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 ...

Financial Data and Key Metrics Changes - In Q3 2024, the company reported R&D expenses of $6.8 million, a decrease from $8.7 million in Q3 2023, primarily due to fewer CRO milestone payments related to the DRAGON trial, partially offset by increased expenses in the DRAGON 2 trial [26] - G&A expenses increased to $2.9 million in Q3 2024 from $2.2 million in the same period in 2023, mainly due to increased share-based compensation [26] - The net loss for Q3 2024 was $8.7 million, compared to $10.9 million for the same period in 2023 [26] - The company maintains a strong balance sheet with total cash of $109 million, providing an estimated four years of cash runway to execute key milestones [27] Business Line Data and Key Metrics Changes - The company is advancing its lead product, Tinlarebant, in clinical trials for Stargardt disease and geographic atrophy, with significant progress reported in both the DRAGON and PHOENIX trials [10][13] - The DRAGON 2 trial is fully enrolled with 104 patients, and the DRAGON trial is expected to enroll 60 subjects across the U.S., U.K., and Japan [11][12] Market Data and Key Metrics Changes - The company has received multiple designations for Tinlarebant, including Rare Pediatric Disease and Fast Track designations in the U.S., and orphan drug designations in the U.S., E.U., and Japan, highlighting the significant unmet need for treatments in these indications [9][10] Company Strategy and Development Direction - The company is focused on early intervention in retinal diseases, aiming to slow or stop the progression of Stargardt disease and geographic atrophy through Tinlarebant [8] - The management emphasizes the importance of their clinical programs and the potential of Tinlarebant to address significant unmet medical needs in ophthalmology [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the clinical programs and the potential of Tinlarebant, particularly in light of the promising data from ongoing trials [6][10] - The company is optimistic about the future, with plans to continue advancing its clinical trials and addressing the needs of patients suffering from retinal diseases [28] Other Important Information - The company welcomed Dr. Hendrik Scholl as the new Chief Medical Officer, who brings extensive expertise in retinal diseases and will lead the clinical program [5][6] Q&A Session Summary Question: Market research on GA patients' tolerance to ocular AEs - Management expects a higher incidence of ocular AEs in GA patients due to more extensive disease involvement, but believes patients will tolerate these AEs well [29][30] Question: Discontinuation rates in the PHOENIX trial - The discontinuation rate is around 15%, which includes all reasons, not just ocular AEs [32] Question: Interim analysis for the DRAGON trial - The interim analysis will occur once all subjects complete their month 12 visit, with data cleaning and DSMB review expected around December or early January [33][34] Question: Enrollment status in DRAGON 2 - Enrollment is ongoing, with expectations to complete by Q2 next year [35] Question: Baseline characteristics of PHOENIX trial subjects - The subjects in the PHOENIX trial have similar baseline characteristics to those in approved GA therapies, with a focus on smaller lesion sizes [38][39] Question: Differences between Tinlarebant and other therapies - Tinlarebant reduces the accumulation of toxic retinal byproducts, while other therapies may increase vitamin A levels, leading to different mechanisms of action [49][50]

Company Overview - Belite Bio, Inc is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases with significant unmet medical needs, including Stargardt Disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD) [3] Upcoming Events - The company will host a webcast on November 12, 2024, at 4:30 p.m. Eastern Time to discuss its financial results and provide a business update for the third quarter ended September 30, 2024 [1][2] Product Development - Belite's lead candidate, Tinlarebant, is an oral therapy aimed at reducing the accumulation of toxins in the eye and is currently being evaluated in multiple studies: 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]

SAN DIEGO, Nov. 03, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announces the entry into a definitive agreement for the immediate exercise of certain outstanding warrants to purchase up to an aggregate of 651,380 ordinary shares of the Company originally issued in April 2024, a ...

Scholl , Sept. 04, 2024 (GLOBE NEWSWIRE) -- Belite Bio, IncBLTE"Belite Bio""" Hendrik P. N. Scholl Scholl AMD Belite Bio Tinlarebant Belite BioTom Lin"Hendrik Scholl Belite BioScholl 2 3 Belite Bio Tinlarebant TinlarebantHendrik Scholl" " Belite Bio"Scholl "Belite Bio, " Scholl IOB EVICR.net Swiss Association for Research in Vision and Ophthalmology, ARVO-SWISS Scholl · Wilmer · Wilmer · Hendrik SchollProgStar StudyProgStar Study 365 10 280W. Richard GreenPaul HenkindAlfred-Vogt ARVOKupfer 25 Scholl Pro Re ...

Dr. Scholl is a globally recognized leader in the field of ophthalmology and the coordinating principal investigator of the largest ever natural history study of Stargardt disease SAN DIEGO, Sept. 01, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) ("Belite Bio" or the "Company"), 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 its board of direc ...

| --- | --- | --- | |-------|--------------------------------------------------------------------------------------|-----------------------------------------------| | | | | | | Q2 2024 Financial Results Conference Call August 12, 2024, 4:30 p.m. ET Nasdaq: BLTE | | | | | For more info please visit: www.belitebio.com | 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, r ...

Belite Bio, Inc (NASDAQ:BLTE) Q2 2024 Earnings Conference Call August 12, 2024 4:30 PM ET Company Participants Tom Lin - Chairman and Chief Executive Officer Nathan Mata - Chief Scientific Officer Hao-Yuan Chuang - Chief Financial Officer Conference Call Participants Basma Radwan - Leerink Partners Jennifer Kim - Cantor Yi Chen - H.C. Wainwright Bruce Jackson - Benchmark Operator Hello, and thank you for joining us to discuss Belite Bio's Second Quarter 2024 Financial Results. Joining the call today are Dr. ...