Tinlarebant

Designation is based on the pivotal Phase 3 DRAGON trial interim analysis results demonstrating Tinlarebant’s efficacy and favorable safety profileTrial completion expected by Q4 2025 (including a three-month follow-up period)Tinlarebant has previously been granted Fast Track and Rare Pediatric Disease Designations in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and the Pioneer Drug Designation in Japan for Stargardt disease SAN DIEGO, May 21, 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 U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tinlarebant for the treatment of Stargardt disease (STGD1) based on the previously reported interim data from the ongoing Phase 3 DRAGON trial. There are currently no approved treatments for STGD1. “Breakthrough Therapy Designation is an exciting milestone that underscores Tinlarebant’s potential to address a serious unmet need for patients with STGD1 — a condition where there are currently no approved therapies,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “We remain deeply committed to the Stargardt community and to advancing Tinlarebant as we prepare for the DRAGON study readout expected by the end of this year.” The Breakthrough Therapy Designation is supported by a pre-specified interim analysis of the pivotal, global Phase 3 DRAGON trial data of Tinlarebant in adolescent STGD1 patients. The designation is based on preliminary clinical evidence indicating that a drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. In addition, according to the Data Safety and Monitoring Board (DSMB), Tinlarebant continued to demonstrate a favorable safety profile consistent with prior findings and its mechanism of action. Importantly, visual acuity remained stable in the majority of participants, with mean changes from baseline of less than three letters over the course of the two-year study. “This granting represents a significant achievement for Belite Bio and serves as validation of a therapeutic approach that directly targets the underlying pathophysiology of STGD1 in order to slow or halt the disease process,” stated Dr. Nathan L. Mata, Belite Bio’s Chief Scientific Officer. Dr. Mata noted further, “Although it has been more than 26 years since the development of the first STGD1 mouse model, it is encouraging to know that the learnings from this model, and the many years spent evaluating the therapeutic benefit of targeting retinol binding protein 4 (RBP4) to reduce the accumulation of cytotoxic byproducts of vitamin A, have advanced our understanding of the disease and have led us closer than ever to the realization of an approved treatment for patients living with STGD1.” “We are very encouraged by the FDA’s decision. STGD1 is a progressive condition that typically begins in adolescence and inevitably leads to legal blindness. People living with STGD1 experience a severe loss of quality of life even though they typically have decades of their lives ahead of them,” said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. “Breakthrough Therapy Designation reinforces the importance of our work to develop a potential therapy for people who currently have limited options. We look forward to continuing to evaluate Tinlarebant’s safety and efficacy as the DRAGON trial progresses.” About FDA Breakthrough Therapy DesignationThe FDA grants Breakthrough Therapy Designation to expedite the development and regulatory review of drugs that are intended for serious or life-threatening conditions. The designation is based on preliminary clinical evidence indicating that a drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Breakthrough Therapy Designation affords all of the benefits of the fast-track program, including eligibility for rolling review and priority review, as well as additional regulatory engagement to facilitate expedited development, with the aim to bring therapies to patients more quickly. About Phase 3 DRAGONThe pivotal Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, global study designed to evaluate the safety and efficacy of Tinlarebant in adolescent patients with Stargardt disease. The trial enrolled 104 subjects across 11 jurisdictions, including the United States, United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization (Tinlarebant:placebo). The primary efficacy endpoint is the growth rate of atrophic lesions, alongside the assessment of safety and tolerability. About TinlarebantTinlarebant is an orally administered, once-a-day tablet intended as an early intervention for maintaining the health and integrity of retinal tissues in Stargardt disease type 1 (STGD1) and Geographic Atrophy (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. About Belite BioBelite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on X, Instagram, LinkedIn, and Facebook or visit us at www.belitebio.com. Important Cautions Regarding Forward Looking StatementsThis press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, interim analysis and recommendation from DSMB; Belite Bio’s advancement of, and anticipated future activities on preclinical studies, clinical development, regulatory milestones, and commercialization of its product candidates; and any other statements containing the words “expect”, “hope”, “indicate”, “look forward to”, and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio’s ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing of potential submission with FDA; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the “Risk Factors” section in Belite Bio’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. Media and Investor Relations Contact:Jennifer Wu ir@belitebio.com Julie Fallonbelite@argotpartners.com

Following a pre-specified interim analysis, an independent Data Safety Monitoring Board (DSMB) recommended the pivotal Phase 3 trial (DRAGON) of Tinlarebant in adolescent Stargardt disease (STGD1) patients proceed without any modification; trial completion expected Q4 2025 (including a three-month follow-up period)DSMB also recommended the Company submit the interim data for further regulatory review for drug approvalA pivotal global Phase 3 trial (PHOENIX) of Tinlarebant in geographic atrophy (GA) patients is ongoing with 464 of targeted 500 subjects enrolledRaised $15 million in gross proceeds in a registered direct offering on February 5, 2025Conference call and webcast on Wednesday, May 14, 2025, at 4:30 p.m. ET SAN DIEGO, May 13, 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 its financial results for the first quarter ended March 31, 2025, and provided a business update. “We continue to advance the clinical development of Tinlarebant, reaching a major milestone with the favorable interim analysis of our Phase 3 DRAGON trial earlier this year,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “We are excited by the encouraging feedback from the DSMB on the safety and efficacy outcomes in DRAGON as we work toward trial completion by the end of 2025. We are focused on maintaining strong execution across our late-stage clinical programs as we aim to deliver new treatment options for people living with degenerative retinal diseases, where there is significant unmet need.” First Quarter 2025 Business Highlights and Upcoming Milestones: Clinical Highlights Tinlarebant is an oral, once-daily, potent retinol binding protein 4 (RBP4) antagonist that decreases RBP4 levels in the blood and reduces vitamin A (retinol) delivery to the eye without disrupting systemic retinol delivery to other tissues. Vitamin A is critical for normal vision but can accumulate as toxic byproducts (bisretinoids) in individuals affected with STGD1 and GA, the advanced form of dry age-related macular degeneration (AMD), leading to retinal cell death and loss of vision. Stargardt disease (STGD1): Accumulation of cytotoxic bisretinoids compounds has been implicated in the onset and progression of STGD1, for which there are no approved treatments. Tinlarebant has been granted Fast Track and Rare Pediatric Disease Designations in the U.S.; Orphan Drug Designation in the U.S., Europe, and Japan; and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1. DRAGON Trial: Ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 patients Following a pre-specified interim analysis, an independent DSMB recommended trial continuation without modifications, maintaining a sample size of 104 subjectsIn addition, the DSMB recommended submitting the data for further regulatory review for drug approvalPrimary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessedTrial completion expected by Q4 2025 (including a three-month follow-up period) DRAGON II Trial: Combination of a Phase 1b open-label trial to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in adolescent Japanese STGD1 patients and a Phase 2/3, 24-month, randomized (1:1, active: placebo), double-masked, placebo-controlled, multicenter trial in adolescent STGD1 patients Enrolled 16 subjects in the Phase 2/3 trial, with a target enrollment of approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects; data from the Japanese subjects is intended to facilitate a future new drug application in JapanPrimary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed Geographic Atrophy (GA): GA is a chronic degenerative disease of the retina that leads to blindness in the elderly. Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the progression of GA. There are currently no FDA-approved, orally administered treatments for GA. PHOENIX Trial: Ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in GA patients 464 of the targeted 500 subjects have been enrolled to datePrimary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessedCompany expects to conduct an interim analysis Corporate Highlights In February 2025, Belite completed a registered direct offering priced at the market, raising gross proceeds of $15 million, with the potential for additional proceeds of approximately $15 million from the exercise of five-year warrants issued in the offering. First Quarter 2025 Financial Results: Current Assets: As of March 31, 2025, the Company had $157.4 million in cash, liquidity funds, time deposits, and U.S treasury bills. R&D Expenses: For the three months ended March 31, 2025, research and development expenses were $9.4 million compared to $6.8 million for the same period in 2024. The increase in research and development expenses was primarily attributable to (i) share-based compensation granted in the third quarter of 2024 and first quarter of 2025, (ii) slightly higher clinical trial expenses related to the PHOENIX trial. G&A Expenses: For the three months ended March 31, 2025, general and administrative expenses were $6.1 million compared to $1.6 million for the same period in 2024. The increase resulted primarily from an increase in share-based compensation granted in the third quarter of 2024 and first quarter of 2025. Other Income: For the three months ended March 31, 2025, other income was $1.2 million compared to $0.5 million for the same period in 2024. The increase in other income was attributable to accrued interest from time deposits and U.S. treasury bills. Net Loss: For the three months ended March 31, 2025, the Company reported a net loss of $14.3 million, compared to a net loss of $7.9 million for the same period in 2024. Webcast Information Belite Bio will host a webcast on Wednesday, May 14, 2025, at 4:30 p.m. Eastern Time to discuss the Company’s financial results and provide a business update. To join the webcast, please visit https://events.q4inc.com/attendee/137642555. A replay will be available for approximately 90 days following the event at the Company’s Investor Relations website at https://investors.belitebio.com/presentations-events/events. About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical need, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 patients and a Phase 3 study (PHOENIX) in patients with GA. For more information, follow us on X, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. Important Cautions Regarding Forward Looking Statements This press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, and Belite Bio’s advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, and any other statements containing the words “expect”, “hope” and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio’s ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the “Risk Factors” section in Belite Bio’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. BELITE BIO, INC UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Amounts in thousands of US Dollars, except share and per share amounts) For the Three MonthsEnded March 31, 2024 2025 Expenses Research and development 6,765 9,396 General and administrative 1,563 6,121 Total operating expenses 8,328 15,517 Loss from operations (8,328) (15,517)Other income: Total other income, net 463 1,240 Loss before income tax (7,865) (14,277)Income tax expense 6 - Net loss (7,871) (14,277)Other comprehensive income (loss) Foreign currency translation adjustments, net of nil tax (96) 18 Total comprehensive loss (7,967) (14,259)Weighted average number of ordinary shares used in per share calculation: - Basic and Diluted 29,677,173 32,084,106 Net loss per ordinary share - Basic and Diluted $(0.27) $(0.45) BELITE BIO, INC UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (Amounts in thousands of US Dollars, except share amounts) December 31, March 31, 2024 2025 Current assets $147,073 $159,287 Other assets 5,059 4,914 TOTAL ASSETS $152,132 $164,201 TOTAL LIABILITIES $6,311 $6,131 TOTAL SHAREHOLDERS’ EQUITY 145,821 158,070 TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY $152,132 $164,201 Ordinary shares authorized 400,000,000 400,000,000 Ordinary shares issued 31,857,802 32,595,001 Ordinary shares outstanding 31,826,549 32,544,784 Media and Investor Relations Contact: Jennifer Wu ir@belitebio.com Julie Fallon belite@argotpartners.com