Aldeyra Therapeutics, Inc. · NASDAQ:ALDX
Aldeyra Therapeutics — ARVO Annual Meeting 2019
21 slides from Aldeyra Therapeutics's conference presentation, May 1, 2019
Slide 1: Phase 2b Dry Eye Disease Clinical Trial of Topical Ocular Reproxalap
A Multi-Center, Randomized, Double-Masked, Parallel-Group, Vehicle-Controlled Phase 2b Dry Eye Disease Clinical Trial to Evaluate the Safety and Efficacy of Topical Ocular Reproxalap, a Novel RASP Inhibitor. John D Sheppard, MD, MMSc, FACS Professor of Ophthalmology, Microbiology & Molecular Biology
Slide 2: Disclosures
Disclosures • Alcon: Research Grants, Speaker, Advisor • Lacrisciences: Shareholder, Advisor • Rutech: Clinical Investigator, Advisory Board • Aldeyra Pharmaceuticals: Advisory Board, Clinical Research • LayerBio: Advisor, Investor • Santen: Research, Speaker, Advisory Board • Allergan: Research Gra
Slide 3: Blockade of RASP: A Novel Therapeutic Approach
Blockade of RASP: A Novel Therapeutic Approach • Reproxalap rapidly binds to RASP in a reaction that is RASP essentially irreversible in vivo Binding • Reproxalap out-competes endogenous RASP targets Adduct • Reproxalap-RASP adducts are transported Transport inside the cell Adduct • Reproxalap-RASP
Slide 4: RASP Are Elevated in Tears of Dry Eye Disease Patients
RASP Are Elevated in Tears of Dry Eye Disease Patients Curr Eye Res. 41:1143-9, 2016. Graefe's Arch Clin Exp Ophthalmol. 233:694-8, 1995. 4
Slide 5: Reproxalap Demonstrated RASP Reduction in DED Phase 2a Trial
Reproxalap Demonstrated RASP Reduction in DED Phase 2a Trial Which Correlated with Sign Improvement in Individual Patients Pre-Treatment = Day 0, Post-Treatment = Day 28. Pooled data from Phase 2a clinical trial presented at ARVO 2018 5
Slide 6: Reproxalap's Novel Mechanism of Action Addresses Two Major Forms of Dry Eye
Reproxalap’s Novel Mechanism of Action has the Potential to Address the Two Major Forms of Dry Eye Disease Produces aqueous fluid AQUEOUS DEFICIENT Lacrimal gland DRY EYE RASP- RASP inhibitors Inflammatory Mediated cytokines Tear Film Insufficient tear volume and Cell death increased evaporation
Slide 7: Phase 2b Dry Eye Disease Clinical Trial Design
Phase 2b Dry Eye Disease Clinical Trial Design • Primary objective: – Evaluate efficacy of reproxalap ophthalmic solutions vs. baseline and vehicle to confirm endpoint selection and sample size for Phase 3 clinical trials • Inclusion/exclusion highlights: – History of dry eye disease for at least 6
Slide 8: Phase 2b Dry Eye Disease Clinical Trial Design
Phase 2b Dry Eye Disease Clinical Trial Design Phase 2b Dry Eye Disease Clinical Trial Visit 1 Visit 2 Visit 3 Visit 4 Visit 5 Visit 6 Week -2 Day 1 Week 2 Week 4 Week 8 Week 12 Screening Treatment (QID) Vehicle (N = 100) Vehicle Reproxalap 0.1% (N = 100) Reproxalap 0.25% (N = 100) -Wk 2 -Wk 1 Wk 1
Slide 9: Reproxalap Improved Ocular Dryness vs. Vehicle
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Reproxalap Improved Ocular Dryness vs. Vehicle OD & 4-Symptom Questionnaire: Dryness (0-5) ITT Population with Observed Data Only Average Baseline Score = 3.1 p values subject to change based on quality control analysis Source: Re
Slide 10: Drug Potency Supported by Ocular Dryness Improvement in Higher Baseline Patients
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Drug Potency Supported by Ocular Dryness Improvement vs. Vehicle in Higher Baseline Patients OD & 4-Symptom Questionnaire: Dryness (0-5) Total Population (N=100|100|100) Above Median Baseline Population (N=79|69|69) ITT Population
Slide 11: Ocular Discomfort Symptom Results Support Improvement in Ocular Dryness Score
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Ocular Discomfort Symptom Results Support Observed Improvement in Ocular Dryness Score OD & 4-Symptom Questionnaire: Overall Ocular Discomfort (0-5) ITT Population with Observed Data Only Average Baseline Score = 2.9 p values subj
Slide 12: Drug Potency Supported by Ocular Discomfort Improvement in Higher Baseline Patients
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Drug Potency Supported by Ocular Discomfort Improvement vs. Vehicle in Higher Baseline Patients OD & 4-Symptom Questionnaire: Overall Ocular Discomfort (0-5) Total Population (N=100|100|100) Above Median Baseline Population (N=69|
Slide 13: Reproxalap Improved Ocular Staining vs. Vehicle
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Reproxalap Improved Ocular Staining vs. Vehicle Fluorescein Staining: Nasal (0-4) ITT Population with Observed Data Only Average Baseline Score = 1.9 0.25% reproxalap vs. vehicle MMRM Week 2-12 p=0.02 p values subject to change ba
Slide 14: Drug Potency Supported by Ocular Staining Improvement in Higher Baseline Patients
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Drug Potency Supported by Ocular Staining Improvement vs. Vehicle in Higher Baseline Patients Fluorescein Staining: Nasal (0-4) Total Population (N=100|100|100) Above Median Baseline Population (N=59|56|62) ITT Population with Obs
Slide 15: Ocular Staining Responder Analyses Demonstrate Statistical Superiority over Vehicle
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Ocular Staining Responder Analyses Demonstrate Statistical Superiority of Reproxalap over Vehicle Fluorescein Staining (Nasal) OD&4S: Ocular Dryness and Fluorescein Staining (Nasal) ITT Population with Observed Data Only ITT Popul
Slide 16: Differentiated Product Profile Evidenced by Responder Analyses: Rapid and Symptom-Free
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Reproxalap’s Differentiated Product Profile Evidenced by Responder Analyses – Rapid and Symptom-Free (Ocular Dryness) OD & 4-Symptom Questionnaire: Dryness ITT Population with Observed Data Only Probability of Response (Improvemen
Slide 17: Broad Pattern of Drug Activity Across Symptoms and Signs
Broad Pattern of Drug Activity Across Dry Eye Disease Symptoms and Signs Supports Differentiated Product Profile Improvement Effect Size at Week 12 ITT Population with Observed Data Only Dry Eye Disease Symptoms 0.25% reproxalap 4-Symptom: Ocular Discomfort Vehicle 4-Symptom: Dryness 4 Symptom: Grit
Slide 18: Conclusions
Conclusions Positive Phase 2b Clinical Trial Results • Primary objective achieved: Endpoint selection and sample size powering confirmed for Phase 3 clinical trials • Reproxalap demonstrated statistically significant improvements versus vehicle across multiple symptom and sign measures, consistent w
Slide 19: Adaptive Phase 3 Dry Eye Disease Clinical Trial Design
CONFIDENTIAL – NOT FOR DISTRIBUTION; PROPERTY OF ALDEYRA THERAPEUTICS Adaptive Phase 3 Dry Eye Disease Clinical Trial Design Phase 3 Dry Eye Disease Clinical Trial: Part 1 Phase 3 Dry Eye Disease Clinical Trial: Part 2 Visit 1 Visit 2 Visit 3 Visit 4 Visit 5 Visit 6 Visit 7 Visit 8 Visit 9 Week -2 D
Slide 20: Phase 3 Primary Endpoint Strategy: Symptom and Sign Endpoints
Phase 3 Primary Endpoint Strategy: Dry Eye Disease Symptom and Sign Endpoints Achieved in Phase 2b Clinical Trial with MMRM Primary Symptom Endpoint for Phase 3 DED Primary Sign Endpoint for Phase 3 DED OD & 4-Symptom Questionnaire: Dryness (0-5) Fluorescein Staining: Nasal (0-4) Baseline Score > 3
Slide 21: Thank You
Thank You ARVO May 1, 2019 Vancouver, BC 21