Belite Bio, Inc. · NASDAQ:BLTE
Belite Bio — Investor Presentation
32 slides from Belite Bio's investor presentation
Slide 1: Early Intervention with an Oral Treatment for Macular Degeneration
Early Intervention with an Oral Treatment for Macular Degeneration Mission for Vision Nasdaq: BLTE BELITE BIO / 1
Slide 2: Forward-Looking Statements and Legal Disclaimer
Forward-Looking Statements and Legal Disclaimer 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,
Slide 3: Belite Bio Executive Team
Belite Bio Executive Team Belite Management Team Tom Lin, MMED, PhD, MBA Hendrik Scholl, MD, MA Nathan Mata, PhD Hao-Yuan Chuang, CFA, MBA, FRM (Chairman, CEO) (CMO) (CSO) (CFO) • 16+ years of executive management • 25+ years of expertise in treating retinal • 15+ years of ophthalmic drug • 16+ year
Slide 4: Belite Bio Pipeline Overview
Belite Bio Pipeline Overview PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NDA Stargardt Disease (STGD1) • NDA accepted by US FDA and granted Priority Review; PDUFA date of February 12, 2027 • Ph3, 2-year treatment, global trial (“DRAGON” Study) completed (104 subjects) Tinlarebant • Ph2/3, 2-year treatment,
Slide 5: Tinlarebant Overview
Tinlarebant Overview BELITE BIO / 5
Slide 6: Market Opportunity
Market Opportunity Tinlarebant STARGARDT GEOGRAPHIC ATROPHY 1 in 8,800 (1,2) DISCOVERY PRE-CLINICAL The most common inherited PHASE I macular dystrophy PHASE II Patient population with PHASE III Stargardt Disease: MARKET 53k 109k (2) (2) US China Columbia University + NIH Blueprint “a promising firs
Slide 7: Visual Cycle
Normal Processing of Vitamin A in the Tinlarebant Induced Visual Cycle Enzymes Down-Regulation Tinlarebant Visual Pigment Retinoids Visual Chromophore Rhodopsin 11c-Ral at-Ral PHOTORECEPTORS (PR) ABCA4 11c-RDH at-RDH RETINAL PIGMENT 11c-Rol RPE65 at-RE LRAT at-Rol EPITHELIUM (RPE) RBP Receptor RBP4
Slide 8: Stargardt Disease 1 (STGD1)
Formation of Toxic Vitamin A Byproducts in Tinlarebant Induced Stargardt Disease 1 (STGD1) Enzymes Down-Regulation Tinlarebant Visual Pigment Retinoids Visual Chromophore Rhodopsin 11c-Ral at-Ral A2E STARGARDT PHOTORECEPTORS Gene mutation causes loss of (PR) ABCA4 ABCA4 transporter 11c-RDH function
Slide 9: Mechanism of Tinlarebant Action
Tinlarebant Mechanism of Tinlarebant Action Tinlarebant Induced Down-Regulation Visual Pigment Enzymes Retinoids Bisretinoids are derived from vitamin A (retinol). Therefore, reducing the delivery of retinol to the eye is Visual Chromophore expected to reduce bisretinoid levels in the eye leading to
Slide 10: Similar Pathology in STGD1 & GA
Tinlarebant Similar Pathology in STGD1 & GA • STGD1 and GA show a very similar phenotype, where loss of RPE and photoreceptors in the center of the retina, the macula, leads to loss of vision • Fundus autofluorescence imaging, visualizing bisretinoid content in the RPE, shows loss of signal in degen
Slide 11: DRAGON Clinical Trial
DRAGON Clinical Trial BELITE BIO / 11
Slide 12: DRAGON & DRAGON II Clinical Trial Design in STGD1
Tinlarebant DRAGON & DRAGON II Clinical Trial Design in STGD1 Reduction in lesion growth rate (DDAF) as measured by retinal imaging is the FDA accepted primary endpoint in STGD1 and GA STGD1 “DRAGON” Phase 3(1) STGD1 “DRAGON II” Phase 1b/2/3 Enrollment 104 subjects (have DDAF) 60 subjects (have DDAF
Slide 13: Tinlarebant Treatment Led to 80% Reduction in RBP4, Well Above Goal of 70%
Efficacy Tinlarebant Treatment Led to 80% Reduction in RBP4, Well Above Goal of 70%* Results Biomarker: RBP4 - Percent Reduction from Baseline PD Set N=92 70% Reduction Target * In a prior study of a surrogate RBP4 antagonist (fenretinide) in patients with Geographic Atrophy, an RBP4 reduction of ≥7
Slide 14: DDAF Represents Well-demarcated Areas of Complete RPE Loss
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 autofluorescence): between 50
Slide 15: DDAF Progression Rate in Stargardt
DDAF Progression Rate in Stargardt Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study Overall DDAF growth rate in the ProgStar cohort over 24 months: 0.74 mm2/year (confidence interval: 0.64 - 0.85 mm2/year) Strauss RW, Ho A, Jha A, Fujinami K, Michaelides
Slide 16: Primary Endpoint: DDAF in the Study Eye
Efficacy Primary Endpoint: DDAF in the Study Eye (Change from Baseline) Results • 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 consist
Slide 17: Primary Endpoint Showed a Statistically Significant & Clinically Meaningful Outcome
Efficacy Primary Endpoint Showed a Statistically Significant Results & Clinically Meaningful Outcome 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 P = 0.
Slide 18: Statistically Significant Treatment Effect Also Observed in the Fellow Eye
Efficacy A Statistically Significant Treatment Effect Was Also Observed Results in the Fellow Eye for the Primary Endpoint Primary Endpoint: DDAF Change from Baseline (Fellow Eye) mFAS N = 95 Tinlarebant slowed DDAF lesion growth in the fellow eye by 33.6% compared to placebo (P = 0.041) BELITE BIO
Slide 19: Tinlarebant Slowed DAF Lesion Growth in the Study Eye by 33.7%
Efficacy Tinlarebant Slowed DAF Lesion Growth, the Key Secondary Results Endpoint, in the Study Eye by 33.7% Key Secondary Endpoint: DAF Change from Baseline (Study Eye) mFAS N = 96 Tinlarebant slowed DAF lesion growth by 33.7% compared to placebo (P = 0.027) BELITE BIO / 19
Slide 20: Tinlarebant Slowed DAF Lesion Growth in the Fellow Eye by 32.7%
Efficacy Tinlarebant Slowed DAF Lesion Growth, the Key Secondary Results Endpoint, also in the Fellow Eye by 32.7% Key Secondary Endpoint: DAF Change from Baseline (Fellow Eye) mFAS N = 94 Tinlarebant slowed DAF lesion growth in the fellow eye by 32.7% compared to placebo (P = 0.017) BELITE BIO / 20
Slide 21: As Expected, BCVA in Study Eye Did Not Show Any Significant Change
Efficacy As Expected, BCVA in Study Eye Did Not Show Any Significant Change Results 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 change
Slide 22: ProgStar: Visual Acuity Change over 24 Months
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, West SK, Cideciyan AV,
Slide 23: Safety Results
Safety Results BELITE BIO / 23
Slide 24: Tinlarebant Demonstrated a Well Tolerated Safety Profile
Safety Tinlarebant Demonstrated a Well Tolerated Safety Profile Safety Set N = 104 Results Subjects Who Experienced at Least One Non-Ocular Treatment-Emergent Adverse Events (TEAE), N / (%) Tinlarebant 5mg Placebo Category (N=69) (N=35) TEAE 59 (85.5%) 27 (77.1%) Severe TEAE 2 (2.9%) 1 (2.9%) Seriou
Slide 25: The Majority of Ocular Adverse Events were Mild
Safety The Majority of Ocular Adverse Events were Mild Safety Set N = 104 Results Subjects Who Experience at Least One Ocular TEAE, N / (%) Tinlarebant 5mg Placebo Category (N=69) (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
Slide 26: Phase 3 PHOENIX Trial in Geographic Atrophy
Phase 3 PHOENIX Trial in Geographic Atrophy BELITE BIO / 26
Slide 27: Tinlarebant: ≥ 70% Reduction of RBP4
Tinlarebant Tinlarebant: ≥ 70% Reduction of RBP4 Phase 1, 5mg Daily Dosing in Healthy Adults: Mean Percent Reduction of RBP4 (excludes placebo) Final dose 70% Reduction Dosing Period BELITE BIO / 27
Slide 28: Clinical Trial Design Overview in GA
Tinlarebant Clinical Trial Design Overview in GA • Established Efficacy Endpoint – Reduction in GA Phase 3 “PHOENIX” (1) atrophic lesion growth rate as measured by retinal imaging is the FDA accepted primary Enrollment 530 subjects endpoint Sites Global • Early Intervention – Targeting patients with
Slide 29: Summary
Summary BELITE BIO / 29
Slide 30: Summary of Results of the DRAGON Trial
Summary Summary of Results of the DRAGON Trial • The DRAGON trial met its primary endpoint: A 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 be considered clinicall
Slide 31: Tinlarebant has the Potential to be the First-Ever Approved Treatment for Stargardt Disease
Tinlarebant has the Potential to be the First-Ever Approved Treatment for Stargardt Disease Conclusions • First-ever oral therapy in a retinal degenerative disease to demonstrate a clinically meaningful slowdown of neurodegeneration • 36% reduction in DDAF lesion growth rate, representing a robust a
Slide 32: Thank You
Thank You For more info please visit: www.belitebio.com