Financial Data and Key Metrics Changes - In 2024, the company reported R&D expenses of $29.9 million, an increase from $28.8 million in 2023, primarily due to higher royalty payments and increased share-based compensation [32] - General and administrative (G&A) expenses rose to $10.1 million in 2024 from $6.8 million in 2023, driven by increased share-based compensation [33] - The net loss for 2024 was $36.1 million, compared to $31.6 million in 2023 [33] - Cash and investments at the end of 2024 totaled $145.2 million, up from $88.2 million at the end of 2023 [33] Business Line Data and Key Metrics Changes - The company is advancing its lead product, Tinlarebant, in Phase 3 trials for Stargardt disease and geographic atrophy, with significant progress reported in both trials [4][10] - The Phase 3 DRAGON trial for Stargardt disease has maintained a sample size of 104 subjects after an interim analysis recommended proceeding without modifications [8][20] - The PHOENIX trial for geographic atrophy has enrolled over 400 subjects, with plans to increase enrollment from 450 to 500 subjects [10][30] Market Data and Key Metrics Changes - Tinlarebant has received multiple designations, including Rare Pediatric Disease and Fast Track in the U.S., and Pioneer Drug designation in Japan, indicating a significant unmet medical need [6] - The company is uniquely positioned with ongoing global Phase 3 trials, which may lead to the first oral treatment for degenerative retinal diseases [11] Company Strategy and Development Direction - The company aims to position Tinlarebant as a first-in-class oral therapy for Stargardt disease and geographic atrophy, focusing on early intervention to slow disease progression [4][5] - The strategy includes expanding patient enrollment in clinical trials to enhance the chances of regulatory approval and market success [40] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the regulatory review process following the DSMB's recommendation for drug approval based on interim results [38] - The company plans to expedite a second Phase 3 trial for geographic atrophy if positive signals are observed in the ongoing PHOENIX trial [44] Other Important Information - The company raised $15 million in gross proceeds through a registered direct offering in February 2025, ensuring a full year's cash runway [34] - The dropout rate in the PHOENIX trial is approximately 20%, which is considered manageable given the elderly population involved [52] Q&A Session Summary Question: Potential outcomes from regulatory interactions regarding Stargardt disease - Management believes that regulatory agencies will likely align with the DSMB's positive recommendation for drug approval, but if not, they will continue with the study [38] Question: Reason for increasing sample size in the GA trial - The decision to increase the sample size to 500 patients was driven by smooth progress in the GA study and the desire to enhance the chances of success [40] Question: Update on discontinuation rates in the GA trial - The dropout rate for the PHOENIX trial is approximately 20%, which is lower than historical rates for similar studies [52] Question: Approval expectations for Stargardt disease patients - Management indicated that if efficacy is demonstrated in adolescents, it should facilitate approval for adults as well [56] Question: Current status of regulatory submissions - The company is working with multiple CROs for regulatory submissions, and the process is currently underway [78] Question: Expected treatment effect in the DRAGON study - The DRAGON study is powered for a 40% treatment effect with 80% power to detect that effect at the second year [84]

Financial Performance - The company reported a net loss of $36.1 million or ($1.18) per share for the year ended December 31, 2024, compared to a net loss of $31.6 million or ($1.19) per share in 2023[13]. - Other income for the year ended December 31, 2024, was $3.9 million, significantly up from $0.05 million in 2023, primarily from interest on cash and investments[12]. - The total operating expenses for the year ended December 31, 2024, were $39.996 million, compared to $31.668 million in 2023[17]. Research and Development - For the year ended December 31, 2024, research and development expenses increased to $29.9 million from $24.8 million in 2023, primarily due to increased royalty payments and share-based compensation[10]. - The ongoing Phase 3 DRAGON trial for Tinlarebant is expected to complete by Q4 2025, with the Data Safety Monitoring Board recommending continuation without modifications[4]. - The company completed the Phase 1b portion of the DRAGON II trial with six subjects evaluated in Japan in Q3 2024, targeting approximately 60 subjects for the Phase 2/3 trial[5]. - The PHOENIX trial for geographic atrophy subjects has enrolled 406 out of an increased target of 500 subjects[7]. Cash and Assets - As of December 31, 2024, the company had $31.7 million in cash, down from $88.2 million on December 31, 2023[8]. - Total assets increased from $94,642 million in 2023 to $152,132 million in 2024, representing a growth of 60.5%[19]. - Total current assets rose from $89,940 million in 2023 to $147,073 million in 2024, an increase of 63.5%[19]. - Cash reserves decreased significantly from $88,157 million in 2023 to $31,677 million in 2024, a decline of 64.1%[19]. Equity and Liabilities - Shareholders' equity grew from $90,431 million in 2023 to $145,821 million in 2024, reflecting a 61.2% increase[19]. - Additional paid-in capital increased from $162,305 million in 2023 to $254,125 million in 2024, a rise of 56.5%[19]. - Accumulated deficit widened from $71,503 million in 2023 to $107,647 million in 2024, indicating a deterioration of 50.6%[19]. - Total liabilities increased from $4,211 million in 2023 to $6,311 million in 2024, marking a growth of 49.9%[19]. - Current liabilities rose from $3,633 million in 2023 to $6,050 million in 2024, an increase of 66.5%[19]. - Operating lease liabilities (non-current) decreased from $578 million in 2023 to $261 million in 2024, a reduction of 54.8%[19]. - Other receivables due from related parties were recorded at $18 million in 2023, with no amounts reported in 2024[19]. Fundraising - The company raised $15 million in gross proceeds from a registered direct offering completed on February 5, 2025[6].

Product Development and Regulatory Approval - The company is highly dependent on the success of its lead product candidate, Tinlarebant, which is currently in clinical trials for the treatment of autosomal recessive Stargardt disease (STGD1) [28]. - The company currently has no products approved for commercial sale and may never be able to develop marketable products, making investment riskier compared to companies with multiple late-stage product candidates [28]. - Significant delays in obtaining regulatory approvals for Tinlarebant could adversely affect the company's business and financial condition [42]. - The success of Tinlarebant will depend on successful completion of ongoing clinical trials and timely receipt of marketing approvals from regulatory authorities [30]. - The company faces risks related to the regulatory approval processes, which are time-consuming and may evolve over time [25]. - The regulatory approval process for the company's product candidates is expensive and may take many years, with potential delays due to additional required clinical trials [72]. - The company has never obtained marketing approval for any product candidate, and regulatory authorities may require additional trials or studies before reconsidering applications [73]. - The company may need to conduct additional clinical trials if initial results are not positive, which could delay regulatory approval and commercialization prospects [58]. - The inability to obtain marketing approvals could prevent the commercialization of product candidates, significantly harming the business [74]. - Regulatory authorities may impose additional restrictions or require recalls if product candidates are found to be less effective or cause undesirable side effects post-approval [75]. - The company may face challenges in obtaining regulatory approvals from authorities such as the FDA, TGA, NMPA, EMA, and PMDA, which can be time-consuming and uncertain [121]. - Regulatory approval processes may require additional preclinical or clinical data, potentially delaying commercialization plans [124]. - Ongoing regulatory obligations post-approval may lead to significant additional expenses and potential penalties for non-compliance [138]. Financial Performance and Funding - The company has incurred significant net operating losses of approximately US$12.6 million, US$31.6 million, and US$36.1 million for the years ended December 31, 2022, 2023, and 2024, respectively, with an accumulated deficit of approximately US$107.6 million as of December 31, 2024 [115]. - The company expects to continue incurring substantial expenses related to ongoing clinical trials and research and development efforts, which will require additional funding [107]. - The net cash used in operating activities was approximately US$11.5 million, US$29.8 million, and US$29.2 million for the years ended December 31, 2022, 2023, and 2024, respectively [106]. - The company has no products approved for commercial sale and has not generated any revenue from product sales, indicating a high level of uncertainty regarding future profitability [112]. - The company anticipates that its expenses will increase significantly as it seeks regulatory approvals and commercializes its product candidates [116]. - The company has recorded net cash outflow from operating activities since its inception, indicating a need for additional financing to fund operations [105]. - The company may seek additional funding through equity offerings, debt financings, collaborations, and licensing arrangements, which could dilute existing shareholders' ownership [119]. Clinical Trials and Patient Enrollment - The timely completion of clinical trials is contingent on the ability to enroll a sufficient number of patients, which may be affected by various factors [48]. - Clinical trials may face competition for patient enrollment, which could limit the number and types of patients available for the company's trials [53]. - The company may incur additional costs or experience delays if clinical trials do not demonstrate safety and efficacy to the satisfaction of regulatory authorities [54]. - Manufacturing issues, including supply quality and compliance with good manufacturing practices, could significantly impact the company's clinical trials and product availability [71]. Intellectual Property and Competition - The company holds a portfolio of 23 issued U.S. patents and 9 pending U.S. patent applications, along with 23 issued foreign patents and 44 pending foreign patent applications, providing protection in multiple regions [151]. - The patent position of the company is uncertain, and challenges to patent validity could limit competitive advantages [152]. - The company may face increased competition if a competitor's drug is approved earlier for the same condition, potentially impacting market share despite orphan drug exclusivity [134]. - Competition in the biopharmaceutical industry is intense, with competitors potentially achieving regulatory approval and market penetration faster [82]. - The company may face claims regarding the wrongful use of trade secrets from former employers of its employees, which could lead to litigation and loss of intellectual property rights [185]. - There is a risk of costly litigation if third parties claim that the company's products infringe on their intellectual property rights [193]. Operational Risks and Management - The company must maintain an effective system of internal controls to accurately report its results and prevent fraud, as failure to do so could adversely affect investor confidence [25]. - The adequacy of the company's internal control over financial reporting is crucial for maintaining investor confidence and preventing material misstatements [205]. - The company may experience difficulties in managing growth as it plans to significantly increase the number of employees and consultants [216]. - The company is exposed to risks of misconduct by employees and contractors, which could harm its reputation and financial standing [215]. - The company may not be able to effectively manage outsourced activities, potentially leading to delays in regulatory approvals [217]. Market and Commercialization Challenges - Market acceptance of product candidates is crucial for commercial success, and failure to achieve this could result in insufficient revenue generation [79]. - Reimbursement for approved product candidates may be limited or unavailable, impacting the ability to commercialize successfully [89]. - The potential market opportunity for the company's product candidates may be limited, depending on regulatory approvals and market acceptance [98]. - Legislative changes, such as the ACA, may impose additional costs and requirements on the company, affecting marketing approvals and pricing strategies [146]. Risks Related to Manufacturing and Supply Chain - The company relies entirely on third-party contractors for cGMP manufacturing capabilities, which poses risks of delays in clinical supply and commercialization if issues arise [70]. - The company relies on third parties for conducting clinical trials and manufacturing, and any failure in these relationships could harm its business [25]. - The company maintains liability insurance for clinical trials, but coverage may be insufficient for product liability claims [219].

Core Insights - Belite Bio, Inc is advancing its clinical development of Tinlarebant, a novel therapeutic for degenerative retinal diseases, with significant progress in the Phase 3 DRAGON trial for Stargardt disease type 1 (STGD1) [2][3][4] Financial Results - As of December 31, 2024, the company reported cash of $31.7 million, a decrease from $88.2 million on December 31, 2023 [8][20] - The company had $113.5 million in investments as of December 31, 2024, compared to none on December 31, 2023 [8][20] - For the year ended December 31, 2024, research and development expenses were $29.9 million, up from $24.8 million in 2023 [10][18] - General and administrative expenses for the year ended December 31, 2024, were $10.1 million, compared to $6.8 million in 2023 [11][18] - The net loss for the year ended December 31, 2024, was $36.1 million, or ($1.18) per share, compared to a net loss of $31.6 million, or ($1.19) per share in 2023 [13][18] Clinical Development Highlights - The DRAGON trial for STGD1 subjects is ongoing, with completion expected in Q4 2025, following a positive interim analysis by the Data Safety Monitoring Board (DSMB) [4][5] - The PHOENIX trial for geographic atrophy (GA) subjects is also ongoing, with 406 subjects enrolled and an increased sample size target of 500 [14] - Tinlarebant has received multiple designations, including Fast Track and Orphan Drug Designation in the U.S. for STGD1 [5][16] Corporate Actions - The company raised $15 million in gross proceeds through a registered direct offering on February 5, 2025, with potential additional proceeds from warrant exercises [4][7] - A conference call and webcast were scheduled for March 17, 2025, to discuss financial results and business updates [15]

Group 1 - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing therapies for degenerative retinal diseases and specific metabolic diseases [2] - The company has filed its annual report on Form 20-F for the year ended December 31, 2024, with the Securities and Exchange Commission [1] - Belite's lead candidate, Tinlarebant, is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for Stargardt disease type 1, as well as a Phase 3 study (PHOENIX) for Geographic Atrophy [2] Group 2 - The annual report is available on the company's website, and shareholders can request a hard copy free of charge [1] - Belite Bio targets significant unmet medical needs in retinal diseases, such as Stargardt disease type 1 and Geographic Atrophy in advanced dry age-related macular degeneration [2]

Group 1 - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs [1][3] - The company will host a webcast on March 17, 2025, at 4:30 p.m. Eastern Time to discuss its financial results and provide a business update for Q4 and the full year ended December 31, 2024 [1][2] - Belite's lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye and is currently undergoing multiple clinical trials, including Phase 3 studies [3]

Company Overview - Belite Bio, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs [3] - The company is targeting conditions such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) associated with advanced dry age-related macular degeneration (AMD) [3] Product Development - Belite's lead candidate, Tinlarebant, is an oral therapy designed to reduce toxin accumulation in the eye [3] - Tinlarebant is currently undergoing multiple clinical trials: a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for adolescent STGD1 subjects, as well as a Phase 3 study (PHOENIX) for subjects with GA [3] Upcoming Events - The executive management team will participate in the Leerink Partners Global Healthcare Conference from March 10-12, 2025, in Miami, FL [1] - A fireside chat hosted by the company is scheduled for March 10, 2025, at 3:00 pm ET [1] - A webcast of the fireside chat will be available on the investor relations section of the Belite Bio website and will be archived for 90 days post-presentation [2]

Core Insights - Belite Bio, Inc has announced that the Data Safety Monitoring Board (DSMB) has recommended the continuation of the Phase 3 "DRAGON" trial for Tinlarebant in adolescent Stargardt disease patients without modifications following an interim analysis [1][7] - The trial is expected to be completed by Q4 2025, including a three-month follow-up period [4][7] Trial Design and Recommendations - The DRAGON trial employs an adaptive sample size re-estimation to enhance statistical power based on treatment effects observed during the interim analysis [2] - The DSMB's recommendation indicates that an increase in sample size is not necessary, and they have advised submitting the data for further regulatory review [2] Safety and Efficacy Observations - Tinlarebant has shown a well-tolerated safety profile consistent with previous data, with visual acuity stabilized in most subjects, showing a mean change from baseline of less than three letter scores under both standard and low luminance conditions [3][4] - The primary efficacy endpoint of the trial is the growth rate of atrophic lesions, alongside safety and tolerability assessments [4][7] Regulatory Designations - Tinlarebant has received multiple designations, including Orphan Drug Designation in the U.S., Europe, and Japan, Rare Pediatric Disease designation, Fast Track designation in the U.S., and Sakigake (Pioneer Drug) designation in Japan [4][7] Company Overview - Belite Bio focuses on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs, including Stargardt disease and Geographic Atrophy in advanced dry age-related macular degeneration [6]

Core Viewpoint - Belite Bio, Inc has announced a securities purchase agreement for the sale of 258,309 American Depositary Shares (ADSs) and warrants, aiming to raise approximately $15 million in gross proceeds, with potential additional proceeds from warrant exercises [1][2]. Group 1: Offering Details - The purchase price for the ADSs and accompanying warrants is set at $58.07 per ADS, equivalent to the current closing price [1]. - The offering is expected to close around February 7, 2025, pending customary closing conditions [1]. - The net proceeds from the offering will be utilized for working capital and general corporate purposes [2]. Group 2: Regulatory Information - The offering is conducted under a shelf registration statement on Form F-3, which was filed with the U.S. Securities and Exchange Commission (SEC) and became effective on January 27, 2025 [3]. - A prospectus supplement will be filed with the SEC and will be available on the SEC's website [3]. Group 3: Company Overview - Belite Bio, Inc is focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs, including Stargardt disease and Geographic Atrophy in advanced dry age-related macular degeneration [5].

| --- | --- | --- | --- | |-------|----------------------------------------------------------------------------|-------|-------| | | | | | | | | | | | | Early Intervention with | | | | | Mission for Vision Nasdaq: BLTE an Oral Treatment for Macular Degeneration | | | Forward-Looking Statements and Legal Disclaimer This presentation (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or so ...