Date: Wednesday, May 14, 2025 Time: 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) Webcast Link: https://events.q4inc.com/attendee/137642555 Media and Investor Relations Contact: Jennifer Wu ir@belitebio.com Webcast Link Instructions You can join the live webcast by visiting the link above or the "Presentations & Events" section of the Company's Investor Relations website at https://investors.belitebio.com/presentations-events/events. A replay will be available for approximately 90 days after the event. Ab ...

SAN DIEGO, April 29, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc. (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the Company will give two poster presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting being held May 4–8, 2025, in Salt Lake City, Utah. The presentations will highlight baseline patient ...

Financial Data and Key Metrics Changes - In 2024, R&D expenses were $29.9 million, up from $28.8 million in 2023, primarily due to increased royalty payments and share-based compensation [33] - G&A expenses rose to $10.1 million in 2024 from $6.8 million in 2023, driven by share-based compensation [34] - The net loss for 2024 was $36.1 million, compared to $31.6 million in 2023 [34] - Cash and investments at the end of 2024 totaled $145.2 million, up from $88.2 million at the end of 2023 [34] Business Line Data and Key Metrics Changes - The company is advancing Tinlarebant in Phase 3 trials for Stargardt disease and geographic atrophy, with significant progress reported in both trials [5][9] - The Phase 3 DRAGON trial for Stargardt disease has maintained a sample size of 104 subjects after an interim analysis recommended no modifications [9][21] - The PHOENIX trial for geographic atrophy has enrolled over 400 subjects, with plans to increase enrollment to 500 [11][31] 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 need in the market [7] - The company is uniquely positioned with ongoing global Phase 3 trials, which may lead to the first oral treatment for degenerative retinal diseases [12] 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 [5][6] - The strategy includes expanding the sample size in the PHOENIX trial to enhance the chances of success [41] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding regulatory reviews following the DSMB's recommendations, anticipating alignment with regulatory agencies [39] - The company remains focused on completing trials and maintaining a full-year cash runway, with expectations for continued progress in clinical studies [35] Other Important Information - The withdrawal rate in the DRAGON trial was 9.6%, with ocular adverse events accounting for 3.8% of withdrawals [20] - The company raised $15 million in gross proceeds in February 2025, enhancing its financial position [35] Q&A Session Summary Question: Potential outcomes from regulatory interactions regarding Stargardt - Management believes the regulatory agencies will likely align with the DSMB's positive recommendation for drug approval [39] Question: Reason for increasing sample size in GA trial - The decision was made to boost chances of success based on smooth enrollment progress [41] Question: Current discontinuation rates in the GA trial - The dropout rate for the PHOENIX trial is approximately 20%, which is common for elderly populations [53] Question: Approval potential for broader patient populations - Management indicated that efficacy in adolescents could facilitate approval for adults as well [57] Question: Difference in lesion growth between placebo and treatment in DRAGON study - The study is powered for a 40% treatment effect with 80% power to detect that effect at the second year [84]

Belite Bio, Inc. (NASDAQ:BLTE) Q4 2024 Results Conference Call March 17, 2025 4:30 PM ET Company Participants Julie Fallon - Investor Relations Tom Lin - Chairman and Chief Executive Officer Nathan Mata - Chief Scientific Officer Hendrik Scholl - Chief Medical Officer Hao-Yuan Chuang - Chief Financial Officer Conference Call Participants Jennifer Kim - Cantor Basma Radwan - Leerink Partners Yi Chen - H.C. Wainwright Bruce Jackson - Benchmark Michael Okunewitch - Maxim Group Operator Ladies and gentlemen, th ...

Exhibit 99.1 · Following a pre-specified interim analysis, the Data Safety Monitoring Board (DSMB) recommended the DRAGON trial, a pivotal Phase 3 trial in adolescent Stargardt 1 (STGD1) subjects, proceed without any modification; trial completion expected Q4 2025 (including a three-month follow-up period) · Pivotal global Phase 3 PHOENIX trial of Tinlarebant in geographic atrophy (GA) subjects is ongoing with 406 subjects enrolled · Raised $15 million in gross proceeds in a registered direct of ering on Fe ...

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]