds from its March 2021 IPO - There were **no unregistered sales of equity securities** in the period[469](index=469&type=chunk) - There has been **no material change** in the planned use of proceeds from the March 2021 IPO, which raised net proceeds of **$40.5 million**[470](index=470&type=chunk)[471](index=471&type=chunk) [Other Items (3, 4, 5)](index=81&type=section&id=Other%20Items%20(3,%204,%205)) Item 3 (Defaults Upon Senior Securities), Item 4 (Mine Safety Disclosures), and Item 5 (Other Information) are all noted as not applicable for this reporting period - Items 3, 4, and 5 are **not applicable**[472](index=472&type=chunk)[473](index=473&type=chunk)[474](index=474&type=chunk) [Item 6. Exhibits](index=82&type=section&id=Item%206.%20Exhibits) Lists exhibits filed with the Form 10-Q, including corporate governance documents, executive agreements, and officer certifications - Lists exhibits filed with the report, including corporate governance documents, executive agreements, and required officer certifications[476](index=476&type=chunk)

Table of Contents 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, 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: 001-40237 GAIN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 85-1726310 (Stat ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark one) 4800 Montgomery Lane, Suite 220 (Address of principal executive offices) (Zip Code) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 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: 001-40237 GAIN THERAPEUTICS, ...

Part I [Business](index=7&type=section&id=Item%201.%20Business) Gain Therapeutics is a preclinical biotech developing novel **STARs** for protein misfolding diseases via its **SEE-Tx® platform**, with lead programs in **Parkinson's** and **Gaucher disease** [Overview](index=7&type=section&id=Overview) Gain Therapeutics uses its **SEE-Tx® platform** to discover **STARs** for **LSDs** and **CNS** disorders, with lead programs for **Parkinson's** and **Gaucher disease** in preclinical development - The company uses its **Site-Directed Enzyme Enhancement Therapy (SEE-Tx®) platform** to discover novel allosteric binding sites on proteins and identify proprietary small molecules (**STARs**) to treat diseases like **LSDs** and **CNS** disorders[14](index=14&type=chunk) - Lead compounds for **Parkinson's disease** and **Gaucher disease** are in preclinical studies, with **positive in vitro data** showing **increased GCase protein levels** and **reduction of toxic substrates**[16](index=16&type=chunk) - The company plans to submit a **regulatory dossier** to initiate a **first-in-human Phase 1 clinical trial** for its **Parkinson's disease** program in the **second half of 2022**[17](index=17&type=chunk) - Completed its **Initial Public Offering (IPO)** on **March 17, 2021**, raising net proceeds of **approximately $40.5 million**[19](index=19&type=chunk)[20](index=20&type=chunk) [Our Platform for Computational Target and Drug Discovery](index=9&type=section&id=Our%20Platform%20for%20Computational%20Target%20and%20Drug%20Discovery) The **SEE-Tx® platform** discovers novel allosteric binding sites, identifying **STARs** to modulate protein function with high specificity and a **14%** validated compound hit rate - The **SEE-Tx® platform** uses **computational methods** to identify novel **allosteric binding sites** on proteins, which are distinct from the active site[22](index=22&type=chunk)[23](index=23&type=chunk) - Targeting **allosteric sites** allows for **improved specificity** and **multiple mechanisms of action**, including **protein stabilization**, **destabilization**, and **targeted degradation**[25](index=25&type=chunk)[29](index=29&type=chunk) - The platform's **validated-target approach** is **faster and more efficient** than traditional **high-throughput screening**, with an **average hit rate** for **validated compounds** of **14%**, a significant improvement over traditional methods[28](index=28&type=chunk) [Our Pipeline of STARs](index=15&type=section&id=Our%20Pipeline%20of%20STARs) The company's **STARs** pipeline targets **LSDs**, **CNS** disorders, metabolic diseases, and oncology, with lead preclinical programs **GT-02287** for **Parkinson's** and **GT-02329** for **Gaucher Disease** - The pipeline is built on the **SEE-Tx® platform** and is **disease-agnostic**, with an **initial focus** on **LSDs** and **CNS disorders**[37](index=37&type=chunk) - The lead compound for **Parkinson's Disease**, **GT-02287**, has demonstrated the ability to **increase GCase activity**, **improve lysosomal delivery**, and reduce toxic **alpha-synuclein** in preclinical models, showing **high brain exposure** with a **brain-to-plasma ratio greater than one**[48](index=48&type=chunk)[50](index=50&type=chunk)[55](index=55&type=chunk) - The lead compound for **Gaucher Disease**, **GT-02329**, has been shown to **increase GCase levels**, reduce toxic **glucosylsphingosine**, and **enhance GCase activity** in the brain and plasma in preclinical studies[63](index=63&type=chunk)[66](index=66&type=chunk)[68](index=68&type=chunk) - **Research-stage programs** are targeting enzymes related to **GM1 Gangliosidosis (GLB)**, **MPS1 (IDUA)**, **Krabbe Disease (GALC)**, and **Metachromatic Leukodystrophy (MLD)**[69](index=69&type=chunk)[70](index=70&type=chunk)[77](index=77&type=chunk)[87](index=87&type=chunk)[95](index=95&type=chunk) [Competition](index=35&type=section&id=Competition) The company faces intense competition from gene therapy developers and other firms like **Axovant**, **Lysogene**, **Prevail**, and **Apollo**, many with superior resources - The company faces **competition** from firms developing **gene therapy approaches** and other treatments for its target indications, including **Axovant**, **Lysogene**, **Prevail Therapeutics**, and **Apollo Therapeutics**[108](index=108&type=chunk) - Many competitors have **greater financial, R&D, and manufacturing resources**, which could allow them to establish a **strong market position** before Gain Therapeutics can enter the market[109](index=109&type=chunk) [Strategic Transactions; Collaboration and Licensing Arrangements](index=35&type=section&id=Strategic%20Transactions%3B%20Collaboration%20and%20Licensing%20Arrangements) The company's strategy includes collaborations and licensing, notably an **exclusive SEE-Tx® platform** license from **Minoryx** and a multi-target oncology collaboration with **Zentalis Pharmaceuticals** - The company has an **exclusive, worldwide license** from **Minoryx Therapeutics, S.L.** to use the **SEE-Tx® discovery platform**, which requires **royalty payments** on net revenues and a **1.25% milestone payment** on any consideration received from a **sale of the company**[117](index=117&type=chunk)[118](index=118&type=chunk)[119](index=119&type=chunk) - In **April 2021**, entered into a **multi-target collaboration** with **Zentalis Pharmaceuticals** to discover new **cancer treatments**, with the agreement including initiation fees, **R&D cost reimbursement**, and potential **milestone payments up to $41.5 million** plus **royalties**[112](index=112&type=chunk)[116](index=116&type=chunk) - A strategic research collaboration with **Sumitomo Dainippon Pharma** was **terminated**, with a formal notice expected after **March 22, 2022**[111](index=111&type=chunk) [Intellectual Property](index=39&type=section&id=Intellectual%20Property) The company protects its technology through patents and trade secrets, with **five pending European patent applications** and key in-licensed patents for **SEE-Tx®** (**expiring 2032**) and **GLB/GBA** programs (**expiring 2037**) - The company's **patent portfolio** consists of **five pending European patent applications** and related **national stage applications** as of **February 2022**[122](index=122&type=chunk) - Key **in-licensed patents** include one for the **SEE-Tx® Technology** (**expiring 2032**) and patent families for the **GLB** and **GBA programs** (applications, if issued, **expiring 2037**)[122](index=122&type=chunk)[123](index=123&type=chunk)[124](index=124&type=chunk) - The company also relies on **trade secrets**, **know-how**, and **confidentiality agreements** with **employees and collaborators** to protect its **proprietary information**[126](index=126&type=chunk) [Government Regulation](index=41&type=section&id=Government%20Regulation) The company's products face extensive **FDA** regulation, involving lengthy preclinical and clinical trials, with potential for **Orphan Drug Designation** and ongoing compliance with **cGMP**, **ACA**, and anti-fraud laws - The **FDA drug approval process** involves extensive **preclinical studies**, an **IND submission**, and typically **three phases of clinical trials (Phase I, II, III)** to establish **safety and efficacy**[132](index=132&type=chunk)[138](index=138&type=chunk)[139](index=139&type=chunk)[140](index=140&type=chunk) - The company may seek **Orphan Drug Designation**, which is for diseases affecting **fewer than 200,000 individuals** in the U.S. and provides a **seven-year period of marketing exclusivity** if the product is the first approved for that indication[154](index=154&type=chunk)[155](index=155&type=chunk) - The company is subject to **healthcare laws** such as the **Affordable Care Act (ACA)**, which impacts **drug pricing and reimbursement**, and federal/state **anti-kickback** and **false claims laws**[172](index=172&type=chunk)[179](index=179&type=chunk)[181](index=181&type=chunk) - Marketing products outside the U.S., particularly in the **European Economic Area (EEA)**, requires authorization through centralized or national procedures and compliance with regulations like the **GDPR for data privacy**[190](index=190&type=chunk)[191](index=191&type=chunk)[201](index=201&type=chunk) [Employees and Human Capital Resources](index=65&type=section&id=Employees%20and%20Human%20Capital%20Resources) As of **December 31, 2021**, the company had **29 employees** (**20** in **R&D**) across **Barcelona**, **Lugano**, and **Bethesda** - As of **December 31, 2021**, the company had **29 employees**, with **20 in R&D** and **9 in general and administrative roles**[206](index=206&type=chunk) - Employees are located in **Barcelona, Spain (12)**, **Lugano, Switzerland (14)**, and **Bethesda, Maryland (3)**[206](index=206&type=chunk) [Risk Factors](index=67&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including **operating losses**, need for **additional capital**, uncertain clinical trial outcomes, **SEE-Tx® platform** novelty, **Minoryx** license dependency, competition, and regulatory complexities - The company has a history of **operating losses** and expects to incur losses for the foreseeable future, with no guarantee of achieving profitability[213](index=213&type=chunk) - The company's success is highly dependent on its ability to raise **additional capital**; failure to do so could force delays or termination of development programs[221](index=221&type=chunk)[274](index=274&type=chunk) - The company's product candidates are novel and in early development, with success in **preclinical studies** not being indicative of future **clinical trial results**, and **clinical trials** are expensive, time-consuming, and have an uncertain outcome[231](index=231&type=chunk)[236](index=236&type=chunk)[242](index=242&type=chunk) - The business is significantly dependent on an **exclusive license** from **Minoryx Therapeutics** for its core **SEE-Tx® platform**; termination of this license would have a **material adverse effect** on operations[290](index=290&type=chunk)[291](index=291&type=chunk) - The ongoing **COVID-19 pandemic** and **global instability**, such as the conflict in **Ukraine**, could adversely affect operations, disrupt supply chains, and impact the ability to access capital[280](index=280&type=chunk)[344](index=344&type=chunk) - The company has identified and is remediating **material weaknesses** in its **internal control over financial reporting**, which could affect the accuracy and timeliness of its **financial statements**[341](index=341&type=chunk)[342](index=342&type=chunk)[343](index=343&type=chunk) [Unresolved Staff Comments](index=125&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) The company reports no unresolved staff comments from the **Securities and Exchange Commission** - None[395](index=395&type=chunk) [Properties](index=125&type=section&id=Item%202.%20Properties) The company leases facilities in **Bethesda**, **Lugano**, and **Barcelona** for its operations, with potential future expansion needs - Leases **1,568 sq ft** of **office space** in **Bethesda, Maryland** (**lease expires Sept 2024**)[396](index=396&type=chunk) - Leases **2,992 sq ft** of **office space** in **Lugano, Switzerland** (**lease expires May 2026**)[397](index=397&type=chunk) - Leases a total of **2,819 sq ft** of lab and **office space** in **Barcelona, Spain** across two agreements (**leases expire Dec 2025** and **Nov 2026**)[397](index=397&type=chunk)[398](index=398&type=chunk) [Legal Proceedings](index=126&type=section&id=Item%203.%20Legal%20Proceedings) The company is not currently involved in any **legal proceedings** - None[399](index=399&type=chunk) [Mine Safety Disclosures](index=126&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is **not applicable** to the company's business - Not applicable[400](index=400&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=127&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company's **common stock** trades on **Nasdaq** under **GANX**, with **54 holders** as of **February 28, 2022**, and no plans for **dividends** - **Common Stock** trades on the **Nasdaq Global Market** under the symbol "**GANX**"[402](index=402&type=chunk) - As of **February 28, 2022**, there were **approximately 54 holders of record**[402](index=402&type=chunk) - The company has no intention to declare or pay a **dividend** in the foreseeable future[403](index=403&type=chunk) [Selected Financial Data](index=127&type=section&id=Item%206.%20Selected%20Financial%20Data) This section has been **reserved** and contains no information - Reserved[404](index=404&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=127&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) For **2021**, the company reported a **net loss of $13.9 million**, driven by increased **R&D** and **G&A** expenses, with **$36.9 million** in cash as of **December 31, 2021**, expected to fund operations into **H2 2023** | Financial Metric | 2021 | 2020 | | :--- | :--- | :--- | | **Total Revenues** | **$164,994** | **$28,881** | | **Research and Development Expenses** | **$7,164,229** | **$2,259,204** | | **General and Administrative Expenses** | **$6,826,938** | **$1,249,126** | | **Loss from Operations** | **$(13,826,173)** | **$(3,479,449)** | | **Net Loss** | **$(13,890,606)** | **$(3,577,682)** | | **Net Loss Per Share** | **$(1.37)** | **$(1.33)** | - The increase in **R&D expenses** was primarily due to expanded research activities, including external collaborations for chemical synthesis and toxicology studies, and higher personnel costs[436](index=436&type=chunk) - The increase in **G&A expenses** was driven by costs associated with being a public company, including legal, accounting, and investor relations fees, as well as increased personnel and stock-based compensation costs[438](index=438&type=chunk) - As of **December 31, 2021**, the company had **cash and cash equivalents of $36.9 million** and an **accumulated deficit of $20.9 million**[412](index=412&type=chunk)[440](index=440&type=chunk) - The company believes its existing cash, including **IPO proceeds**, will fund **operating expenses** and **capital requirements** into the **second half of 2023**[454](index=454&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=145&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company faces **interest rate** and **foreign currency risk**, with minimal **interest rate risk** and **foreign currency risk** from **CHF** and **EUR** operations, where a **10% USD-CHF** change would impact **2021 net loss** by **$587,000** - Primary market risk exposures are **interest rate sensitivity** and **foreign currency exchange risk**[471](index=471&type=chunk)[473](index=473&type=chunk) - **Interest rate risk is minimal** due to the **short-term nature of cash equivalents** and **zero-interest debt**[471](index=471&type=chunk)[472](index=472&type=chunk) - **Foreign currency risk exists** due to **operations in Switzerland (CHF) and Spain (EUR)**, where a **hypothetical 10% change** in the **USD-CHF exchange rate** would have changed the **2021 net loss by approximately $587,000**[473](index=473&type=chunk) [Financial Statements and Supplementary Data](index=147&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section presents the company's **audited consolidated financial statements** for **2021** and **2020**, including **balance sheets**, **statements of operations**, and **cash flows**, with **total assets of $39.0 million** and **total liabilities of $4.2 million** as of **December 31, 2021** | Balance Sheet Item | Dec 31, 2021 | Dec 31, 2020 | | :--- | :--- | :--- | | **Total Current Assets** | **$37,722,044** | **$8,987,828** | | **Total Assets** | **$38,985,071** | **$9,604,358** | | **Total Current Liabilities** | **$2,552,546** | **$2,114,831** | | **Total Liabilities** | **$4,167,525** | **$3,402,369** | | **Total Stockholders' Equity** | **$34,817,546** | **$6,201,989** | | Cash Flow Item | Year Ended Dec 31, 2021 | Year Ended Dec 31, 2020 | | :--- | :--- | :--- | | **Cash used in operating activities** | **$(12,365,670)** | **$(3,240,237)** | | **Cash used in investing activities** | **$(94,212)** | **$(20,826)** | | **Cash provided by financing activities** | **$41,766,775** | **$10,486,961** | [Changes in and Disagreements with Accountants on Accounting and Financial Disclosure](index=195&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20with%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) The company reports no changes or disagreements with its accountants regarding **accounting and financial disclosure** - None[634](index=634&type=chunk) [Controls and Procedures](index=195&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded **disclosure controls** were ineffective as of **December 31, 2021**, due to an unremediated **material weakness** in procedures for **timely financial statement preparation**, though personnel issues were **remediated** - Management concluded that **disclosure controls and procedures were not effective** as of **December 31, 2021**, due to a **material weakness** in **internal control over financial reporting**[635](index=635&type=chunk) - Of the two **material weaknesses** identified in **2020**, the one related to a lack of sufficient **accounting and supervisory personnel** was **remediated** as of **December 31, 2021**[638](index=638&type=chunk)[640](index=640&type=chunk) - The **material weakness** related to a lack of **adequate procedures and controls** to ensure accurate and **timely financial statement preparation remains unremediated**[638](index=638&type=chunk)[640](index=640&type=chunk) [Other information](index=197&type=section&id=Item%209B.%20Other%20information) **Not applicable** - Not applicable[643](index=643&type=chunk) Part III [Directors, Executive Officers and Corporate Governance](index=198&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information on directors, executive officers, and corporate governance will be in the **2022 Proxy Statement**, filed within **120 days** of **December 31, 2021**, with a **code of ethics** available online - Information is incorporated by reference to the company's **Proxy Statement** for the **2022 annual meeting of stockholders**[646](index=646&type=chunk) - The company has a **code of ethics** posted on its website[647](index=647&type=chunk) [Executive Compensation](index=198&type=section&id=Item%2011.%20Executive%20Compensation) Information on **executive compensation** is incorporated by reference from the **2022 Proxy Statement**, to be filed within **120 days** of fiscal year-end - Information is incorporated by reference to the company's **Proxy Statement** for the **2022 annual meeting of stockholders**[649](index=649&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=198&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Information on **security ownership** is incorporated by reference from the **2022 Proxy Statement**, to be filed within **120 days** of fiscal year-end - Information is incorporated by reference to the company's **Proxy Statement** for the **2022 annual meeting of stockholders**[650](index=650&type=chunk) [Certain Relationships and Related Transactions and Director Independence](index=198&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%20and%20Director%20Independence) Information on **related party transactions** and **director independence** is incorporated by reference from the **2022 Proxy Statement**, to be filed within **120 days** of fiscal year-end - Information is incorporated by reference to the company's **Proxy Statement** for the **2022 annual meeting of stockholders**[651](index=651&type=chunk) [Principal Accountant Fees and Services](index=198&type=section&id=Item%2014.%20Principal%20Accountant%20Fees%20and%20Services) Information on **principal accountant fees and services** is incorporated by reference from the **2022 Proxy Statement**, to be filed within **120 days** of fiscal year-end - Information is incorporated by reference to the company's **Proxy Statement** for the **2022 annual meeting of stockholders**[652](index=652&type=chunk) Part IV [Exhibits and Financial Statement Schedules](index=199&type=section&id=Item%2015.%20Exhibits%20and%20Financial%20Statement%20Schedules) This section lists **exhibits** for the **Annual Report on Form 10-K**, with **financial statements** under **Item 8**, omitted schedules, and an **exhibit list** including **organizational documents** and **material contracts** - The **financial statements** required by this item are filed under **Item 8** of this report[654](index=654&type=chunk) - All **financial statement schedules** have been omitted as they are **not applicable** or the required information is included in the **financial statements** or notes[654](index=654&type=chunk) - A **list of exhibits** filed with the report is provided, including **organizational documents**, **material contracts**, and required **certifications**[655](index=655&type=chunk) [Form 10-K Summary](index=201&type=section&id=Item%2016.%20Form%2010-K%20Summary) The company has **not provided** a **summary** for **Form 10-K** - None[660](index=660&type=chunk)

Table of Contents 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 September 30, 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: 001-40237 GAIN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 85-1726310 ...

Table of Contents 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, 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: 001-40237 GAIN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 85-1726310 (Stat ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Table of Contents 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, 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: 001-40237 GAIN THERAPEUTICS, INC. Securities registered pursuant to Section 12(b) of the Act: | Title of each cla ...