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Belite Bio 2026 Commercial Day September 2nd, 2026 8am-9:30am ET
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B E L I T E B I O / 2 Forward-Looking Statements and Legal Disclaimer ATTENTION: The following disclaimer applies to the attached Presentation and you must read this disclaimer page carefully before reading, accessing or making any other use of the attached Presentation. In accessing the attached Presentation, you agree to be bound by the following terms and conditions. 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 solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities of Belite Bio Inc (“Belite Bio” or the “Company”) from any investor or in any jurisdiction in which such an offer or solicitation is not authorized or would be unlawful. No shares or other securities of Belite Bio are being offered to the public by means of this Presentation. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This Presentation is being given on the condition that it is for use by the recipient for information purposes and to evaluate Belite Bio only. Any failure to comply with these restrictions may constitute a violation of applicable securities laws. Any statements in this Presentation about Belite Bio’s future expectations, plans and prospects, as well as other statements regarding matters that are not historical facts constitute forward-looking statements within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking 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, the ability or potential of tinlarebant to treat STGD1 and GA, the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the timing to obtain regulatory approval for tinlarebant, the potential benefits of any regulatory designations, commercialization preparation activities and hiring progress, as well as any other statements regarding matters that are not historical facts, and any other statements containing the words “expect”, “believe”, “anticipate”, “intend”, “target”, “plan”, “hope”, “potential”, “estimate”, “project”, “may”, “will”, “could”, “should”, “would”, “on track”, and other similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors related to Belite Bio’s business, 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; expectations for the timing of initiation, enrollment and completion of, and data relating to, its clinical trials; the possibility that the FDA or other regulatory authorities may require additional clinical trials; the timing to complete any ancillary clinical trials and/or to receive the interim/final data of such clinical trials; the timing to communicate with and submit trial data to regulatory authorities for drug approval in various jurisdictions; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; Belite Bio’s limited experience in launching and marketing product candidates; Belite Bio’s ability to attract, retain and motivate senior management and qualified employees; timing for Belite Bio to share additional data at upcoming medical meetings, 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. Market data and industry information used throughout this Presentation are based on the knowledge of the industry and the good faith estimates of Belite Bio’s management. The Company also relied, to the extent available, upon management’s review of independent industry surveys and publications and other publicly available information prepared by a number of third-party sources. All of the market data and industry information used in this Presentation involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. 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B E L I T E B I O / 3 Belite Commercial Day Agenda Section Length Speaker(s) Opening 5 mins • Tom Lin (Chief Executive Officer) Living with Stargardt Disease 15 mins • Tracey S. • Havah F. Disease and Clinical Overview 15 mins • Dr. Michel Michaelides U.S. Key Opinion Leader 10 mins • Dr. Paul Bernstein Tackling an Important Cause of Blindness 5 mins • Dr. Hendrik Scholl (Chief Medical Officer) U.S. Commercial 15 mins • Kelly Kilpatrick (Chief Commercial Officer) Live Q&A 25 mins
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Opening Tom Lin Chairman, CEO
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Mission: To preserve vision and pioneer degenerative retinal disease treatments where few or none exist today Vision: To shine light on the shadows of vision loss and degenerative retinal disease by changing how it is recognized and treated
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B E L I T E B I O / 6 FDA PRIORITY REVIEW PDUFA: Feb. 12, 2027 POTENTIAL FIRST & ONLY TREATMENT Stargardt disease type 1 U.S. LAUNCH READINESS Building capabilities to support patients if approved COMMERCIAL-STAGE TRANSFORMATION Positioned for transition to a commercial-stage biotech Key Highlights
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B E L I T E B I O / 7 Joining Today T om Lin, MMED, PhD, MBA Chairman, CEO Hao-Yuan Chuang, CFA, MBA, FRM CFO Kelly Kilpatrick CCO Hendrik Scholl, MD, MA CMO
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B E L I T E B I O / 8 Guest Speakers • Val A. and Edith D. Green Presidential Endowed Chair in Ophthalmology and Visual Sciences • Vice-Chair for Clinical Research • Chief of the Retina Division, Moran Eye Center • University of Utah School of Medicine Paul S. Bernstein, MD, PhD • Consultant Ophthalmologist at Moorfields Eye Hospital in the Departments of Medical Retina, Inherited Eye Disease and Pediatric Ophthalmology • Professor of Ophthalmology at the UCL Institute of Ophthalmology Michel Michaelides, BSc MB BS MD(Res) FRCOphth FACS
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Living with Stargardt Disease Havah F.Tracey S.
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B E L I T E B I O / 10 Q1: What signs and symptoms led to your diagnosis of Stargardt disease? Living with Stargardt Disease
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B E L I T E B I O / 11 Q2: What were you told to expect when you were diagnosed with Stargardt disease? Living with Stargardt Disease
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B E L I T E B I O / 12 Q3: How does living with Stargardt disease impact your day-to-day life? Living with Stargardt Disease
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B E L I T E B I O / 13 Q4: What is most challenging for you about living with Stargardt disease? Living with Stargardt Disease
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B E L I T E B I O / 14 Q5: What are your hopes for the future for people living with Stargardt disease? Living with Stargardt Disease
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Disease and Clinical Overview Michel Michaelides BSc MB BS MD(Res) FRCOphth FACS
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B E L I T E B I O / 16 Stargardt Disease • Most common juvenile macular dystrophy • Described by Karl Stargardt in 1909 • Ocular exam signs: areas of retinal atrophy centrally, and subretinal yellowish flecks more peripherally • Manifestation age range: 1 → 84+ years Stargardt K (1909) Über familiäre, progressive Degeneration in der Maculagegend des Auges (On familial, progressive degeneration in the macular region of the eye). Zeitschrift für Augenheilkunde. 22: 289–306. Lambertus S et al. (2015) Early-onset Stargardt disease: phenotypic and genotypic characteristics. Ophthalmology. 122: 335-44. Lambertus S et al. (2016) Progression of Late-Onset Stargardt Disease. Invest Ophthalmol Vis Sci. 57: 5186-5191.
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B E L I T E B I O / 17 Stargardt Disease Overview Stargardt disease (STGD1) and/or ABCA4-associated retinal dystrophy is a rare and progressive eye disease leading to legal blindness in almost all cases. There is no approved treatment for Stargardt disease. • Autosomal recessive mutations in the ABCA4 gene, impair retinoid transport and lead to toxic bisretinoid buildup in the retinal pigment epithelium (RPE) • Bisretinoid accumulation manifests as yellowish flecks underneath the retina and causes oxidative stress, toxicity, and eventually RPE cell death • RPE damage leads to secondary degeneration of macular photoreceptors • Progressive macular atrophy and retinal degeneration Pathophysiological Changes • Gradual central vision loss in both eyes, often starting in childhood or early adulthood • Blurry or distorted central vision • Central blind spots (scotomas) • Photophobia and delayed dark adaptation • Impaired color vision • Difficulty with detailed tasks like reading or recognizing faces • Peripheral vision preserved in the majority of cases Key Symptoms • Challenges with reading, using screens, and recognizing faces, harming education and work • Typically, inability to drive, reducing mobility and independence • Restrictions in sports, leisure, and simple tasks like avoiding trips • Social struggles, including interactions, relationships, and events • Emotional effects like frustration, worry, anger, and depression • Career changes or job loss, affecting finances and productivity Impact on Activities of Daily Living
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B E L I T E B I O / 18 ProgStar Study: The Natural History of the Progression of Atrophy Secondary to Stargardt Disease University of Utah Salt Lake City, UT, U.S. Cole Eye Institute Cleveland, OH, U.S. Scheie Eye Institute Philadelphia, PA, U.S. Johns Hopkins University Baltimore, MD, U.S. Moorfields Eye Hospital London, U.K. Center for Ophthalmology Tuebingen, Germany Coordination Center DANA Wilmer Eye Institute Baltimore, MD, U.S. Retina Foundation of the Southwest Dallas, TX, U.S. Reading Center DIRC Doheny Eye Institute Los Angeles, CA, U.S. Greater Baltimore Medical Center (GBMC) Baltimore, MD, U.S. Institut de la Vision Paris, France
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B E L I T E B I O / 19 DDAF Represents Well-Demarcated Areas of Complete RPE Loss and Grows Predictably, Making It an Approvable Primary Endpoint ONH = optic nerve head. Strauss RW, Ho A, Jha A, Fujinami K, Michaelides M, Cideciyan AV, Audo I, Birch DG, Sadda S, Ip M, West S, Schönbach EM, Kong X, Scholl HPN; ProgStar Study Group (2023) The progression of Stargardt Disease as determined by fundus autofluorescence over a 24-month period (ProgStar Report No. 17). Am J Ophthalmol. 250: 157-170. Definitely Decreased Autofluorescence Darkness level of at least 90% relative to the ONHDDAF Questionably Decreased Autofluorescence Darkness level between 50% and 90% relative to the ONH QDAF Decreased Autofluorescence The combined sum of DDAF + QDAF DAF DAF DDAF+QDAF DDAF QDAF DAF DDAF+QDAF DDAF QDAF ONH
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B E L I T E B I O / 20 DDAF Progression Rate in Stargardt Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study Strauss RW, Ho A, Jha A, Fujinami K, Michaelides M, Cideciyan AV, Audo I, Birch DG, Sadda S, Ip M, West S, Schönbach EM, Kong X, Scholl HPN; ProgStar Study Group (2023) The progression of Stargardt Disease as determined by fundus autofluorescence over a 24-month period (ProgStar Report No. 17). Am J Ophthalmol. 250: 157-170. Overall DDAF growth rate in the ProgStar cohort over 24 months: 0.74 mm2/year (confidence interval: 0.64 - 0.85 mm2/year) A B C
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B E L I T E B I O / 21 ProgStar: Visual Acuity Change over 24 Months BCVA = best-corrected visual acuity; LogMAR = logarithm of the minimum angle of resolution; VI = visual impairment. Kong X, Fujinami K, Strauss RW, Munoz B, West SK, Cideciyan AV, Michaelides M, Sahel JA, Ahmed M, Ervin AM, Schönbach EM, Cheetham JK, Scholl HPN; ProgStar Study Group (2018) Visual Acuity Change over 24 Months and its Association With Foveal Phenotype and Genotype in Individuals With Stargardt Disease: ProgStar Report Number 10. JAMA Ophthalmol. 136: 920-928. Overall rate of BCVA loss was 0.55 letters/year over two years Prospective Cohort (N=434) BCVA of eyes with baseline BCVA between 20/70 and 20/200 declined at a rate of 0.6 letters/year
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B E L I T E B I O / 22 DDAF = Definitely Decreased Autofluorescence; DAF = Decreased Autofluorescence; BCVA = Best-Corrected Visual Acuity; SD-OCT = Spectral-Domain Optical Coherence Tomography Reduction in atrophic lesion growth rate as measured by fundus autofluorescence imaging is the FDA’s accepted primary endpoint in Stargardt disease and geographic atrophy secondary to age-related macular degeneration DRAGON Design Key Inclusion Criteria • Clinical diagnosis of Stargardt disease • 12-20 years old • ≥1 mutation identified in the ABCA4 gene • Atrophic lesion size (DDAF) within 3 disc areas (7.62 mm2) • BCVA of 20/200 or better Primary Measures • Slowing of atrophic lesion growth (DDAF) • Safety and tolerability Secondary Measures • DAF • BCVA • SD-OCT • Microperimetry N=104Randomized 2:1 Placebo n=35 Treatment n=69 Phase 3 Trial 2-Year Duration Double-blinded, global trial of oral tinlarebant 5mg/day DRAGON Clinical Trial Design in Stargardt Disease
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B E L I T E B I O / 23 70% Reduction Target Tinlarebant Treatment Led to 80% Reduction in RBP4, Well Above Goal of 70%* Daily dosing of 5mg/day tinlarebant led to a sustained 80% reduction of RBP4 and RBP4 levels returned to 84% of the baseline value at the End of Study (EOS) * In a prior study of a surrogate RBP4 antagonist (fenretinide) in patients with Geographic Atrophy, an RBP4 reduction of ≥70% was associated with a statistically significant slowing of lesion growth [Mata et al., Retina. 2013; 33(3): 498-507.] Biomarker: RBP4 – Percent Reduction from Baseline PD Set N=92 EOT = End of treatment.
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B E L I T E B I O / 24 Primary Endpoint: DDAF Change from Baseline (Study Eye) mFAS N=96 • Applying an unstructured covariance matrix, the treatment effect size was 35.7% compared to placebo and yielded a p-value of 0.0033 • With a first-order autoregressive covariance matrix, the treatment effect size remained consistent (35.4%) with P<0.0001 • DDAF lesion growth was slowed to 0.38 mm2/year vs. 0.59 mm2/year for placebo and 0.74 mm2/year observed in ProgStar Primary Endpoint Showed a Statistically Significant and Clinically Meaningful Outcome
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B E L I T E B I O / 25 Tinlarebant Slowed DAF Lesion Growth, the Key Secondary Endpoint, in the Study Eye by 33.7% Tinlarebant slowed DAF lesion growth by 33.7% compared to placebo (P=0.027) Key Secondary Endpoint: DAF Change from Baseline (Study Eye) mFAS N=96
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B E L I T E B I O / 26 As Expected, BCVA in Study Eye Did Not Show Any Significant Change ETDRS = Early Treatment Diabetic Retinopathy Study. (1) Parker MA, Choi D, Erker LR, Pennesi ME, Yang P, Chegarnov EN, Steinkamp PN, Schlechter CL, Dhaenens CM, Mohand-Said S, Audo I, Sahel J, Weleber RG, Wilson DJ. Test-Retest Variability of Functional and Structural Parameters in Patients with Stargardt Disease Participating in the SAR422459 Gene Therapy Trial. Transl Vis Sci Technol. 2016 Oct 1;5(5):10. • The overall change of visual acuity was minimal over the period of 24 months in both treatment groups • Test–retest variability for ETDRS change scores in Stargardt disease are known to yield a repeatability coefficient ≈ 8 letters (1) • Such minor changes in average visual acuity over two years are in line with the natural history of Stargardt disease and were observed in the ProgStar Study Tinlarebant 5mg (N=69) Placebo (N=35) BCVA at Baseline 39.9 39.4 BCVA at EOS 39.7 40.0
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B E L I T E B I O / 27 Subjects Who Experienced at Least One Non-Ocular Treatment-Emergent Adverse Event (TEAE), N / (%) • Total of 6 serious adverse events (SAEs) reported in the study – all events were non-ocular, with 4 assessed as unrelated and 2 assessed as unlikely related to the study treatment • Most reported Non-Ocular adverse events (AEs): Nasopharyngitis (all cases were assessed as unrelated/unlikely related to treatment), Headache, and Acne – most events were mild and resolved during the study period Category Tinlarebant 5mg (N=69) Placebo (N=35) TEAE 59 (85.5%) 27 (77.1%) Severe TEAE 2 (2.9%) 1 (2.9%) Serious TEAE 2 (2.9%) 4 (11.4%) Study Drug-Related TEAE 14 (20.3%) 4 (11.4%) Study Drug-Related Serious TEAE 0 (0.0%) 0 (0.0%) TEAE Leading to Study Drug Discontinuation 0 (0.0%) 0 (0.0%) TEAE Leading to Study Discontinuation 0 (0.0%) 0 (0.0%) TEAE Leading to Death 0 (0.0%) 0 (0.0%) Tinlarebant Demonstrated a Well Tolerated Safety Profile Safety Set N=104
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B E L I T E B I O / 28 Subjects Who Experienced at Least One Ocular TEAE, N / (%) • Most reported Ocular AEs: Xanthopsia, Delayed dark adaptation, and Night Vision Impairment • The majority of the events were mild, and most resolved while on study • There were no serious ocular TEAEs – 4 TEAEs led to study drug discontinuation and 2 TEAEs led to study discontinuation Category Tinlarebant 5mg (N=69) Placebo (N=35) TEAE 53 (76.8%) 8 (22.9%) Severe TEAE 2 (2.9%) 0 (0.0%) Serious TEAE 0 (0.0%) 0 (0.0%) Study Drug-Related TEAE 49 (71.0%) 8 (22.9%) Study Drug-Related Serious TEAE 0 (0.0%) 0 (0.0%) TEAE Leading to Study Drug Discontinuation 4 (5.8%) 0 (0.0%) TEAE Leading to Study Discontinuation 2 (2.9%) 0 (0.0%) TEAE Leading to Death 0 (0.0%) 0 (0.0%) The Majority of Ocular Adverse Events Was Mild Safety Set N=104
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B E L I T E B I O / 29 Association of QDAF, DDAF and DAF with Visual Acuity in Stargardt Disease • For QDAF lesions, every 1mm2 larger QDAF area was associated with 3.3 letters worse VA (p<0.001) • For DDAF lesions and lesion area <11mm2, every 1mm2 larger DDAF area was associated with 1.5 letters worse VA (p=0.02) • For DAF lesions (all eyes) and lesion area <11mm2, every 1mm2 larger DAF area was associated with 0.9 letters worse VA (p=0.003) Secondary cross-sectional data analysis using data from 301 ProgStar Study participants (566 study eyes) QDAF DDAF DAF Scholl HPN (2024) What's new in Retina and EVICR.net Developments in the Treatment of Retinal Dystrophies. EURETINA 2024, Barcelona – EVICR.net Retina Symposium. 22/09/2024
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B E L I T E B I O / 30 Severe Loss of Light Sensitivity within DDAF Microperimetry as an exploratory outcome measure in the DRAGON trial • Within DDAF lesions in the study eye, mean retinal sensitivity (at baseline) was 4.26 ± 5.59 dB for the tinlarebant group and 4.28 ± 7.07 dB in the placebo group • This corresponds to a marked reduction of approximately 2.2-2.6 log units (up to 400-fold) compared with an unaffected retina Clinical Study Report, Version 1.1 (15 Apr 2026), Table 14.2.3.4.1.1
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B E L I T E B I O / 31 Slow but Inevitable Loss of Visual Acuity in Stargardt Disease Kaplan-Meier curve for the time to reach a visual acuity of 20/200 or worse in Stargardt patients with visual acuity of 20/40 or better at initial visit Rotenstreich Y, Fishman GA, Anderson RJ (2003) Visual acuity loss and clinical observations in a large series of patients with Stargardt disease. Ophthalmology 110: 1151-8. Retrospective Mono-Center Study of N=361 STGD1 Patients at the Department of Ophthalmology and Visual Sciences, University of Illinois at Chicago Probability of maintaining visual acuity better than 20/200 1.0 0.8 0.6 0.4 0.2 0.0 0 5 10 15 20 25 3022 0.5 Length of follow-up (years)
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B E L I T E B I O / 32 Tinlarebant Has the Potential to Be the First-Ever Approved Treatment for Stargardt Disease 36% reduction in DDAF lesion growth rate, representing a statistically and clinically significant treatment effect in Stargardt disease Well tolerated safety and tolerability profile across two years of treatment Addresses the root pathogenic mechanism (bisretinoid accumulation), offering a rational, disease-modifying approach where no approved therapies currently exist Broad applicability across disease stages, from early ABCA4-mediated changes to more advanced atrophy First-ever oral therapy in a retinal degenerative disease to demonstrate a clinically meaningful slowdown of neurodegenerationTinlarebant Efficacy Safety Mechanism of Action Applicability
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U.S. Key Opinion Leader (KOL) Paul S. Bernstein MD, PhD
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B E L I T E B I O / 34 Undiagnosed STGD1 patient The Current Stargardt Disease Patient Journey Involves Multiple Specialists Source: National Eye Institute; American Academy of Ophthalmology; Review of Ophthalmology; FDA; L.E.K. interviews HCPs INVOLVED Retina specialist Inherited retinal disease specialist Genetic counselor Pediatrician Optometrists General ophthalmologist DiagnosisPrevalence ManagementPresentation Clinical Examination Clinical Diagnosis / Genetic Confirmation Disease ManagementSymptom / Screening Early symptoms Poor vision and difficulties reading Vision screening Visual problems detected at routine screening Conclusive STGD1 patients Suspicion of Stargardt disease (STGD1) Confirmatory Genetic test Initial clinical diagnosis
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B E L I T E B I O / 35 Retina Specialists’ Diagnosis of STGD1 is Aided by Genetic Testing Commonly Used Diagnostic Tests Retinal Imaging Modalities e.g., FAF, SD-OCT and Fundus Photography Visual Function Tests e.g., BCVA, Microperimetry, Color Vision Testing Genetic Testing e.g., gene panel or genomic sequencing Retina specialists are very familiar with Stargardt disease and comfortable with genetic testing, but general ophthalmologists – the referral entry point for most patients – have more limited knowledge of both Stargardt disease and genetic testing. IRD or retina specialists conduct regular follow-up, using functional tests and retinal imaging to monitor progression and document visual decline Follow-up is specialist-led: Most patients see a specialist yearly post- diagnosis, though many gradually extend the interval between visits Visit frequency tapers over time: Because of the presence of disease phenocopies, genetic testing is a critical component to confirm the STGD1 diagnosis Genetic testing is critical: IRD: Inherited Retinal Disease
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B E L I T E B I O / 36 Progressive Retinal Degeneration Leads to Visual Decline in Stargardt Disease Cremers FPM, Lee W, Collin RWJ & Allikmets R (2020) Clinical spectrum, genetic complexity and therapeutic approaches for retinal disease caused by ABCA4 mutations. Prog Retin Eye Res. 79: 100861. Funduscopy / Fundus Photography Fundus Auto-fluorescence (FAF) Optical Coherence Tomography (OCT)
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B E L I T E B I O / 37 Critical Gap in Care: No Current Standard of Treatment for Stargardt Disease Care today can help patients adapt to vision loss – but it does not impact the disease itself, leaving patients and caregivers to navigate a profound burden largely alone. Stargardt Management Today – No Approved Disease-Modifying Therapy Current Patients and Caregivers Unmet Need Prescribe low-vision aids (e.g., magnifiers) Counseling on lifestyle and career choices Recommend avoidance of Vitamin A supplementation Recommend UV-blocking glasses/sunglasses Difficulty reading Loss of independence – most profound impact Ongoing social challenges
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Hendrik Scholl Chief Medical Officer Tackling an Important Cause of Blindness
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B E L I T E B I O / 39 Ranking of Serious Health Conditions Scott AW, Bressler NM, Ffolkes S, Wittenborn JS & Jorkasky J (2016) Public Attitudes About Eye and Vision Health. JAMA Ophthalmol. 134: 1111-1118.
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B E L I T E B I O / 40 Conditions With the Greatest Effect on Day-to-Day Life Scott AW, Bressler NM, Ffolkes S, Wittenborn JS & Jorkasky J (2016) Public Attitudes About Eye and Vision Health. JAMA Ophthalmol. 134: 1111-1118.
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B E L I T E B I O / 41 DDAF Progression and Visual Loss in Stargardt Disease • 37-year-old male patient • Compound heterozygous for mutations in ABCA4 – Visual acuity (baseline): 20/40 – Visual acuity (at 4Y): 20/80 Atrophy Progression over 4 Years
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B E L I T E B I O / 42 Estimated Patient Population Sizes of STGD1 gnomAD = Genome Aggregation Database. Mata NL, Weng S, Michaelides M, Charbel Issa P, Quinodoz M, Rivolta C, Scholl HPN. Bisretinoids as a Source of Early Photoreceptor Pathology in Stargardt Disease. Ophthalmic Res. 2025;68(1):555-572. Europe Australia China Japan United States gnomAD Ethnicity European European East Asian East Asian Mixed Population (N) 742,000,000 26,640,000 1,411,000,000 124,500,000 334,900,000 STGD1 Patients (N) High Estimate 115,345 4,141 122,135 10,777 59,235 Low Estimate 95,517 3,429 94,940 8,377 46,767
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B E L I T E B I O / 43 ABCA4: One of the Largest Genetically Defined Rare Disease Opportunities Illustrative epidemiologic comparison; estimates represent published incidence/prevalence and are intended for order-of-magnitude benchmarking. References: Hanany M, Rivolta C, Sharon D (2020) Worldwide carrier frequency and genetic prevalence of autosomal recessive inherited retinal diseases. Proc Natl Acad Sci U S A. 117: 2710-2716. Al-Khuzaei S, Broadgate S, Foster CR, et al. Genes (Basel). 2021;12:1241. (ABCA4/Stargardt) Hu P, Dharmayat KI, Stevens CAT, et al. Circulation. 2020;141:1742-1759. (Familial hypercholesterolemia) Romitti PA, Zhu Y, Puzhankara S, et al. Prevalence of Duchenne and Becker muscular dystrophies in the United States. Pediatrics. 2015;135(3):513-521. doi:10.1542/peds.2014-2044. Illustrative comparison with selected commercially important monogenic diseases 1 in 30,000 1 in 17,500 1 in 10,000 1 in 6,578 1 in 5,000 1 in 4,250 1 in 2,000 1 in 311 Cystic fibrosis Phenylketonuria Spinal muscular atrophy Stargardt disease (ABCA4) Hemophilia A Duchenne muscular dystrophy Sickle cell disease Familial hypercholesterolemia Carrier frequency vs. selected monogenic diseases 30% 37% 12% 8% 3% 2% 2% 2%2% 2% Share of inherited retinal disease by gene ABCA4 Other genes USH2A EYS CDH23 TUBGCP6 CNGB1 CEP290 GPR179 CNGA3 • ABCA4 frequency: ~1 in 6,578 (Hanany M, Rivolta C, Sharon D, PNAS 2020) • Largest inherited retinal disease gene worldwide (Hanany M, Rivolta C, Sharon D, PNAS 2020) • Comparable patient population to several successful orphan-drug markets • Normal life expectancy creates a large prevalent patient pool
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U.S. Commercial Kelly Kilpatrick Chief Commercial Officer
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B E L I T E B I O / 45 Market Opportunity: There is a Strong Existing Diagnosed Patient Base Mata NL, Weng S, Michaelides M, Charbel Issa P, Quinodoz M, Rivolta C, Scholl HPN. Bisretinoids as a Source of Early Photoreceptor Pathology in Stargardt Disease. Ophthalmic Res. 2025;68(1):555-572. doi: 10.1159/000549368. Epub 2025 Nov 7. PMID: 41208544; PMCID: PMC12700588. Clinically diagnosed estimates sourced via Verana Health / IRIS registry Stargardt Disease Type 1 is the most common inherited retinal disease (IRD). ~53,000 Estimated Prevalent STGD1 population in the U.S. ~20,000 Estimated Clinically diagnosed STGD1 patients in the U.S. since 2016
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B E L I T E B I O / 46 The Commercial Organization is Built to Directly Engage with Key Stakeholders 50+ combined rare disease product launches amongst the commercial organization's current team members GENERAL / PED. OPHTH. COMMUNITY PAYERS PATIENTS AND CAREGIVERS OPTOMETRISTS AND GENETIC COUNSELORS IRD AND RETINA SPECIALISTS KEY STGD1 STAKEHOLDERS BELITE COMMERCIAL ORGANIZATION SALES TEAM PATIENT SUPPORT TEAM DIAGNOSTIC SPECIALIST TEAM MARKET ACCESS TEAM MARKETING TEAM COMMERCIAL OPERATIONS TEAM
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B E L I T E B I O / 47 Market Research Insights Source: Qualitative and Quantitative Market Research IDIs and Surveys Retinal specialists will be the primary prescribers and managers of tinlarebant There is a high unmet need in the Stargardt Disease Type 1 space UNMET NEED Disease awareness and genetic testing rates are high among retinal specialists AWARENESS Tinlarebant profile is very well received PERCEPTION The patient journey is convoluted and emotionally frustrating for patients PRESCRIBER PATIENT JOURNEY HCP Insights Patient and Caregiver Insights
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B E L I T E B I O / 48 U.S. Commercial Focus Areas Build Awareness of Stargardt Disease and IRD Genetic Testing Options Adopt tinlarebant as the New Standard of Care in STGD1 Ensure Optimal Market Access to tinlarebant Deliver an Optimal Patient Experience for Seamless Onboarding and Adherence APPROVAL
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B E L I T E B I O / 49 Build Awareness of Stargardt Disease Awareness Partner with Advocacy Groups Unbranded awareness campaign through patient advocacy organizations Pre-Approval Information Exchange Strengthen market awareness of Stargardt disease, its biology and unmet needs with payer audience Disease Education Educate stakeholders to recognize, understand, and address Stargardt disease
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B E L I T E B I O / 50 Build Awareness of IRD Genetic Testing Options Awareness Sources for IRD Genetic Testing Specialist Visit Order Test Gene Sequencing Test Report Next Steps How Genetic Testing Is Obtained 1 2 3 4 5 Academic and Private Labs Commercial Testing Labs Advocacy/Non-Profit Sponsored Program Manufacturer-Sponsored Program e.g., UCSF, MUSC, UCLA, OSU e.g., My Retina Tracker (Foundation Fighting Blindness) e.g., Genentech, Opus e.g., Blueprint Genetics, Prevention Genetics, GeneDx
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B E L I T E B I O / 51 Access Ensuring Optimal Market Access to tinlarebant ABCA4 Test Confirmatory test expected Pre-approval Info Clinical data to payers, from 4Q Payer Mix Primarily commercial, then Medicare and Medicaid Payer Team Across commercial, Medicare and Medicaid
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B E L I T E B I O / 52 Adopt Adopt tinlarebant as the New Standard of Care in STGD1 Personal Promotion Non-Personal Promotion Prioritize top centers and key HCPs. Engage across every channel. Establish the standard of care. Total Priority HCP Universe: ~6,000 HCPs IRD Specialists ~100 Retina Specialists ~3,000 Ophthalmologists, Optometrists and Others ~2,900 Websites Paid Social Media Paid Search Conferences Print Journals Digital Banner Ads Email Marketing
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B E L I T E B I O / 53 Deliver Optimal Patient Experience for Seamless Onboarding and Adherence Onboarding and Adherence Patient Support Pillars Ideal Post Rx Patient Journey HCP Writes Prescription Diagnosis to treatment decision Specialty Pharmacy Prescription routed for fulfillment Patient Services Onboarding into support programs Seamless Onboarding Frictionless start to treatment Financial Support Programs for Eligible Patients Cost should not be the reason a patient does not receive treatment Access Continuity Treatment continues without interruption Adherence Daily treatment is the key to success
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B E L I T E B I O / 54 Belite Bio Has Been Preparing for This Moment … And We Are Prepared to Launch … and we are excited to potentially bring the first-in-class treatment for STGD1 patients to market! We have done the work, developed the plan, built the team… PDUFA February 2027 NDA Acceptance and Priority Review August 11, 2026 Launch March 2027 NDA Submission June 12, 2026
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Live Q&A
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Thank you!