You should read the following discussion and analysis of our financial condition and results in conjunction with our financial statements and the related notes appearing elsewhere in this Annual Report. This discussion may contain forward-looking statements that involve risks and uncertainties. Our actual results and the timing of selected events may differ materially from those described in or implied by the forward-looking statements as a result of various factors, including those set forth under “Risk Factors” and elsewhere in this Annual Report. We incorporated by reference the discussion of the operating results comparing the years ended December 31, 2023 and 2024 which was previously included in Item 5: Operating And Financial Review and Prospects of our annual report on Form 20-F filed with the SEC on March 17, 2025. A.Operating Results Overview We are a clinical stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases which have significant unmet medical need such as (i) Geographic Atrophy (GA), the late atrophic (“dry”) form of age-related macular degeneration (AMD), and (ii) Stargardt disease type 1 (STGD1). Both GA and STGD1 cause progressive loss of vision leading to permanent blindness in almost all cases. In addition to our lead product candidate (tinlarebant) which is intended for the treatment of GA and STGD1, our drug development pipeline also includes a small molecule, orally administered compound, LBS-009, which is intended for the treatment of metabolic diseases such as non-alcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), type 2 diabetes (T2D), and gout. Our lead product candidate, tinlarebant (a/k/a LBS-008), is an orally administered, once-a-day tablet intended as an early intervention for maintaining the health and integrity of retinal tissues in STGD1 and GA patients. Currently, there are no FDA approved treatments for STGD1 and no approved orally administered treatments for GA. Therefore, if approved, tinlarebant would be a novel oral therapeutic addressing an unmet medical need in both STGD1 and GA. Since our inception in 2016, our operations have focused on organizing and staffing our Company, business planning, raising capital, acquiring rights to product candidates, developing our product candidates, including conducting preclinical studies and clinical trials, and establishing our intellectual property portfolio. Lin BioScience, Inc. (the parent company of our largest shareholder, Lin Bioscience International Ltd.) obtained from Columbia University an exclusive worldwide license to the RBP4 IP Portfolio, which contains disclosure directed to over 400 structurally distinct compounds including our lead product candidate, tinlarebant, in September 2016, which was initially assigned to our largest shareholder, Lin Bioscience International Ltd., and subsequently assigned to our Cayman Island holding company, Belite Bio, Inc, in 2018. In October 2022, we entered into a subscription agreement with our wholly owned subsidiary, Belite Bio (HK) Limited (“Belite HK”), for assignment of our rights, title, interests and obligations under the Columbia License Agreement, in consideration for subscription of Belite HK’s ordinary shares. In November 2025, Belite HK entered into a contribution agreement with its wholly owned subsidiary Belite Bio (Swiss) AG (“Belite Swiss”), for assignment of Belite HK’s rights, title, interests and obligations under the Columbia License Agreement, as part of the consideration for the allocation to Belite Swiss’s capital contribution reserve. The foregoing assignments were aimed to optimize the tax structure of the Company and, in the event that the Company enters into any sub-licensing or collaboration in the future, to fulfill the economic substance requirements under the Cayman Islands law. This assignment does not affect our business and operations. 137 Table of Contents To date, we have not generated any revenues. We have financed our operations primarily through the issuance of our ordinary shares, including in the form of ADSs, pursuant to initial public offering, follow-on offerings, warrants, registered direct offerings, ATM offering program and PIPE offering. We have incurred annual net operating losses in each year since our inception and expect to continue to incur net operating losses for the foreseeable future. Our net losses were approximately US$31.6 million, US$36.1 million and US$77.6 million for the years ended December 31, 2023, 2024 and 2025, respectively. As of December 31, 2025, we had an accumulated deficit of approximately US$185.3 million. We expect to continue to incur significant expenses and increasing operating losses for the next several years. Our net losses may fluctuate significantly from quarter to quarter and year to year depending on the progress of our clinical trials and the payment schedule between us and the CROs engaged by us. We anticipate that our expenses will increase significantly if and as we continue to develop and conduct clinical trials in our current indications with tinlarebant, including our ongoing Phase 2/3 DRAGON II trial in STGD1 and Phase 3 PHOENIX trial in GA associated with dry AMD; initiate and continue research and preclinical and clinical development efforts for any future product candidates; leverage our exclusive RBP4 IP Portfolio to identify and develop additional product candidates and/or for additional indications; further expand our product pipeline through in-licensing or collaboration arrangements; seek regulatory and marketing approvals for our product candidates and complete clinical trials, if any; establish strategic collaborations or sales, marketing distribution and other commercial infrastructure in the future to commercialize any products for which we may obtain marketing approval; require the manufacture of larger quantities of product candidates for clinical development and, potentially, commercialization; maintain, expand and protect our intellectual property portfolio; hire and retain additional personnel, such as clinical and scientific personnel; add operational, financial and management information systems and personnel, including personnel to support our product development and help us comply with our obligations as a public company; and add equipment and physical infrastructure to support our research and development programs. Our ability to become and remain profitable depends on our ability to generate product revenue. We do not expect to generate product revenue unless, and until, we enter into an out-license and/or collaboration agreement with others for, or we obtain marketing approval for, and commercialize, a product candidate, and we cannot assure you that we will ever generate revenue or profits. Our current clinical development, manufacturing and commercialization plans and any of our future strategies for growth will be based on our ongoing assessment of macroeconomic trends and the evolving regulatory landscape. Key Factors Affecting Our Results of Operations Our results of operations, financial condition, and the year-to-year comparability of our financial results have been, and are expected to continue to be, principally affected by the below factors: Costs and Expenses Structure Our results of operations are significantly affected by our cost structure, which primarily consists of research and development expenses, and selling, general and administrative expenses. Research and development activities are central to our business model. We believe our ability to successfully develop product candidates will be the primary factor affecting our long-term competitiveness, as well as our future growth and development. Developing high-quality product candidates requires a significant investment of resources over a prolonged period of time, and a core part of our strategy is to continue making sustained investments in this area. Since our inception, we have focused our resources on our research and development activities, including developing our RBP4 IP Portfolio, conducting preclinical studies and clinical trials, and engaging in activities related to regulatory filings for our product candidates. Clinical studies become increasingly more expensive from Phase 1b/2 and onwards due to an increase in the number of subjects enrolled and longer study duration in such studies. Research and development costs are expensed as incurred. Costs for certain activities, such as activities performed by third-party contractors relating to the manufacturing and preclinical studies and clinical trials of our product candidates, are generally accrued based on our estimates of the actual services performed for a given period. These estimates are based on our evaluation of the progress to completion of specific tasks to be performed using information and data provided to us by our third-party contractors and vendors. 138 Table of Contents At this time, we cannot reasonably estimate the nature, timing and estimated costs of the efforts that will be necessary to complete the development of, or the period, if any, in which material net cash inflows may commence from, any of our product candidates. This is due to the numerous risks and uncertainties associated with developing such product candidates, including, but not limited to, the uncertainty of: ● the scope, rate of progress and expense of our research and development activities; ● successful completion of clinical trials, including the successful enrollment in such clinical trials, and clinical trial results; ● our ability to market, commercialize and achieve market acceptance for any of our product candidates, whether alone or in collaboration with others; ● our ability to establish manufacturing capabilities and capacities, whether internally or through CMOs, to the specifications of our product candidates for clinical supply; ● the potential benefits of our product candidates over other therapies; ● obtaining and maintaining patent, trade secret and other intellectual property protection and/or regulatory exclusivity for our product candidates; ● the terms and timing of regulatory approvals; ● successful completion of all safety studies required to obtain regulatory approval in the United States and other applicable jurisdictions for our product candidates; and ● maintaining a continued acceptable safety profile of the product candidates following regulatory approval. A change in the outcome of any of these variables with respect to the development of any of our product candidates could mean a significant change in the costs, timing and viability of the development of that product candidate. For example, if we are required to conduct additional clinical trials or other testing of any of our product candidates beyond those that are contemplated or if we experience significant delays in enrollment in any clinical trials, we could incur significant additional costs and the clinical development timeline for our product candidates may be delayed. We expect our research and development expenses to continue to increase for the foreseeable future, as we continue to advance our lead product candidate, tinlarebant, toward later stages, develop our other product candidates and potentially initiate new pipelines, as well as continue to expand our operations. Our selling, general and administrative expenses consist primarily of employee salaries and related benefit costs, including share-based compensation expenses, for personnel in executive, commercialization, finance, accounting and administrative functions. Other selling, general and administrative expenses include professional fees for financial advisory, auditing and legal services. Our selling, general and administrative headcount and related expenses have increased and are expected to increase in the future to support our clinical program and research and development efforts, and the commercialization of our product candidates in the event approval is obtained. Funding for Our Operations During the periods presented, we have funded our operations primarily through the issuance of our ordinary shares, including in the form of ADSs, pursuant to warrants, registered direct offering, ATM offering programs and PIPE offering. However, if our business and our product candidate pipeline continue to expand, we may require further funding through public or private offerings, debt financing, collaboration, and licensing arrangements or other sources. Any fluctuation in our ability to fund our operations will impact our development plan, operating plan and our results of operations. In the event of the successful development and commercialization of one or more of our product candidates, we expect to fund our operations in part with revenue generated from sales of our commercialized drug products. In the event that we enter into out-license and/or collaboration arrangements with others, we expect to fund our operations in part with revenue received from such out-license and/or collaboration arrangements. 139 Table of Contents Our Ability to Commercialize and/or Out-License Our Product Candidates Our business and results of operations depend on our ability to out-license our product candidates or, in the event any of our product candidates are approved for marketing by the respective regulatory authority in a country, commercialize such product candidates. Currently, our pipeline consists of our lead product candidate, tinlarebant, which is currently in clinical development, and LBS-009, which is currently in preclinical development. Although we currently do not have any product approved for commercial sale and have not generated revenue from product sales or out-licensing, we expect to generate revenue either from sales of a product candidate if we complete the clinical development, obtain regulatory approval, and successfully commercialize such product candidate, or from out-licensing a product candidate if we enter into an out-license and/or collaboration agreement for any product candidate. Key Components of Results of Operations Revenue To date, we have not generated any revenue. Our ability to generate revenue and to become profitable will depend upon the successful commercialization of, and/or our successful entry into out-license and/or collaboration arrangements in connection with, one or more of our product candidates. Because of the numerous risks and uncertainties associated with product development and regulatory approval, and out-license and/or collaboration arrangements, we are unable to predict the amount or timing of product revenue or out-license and/or collaboration revenue. Research and Development Expenses Research and development expenses consist of costs associated with planning and conducting clinical trials of our product candidates. Our research and development expenses primarily consist of: ● payroll, share-based compensation and other related costs of personnel engaged in research and development activities; ● in-licensed patent rights fee of exclusive development rights of drugs granted to us under the Columbia License Agreement; ● costs for preclinical testing of our technologies and clinical trials such as payments to CROs and CMOs, investigators and clinical trial sites that conduct the clinical studies; ● costs to develop our product candidates, including raw materials and supplies, product testing, clinical trial equipment and its depreciation, and facility related expenses; and ● other research and development expenses. 140 Table of Contents Clinical trial costs are a significant component of our research and development expenses. Our current research and development activities primarily relate to the clinical development of tinlarebant for the following indications: ● STGD1. We are developing tinlarebant as an orally administered, once-a-day tablet treatment for STGD1. STGD1 is an inherited juvenile form of macular degeneration. Currently, there is no FDA approved therapy for STGD1. We initiated an open-label, dose-finding Phase 1b/2 clinical trial in adolescent STGD1 patients in mid-2020 in Australia and Taiwan. Based on the Phase 1b/2 data, we initiated our Phase 3 clinical trial to evaluate the safety and efficacy of tinlarebant in adolescent STGD1 patients in 2022. This Phase 3 STGD1 trial named “DRAGON” has commenced in the United States, the United Kingdom, Germany, Netherlands, France, Belgium, Switzerland, China, Hong Kong, Taiwan, and Australia and has completed enrollment of 104 subjects. In September 2025, we completed the last subject visit in the Phase 3 DRAGON trial. In December 2025, we announced the top-line results of the Phase 3 DRAGON trial. In addition to the completed Phase 3 DRAGON clinical trial, we are conducting a clinical trial of tinlarebant in adolescent STGD1 patients aged 12 to 20 years old in Japan, the United States and the United Kingdom (“DRAGON II”). The DRAGON II trial is a combination of a Phase 1b open-label study to evaluate the pharmacokinetics and pharmacodynamics of tinlarebant in adolescent Japanese STGD1 subjects and a 24-month, Phase 2/3, multi-center, randomized, double-masked, placebo-controlled study designed to evaluate the efficacy, safety and tolerability of tinlarebant. We have completed the Phase 1b portion of DRAGON II, and have completed the enrollment of the Phase 2/3 portion of the trial with a total of 73 subjects, including 15 Japanese subjects. The data from Japanese subjects is intended to facilitate future NDA applications in Japan. ● GA. We are developing tinlarebant as an orally administered, once-a-day tablet treatment for GA, the advanced stage of dry AMD. Age-related macular degeneration, or AMD, is a common eye disorder among people over 50. ‘Wet’ AMD represents approximately 10% of all AMD cases. Currently, there are no FDA approved orally administered therapies for GA and no FDA approved therapies for the other stages of dry AMD other than GA. We have completed the Phase 1b dose-finding clinical trial, which is an open-label, parallel, single-dose clinical trial designed to evaluate the PK and PD of tinlarebant in healthy subjects aged between 50 to 85. In addition, we have initiated our Phase 3 trial named “PHOENIX” to evaluate the safety and efficacy of tinlarebant in patients with GA associated with dry AMD, and have randomized the first subject in July 2023. The PHOENIX trial has commenced in the United States, the United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia. As of the Latest Practicable Date, we have completed patient enrollment with a total of 530 subjects for this trial. We incurred research and development expenses of approximately US$24.8 million, US$29.9 million and US$45.4 million for the years ended December 31, 2023, 2024 and 2025, respectively, representing approximately 78.5%, 74.9% and 53.9%, respectively, of our total operating expenses for the corresponding periods. Our research and development expenses may vary substantially from period to period according to the status of our research and development activities. The timing of expenses is impacted by the commencement of clinical trials and enrollment of patients in clinical trials. Clinical studies become increasingly expensive from Phase 1b/2 and onwards. We expect our research and development expenses to continue to increase for the foreseeable future, as we continue to advance our lead product candidate, tinlarebant, toward later stages, develop our other product candidates and potentially initiate new pipelines, as well as continue to expand our operations. Selling, General and Administrative Expenses Our selling, general and administrative expenses consist primarily of employee salaries and related benefit costs, including share-based compensation expenses, for personnel in executive, commercialization, finance, accounting and administrative functions. Other selling, general and administrative expenses include professional fees for financial advisory, auditing and legal services. For the years ended December 31, 2023 and 2024 and 2025, our general and administrative expenses amounted to approximately US$6.8 million, US$10.1 million and US$38.8 million, respectively, representing approximately 21.5%, 25.1% and 46.1% respectively, of our total operating expenses for the corresponding periods. Our selling, general and administrative expenses have increased and are expected to continue to increase for the foreseeable future, as we continue to incentivize and retain our management, expand our team (including building up operational teams across various jurisdictions), and incur additional expenses as a result of operating as a public company, including expenses related to compliance with the rules and regulations of the SEC, additional insurance expenses, investor relations activities and other related administrative and professional services. 141 Table of Contents Additionally, if we obtain regulatory and marketing approvals for our products, we expect to incur additional selling expenses in connection with establishing our in-house commercialization team, sales network and systems, and these expenses are expected to increase for the foreseeable future as we advance our commercialization efforts. However, there can be no assurance that we will obtain regulatory and marketing approvals for tinlarebant or other product candidates or future products in a timely manner, or at all. For details, see “Item 3. Key Information—D. Risk Factors—Our product candidates are subject to extensive regulation—Our product candidates are subject to extensive regulation. However, we have never obtained marketing approval for a product candidate and we may be unable to obtain, or may be delayed in obtaining, marketing approval for any of our product candidates.” Other Income Other income consists primarily of interest income. Interest income was derived from cash in banks and our investments in money market funds, debt securities issued by the United States Government and time deposits from financial institutions with a range of maturity dates over 3 months or greater than 12 months from the origination date. Taxation Cayman Islands We are an exempted company with limited liability incorporated in the Cayman Islands. Under the current laws of the Cayman Islands, we are not subject to tax on income or capital gain. Additionally, upon payments of dividends by us to our shareholders, if any, no Cayman Islands withholding tax will be imposed. However, we may be subject to taxation in other jurisdictions in which we operate, in particular, the United States, Australia, Hong Kong, Taiwan, the PRC, Switzerland and Japan where eight of our wholly-owned subsidiaries are incorporated, if certain conditions are met under the laws and regulations of the Cayman Islands. United States Our subsidiaries, Belite Bio Holdings Corp. and Belite Bio, LLC, which are both incorporated or formed in Delaware, United States, are subject to statutory U.S. Federal corporate income tax at a maximum rate of 21% for the years ended December 31, 2023, 2024 and 2025. Both entities are also subject to state income tax in California at a rate of 8.84% for the years ended December 31, 2023, 2024 and 2025. As a result of the Tax Cuts and Jobs Act of 2017 (TJCA), the Company evaluated whether it has an additional tax liability from the Global Intangible Low Tax Income (“GILTI”) inclusion on current earnings and profits of its foreign controlled corporations. As of December 31, 2023, 2024 and 2025, the Company does not have any aggregated positive tested income; and as such, did not record a tax liability with respect to GILTI. Australia Our subsidiary, RBP4 Pty Ltd, is subject to Australia profits tax on the taxable income as reported in its respective statutory financial statements adjusted in accordance with the relevant Australia tax laws. The applicable tax rate in Australia is 30%. RBP4 Pty Ltd had no taxable income for the periods presented, therefore, no provision for income taxes has been provided. Hong Kong Our subsidiary, Belite Bio (HK) Limited, is subject to Hong Kong profits tax at a tax rate of 8.25% for assessable profits on the first HK$2 million and 16.5% for any assessable profits in excess. No Hong Kong profit tax was provided for as there was no estimated assessable profit that was subject to Hong Kong profits tax for the years ended December 31, 2023, 2024 and 2025. Taiwan Our subsidiary Belite Bio (Taiwan) Inc is subject to Taiwan profit tax on the taxable income as reported in the respective statutory financial statements adjusted in accordance with the relevant Taiwan tax laws. The applicable tax rate in Taiwan is 20%. 142 Table of Contents PRC Our subsidiary, Belite Bio (Shanghai) Limited is subject to the statutory rate of 25%, in accordance with the Enterprise Income Tax Law (“EIT Law”). Under the EIT Law, dividends, interests, rent or royalties payable by Belite Bio (Shanghai) Limited to non-PRC resident enterprises, and proceeds from any such non-PRC resident enterprise investor’s disposition of assets (after deducting the net value of such assets) shall be subject to a 10% withholding income tax, unless the respective non-PRC resident enterprise’s jurisdiction of incorporation has a tax treaty or arrangements with China that provides for a reduced withholding income tax rate or an exemption from withholding income tax. No provision for PRC corporate income tax has been made for the years ended December 31, 2023, 2024 and 2025, as Belite Bio (Shanghai) Limited had no such assessable profit for the years then ended. Switzerland Belite Bio (Swiss) AG is subject to Switzerland Swiss federal, cantonal and municipal income. The combined statutory tax rate applicable to Belite Bio (Swiss) AG is approximately 12.47% depending on the applicable cantonal and municipal rates. Japan Belite Bio Japan Inc., is subject to Japanese corporate income taxes, which consist of national corporate tax, local corporate tax, enterprise tax and inhabitant tax. The combined statutory tax rate in Japan is approximately 30%, with the actual effective rate varying based on the local tax jurisdictions and the level of taxable income. The Company and its subsidiaries file separate income tax returns. As of December 31, 2025, the tax returns of Belite Bio Holdings Corp. and Belite Bio, LLC for the tax year 2019 to 2024 are subject to examination by United States and states authorities. The tax returns of Belite Bio (HK) Limited for the tax year 2021 to 2024 are subject to examination by Hong Kong tax authorities. The tax returns of RBP4 Pty Ltd for the tax year 2018 to 2024 are subject to examination by Australia authorities. The tax return of Belite Shanghai for tax year 2022 to 2024 are subject to examination by PRC tax authorities. There are currently no pending examinations. Results of Operations Comparison of the Fiscal Years Ended December 31, 2023 and 2024 We incorporate by reference the discussion of the operating results comparing the year ended December 31, 2023 and 2024 which was previously included in Item 5: Operating And Financial Review and Prospects of our registration statement on Form 20-F filed with the SEC on March 17, 2025. 143 Table of Contents Comparison of the Fiscal Years Ended December 31, 2024 and 2025 The following table sets forth a summary of our consolidated results of operations for the years ended December 31, 2024 and 2025. This information should be read together with our consolidated financial statements and related notes included elsewhere in this Annual Report. The operating results in any period are not necessarily indicative of the results that may be expected for any future period. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the Years Ended December 31, ​ ​ ​ ​ ​ ​ ​ ​ 2024 ​ ​ ​ 2025 ​ ​ ​ Change ​ ​ (Amounts in thousands of US$) ​ ​ ​ ​ ​ ​ (%) Expenses ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Research and development(1) ​ ​ 29,939 ​ ​ 45,377 ​ ​ 15,438 ​ ​ 51.6 Selling, general and administrative(1) ​ ​ 10,057 ​ ​ 38,831 ​ ​ 28,774 ​ ​ 286.1 Total operating expenses ​ ​ 39,996 ​ ​ 84,208 ​ ​ 44,212 ​ ​ 110.5 Loss from operations ​ ​ (39,996) ​ ​ (84,208) ​ ​ (44,212) ​ ​ 110.5 Other income: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Interest income ​ ​ 3,737 ​ ​ 6,640 ​ ​ 2,903 ​ ​ 77.7 Interest expense ​ ​ (20) ​ ​ (2) ​ ​ 18 ​ ​ (90.0) Other income (expense) ​ ​ 141 ​ ​ (41) ​ ​ (182) ​ ​ (129.1) Total other income, net ​ ​ 3,858 ​ ​ 6,597 ​ ​ 2,739 ​ ​ 71.0 Loss before income tax ​ ​ (36,138) ​ ​ (77,611) ​ ​ (41,473) ​ ​ 114.8 Income tax expense ​ ​ 6 ​ ​ - ​ ​ (6) ​ ​ (100) Net loss ​ ​ (36,144) ​ ​ (77,611) ​ ​ (41,467) ​ ​ 114.7 Other comprehensive income (loss) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Foreign currency translation adjustments, net of nil tax ​ ​ (286) ​ ​ 124 ​ ​ 410 ​ ​ (143.4) Total comprehensive loss ​ US$ (36,430) ​ US$ (77,487) ​ US$ (41,057) ​ ​ 112.7 (1) Total share-based compensation costs were recognized as follows for the years ended December 31, 2024 and 2025: ​ ​ ​ ​ ​ ​ ​ ​ ​ Years ended December 31, (Amounts in thousands of US$) ​ ​ ​ 2024 ​ ​ ​ 2025 Research and development ​ 3,775 ​ 9,147 Selling, general and administrative ​ 5,212 ​ 29,772 Total US$ 8,987 US$ 38,919 ​ Revenue We did not generate any revenue during the years ended December 31, 2024 and 2025. Research and Development Expenses The following table sets forth a breakdown of the major components of our research and development expenses in absolute amounts and as a percentage of our total research and development expenses for the years ended December 31, 2024 and 2025: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the Years Ended December 31, (Amounts in thousands of US$, except percentages) ​ 2024 ​ ​ ​ 2025 ​ ​ US$ ​ ​ ​ % ​ US$ ​ ​ ​ % Contracted research expenses and clinical trial expenses 18,057 ​ 60.3 28,334 ​ 62.4 Consultancy and professional service fees 1,698 ​ 5.7 3,986 ​ 8.8 Royalties 3,353 ​ 11.2 120 ​ 0.3 Other expenses 912 ​ 3.0 1,168 ​ 2.6 Wages and salaries 5,919 ​ 19.8 11,769 ​ 25.9 Total 29,939 ​ 100.0 45,377 ​ 100.0 ​ 144 Table of Contents Our research and development expenses increased by 51.6% from approximately US$29.9 million for the year ended December 31, 2024, to approximately US$45.4 million for the year ended December 31, 2025. In the year ended December 31, 2025, approximately 62.4% of our total research and development expenses were attributable to contracted research expenses and clinical trial expenses, and in the year ended December 31, 2024, approximately 60.3% of our total research and development expenses were attributable to contracted research expenses and clinical trial expenses. The increase in research and development expenses was primarily attributable to the increases in (i) expenses of US$6.5 million related to the PHOENIX trial; (ii) share-based compensation expenses of US$5.4 million; and (iii) Active Pharmaceutical Ingredient (“API”) manufacturing expenses of US$3.1 million, partially offset by US$3 million the non-recurrence of a royalty payment recognized in 2024 for completion of a Phase 2 trial. Selling, General and Administrative Expenses Our selling, general and administrative expenses increased by 286.1% from approximately US$10.1 million for the year ended December 31, 2024, to approximately US$38.8 million for the year ended December 31, 2025, which was primarily due to an increase in share-based compensation expenses of US$24.6 million and professional service fees of US$1.7 million. Other Income Our other income, increased from approximately US$3.9 million for the year ended December 31, 2024, to approximately US$6.6 million for the year ended December 31, 2025, which was primarily due to increases in interest income from bank deposits, time deposits, money market funds, U.S. treasury bills and U.S. treasury notes total of US$2.9 million. B. Liquidity and Capital Resources The following summarizes the key components of our cash flows for the years ended December 31, 2023, 2024 and 2025. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the Year Ended ​ ​ ​ December 31, ​ ​ ​ ​ 2023 ​ ​ ​ 2024 ​ ​ ​ 2025 ​ ​ (Amounts in thousands of US$) ​ ​ ​ ​ ​ ​ ​ Net cash used in operating activities (29,837) (29,234) (40,671) ​ Net cash used in investing activities (63) (110,572) (301,424) ​ Net cash provided by financing activities 75,959 83,595 663,233 ​ Effects of exchange rate on cash and cash equivalent 9 (269) 117 ​ NET (DECREASE) INCREASE IN CASH AND CASH EQUIVALENT 46,068 (56,480) 321,255 ​ CASH AND CASH EQUIVALENT AT BEGINNING OF THE YEAR 42,089 88,157 31,677 ​ CASH AND CASH EQUIVALENT AT END OF THE YEAR 88,157 31,677 352,932 ​ ​ ​ ​ ​ ​ ​ ​ ​ SUPPLEMENTAL DISCLOSURE OF CASH FLOW INFORMATION ​ ​ ​ ​ Interest paid — — 1 ​ Cash paid for income tax 9 6 — ​ SUPPLEMENTAL DISCLOSURE OF NON-CASH INVESTING AND FINANCING ACTIVITIES ​ ​ ​ ​ Follow-on offering underwriting discounts reclassed to additional paid-in-capital ​ 1,800 ​ — ​ — ​ ATM offering sales commissions reclassed to additional paid-in-capital ​ 858 ​ 509 ​ 896 ​ ATM proceeds not yet received in cash as other receivable ​ 762 ​ — ​ — ​ Non-cash exercise of Representative’s Warrants ​ — * — ** — ​ Right-of-use assets obtained in exchange of lease liability ​ 265 ​ 388 ​ 529 ​ Right-of-use assets in exchange of lease liabilities from an operating lease modification ​ — ​ 385 ​ 361 ​ *The Company issued 65,399 shares of ordinary shares in a non-cash exercise of 84,651 shares of Representative’s Warrants. **The Company issued 73,800 shares of ordinary shares in a non-cash exercise of 84,651 shares of Representative’s Warrants. 145 Table of Contents ​ To date, we have not generated any revenue. We incurred net losses of approximately US$31.6 million, US$36.1 million and US$77.6 million for the years ended December 31, 2023, 2024 and 2025, respectively. Our primary use of cash is funding our research and development expenses. We used approximately US$29.8 million, US$29.2 million and US$40.7 million in cash for our operating activities for the years ended December 31, 2023, 2024 and 2025, respectively. We invested in money market funds, U.S. treasury bills, U.S. treasury notes, liquidity funds and time deposits. As a result, net cash used in investing activities was US$63 thousand, US$110.6 million and US$301.4 million for the years ended December 31, 2023, 2024 and 2025, respectively. We have financed our operations primarily through the issuance of our ordinary shares, including in the form of ADSs, pursuant to initial public offering, follow-on offerings, warrants, registered direct offerings, ATM offering program and PIPE offering. Net cash provided by financing activities amounted to US$76.0 million, US$83.6 million and US$663.2 million for the years ended December 31, 2023, 2024 and 2025, respectively. As of December 31, 2025, we held (i) cash and cash equivalents of approximately US$352.9 million primarily consisting of cash and money market accounts which are unrestricted as to withdrawal and use which have original maturities of less than three months and (ii) investments of approximately US$139.5 million in U.S. Treasury bills (with maturities greater than three months but less than one year) and US$280.2 in U.S. Treasury notes (with maturities over twelve months). Our lead product candidate, tinlarebant, is still in clinical development. We expect our expenses to increase substantially as compared to prior periods in connection with our ongoing activities, particularly as we continue to advance the development of and seek marketing approval for tinlarebant, develop our other product candidates and potentially initiate new pipelines. In addition, if we obtain marketing approval for tinlarebant or any other product candidates that we develop and if we choose to commercialize such product candidates ourselves, we expect to incur significant commercialization expenses related to product sales, marketing, distribution and manufacturing. Furthermore, since the closing of our initial public offering in April 2022, we have incurred additional costs associated with operating as a public company. Accordingly, we anticipate that we will need substantial additional funding in connection with our continuing operations. We expect that our expenses will continue to increase substantially and that we will continue to incur significant operating losses and negative operating cash flows as we fund both ongoing research and development activities and new activities (including commercialization activities if we obtain marketing approval for tinlarebant or any other product candidate that we develop) as well as working capital needs. We have based our estimates on assumptions that may prove to be wrong, and we may use our available capital resources sooner than we currently expect or on alternative uses. Because of the numerous risks and uncertainties associated with the development and commercialization of our product candidates, we are unable to estimate the amounts of increased capital outlays and operating expenditures necessary to complete the development and commercialization of our product candidates. We believe that our cash and cash equivalents, together with our cash generated from our public offerings and private placements, will be sufficient to meet our current and anticipated needs for general corporate purposes for at least the next 12 months. We may, however, consider to raise additional capital to fund future operations, and our future capital requirements will depend on many factors, including: ● the number and development requirements of the product candidates we pursue; ● the scope, progress, timing, results and costs of discovering, researching and developing product candidates, and conducting preclinical studies and clinical trials; ● the scope, prioritization and number of our research and development programs; ● the costs, timing and outcome of regulatory review of our product candidates; ● the cost of manufacturing our product candidates and any products we commercialize, including costs associated with expanding our supply chain; ● the cost and timing of future commercialization activities, including product manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive regulatory approval; ● the cash received, if any, from commercial sales of any product candidates for which we receive regulatory approval; 146 Table of Contents ● our ability to establish and maintain strategic collaborations, licensing or other arrangements and the financial terms of such collaborations and arrangements; ● the extent to which we acquire or in-license other product candidates and technologies; ● our headcount growth and associated costs; ● the costs, timing and outcome of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending any intellectual property-related claims; ● resources required to develop and implement policies and processes to promote ongoing compliance with applicable healthcare laws and regulations; and ● the costs of operating as a public company in the United States. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution or licensing arrangements. To the extent that we raise additional capital through the sale of equity or warrant exercise your ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a holder of our ADSs. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. There can be no assurances, however, that our current operating plan will be achieved or that additional funding will be available on terms acceptable to us, or at all. If we are unable to obtain sufficient funding, it could delay our development efforts, limit activities and reduce research and development costs, which could adversely affect our business prospects. For additional information regarding the risks related to our need to obtain additional capital, see “Item 3. Key Information—D. Risk Factors — Risks Related to Our Financial Position and Need for Additional Capital —  We have recorded net cash outflow from operating activities since our inception. Although we have consummated our initial public offering, we will need to obtain additional financing to fund our operations. If we are unable to obtain such financing, we may be unable to complete the development and commercialization of our product candidates.” And “Item 3. Key Information—D. Risk Factors — Risks Related to Our Financial Position and Need for Additional Capital — Raising additional capital may cause dilution to holders of our ADSs and our shareholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.” Operating Activities Net cash used in operating activities was approximately US$29.8 million for the year ended December 31, 2023 and consisted primarily of a net loss of approximately US$31.6 million, after non-cash US$4.2 million add backs, offset primarily by an increase in accrued expenses of approximately US$1.4 million and an increase in prepayments of approximately US$3.5 million. Net cash used in operating activities was approximately US$29.2 million for the year ended December 31, 2024 and consisted primarily of a net loss of approximately US$36.1 million, after non-cash US$5.8 million add backs, offset primarily by an increase in accrue expenses of approximately US$2.5 million, and an increase in prepayments of approximately US$1.1 million. Net cash used in operating activities was approximately US$40.7 million for the year ended December 31, 2025 and consisted primarily of a net loss of approximately US$77.6 million, after non-cash US$34.4 million add backs, offset primarily by an increase in accrue expenses of approximately US$3.8 million, an increase in prepayments and other assets of approximately US$0.8 million and an increase in lease liabilities of approximately US$0.3 million. Investing Activities Net cash used in investing activities during the year ended December 31, 2023 was approximately US$63 thousand which resulted primarily from the purchase of fixed assets. 147 Table of Contents Net cash used in investing activities during the year ended December 31, 2024 was approximately US$110.6 million which resulted primarily from the net of acquisition of investments and proceeds from maturities of investments of U.S. treasury bills and time deposits. Net cash used in investing activities during the year ended December 31, 2025 was approximately US$301.4 million which resulted primarily from the acquisition of investments in U.S. treasury bills and U.S. treasury notes. Financing Activities Net cash provided by financing activities was approximately US$76.0 million for the year ended December 31, 2023, which consisted primarily net proceeds from follow-on offering of US$27.3 million, net proceeds from ATM offering of US$26.6 million, net proceeds from warrant exercise of US$21.7 million and exercise of stock options of US$0.3 million. Net cash provided by financing activities was approximately US$83.6 million for the year ended December 31, 2024, which consisted primarily of net proceeds from ATM offering of US$16.3 million, proceeds from registered direct offering of US$25.0 million, net proceeds from warrant exercise of US$39.0 million and proceeds from exercise of stock options of US$2.8 million. Net cash provided by financing activities was approximately US$663.2 million for the year ended December 31, 2025, which consisted primarily of net proceeds from underwritten public offering and over-allotment exercise of US$378.4 million, net proceeds from PIPE offering of US$118.2 million, and net proceeds from warrant exercise of US$103.0 million. Material Cash Requirements Contractual Obligations and Commitments We are party to an exclusive license agreement with Columbia University, as amended, under which we license specified intellectual property from Columbia University. The patent rights licensed to us by Columbia University include issued patents with claims that recite a class of compounds directed to covering our planned lead compound, tinlarebant, and specifically recite tinlarebant. The license agreement requires us to make minimum annual royalty payments to Columbia University of (i) US$2.5 million on each of the second, third and fourth anniversaries of the first commercial sale of a licensed product and (ii) US$5 million on each anniversary of the first commercial sale of a licensed product, commencing on the fifth anniversary of such sale. We will also be obligated to pay single-digit earned royalties to Columbia University based on net sales of each licensed product by us and our affiliates and sublicensees. The minimum royalty payments will be creditable against the earned royalties accrued during the same calendar year. The license agreement obligates us to use commercially reasonable efforts to research, discover, develop and market licensed products for commercial sale and distribution and achieve certain development and regulatory approval milestones within certain timeframes, if certain milestones are achieved. We are also obligated to periodically inform Columbia University of our progress in meeting such milestones. The failure to achieve any such milestone would constitute a breach of the Columbia License Agreement. If we pay Columbia University the required fee, we will be granted a 6-month extension. As of the Latest Practicable Date, we have complied with the development and regulatory approval milestones under the Columbia License Agreement and requested no extensions. In the event that, after completion of Phase I trials, there are unforeseen changes in clinical or regulatory development that we believe would affect the timely achievement of any milestone, we may request an extension from Columbia University for such milestone, which will not be unreasonably withheld or delayed. In the event that Columbia University does not agree to extend the deadlines to meet the milestones, and we are in breach for failure to timely meet the milestones or to make milestone or royalty payments, Columbia University may elect to convert our license to a nonexclusive license without the right to sublicense or initiate legal proceedings against third party patent infringers or terminate our license. We are also obligated to make payments to Columbia University in an aggregate amount of up to US$20 million based on achieving specified development and regulatory approval milestones and in an amount of up to US$25 million based on achieving a specified cumulative sales milestone with respect to the first licensed product. In addition, we are obligated to pay Columbia University a specified portion of revenue (other than royalties) we receive from sublicensees and a percentage of revenue in the low double-digits received from any sale of a priority review voucher by us or a sublicensee. In the event that we or a sublicensee do not sell a priority review voucher within one year of receipt, the earned royalties that we would be obligated to pay to Columbia University based on net sales of licensed products would increase by 1%. We cannot reasonably estimate whether, when and in what amount any of such payments shall be made, but believe we are in compliance with the terms of the license. From inception through December 31, 2025, the Company has made a payment of $4 million to Columbia University resulting from this license agreement, which was triggered by the completion of certain clinical trials, and expects to make additional payments of $6 million upon the near term expected completion of development milestones. 148 Table of Contents We also enter into cancelable contracts in the normal course of business with CROs for clinical trials, preclinical studies, manufacturing and other services and products for operating purposes. As of the Latest Practicable Date, the remaining contractual costs expected to be incurred in future periods for our clinical trials in STGD1 and GA are approximately US$31.9 million. Off-Balance Sheet Arrangements We are not party to any off-balance sheet arrangements. We have not entered into any financial guarantees or other commitments to guarantee the payment obligations of any third parties. In addition, we have not entered into any derivative contracts that are indexed to our shares and classified as shareholder’s equity or that are not reflected in our consolidated financial statements included elsewhere in this Annual Report. Furthermore, we do not have any retained or contingent interest in assets transferred to an unconsolidated entity that serves as credit, liquidity or market risk support to such entity. We do not have any variable interest in any unconsolidated entity that provides financing, liquidity, market risk or credit support to us or engages in leasing, hedging or product development services with us. C.Research and Development, Patents and Licenses, etc. We have established research and development policies for all stages of our research and development activities, through our internal research project initiation processes and our preclinical and clinical development programs. Our in-house discovery, research and development team focuses on identifying small-molecule compounds in our core therapeutic areas of macular degeneration and age-related metabolic diseases. We have relied on, and plan to continue to rely on, third-party CROs to monitor and manage data for some of our ongoing preclinical studies and clinical trials. We have incurred research and development expenses of US$24.8 million, US$29.9 million and US$45.4 million in 2023, 2024 and 2025, respectively. See “Item 4. Information on the Company—B. Business Overview” and “—A. Operating Results.” D.Trend Information Other than as disclosed elsewhere in this Annual Report, we are not aware of any trends, uncertainties, demands, commitments or events since January 1, 2025 that are reasonably likely to have a material adverse effect on our net revenues, income, profitability, liquidity or capital resources, or that caused the disclosed financial information to be not necessarily indicative of future operating results or financial conditions. For a detailed discussion of trend information, see “—A. Operating Results—Key Factors affecting our results of operations.” E.Critical Accounting Estimates Our consolidated financial statements are prepared in accordance with U.S. GAAP. The preparation of our consolidated financial statements requires us to make estimates, assumptions and judgments that affect the reported amounts of assets, liabilities, costs and expenses. We base our estimates and assumptions on historical experience and other factors that we believe to be reasonable under the circumstances. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates. Some of our accounting estimates require a higher degree of judgment than others in their application. When reading our consolidated financial statements, you should consider our use of accounting estimates, the judgment and other uncertainties affecting such estimates and the sensitivity of reported results to changes in conditions and assumptions. Our accounting estimates include but are not limited to, accrued research and development expenses related to payments to contract research organizations (“CRO”), valuation of ordinary shares, valuation allowance for deferred income tax assets, useful lives for property and equipment, discount rate for right-of-use (“ROU”) assets and operating lease liabilities, the fair value of share-based compensation and warrants.; See Note 2—Summary of Significant Accounting Policies to our consolidated financial statements for the disclosure of these accounting estimates. We consider an accounting estimate to be critical if: (i) the accounting estimate requires us to make assumptions about matters that were highly uncertain at the time the accounting estimate was made, and (ii) changes in the estimate that are reasonably likely to occur from period to period or use of different estimates that we reasonably could have used in the current period, would have a material impact on our financial condition or results of operations. We believe the following critical accounting estimates involve the most significant judgments used in the preparation of our financial statements. 149 Table of Contents Accrued Research and Development Expenses We incur expenses associated with preclinical development and clinical trials. Accounting for research and development activities performed by CROs and other external clinical service providers requires management to apply significant estimates, particularly with respect to the timing of services rendered and the related expense recognition. These activities include sponsored research, preclinical studies and clinical trial-related services. The diverse nature of CRO arrangements, varying compensation structures, and the lack of real-time visibility into certain ongoing clinical activities make the estimation of accrued research and development expenses inherently complex. We record the estimated costs of research and development activities based on the progress of the preclinical or clinical study as measured by the timing of various aspects of the study or related activities and determine accrual estimates through review of the underlying contracts along with discussions with internal personnel and external service providers regarding the progress or stage of completion of contracted services and consider the agreed-upon contractual fees for such services. In this process, we conduct a detailed review of open contracts and an assessment of services that have been performed for us but for which invoices have not yet been received. In evaluating the duration and cost of clinical studies, we consider key phases such as start-up treatment, follow-up and wrap-up activities. We base our estimates on the best information available at the time; however, if additional information becomes available regarding the status or progression of a study, we adjust our rate of expense recognition when actual results differ from our estimates. If we underestimate or overestimate the level of services performed or the associated costs, our actual research and development expenses could differ from the amounts recorded. There were no such significant changes for the years ended December 31, 2023, 2024 and 2025. Share-Based Compensation We grant share‑based compensation to employees, management and directors through stock options and restricted stock units (“RSUs”). Option awards are valued using an option‑pricing model, and RSUs with market conditions are measured using a Monte Carlo simulation. Other share-based awards with non‑market performance conditions require ongoing assessments of the likelihood of achievement, to the extent that the achievement of performance conditions associated with such awards, as applicable, are determined to be “probable.” The fair value of share-based awards is measured on the grant date. For stock options, we determine fair value using the Binomial Option Pricing Model, which requires assumptions about the fair value of our common shares, expected volatility, expected term and exercise behavior, the risk‑free interest rate and expected dividends. Expected volatility is based on the historical volatilities of comparable public companies in our industry and, where relevant, our own trading history. The expected term of the award requires us to make assumptions regarding the post-vesting behavior of the recipients, which is based off available evidence. The risk‑free interest rate is derived from U.S. Treasury yields with maturities that correspond to the expected term, and we currently assume no dividends. For awards that include market conditions, we determine grant‑date fair value using a Monte Carlo simulation, which models the likelihood and timing of achieving the specified market targets. The simulation also determines the derived service period, which reflects the expected time required to satisfy the market condition. As of December 31, 2025, all market-based stock unit awards had fully vested, as the applicable market conditions were achieved within the expected timeframe. For service‑only RSUs, grant‑date fair value generally equals the market price of our common shares on the grant date. We recognize share-based compensation cost for awards to employees with a performance condition at the fixed fair value on date of grant over the period based on the expected milestone achievement dates as the derived service period (usually the vesting period), on a straight-line basis. We consider the probable outcome of that performance condition when determining share-based compensation expenses and will recognize a cumulative true-up adjustment if the probability of the conditions has changed. To the extent the required vesting conditions are not met resulting in the forfeiture of the share-based awards, previously recognized compensation expense relating to those awards are reversed. The Company elected to account for the forfeitures as occurred. The granted stock options reflected an expected term based on our expectations for exercise activity. Changes in any of these assumptions could result in a revised estimate of fair value of the granted stock options, which would impact the amount of expense recognized over the requisite service period, and could materially affect the total fair value or the amount of expense recognized in a particular period. 150 Table of Contents As of December 31, 2025, total unrecognized share‑based compensation amounted to US$1.7 million under the 2020 Plan and US$19.8 million under the 2022 Plan, with these amounts expected to be recognized over weighted‑average periods of 1.6 years and 1.33 years, respectively. We also had US$10 million of unrecognized compensation cost related to service‑only RSUs granted under the 2022 Plan. All such unrecognized amounts may be adjusted for actual forfeitures occurring in the future. A 10% change in our share-based compensation expense for the year ended December 31, 2025, would have affected pre-tax earnings by approximately $ 3.9 million. Recent Accounting Pronouncements A list of recently issued accounting pronouncements that are relevant to us is included in Note 2 “Summary of Significant Accounting Policies — Recent Accounting Pronouncements” of our consolidated financial statements beginning on page F - 18 of this Annual Report.