Tinlarebant

Tinlarebant is Belite Bio’s orally administered tablet 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) Data from a 24-month Phase 2 trial in adolescent STGD1 subjects 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, 5 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 QDAF was observed A novel lesion size quantification method that can better detect atrophic lesions revealed 8 of 12 subjects with atrophic lesion involvement within the macula at baseline; growth of these lesions into the macula was halted during the second year Interim data from the pivotal global Phase 3 trial of Tinlarebant in adolescent STGD1 subjects (“DRAGON”) expected in 4Q 2024 Initiated DRAGON II trial of Tinlarebant in adolescent STGD1 patients Pivotal global Phase 3 trial of Tinlarebant in GA subjects (“PHOENIX”) is ongoing Raised $25 million in gross proceeds in a registered direct offering in April 2024 Conference Call and Webcast Tuesday, May 14, 2024, at 4:30 p.m. ET SAN DIEGO, May 13, 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 first quarter ended March 31, 2024 and provided a general business update. “It has been a strong start to 2024 with the initiation of the DRAGON II trial and the recent closing of our $25 million registered direct offering in April 2024,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “At the annual meeting of The Association for Research in Vision and Ophthalmology (ARVO), we announced additional analysis from our completed Phase 2 trial of Tinlarebant in adolescent STGD1 supporting its potential to slow or even halt disease progression. We are encouraged by these findings and look forward to building on this momentum as we continue to evaluate the potentially life-changing impact of this therapy in our ongoing STGD1 trials, DRAGON & DRAGON II, as well as in GA through our PHOENIX trial.” First Quarter 2024 Business Highlights and Upcoming Milestones: Clinical Highlights Tinlarebant (LBS-008) is designed to be 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 and Rare Pediatric Disease (RPD) designations by the U.S. Food and Drug Administration (FDA), and orphan drug designation (ODD) in the U.S., Europe, and Japan for STGD1. There are currently no FDA approved treatments for STGD1. LBS-008-CT02 trial: Completed, open label, 24-month Phase 2 trial in adolescent STGD1 subjects and presented end of trial data at the AAO annual meeting in November 2023 and additional analysis at ARVO 2024 Tinlarebant was safe and well tolerated with no withdrawals due to adverse eventsThe 24-month data 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 5 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 QDAF was observedA novel lesion size quantification method that can better detect atrophic lesions revealed 8 of 12 subjects with atrophic lesion involvement within the macula at baseline; growth of these lesions into the macula was halted during the second yearVisual acuity was stabilized in the majority of subjects during the trial with a mean loss of five letters following 24 months of treatment (a loss of <10 letters is not considered clinically significant)In 6 subjects with prior bilateral BCVA loss (mean ~10 letters/year), mean BCVA loss over 24 months was 1.9 letters/yearSibling subjects with identical ABCA4 mutations showed different rates of QDAF/DDAF lesion growth and BCVA lossAdditional analysis of visual acuity loss, genotypic profiles, and retinal imaging data provides further support for the potential of Tinlarebant to slow and potentially halt lesion growth 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 data 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 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. The data from Japanese subjects is intended to facilitate future NDA applications in JapanInitiated in Japan, the U.S., and the U.K.Primary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessed Geographic Atrophy (GA): GA, the advanced form of dry AMD, 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 assessedHas enrolled 99 subjectsInterim data expected at mid-point of the trial Corporate Highlights For the three months ended March 31, 2024, raised $12.5 million from the exercise of the warrants issued in the Follow-on Offering and ATM Offering. On April 25, 2024 announced $25 million Registered Direct Offering, with the purchase and sale of up to an aggregate of 651,380 American Depositary Shares of the Company (“ADSs”), each ADS representing one ordinary share of the Company, at a purchase price of $38.38 per ADS, and the warrants to purchase up to an aggregate of 651,380 ADSs, which will have an initial exercise price equal to $44.14 per ADS and will be immediately exercisable and expire on the five-year anniversary of the issuance. First Quarter 2024 Financial Results: Current Assets: As of March 31, 2024, the Company had $95.5 million in cash and U.S treasury bills. R&D Expenses: For the three months ended March 31, 2024, research and development expenses were $6.8 million compared to $5.7 million for the same period in 2023. The increase in research and development expenses was primarily attributable to (i) conducting the DRAGON trial, (ii) initiating the DRAGON II trial, and (iii) increased wages and salaries due to our R&D team expansion and share-based compensation granted in the third quarter of 2023. G&A Expenses: For the three months ended March 31, 2024, general and administrative expenses were $1.6 million compared to $1.2 million for the same period in 2023. The increase resulted primarily from an increase in share-based compensation granted in the third quarter of 2023. Net Loss: For the three months ended March 31, 2024, the Company reported a net loss of $7.9 million, compared to a net loss of $6.9 million for the same period in 2023. Webcast Information Belite Bio will host a webcast on Tuesday, May14, 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/blte3/1420956. 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 which 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 Ended March 31, 2023 2024 Expenses Research and development 5,723 6,765 General and administrative 1,158 1,563 Total operating expenses 6,881 8,328 Loss from operations (6,881) (8,328)Other income (expense): Total other (expense) income, net (8) 463 Loss before income tax (6,889) (7,865)Income tax expense 6 6 Net loss (6,895) (7,871)Other comprehensive income (loss) Foreign currency translation adjustments, net of nil tax 16 (96)Total comprehensive loss $(6,879) (7,967)Weighted average number of ordinary shares used in per share calculation: - Basic and Diluted 20,950,240 29,677,173 Net loss per ordinary share - Basic and Diluted $(0.33) (0.27) BELITE BIO, INCUNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS(Amounts in thousands of US Dollars, except share amounts) December 31, March 31, 2023 2024 Current assets $89,940 $96,468 Other assets 4,702 4,679 TOTAL ASSETS $94,642 $101,147 TOTAL LIABILITIES $4,211 $4,677 TOTAL SHAREHOLDERS’ EQUITY 90,431 96,470 TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY $94,642 $101,147 Ordinary shares authorized 400,000,000 400,000,000 Ordinary shares issued 29,184,475 29,802,332 Ordinary shares outstanding 29,149,444 29,794,433 Media and Investor Relations Contact:Jennifer Wu /ir@belitebio.comJulie Fallon /belite@argotpartners.com

Additional analysis of visual acuity loss, genotypic profiles, and retinal imaging data from a 24-month Phase 2 trial of Tinlarebant in adolescent Stargardt disease patients provides further support for the potential of Tinlarebant to slow and potentially halt lesion growthIn 5 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 QDAF was observedA novel lesion size quantification method that can better detect atrophic lesions revealed 8 of 12 subjects with atrophic lesion involvement within the macula at baseline; growth of these lesions into the macula was halted during the second year In 6 subjects with prior bilateral BCVA loss (mean ~10 letters/year), mean BCVA loss over 24 months was 1.9 letters/year Sibling subjects with identical ABCA4 mutations showed different rates of QDAF/DDAF lesion growth and BCVA loss Tinlarebant is currently being evaluated in adolescent Stargardt disease in a Phase 3 DRAGON study, with interim data expected in 4Q 2024, and a combination Phase 1b and Phase 2/3 DRAGON II trial, and in Geographic Atrophy in a Phase 3 PHOENIX study SAN DIEGO, May 06, 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 additional findings from the 24-month Phase 2 study of Tinlarebant in adolescent Stargardt disease (STGD1) at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with STGD1 and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD). Genetic profiling was performed on the 13 adolescent STGD1 patients enrolled in a Phase 2 study of Tinlarebant. Eleven of these subjects (85%) harbored severe pathogenic/likely pathogenic ABCA4 variants. Despite these severe variants, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop incident atrophic (definitely decreased autofluorescence, DDAF) retinal lesions and no change in questionably decreased autofluorescence (QDAF) was observed during the 24-month treatment period. Incident atrophic lesions appeared in seven subjects at different timepoints over 24 months; four of these subjects developed DDAF lesions after month 12. The mean DDAF lesion growth at month 24 was 0.51 mm2 with a range of variation of 0.4 mm2, a growth rate that is significantly lower than what has been observed in natural history studies. Importantly, six STGD1 patients who had a mean bilateral best corrected visual acuity (BCVA) loss of 10 letters per year prior to enrollment in the Phase 2 study showed a mean BCVA loss equivalent to 1.9 letters per year during the 24-month treatment period. Analysis of genotype-phenotype relationships revealed that sibling subjects with identical ABCA4 mutations had different rates of lesion growth and BCVA loss indicating that identical genotypes do not necessarily predict an identical course of disease. Notably, retinal imaging data from the Phase 2 study was reanalyzed using a novel lesion size quantification method that utilizes a mathematical classification of lesions to reduce subjective reader bias and provide enhanced accuracy and superior precision compared to the traditional method of DDAF lesion quantification. This analysis revealed DDAF lesions within the macula in 12 eyes of eight subjects at baseline. Analysis of change in atrophic lesion area within the macula of these eyes over 24 months showed a halt in lesion growth into the macula after 16 months. This finding is consistent with the observed stabilization of visual acuity. “Data from the genotype and lesion growth analyses from our completed Phase 2 trial continues to demonstrate the efficacy of Tinlarebant,” said Dr. Nathan Mata, CSO of Belite Bio. “The stabilization of visual acuity, particularly in patients with significant visual acuity loss prior to study enrollment, as well as the observation that 42% of the Tinlarebant-treated subjects did not develop atrophic retinal lesions during the course of treatment despite the presence of severe pathogenic ABCA4 variants, suggests that Tinlarebant holds great promise toward slowing or even preventing the development of atrophic lesions to alter the course of disease and provide a meaningful benefit for patients.” “Traditional measurement of DDAF relies heavily on subjective assessment of the grader without a specific focus on key regions, potentially leading to less precise measurements,” added Professor John Grigg, Principal Investigator, Head Specialty of Ophthalmology at the University of Sydney and Consultant Ophthalmologist at the Sydney Children’s Hospitals Network at Westmead and Sydney Eye Hospital who presented the data. “The recent analysis of retinal images using a novel lesion size quantification method that focuses solely on the macular region or even smaller areas with greater precision and accuracy, revealed that 8 subjects did have atrophic lesions within the macula at the time of enrollment. Moreover, analysis of change in atrophic lesion area within the macula in these subjects over 24 months reveals that the lesion growth has stopped after month 16. These findings suggest that the baseline character of the subjects in the Phase 2 trial is similar to the those in the ongoing Phase 3 trial and similar outcome may be expected in the ongoing Phase 3 trials, and Tinlarebant may not only prevent healthy retinal cells from A2E damage, but also potentially even halt disease progression.” These new findings continue to support Tinlarebant as a promising oral treatment for STGD1 patients. The presentation is now available in the “Presentations & Events” section of Belite Bio’s website: https://investors.belitebio.com/presentations-events/events. About Tinlarebant (a/k/a LBS-008re) 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., and Orphan Drug Designation in the U.S. Europe, and Japan for the treatment of STGD1. Stargardt Disease (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). About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which 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 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, 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.comJulie Fallon /belite@argotpartners.com