RBP4

SAN DIEGO, Oct. 14, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite” 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 the Company will present data from its completed Phase II adolescent Stargardt disease trial at the American Academy of Ophthalmology Annual Meeting (AAO 2024) being held October 18-21, 2024, in Chicago, IL. Podium Presentation: Title: Tinlarebant (LBS-008) for Adolescents with Stargardt DiseasePresentation Type: Late breaking developments (live broadcast)Presenting Author: Quan Dong Nguyen, MD, MScSession: RET16Date/Time: Saturday, October 19, 2024; 10:33 AM – 10:40 AM CDTLocation: Arie Crown Theater (Lakeside Center building, 1F), Chicago, IL E-poster Presentation Title: Investigation of an Oral Retinol Binding Protein 4 Antagonist in the Treatment of Childhood-onset Stargardt DiseasePresenting Author: Nathan L Mata, PHDDate/Time: On-demand About Tinlarebant (a/k/a LBS-008) Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in Stargardt Disease type 1 (STGD1) and also contribute to disease progression in geographic atrophy, or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S. Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. Stargardt Disease (STGD1) STGD1 is the most common inherited macular dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1. Importantly, the appearance of bisretinoids, followed by retinal cell death, and progressive loss of vision is also observed in geographic atrophy (GA) patients. Therefore, Belite Bio is evaluating safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). 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 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. Safety and efficacy of Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of vitamin A-based toxins in the eye, is currently being evaluated in three late-stage clinical trials. In STGD1, a 2-year, Phase 3 study (DRAGON) and a 2-year, Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects are currently ongoing. In GA, a 2-year, Phase 3 study (PHOENIX) is ongoing. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. 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. Media and Investor Relations Contact: Jennifer Wu / ir@belitebio.com Julie Fallon / belite@argotpartners.com

Phase 2/3 of the DRAGON II trial will evaluate the efficacy, safety, and tolerability of Tinlarebant in approximately 60 adolescent STGD1 subjects across the U.S., U.K., and Japan Phase 1b of DRAGON II trial to evaluate pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese subjects was recently completed Tinlarebant granted Orphan Drug and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1 Pivotal global Phase 3 trial of Tinlarebant in GA subjects (“PHOENIX”) is ongoing and more than 200 subjects have been enrolled Sept. 10, 2024 -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite” 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 the first patient has been dosed at the Tokyo Medical Center in the Phase 2/3 portion of its DRAGON II clinical trial evaluating Tinlarebant for the treatment of STGD1. Tinlarebant is a novel oral therapy designed to reduce the accumulation of vitamin A based toxins that cause retinal disease, and this Phase 2/3 study will evaluate the efficacy, safety, and tolerability of Tinlarebant in approximately 60 adolescent STGD1 subjects across the U.S., U.K., and Japan. “We are pleased to have successfully dosed the first subject in the Phase 2/3 portion of our DRAGON II trial. This milestone is a significant step forward in our mission to address the unmet needs of people living with Stargardt Disease,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “We recently completed a Phase 1b study of Tinlarebant in STGD1 patients at the Tokyo Medical Center and are happy to be continuing to partner with them for this next trial. With enrollment now also underway at sites in the U.S. and U.K., we are making meaningful progress toward our goal of delivering a life-changing oral therapy to those affected by this condition.” “DRAGON II is the first global Stargardt disease trial in Japan. As such, it provides the unprecedented opportunity for Japanese patients and their families to gain access to a potential therapy for this historically untreatable disease,” added Professor Kaoru Fujinami, Principal Investigator at National Hospital Organization, Tokyo Medical Center. “The level of interest we have received in this trial speaks to the urgency of the unmet need for patients living with STGD1. Following Tinlarebant’s receipt of Sakigake designation from Japan’s Ministry of Health, we feel well positioned to rapidly advance this trial and are working diligently to get this much needed therapy to patients.” The DRAGON II trial is a combination of Phase 1b open-label study in Japan to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of Tinlarebant, administered daily for 7 days in Japanese adolescent STGD1 subjects and a Phase 2/3, multicenter (U.S., U.K., and Japan), double-masked, placebo-controlled, randomized study designed to evaluate the efficacy, safety, and tolerability of Tinlarebant, administered daily for 24 months in adolescent STGD1 subjects. Approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects, are targeted for enrollment in the Phase 2/3 portion of the trial with a 1:1 randomization (Tinlarebant:placebo). The data from the Japanese subjects is intended to facilitate future NDA applications in Japan. Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in GA, or advanced Dry AMD. Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S. Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1. Importantly, STGD1 and GA, or advanced Dry AMD, share a similar pathophysiology, which is characterized by the excessive accumulation of bisretinoids, retinal cell death, and progressive loss of vision. Vision loss occurs slowly, despite peripheral expansion of “dead retina,” until the disease reaches the center of the eye (the macula). Therefore, Belite Bio is evaluating safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). Belite 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 Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. The content above comes from the network. if any infringement, please contact us to modify.

Tinlarebant, a novel oral therapy, is intended to slow disease progression in patients affected with Stargardt Disease (STGD1) and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD)Phase 1b & 2/3 (“DRAGON II”) trial of Tinlarebant in adolescent STGD1 patients has been initiated and have completed enrollment for Phase 1b with six subjects in JapanTinlarebant granted Orphan Drug and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1Pivotal global Phase 3 trial of Tinlarebant in GA subjects (“PHOENIX”) is ongoing and approximately 200 subjects have been enrolledData from a 24-month Phase 2 trial in adolescents with STGD1 showed a sustained, lower atrophic lesion growth in Tinlarebant-treated subjects compared to ProgStar participants possessing similar baseline characteristics (aged ≤18 years) (p<0.001)In the Phase 2 trial, five of 12 subjects (42%) with known pathogenic ABCA4 mutations, no incident atrophic (DDAF) lesions were formed during the 24-month treatment period and no change in autofluorescent (QDAF) lesions was observedCompany continues to expect interim analysis from the pivotal global Phase 3 trial of Tinlarebant in adolescent STGD1 subjects (“DRAGON”) in 4Q 2024Company to host conference call and webcast on Monday, August 12, 2024, at 4:30 p.m. EDT SAN DIEGO, Aug. 09, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite” 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 its financial results for the second quarter ended June 30, 2024, and provided a general business update. “We continued to make meaningful strides in advancing Tinlarebant this quarter. We initiated our DRAGON II trial in adolescent STGD1 patients and have completed the enrollment for its phase 1b portion, and notably, we received Sakigake designation in Japan, a testament to the groundbreaking potential of this drug and the unmet need it stands to address for people living with STGD1. In GA, we are approaching 200 patients enrolled in our pivotal Phase 3 trial,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “In the quarter, we also bolstered our balance sheet, having raised $25 million in gross proceeds in a registered direct offering in April. As we enter the second half of the year, we are well positioned to execute on key milestones and look forward to sharing interim analysis from our pivotal Phase 3 DRAGON trial in the fourth quarter.” Second Quarter 2024 Business Highlights and Upcoming Milestones: Clinical Highlights Tinlarebant (LBS-008) is an oral, potent, once daily 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 to normal vision but can accumulate as toxic byproducts in individuals affected with STGD1 and GA (the advanced form of Dry AMD) leading to retinal cell death and loss of vision. Stargardt disease (STGD1): Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the onset and progression of STGD1. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease Designation 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 subjects Completed enrollment with 104 subjects in 11 countriesPrimary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessedInterim analysis expected in 4Q 2024 DRAGON II Trial: Combination of Phase 1b open-label trial to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese adolescent STGD1 subjects and a Phase 2/3, double-masked, placebo-controlled, multicenter, trial in adolescent STGD1 subjects Completed Phase 1b enrollment with six subjects in JapanCompany targeting approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects, for enrollment in the Phase 2/3 portion of the trial with a 1:1 randomization, with data from Japanese subjects intended to facilitate future new drug application in JapanPrimary efficacy endpoint is slowing of atrophic lesion growth rate; 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 toxic 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; n~430 subjects), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in patients with GA Primary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessedApproximately 200 subjects have been enrolled as of August 9, 2024Company expects to conduct interim analysis at the mid-point of the trial Corporate Highlights Raised $25 million in gross proceeds in a registered direct offering in April 2024 Second Quarter 2024 Financial Results: Current Assets: As of June 30, 2024, the Company had $112.3 million in cash, time deposits and U.S treasury bills. R&D Expenses: For the three months ended June 30, 2024, research and development expenses were $9.1 million compared to $5.5 million for the same period in 2023. For the six months ended June 30, 2024, research and development expenses were $15.8 million compared to $11.2 million for the same period in 2023. The increase in research and development expenses in both the quarter and year-to-date was primarily attributable to (i) a development milestone payment for completion of a phase 2 trial, and (ii) share-based compensation expense. G&A Expenses: For the three months ended June 30, 2024, general and administrative expenses were $1.4 million compared to $1.4 million for the same period in 2023. For the six months ended June 30, 2024, general and administration expenses were $3.0 million compared to $2.5 million for the same period in 2023. The increase year-to-date is primarily from an increase in share-based compensation expense. Other Income: For the three months ended June 30, 2024, other income was $1.0 million compared to $0.1 million for the same period in 2023. For the six months ended June 30, 2024, other income was $1.4 million compared to $0.1 million for the same period in 2023. The increase in both the quarter and year-to-date is attributed to accrued interest from time deposits and U.S. treasury bills. Net Loss: For the three months ended June 30, 2024, the Company reported a net loss of $9.5 million, compared to a net loss of $6.8 million for the same period in 2023. For the six months ended June 30, 2024, the Company reported a net loss of $17.4 million, compared to a net loss of $13.7 million for the same period in 2023. Webcast Information Belite Bio will host a webcast on Monday, August 12, 2024, 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://wsw.com/webcast/cc/blte4/1422018. A replay will be available for approximately 90 days following the event. About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases that have significant unmet medical needs such as (i) atrophic age-related macular degeneration (AMD), commonly known as Geographic Atrophy (GA) in advanced dry AMD, and (ii) autosomal recessive Stargardt disease type 1, or STGD1, in addition to specific metabolic diseases. For more information, follow us on Twitter, 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, INCUNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(Amounts in thousands of US Dollars, except share and per share amounts) For the Three Months For the Six Months Ended June 30, Ended June 30, 2023 2024 2023 2024 Expenses Research and development 5,516 9,078 11,239 15,843 General and administrative 1,355 1,393 2,513 2,956 Total operating expenses 6,871 10,471 13,752 18,799 Loss from operations (6,871) (10,471) (13,752) (18,799)Other income: Total other income, net 62 977 54 1,440 Loss before income tax (6,809) (9,494) (13,698) (17,359)Income tax expense 3 - 9 6 Net loss (6,812) (9,494) (13,707) (17,365)Other comprehensive income (loss) Foreign currency translation adjustments, net of nil tax (76) (10) (60) (106)Total comprehensive loss (6,888) (9,504) (13,767) (17,471)Weighted average number of ordinary shares used in per share calculation: - Basic and Diluted 25,785,147 30,324,132 25,350,949 30,000,653 Net loss per ordinary share - Basic and Diluted $(0.26) $(0.31) $(0.54) $(0.58) BELITE BIO, INCUNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS(Amounts in thousands of US Dollars, except share amounts) December 31, June 30, 2023 2024 Current assets $89,940 $113,858 Other assets 4,702 4,572 TOTAL ASSETS $94,642 $118,430 TOTAL LIABILITIES $4,211 $3,837 TOTAL SHAREHOLDERS’ EQUITY 90,431 114,593 TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY $94,642 $118,430 Ordinary shares authorized 400,000,000 400,000,000 Ordinary shares issued 29,184,475 30,649,321 Ordinary shares outstanding 29,149,444 30,612,359 Media and Investor Relations Contact: Jennifer Wu /ir@belitebio.com Julie Fallon /belite@argotpartners.com