Slide 1: Phase 3 DRAGON Trial in Stargardt Disease Topline Results
B E L I T E B I O / 1 December 1, 2025, 8:00 a.m. ET Nasdaq: BLTE Phase 3 DRAGON Trial in Stargardt Disease Topline Results
Slide 2: Disclaimer and forward-looking statements
B E L I T E B I O / 2 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, s
Slide 3: Today's Speakers
B E L I T E B I O / 3 Today’s Speakers Tom Lin, MMED, PhD, MBA Chairman & Chief Executive Officer Nathan Mata, PhD Chief Strategy Officer Hendrik Scholl, MD, MA Chief Medical Officer Prof Leopold Schmetterer, PhD Prof Ruifang (Helen) Sui, MD, PhD • Consultant Ophthalmologist at Moorfields Eye Hospit
Slide 4: Agenda
B E L I T E B I O / 4 Agenda Stargardt Disease Overview Study Design Efficacy Results Safety Results Summary KOL Panel Discussion Q&A
Slide 5: Stargardt Disease Overview
B E L I T E B I O / 5 Stargardt Disease Overview
Slide 6: Stargardt Disease Overview
B E L I T E B I O / 6 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
Slide 7: Estimated Patient Population Sizes of Stargardt Disease
B E L I T E B I O / 7 Estimated Patient Population Sizes of Stargardt Disease 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 Nov 7:1-28. doi: 10.1159/000549368. Eu
Slide 8: Standard of Care Fundus Autofluorescence Imaging in Stargardt Disease
B E L I T E B I O / 8 Standard of Care Fundus Autofluorescence Imaging 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
Slide 9: Study Design
B E L I T E B I O / 9 Study Design
Slide 10: DRAGON Clinical Trial Design in Stargardt Disease
B E L I T E B I O / 1 0 DRAGON Clinical Trial Design in Stargardt Disease 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 Study Des
Slide 11: DRAGON Baseline DDAF Lesion Size
B E L I T E B I O / 1 1 DRAGON Baseline DDAF Lesion Size Study Design Tinlarebant (5mg) Placebo N 63 33 Mean 2.251 2.418 SD 2.0871 2.2050 Median 1.546 1.822 Min – Max 0.07 – 7.02 0.06 – 7.29 Baseline DDAF (mm2 ) – Study Eye Baseline DDAF (mm2 ) – Fellow Eye Tinlarebant (5mg) Placebo N 57 29 Mean 2.5
Slide 12: Efficacy Results
B E L I T E B I O / 1 2 Efficacy Results
Slide 13: Sustained 80% Reduction of RBP4 with Tinlarebant
B E L I T E B I O / 1 3 Daily dosing of 5 mg/day Tinlarebant led to a sustained 80% reduction of RPB4 and RPB4 levels returned to 84 % of the baseline value at the End of Study (EOS) Biomarker: RBP4 - Percent Reduction from Baseline PD Set N=92 Efficacy Results 70% Reduction Target Tinlarebant Treat
Slide 14: DDAF Represents Complete RPE Loss and Is an Approvable Primary Endpoint
B E L I T E B I O / 1 4 DDAF Represents Well-demarcated Areas of Complete RPE Loss & Grows Predictably, Making it an Approvable Primary Endpoint • DDAF (definitely decreased autofluorescence): level of darkness close to 100% (at least 90%) in reference to the ONH • QDAF (questionably decreased autof
Slide 15: DDAF Progression Rate in Stargardt
B E L I T E B I O / 1 5 DDAF Progression Rate in Stargardt Overall DDAF growth rate in the ProgStar cohort over 24 months: 0.74 mm2/year (confidence interval: 0.64 - 0.85 mm2/year) Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study Strauss RW, Ho A, Jha A,
Slide 16: Primary Endpoint: DDAF in the Study Eye
B E L I T E B I O / 1 6 Primary Endpoint: DDAF in the Study Eye (Change from Baseline) • Annualized rate of lesion growth in the aggregate area of atrophy (DDAF) from baseline as assessed by fundus autofluorescence imaging at Month 25. • Data is shown for the modified full analysis set (mFAS) which
Slide 17: Primary Endpoint Showed Statistically Significant Outcome
B E L I T E B I O / 1 7 Applying an unstructured covariance matrix, the treatment effect size was 35.7% compared to placebo and yielded a p-value of P = 0.0033 With a first-order autoregressive covariance matrix, the treatment effect size remained consistent (35.4%) with P <0.0001 DDAF lesion growth
Slide 18: Statistically Significant Effect in the Fellow Eye
B E L I T E B I O / 1 8 Tinlarebant slowed DDAF lesion growth in the fellow eye by 33.6% compared to placebo (P = 0.041) Primary Endpoint: DDAF Change from Baseline (Fellow Eye) mFAS N = 95 Efficacy Results A Statistically Significant Treatment Effect Was Also Observed in the Fellow Eye for the Prim
Slide 19: Key Secondary Endpoint: DAF Change (Study Eye)
B E L I T E B I O / 1 9 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 Efficacy Results Tinlarebant Slowed DAF Lesion Growth, the Key Secondary Endpoint, in the Study Eye by 33.7%
Slide 20: Key Secondary Endpoint: DAF Change (Fellow Eye)
B E L I T E B I O / 2 0 Tinlarebant slowed DAF lesion growth in the fellow eye by 32.7% compared to placebo (P = 0.017) Key Secondary Endpoint: DAF Change from Baseline (Fellow Eye) mFAS N = 94 Efficacy Results Tinlarebant Slowed DAF Lesion Growth, the Key Secondary Endpoint, also in the Fellow Eye
Slide 21: BCVA in Study Eye Did Not Show Any Significant Change
B E L I T E B I O / 2 1 As Expected, BCVA in Study Eye Did Not Show Any Significant Change Tinlarebant Placebo BCVA at Baseline 39.9 39.4 BCVA at EOS 39.7 40.0 • The overall change of visual acuity was minimal over the period of 24 months in both treatment groups • Test–retest variability for ETDRS
Slide 22: ProgStar: Visual Acuity Change over Months
B E L I T E B I O / 2 2 ProgStar: Visual Acuity Change over 24 Months Prospective Cohort (N=434) • Overall rate of BCVA loss was 0.55 letters/year over two years • BCVA of eyes with baseline BCVA between 20/70 and 20/200 declined at a rate of 0.6 letters/year Kong X, Fujinami K, Strauss RW, Munoz B,
Slide 23: Safety Results
B E L I T E B I O / 2 3 Safety Results
Slide 24: Tinlarebant Demonstrated a Well Tolerated Safety Profile
B E L I T E B I O / 2 4 Tinlarebant Demonstrated a Well Tolerated Safety Profile Safety Set N = 104 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
Slide 25: The Majority of Ocular Adverse Events was Mild
B E L I T E B I O / 2 5 The Majority of Ocular Adverse Events was Mild Safety Set N = 104 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
Slide 26: Summary
B E L I T E B I O / 2 6 Summary
Slide 27: Summary of Results of the DRAGON Trial
B E L I T E B I O / 2 7 Summary of Results of the DRAGON Trial • The DRAGON trial met its primary endpoint: A highly statistically significant slowing in DDAF lesion growth was observed in subjects treated with 5 mg/day oral Tinlarebant as compared to placebo • The treatment effect was 36% and must
Slide 28: Tinlarebant Potential to Be First Approved Stargardt Treatment
B E L I T E B I O / 2 8 Tinlarebant has the Potential to be the First-Ever Approved Treatment for Stargardt Disease • First-ever oral therapy in a retinal degenerative disease to demonstrate a clinically meaningful slowdown of neurodegeneration • 36% reduction in DDAF lesion growth rate, representin
Slide 29: KOL Panel Discussion
KOL Panel Discussion For more info please visit: www.belitebio.com
Slide 30: Q&A
Q&A For more info please visit: www.belitebio.com