Summary of Belite Bio Conference Call Company Overview - **Company**: Belite Bio (NasdaqCM:BLTE) - **Product**: Tinlarebant, an oral treatment for Stargardt disease Industry Context - **Disease**: Stargardt disease, an orphan disease affecting the retina, leading to blindness - **Prevalence**: Approximately 53,000 patients in the U.S., with a global prevalence of about 1 in 7,000, making it one of the top 10 most prevalent inherited diseases [4][8][5] Core Points and Arguments - **Mechanism of Action**: - Stargardt disease is caused by mutations in the ABCA4 gene, leading to toxic accumulation of all-trans retinal in photoreceptors [12][13] - Tinlarebant works by antagonizing the retinol binding protein (RBP4), reducing retinal delivery to the eye and thereby decreasing toxic accumulation [14][15] - **Clinical Trials**: - **Phase 1B/2 Trial**: Showed stabilization of vision over 2 years with a significant reduction in disease progression compared to natural history studies [21][22] - **Phase 3 DRAGON Trial**: Enrolled 104 patients, demonstrating a 36% reduction in the progression rate of definitely decreased autofluorescence (DDAF) lesions over 2 years, statistically significant with a P value of 0.0033 [27][28] - Secondary endpoint showed a 34% reduction in DAF lesions, also statistically significant [28] - **Safety Profile**: - Reported 6 serious adverse events, with none related to the drug. Common mild side effects included longer dark adaptation times and temporary visual discoloration [37][40] - **Regulatory Plans**: - Anticipated FDA submission in the first half of the year, with potential approval in the first quarter of the following year [43][44] - Plans for submissions to other global regulators, including EMA and PMDA in Japan, with a focus on a single trial for multiple regulatory approvals [45][46] Additional Important Information - **Market Opportunity**: Stargardt disease represents a significant unmet medical need, with no approved treatments currently available [8] - **Geographic Variability**: Prevalence of ABCA4 mutations varies globally, with the highest rates in populations of African descent [58][60] - **Future Studies**: Ongoing PHOENIX trial for geographic atrophy, with expectations to outperform existing therapies [66] - **Commercial Strategy**: Focus on genetic testing to confirm ABCA4 mutations, essential for patient eligibility for Tinlarebant [73] Conclusion Belite Bio is positioned to address a significant unmet need in the treatment of Stargardt disease with its innovative oral therapy, Tinlarebant, backed by promising clinical trial results and a clear regulatory pathway. The company is also preparing for commercialization and expanding its research into related conditions.

Financial Data and Key Metrics Changes - For Q4 2025, R&D expenses were $14.6 million, up from $7.3 million in Q4 2024, primarily due to expenses related to the DRAGON II trial and lower Australian R&D tax incentives [9][10] - SG&A expenses increased to $13.5 million from $4.2 million in Q4 2024, driven by higher share-based compensation and professional service fees [11][12] - The net loss for Q4 2025 was $25.3 million, compared to a net loss of $10.1 million in Q4 2024 [12] - For the full year, R&D expenses were $45.4 million, up from $29.9 million in 2024, while SG&A expenses rose to $38.9 million from $10.1 million [13][14] - The full year net loss was $77.6 million, compared to a net loss of $36.1 million in 2024 [15] Business Line Data and Key Metrics Changes - The DRAGON trial met its primary efficacy endpoint with a 36% reduction in the growth rate of atrophy lesions compared to placebo, positioning the company for regulatory engagement [5] - Enrollment in the phase III PHOENIX trial was completed with 430 subjects [7] Company Strategy and Development Direction - The company aims for a transformative year in 2026, with a planned NDA submission to the FDA in Q2 2026 and commercialization preparations for Stargardt disease [8] - Key leadership positions have been filled, and the company is building its organization in various functions including sales and marketing [8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming NDA submission and the potential for Tinlarebant to be the first approved therapy for Stargardt disease [4][8] - The company is focused on ensuring awareness among physicians and patients regarding the new treatment [33] Other Important Information - The company closed the year with $772.6 million in cash equivalents, significantly up from $145.2 million at the end of 2024, indicating a strong balance sheet [16] Q&A Session Summary Question: NDA submission and cash usage - The NDA will be a rolling submission, with DRAGON II data relevant for Japan only. The company expects R&D activities to cost about $150 million over the next three years, with commercialization costs between $150 million and $200 million [20][21] Question: Pricing guidance - While it's early to set a price, the average price for rare disease drugs in the US is around $350,000, and the company expects to perform better than that [25] Question: Launch timing and challenges - The expected launch is by Q1 2027, with a focus on genetic testing and patient awareness as key factors for success [32][33] Question: Ex-US regulatory strategy - The company prioritizes the FDA for NDA submission, with plans to follow up with EMA and other regions afterward [40][70] Question: Prevalence of Stargardt disease - The estimated prevalence of Stargardt disease in the US is about 53,000 patients, based on genetic databases [76][77]

Financial Data and Key Metrics Changes - For Q4 2025, R&D expenses were $14.6 million, up from $7.3 million in Q4 2024, primarily due to expenses related to the DRAGON II trial and lower Australian R&D tax incentives [7][8] - SG&A expenses increased to $13.5 million from $4.2 million in Q4 2024, driven by higher share-based compensation and professional service fees [8][9] - The net loss for Q4 2025 was $25.3 million, compared to $10.1 million in Q4 2024, while the non-GAAP net loss was $13.6 million, up from $5.9 million in Q4 2024 [9][10] - For the full year, R&D expenses totaled $45.4 million, compared to $29.9 million in 2024, and SG&A expenses were $38.9 million, up from $10.1 million in 2024 [9][10] - The company reported a full-year net loss of $77.6 million, compared to a net loss of $36.1 million in 2024, with a non-GAAP net loss of $38.7 million compared to $27.2 million in 2024 [10] Business Line Data and Key Metrics Changes - The DRAGON trial met its primary efficacy endpoint, showing a 36% reduction in the growth rate of atrophy lesions compared to placebo, which positions the company well for regulatory engagement [5][6] - Enrollment in the DRAGON II study reached 72 subjects, with expectations to finalize between 72 and 75 subjects [5] - The PHOENIX trial completed enrollment with 430 subjects [6] Company Strategy and Development Direction - The company aims for a transformative year in 2026, with a planned NDA submission to the FDA in Q2 2026 and ongoing commercialization preparations for Stargardt disease [6][11] - Key leadership positions have been filled, and the company is building its organization in various functions including sales and marketing [6] - The company expects to allocate approximately $150 million for R&D activities and $200 million for commercialization over the next three years [16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming NDA submission and the potential for Tinlarebant to become the first approved therapy for Stargardt disease [4][6] - The company anticipates launching the product by Q1 2027, focusing on genetic testing and awareness among physicians [27][28] - Management believes that the absence of existing treatments for Stargardt disease will facilitate a straightforward launch [28] Other Important Information - The company completed a $402 million public offering in Q4, significantly strengthening its balance sheet with $772.6 million in cash equivalents at year-end [6][11] - The company is prioritizing the U.S. market for regulatory submissions, with plans to follow up with submissions in Europe and Japan [35][66] Q&A Session Summary Question: Is the NDA submission expected to be rolling, and what role will DRAGON II play? - Management confirmed that the NDA will be a rolling submission, with DRAGON II data specifically for Japan [15] Question: What are the expected uses of cash for trials and commercialization? - The company expects to spend about $150 million on R&D and $200 million on commercialization over the next three years [16] Question: Can you provide guidance on pricing for the drug? - Management indicated that while it's early to set a price, they expect it to be higher than the average rare disease drug price of $350,000 [20] Question: What is the expected launch timing after NDA submission? - The company anticipates launching by Q1 2027, with a focus on genetic testing and physician awareness [27][28] Question: What are the key gating items before NDA submission? - The primary gating item is the finalization of the clinical study report [22] Question: How important is visual acuity for approval and reimbursement? - Visual acuity is important for regulators, but management believes it may not be a realistic target for efficacy signals in Stargardt disease [55] Question: What is the true prevalence of Stargardt disease? - Management estimates about 53,000 patients in the U.S. are affected by ABCA4 mutated retinal disease, including Stargardt disease [71] Question: Are there plans to expand into the pediatric population? - The company has an approved pediatric investigational plan with EMA, set to initiate in April [74]

Financial Data and Key Metrics Changes - For Q4 2025, R&D expenses were $14.6 million, up from $7.3 million in Q4 2024, primarily due to DRAGON II trial expenses and lower Australian R&D tax incentives [6][8] - SG&A expenses increased to $13.5 million from $4.2 million in Q4 2024, driven by higher share-based compensation and professional service fees [8][10] - The net loss for Q4 2025 was $25.3 million, compared to $10.1 million in Q4 2024, while the non-GAAP net loss was $13.6 million, up from $5.9 million in Q4 2024 [8][9] - For the full year, R&D expenses totaled $45.4 million, compared to $29.9 million in 2024, and SG&A expenses were $38.9 million, up from $10.1 million in 2024 [9][10] - The full year net loss was $77.6 million, compared to $36.1 million in 2024, with a non-GAAP net loss of $38.7 million, up from $27.2 million in 2024 [10][11] Business Line Data and Key Metrics Changes - The company achieved significant milestones in 2025, including the successful phase 3 DRAGON trial, which demonstrated a 36% reduction in the growth rate of atrophy lesions compared to placebo [4][5] - Enrollment in the DRAGON II study reached 72 subjects, with expectations to finalize between 72 and 75 subjects [4] - The phase 3 PHOENIX trial in GA completed enrollment with 430 subjects [5] Market Data and Key Metrics Changes - The company closed the year with $772.6 million in cash equivalents, significantly up from $145.2 million at the end of 2024, positioning it well for future objectives [11] Company Strategy and Development Direction - The primary focus for 2026 is the planned NDA submission to the FDA in Q2 2026, with commercialization preparations for Stargardt disease already underway [5][6] - The company aims to build its organization in sales, market access, medical affairs, marketing, regulatory, and operations [6] - The company is prioritizing the U.S. market for regulatory submissions, with plans to follow up with EMA and other regions post-U.S. approval [33][59] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming year, highlighting the transformative potential of Tinlarebant as a treatment for Stargardt disease [3][5] - The management noted that the absence of existing treatments for Stargardt disease could facilitate a straightforward launch process [27] - The company anticipates launching by Q1 2027, with a focus on genetic testing and patient awareness as key factors for success [26][27] Other Important Information - The company completed a $402 million public offering in Q4 2025, which supports its commercialization and pipeline development efforts [5] - Management indicated that pricing for the drug is still under consideration, with expectations that it could exceed the average rare disease drug price of approximately $350,000 [19] Q&A Session Summary Question: NDA submission process and cash usage - The NDA will be a rolling submission, with DRAGON II data relevant only for Japan. The company expects to spend about $150 million on R&D activities and $150-$200 million on commercialization over the next three years [14][15] Question: Pricing guidance and NDA gating items - Pricing is still being determined, but the company expects to set a price higher than the average for rare disease drugs. The key gating item before NDA submission is the finalization of the clinical study report [19][21] Question: Launch timing and challenges - The company expects to launch by Q1 2027, with a sales team focused on genetic testing and brand awareness. Challenges include patient and physician awareness of the treatment [26][27] Question: Ex-U.S. regulatory strategy - The company prioritizes the U.S. FDA submission, with plans to follow up with EMA and other regions after securing U.S. approval [33][59] Question: Stargardt disease prevalence and awareness - The company estimates about 53,000 patients in the U.S. are affected by Stargardt disease, and having a treatment available is expected to increase awareness and diagnosis of undiagnosed patients [64][66] Question: Pediatric studies - The company has an approved pediatric investigational plan with EMA to study safety and efficacy in children aged 3 to 11, with plans to expand into the pediatric population [68][70]

Q4 and FYE 2025 Financial Results Conference Call March 2, 2026, 4:30 p.m. ET Nasdaq: BLTE For more info, please visit: www.belitebio.com Belite Bio Participants Belite Management Team Hao-Yuan Chuang, CFA, MBA, FRM (CFO) B E L I T E B I O / 2 Tom Lin, MMED, PhD, MBA (Chairman, CEO) Hendrik Scholl, MD, MA (CMO) Nathan Mata, PhD (CSO) • 16+ years of executive management roles in biotech • Over 10 new drug developments in multiple therapeutic areas including ophthalmology • University of Sydney, University of ...

Core Insights - Belite Bio has completed enrollment of 60 subjects in the Phase 2/3 DRAGON II clinical trial for tinlarebant, targeting Stargardt disease type 1 (STGD1) [1][2][7] - The trial is a global, 24-month, randomized, double-masked, placebo-controlled study involving participants aged 12 to 20 years from Japan, the United States, and the United Kingdom [2] - The company plans to submit a New Drug Application (NDA) to the FDA for tinlarebant in the first half of 2026 [3][7] Clinical Trial Details - The DRAGON II trial aims to evaluate the efficacy, safety, and tolerability of tinlarebant in adolescent patients with STGD1 [2] - Participants are randomized 1:1 to receive either tinlarebant or placebo [2] - The completion of enrollment reflects strong engagement from the Stargardt disease community [3] Drug Information - Tinlarebant is an oral therapy designed to reduce the accumulation of vitamin A-based toxins (bisretinoids) that cause retinal disease in STGD1 [4] - The drug works by modulating serum retinol binding protein 4 (RBP4) levels, thereby reducing bisretinoid formation [4] - Tinlarebant has received multiple designations, including Breakthrough Therapy Designation and Fast Track Designation in the U.S. [4] Company Overview - Belite Bio focuses on developing novel therapeutics for degenerative retinal diseases and specific metabolic diseases [5] - The company has completed a Phase 3 trial (DRAGON) in adolescent STGD1 subjects and is also evaluating tinlarebant in a Phase 3 trial (PHOENIX) for geographic atrophy [5] - The company is committed to advancing research for adolescents with Stargardt disease globally [3]

Core Viewpoint - Belite Bio, Inc has announced a public offering of 2,272,727 American Depositary Shares (ADSs) at a price of $154.00 per ADS, aiming to raise approximately $350 million for commercialization and pipeline development [1][2]. Group 1: Offering Details - The public offering consists of 2,272,727 ADSs priced at $154.00 each, with an additional option for underwriters to purchase up to 340,909 more ADSs [1]. - The expected gross proceeds from the offering are approximately $350 million before deducting underwriting discounts and commissions [1]. - The closing of the offering is anticipated to occur around December 3, 2025, pending customary closing conditions [1]. Group 2: Use of Proceeds - The net proceeds from the offering will be allocated for commercialization preparation, including building an in-house commercialization team and establishing sales networks [2]. - Funds will also be used for the development and expansion of the company's drug pipeline [2]. - Additional proceeds will support working capital and other general corporate purposes [2]. Group 3: Company Overview - Belite Bio is focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs, such as Stargardt disease type 1 and geographic atrophy in advanced dry age-related macular degeneration [6]. - The company's lead candidate, Tinlarebant, is an oral therapy aimed at reducing bisretinoid toxins in the eye and has completed a Phase 3 trial in adolescent STGD1 subjects [6].

Core Viewpoint - Belite Bio Inc. has received a stock upgrade to "Buy" with a price target increase, driven by positive trial results for its drug Tinlarebant targeting a rare eye disease [1][5]. Group 1: Stock Performance - H.C. Wainwright upgraded Belite Bio's stock from a price target of $98 to $185, reflecting strong market sentiment [1][5]. - Following the successful trial results, the stock price rose to approximately $153.59, marking an increase of about 11.75% or $16.15 [4]. - The stock has fluctuated between $130.06 and $154.99, with the latter being its highest price in the past year [4]. - Belite Bio's market capitalization is approximately $5.36 billion, with a trading volume of 596,581 shares, indicating strong investor interest [4]. Group 2: Clinical Trial Results - The Phase 3 DRAGON trial for Tinlarebant demonstrated a 36% reduction in lesion growth compared to placebo, marking a significant milestone for treating Stargardt disease type 1 (STGD1) [2][5]. - The trial enrolled 104 patients and is noted as the first successful pivotal trial for STGD1, a rare eye disease causing progressive vision loss [2]. Group 3: Future Plans - Belite Bio plans to file a New Drug Application (NDA) with the FDA in the first half of 2026, which is a crucial step towards commercialization [3][5]. - Tinlarebant works by lowering serum retinol binding protein 4 (RBP4) levels, thereby reducing harmful bisretinoids in the eye, offering hope for over 50,000 STGD1 patients in the U.S. [3].

Core Viewpoint - Belite Bio, Inc has initiated an underwritten public offering of American Depositary Shares (ADSs) to support its commercialization and development efforts in the field of degenerative retinal diseases [1][2] Group 1: Offering Details - The public offering consists of ADSs, each representing one ordinary share, with all securities being sold by Belite Bio [1] - The company plans to grant underwriters a 30-day option to purchase additional ADSs at the public offering price, subject to market conditions [1] - The offering is contingent on market conditions, and there is no assurance regarding its completion or the actual size and terms [1] Group 2: Use of Proceeds - Net proceeds from the offering will be allocated for commercialization preparation, including building an in-house commercialization team and establishing sales networks [2] - Funds will also be used for the development and expansion of the company's pipelines [2] - Additional proceeds will support working capital and other general corporate purposes [2] Group 3: Company Overview - Belite Bio is focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs, such as Stargardt disease type 1 and geographic atrophy in advanced dry age-related macular degeneration [6] - The lead candidate, Tinlarebant, is an oral therapy aimed at reducing the accumulation of bisretinoid toxins in the eye and has completed a Phase 3 trial in adolescent STGD1 subjects [6] - The company is currently evaluating Tinlarebant in a Phase 2/3 trial and a Phase 3 trial for different indications [6]

Core Insights - Belite Bio presented topline data from its Phase III trial of Tinlarebant for Stargardt disease, known as the DRAGON study, which is the largest interventional trial conducted in adolescent patients with this condition [2] Group 1: Study Overview - The DRAGON study is a pivotal 2-year trial focusing on the efficacy of Tinlarebant in treating Stargardt disease [2] - The trial involved a significant number of adolescent patients, highlighting its scale and importance in the field of retinal diseases [2] Group 2: Leadership and Expertise - The conference call featured key executives from Belite Bio, including Dr. Tom Lin (Chairman and CEO) and Dr. Hendrik Scholl (Chief Medical Officer), who provided insights into the study [4] - Dr. Hendrik Scholl, a notable figure in retinal disease research, served as the coordinating principal investigator for the ProgStar Study, adding credibility to the DRAGON study [4]