Aldeyra Therapeutics, Inc. · NASDAQ:ALDX
Aldeyra Therapeutics — Investor Day (February 2019)
44 slides from Aldeyra Therapeutics's investor day presentation, Feb 28, 2019
Slide 1: R&D Day 2019 Update on Research Programs
R&D Day 2019 Update on Research Programs February 28, 2019 Exhibit 99.1
Slide 2: Disclaimers and Forward-Looking Statements
Disclaimers and Forward-Looking Statements This presentation and various remarks which may be made during this presentation contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and Section 21E of the Securities Exchange Act of 1934, as amended
Slide 3: Agenda
Agenda Opening WelcomeTodd Brady, CEO Corporate Strategy & Pipeline GrowthDavid McMullin, CCO Proliferative Vitreoretinopathy – A Rare Retinal DiseaseDean Eliott, M.D. Harvard Medical School Mass. Eye and Ear Infirmary Ocular Disease Area Program UpdatesDavid Clark, CMO Ocular Disease Area Market Op
Slide 4: Our Mission
Our Mission Developing Next-Generation Medicines to Improve the Lives of Patients with Immune-Mediated Diseases ~7% Of Western Society Suffer from some form of immune-mediated disease, and incidence is increasing Disease control elusive despite existing therapies, and thus novel approaches are neede
Slide 5: Immune System Imbalance Leads to Immune-Mediated Disease
Immune System Imbalance Leads to Immune-Mediated Disease Stimulation Imbalance Inhibition Imbalance Autoimmune Cancer Immuno-proliferative Allergy Stimulation Inhibition Immune Balance Healthy Imbalance can result in… Immune-Mediated Diseases
Slide 6: Deliberate Focus on Ocular Diseases and Select Systemic Diseases
Deliberate Focus on Ocular Diseases and Select Systemic Diseases 2004 May 2014 2015 2016 2017 2018 2019 … Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis PVR Retinal Disease Sjögren-Larsson Syndrome PTLD Mesothelioma and other rare cancers** Autoimmune Discovery & Preclinical
Slide 7: Our Novel Approaches to Address Immune-Mediated Disease
Our Novel Approaches to Address Immune-Mediated Disease Cell Activation Cell Proliferation Immune Cell Stimulus Hsp90 Inhibitors RASP Inhibitors DHFR Inhibitors RASP = Reactive Aldehyde Species DHFR = Dihydrofolate Reductase Hsp90 = Heat Shock Protein 90 Reproxalap ADX-103 ADX-629 ADX-1612 ADX-1615
Slide 8: Deep and Innovative Pipeline Focused on Immune-Mediated Diseases
Ocular Inflammation Deep and Innovative Pipeline Focused on Immune-Mediated Diseases Ocular Diseases Indication Compound Disease Area Preclinical Phase 1 Phase 2 Phase 3 Next Anticipated Milestone = Positive Phase 2 clinical trial data reported in 2016 – 2018 Trial initiations contingent on funding,
Slide 9: Helio Vision Acquisition Expands Pipeline in Support of Strategic Growth Plans
Helio Vision Acquisition Expands Pipeline in Support of Our Strategic Growth Plans Retinal disease a strategic priority for pipeline growth Novel therapeutic approach leveraging an immunological mechanism that diminishes inflammation and cell proliferation Addition of Phase 3-ready clinical program
Slide 10: Ocular Disease Area Program Updates
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Slide 11: Ocular Disease Area Program Updates
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Slide 12: ADX-2191: Adaptive Phase 3 PVR Clinical Program Expected to Initiate H2 2019
ADX-2191: Adaptive Phase 3 Proliferative Vitreoretinopathy Clinical Program Expected to Initiate H2 2019 Confirm endpoints, safety and tolerability Confirm sample size for subsequent trial Part 1 controlled, randomized trial design Adaptive Phase 3 Program Phase 3 Program Design Elements Adaptive Ph
Slide 13: ADX-2191: Adaptive Phase 3 (Part 1) PVR Clinical Trial Design
Primary objective: Evaluate efficacy of intravitreal ADX-2191 injections for prevention of recurrent retinal detachment due to proliferative vitreoretinopathy (PVR) Design: Multi-center, non-masked, randomized, controlled, two- part, adaptive Phase 3 clinical trial Inclusion highlights: Recurrent re
Slide 14: Ocular Disease Area Program Updates
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Slide 15: Reproxalap: Adaptive Phase 3 Clinical Program Expected to Initiate H1 2019
Reproxalap: Adaptive Phase 3 Dry Eye Disease Clinical Program Expected to Initiate H1 2019 Adaptive Phase 3 Program Phase 3 Program Design Elements Adaptive DED Phase 3 (Part 1) Adaptive DED Phase 3 (Part 2) Confirmatory DED Phase 3 NDA Illustrative only Expected initiation H1 2019 Phase 3 DED Safet
Slide 16: Reproxalap: Adaptive Phase 3 (Part 1) Clinical Trial Design
Primary objective: Evaluate efficacy of reproxalap ophthalmic solution (0.25%) vs. vehicle to confirm dosing regimen and sample size for Part 2 Inclusion/exclusion criteria: Same as used for Phase 2b Moderate to severe dry eye disease Co-primary endpoints: Ocular dryness score (0-100mm VAS) and fluo
Slide 17: Reproxalap: Adaptive Phase 3 (Part 2) Clinical Trial Design
Reproxalap: Adaptive Phase 3 (Part 2) Dry Eye Disease Clinical Trial Design Vehicle -Wk 2 -Wk 1 Wk 1 Wk 2 Wk 3 Wk 4 Wk 5 Wk 6 Wk 7 Wk 8 Wk 9 Wk 10 Wk 11 Wk 12 Visit 1 Week -2 Visit 2 Day 1 Visit 4 Wk 2 Visit 5 Week 4 Visit 7 Week 8 Visit 9 Week 12 Screening Treatment Regimen tbd (QID or QID to BID)
Slide 18: Reproxalap: Symptom and Sign Endpoints Achieved in Phase 2b Clinical Trial
Reproxalap: Dry Eye Disease Symptom and Sign Endpoints Achieved in Phase 2b Clinical Trial Mean Change from Baseline OD & 4-Symptom Questionnaire: Dryness (0-5) Baseline Score > 3 (N=69|69) Fluorescein Staining: Nasal (0-4) Baseline Score > 2 (N=62|56) Primary Symptom Endpoint for Phase 3 DED Primar
Slide 19: Ocular Disease Area Program Updates
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Slide 20: Reproxalap: ALLEVIATE Phase 3 Trial Design in Allergic Conjunctivitis
Reproxalap: ALLEVIATE Phase 3 Trial Design in Allergic Conjunctivitis Phase 3 Conjunctival Allergen Challenge Trial Primary objective: Evaluate efficacy of reproxalap ophthalmic solutions (0.25% & 0.5%) compared to vehicle for the treatment of ocular itching associated with acute allergic conjunctiv
Slide 21: Reproxalap: AC Endpoints Achieved in Phase 2b Clinical Trial
Reproxalap: AC Ocular Itch Area Under The Curve and Responder Endpoints Achieved in Phase 2b Clinical Trial Source: Reproxalap AC Phase 2b clinical trial results (~30 patients per arm, seasonal allergy) Area Under the Curve: Ocular Itch Score (0-4) Probability of Response: Ocular Itch Score (0-4) Im
Slide 22: Reproxalap's Novel Mechanism of Action Potential for More Durable Activity
Reproxalap’s Novel Mechanism of Action has the Potential to Provide More Durable Activity Than Antihistamines Reproxalap has the potential to be uniquely effective in post-histaminic allergy, for which no drug is approved, and which affects all allergic conjunctivitis patients. Histaminic Phase Post
Slide 23: Reproxalap: Parallel DED and AC Phase 3 Programs Support Concurrent NDA Filings
AC Phase 3 CAC Reproxalap: Parallel Dry Eye Disease and Allergic Conjunctivitis Phase 3 Clinical Programs Support Concurrent NDA Filings Adaptive DED Phase 3 (Part 1) Adaptive DED Phase 3 (Part 2) Confirmatory DED Phase 3 Expected initiation H1 2019 Phase 3 DED Safety Study Phase 3 AC Safety Study A
Slide 24: Ocular Disease Area Program Updates
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Slide 25: Multiple Upcoming Ocular Disease Area Clinical Program Milestones
Multiple Upcoming Ocular Disease Area Clinical Program Milestones 2019 2020 Early 2019: Reproxalap ALLEVIATE Phase 3 allergic conjunctivitis trial Results H1 2019: Reproxalap Phase 3 dry eye disease clinical trial program Initiate H2 2019: Reproxalap SOLACE Phase 3 noninfectious anterior uveitis tri
Slide 26: Ocular Disease Area Market Opportunities
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
Slide 27: We Intend to Target Unmet Medical Needs in Anterior and Posterior Ocular Diseases
Rare Common Low High Under-served Prevalence Unmet Medical Need DED AC We Intend to Target Unmet Medical Needs in Anterior and Posterior Ocular Diseases Anterior Opportunities Posterior Opportunities Rare Common Low High Under-served Prevalence Unmet Medical Need NAU PVR ADX-103 ADX-2191 Future Clea
Slide 28: Ocular Disease Area Market Opportunities
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
Slide 29: PVR: A Rare Sight-Threatening Retinal Disease
4,000 U.S. PVR is a rare disease, with ~4,000 patients per year in the U.S. and nearly twice as many in Europe and Japan PVR is the leading complication of retinal detachment surgery and prevents successful reattachment PVR is an serious condition leading to permanent vision loss in up to 75% of cas
Slide 30: PVR: High Unmet Medical Need With No Approved Therapies
PVR: High Unmet Medical Need With No Approved Therapies ~50% failure rate 4,000 per year >28,000 per year Rhegmatogenous Retinal Detachment Non-traumatic Traumatic (open-globe injury) Proliferative Vitreoretinopathy U.S. Estimates Primary Surgery Etc. Repeat Repeat Repeat PVR Repeat Surgery Addt’l.
Slide 31: ADX-2191: A Unique Approach and Novel Product Candidate for PVR
ADX-2191: A Unique Approach and Novel Product Candidate for PVR A Unique Opportunity PVR: A Sight-Threatening Disease ADX-2191 A novel approach and potential therapeutic breakthrough in PVR treatment Granted U.S. orphan designation Tolerability and reattachment success during study period demonstrat
Slide 32: Ocular Disease Area Market Opportunities
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
Slide 33: DED and AC: Persistently Disturbing and Overlapping Disease Burdens
DED and AC: Persistently Disturbing and Overlapping Disease Burdens Dry Eye Disease Allergic Conjunctivitis DED+AC Comorbidity Women are twice as likely to suffer from DED than men vs Significant negative quality of life impact AC can result in acute, intermittent, and chronic symptoms AC patients e
Slide 34: DED and AC: Chronic Diseases With Inadequate Therapies
DED and AC: Chronic Diseases With Inadequate Therapies Dry Eye Disease Allergic Conjunctivitis DED+AC Comorbidity Current Rx options may take up to six weeks or longer to achieve even modest efficacy Up to 75% of patients with DED are not satisfied with current prescription options Up to 75% Up to 5
Slide 35: DED and AC: Large Market Opportunities With Unmet Medical Needs
DED and AC: Large Market Opportunities With Unmet Medical Needs 10 million 20 million 30 million 100 million Up to 50% comorbidity Allergic Conjunctivitis Unsatisfied With SOC Dry Eye Disease U.S. Patient Estimates Significant negative quality of life Complex, overlapping, and difficult to treat chr
Slide 36: Reproxalap: A Unique and Novel Product Candidate for DED and AC
Effective in post-histaminic allergy, for which no drug is approved Clinically significant and durable symptom response in Phase 2b clinical trial Reproxalap: A Unique and Novel Product Candidate for DED and AC Dry Eye Disease Allergic Conjunctivitis DED+AC Overlap Reproxalap in DED Reproxalap Repro
Slide 37: Ocular Disease Area Market Opportunities
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
Slide 38: NAU: A Severe Ocular Inflammatory Disease
NAU: A Severe Ocular Inflammatory Disease ~50% of NAU patients have recurrent or chronic conditions requiring multiple interventions per year 260K annually NAU is the most common form of uveitis with an estimated 260,000 U.S. patients per year Noninfectious anterior uveitis (NAU) is a severe ocular
Slide 39: NAU: Repeat Episodes and Steroid Toxicity Create Need for Novel Approaches
NAU: Significant Repeat Episodes and Steroid Toxicity Creates the Need for Novel Approaches Acute Recurrent Chronic ~50% ~25% ~25% Single flare within a year Relapse 3-12 months after initial episode Relapse <3 months after initial episode Prevalence: Approximately 260,000 noninfectious anterior uve
Slide 40: Reproxalap: A Unique and Novel Product Candidate for NAU
Reproxalap: A Unique and Novel Product Candidate for NAU A Unique Opportunity NAU: A Serious Inflammatory Disease With Inadequate Current Therapy Reproxalap A novel and differentiated approach to treat NAU Reduced anterior chamber cell count observed in a Phase 2 clinical trial, statistically non-in
Slide 41: Pathway to Commercialization
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
Slide 42: We Intend to Commercialize Directly and Through Partnerships
We Intend to Commercialize Directly and Through Partnerships Estimated U.S. Population* U.S. Healthcare Providers Competitive Value Proposition Infrastructure Requirement Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis Sjögren-Larsson Syndrome Late Stage Programs Ocular Diseas
Slide 43: Conclusion
“Y ALDEYRA THERAPEUTICS™ Conclusion Nasdaq: ALDX Aldeyra Therapeutics, Inc. 2019
Slide 44: Expected Development Milestones: Novel Approaches to Address Immune-Mediated Disease
Expected Development Milestones: Novel Approaches to Address Immune-Mediated Disease *Contingent on funding, regulatory review, and other factors. Reproxalap allergic conjunctivitis ALLEVIATE Phase 3 trial results early 2019 Ocular Diseases: Anticipated Milestones* Systemic Diseases: Anticipated Mil