Aldeyra Therapeutics, Inc. · NASDAQ:ALDX
Aldeyra Therapeutics — Technical Presentation (June 2018)
40 slides from Aldeyra Therapeutics's technical presentation, Jun 26, 2018
Slide 1: Research Day 2018 Update on Research Programs
Research Day 2018 Update on Research Programs June 26, 2018 New York NASDAQ: ALDX ©Aldeyra Therapeutics, Inc. 2018 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: 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 Disease control elusive despite existing therapies, and thus novel approaches are needed Unmet Needs Source: Shurin
Slide 4: Developing Next-Generation Medicines for Immune-Mediated Diseases
Developing Next-Generation Medicines to Improve the Lives of Patients with Immune-Mediated Diseases Deep and Innovative Pipeline focused on immune-mediated diseases Near-Term Development Catalysts support path to commercialization Solid Track Record of development success Solid Cash Position $ Large
Slide 5: Reproxalap: Lead Candidate With Significant Commercial Potential
Reproxalap: Lead Candidate With Significant Commercial Potential Ocular Inflammation Inborn Errors of Metabolism Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis Sjögren-Larsson Syndrome Potential Competitive Advantages2 Rapid onset, broader activity Non-drying, durable activit
Slide 6: Deep and Innovative Pipeline
Deep and Innovative Pipeline Not Disclosed Reproxalap Ocular Reproxalap Dermal ADX-629 Systemic ADX-103 ADX-1612 Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis Sjögren-Larsson Syndrome Autoimmune Disease Retinal Disease Lymphoproliferative Immune Disease Phase 2b results H2-2
Slide 7: 2018 Progress and Near-Term Development Catalysts
2018 Progress and Near-Term Development Catalysts Support Path to Commercialization Initiated reproxalap Phase 2b clinical trial in dry eye disease First patient enrolled in reproxalap Phase 3, Part 1 clinical trial in Sjögren-Larsson Syndrome Q3 2018 Reproxalap dry eye disease Phase 2b clinical tri
Slide 8: Research Day 2018 Update on Research Programs
Research Day 2018 Update on Research Programs June 26, 2018 New York NASDAQ: ALDX
Slide 9: Developing Next-Generation Medicines: Programs Overview
Developing Next-Generation Medicines to Improve the Lives of Patients with Immune-Mediated Diseases Targeting RASP Targeting Hsp90 Partnership Update ADX-629 for Systemic Immune-Mediated Diseases ADX-103 for Inflammatory Retinal Disease ADX-1612 for Lymphoproliferative Immune Disease and Cancer ADX-
Slide 10: Immune System Balance is Complex
Immune System Balance is Complex Stimulation Regulation Immune Balance Healthy Stimulation Imbalance Autoimmune Disease Regulation Imbalance Cancer
Slide 11: Novel Approaches to Address Immune-Mediated Disease
Novel Approaches to Address Immune-Mediated Disease RASP = Reactive Aldehydes Species that are Pro-inflammatory RASP Activated Cell Cell Proliferation Immune Cell Stimulus Hsp90 Inhibitors RASP Inhibitors
Slide 12: Targeting RASP for Systemic Immune-Mediated Diseases ADX-629
Targeting RASP for Systemic Immune-Mediated Diseases ADX-629
Slide 13: Targeting RASP: Mediators of Inflammation and Activators of the Immune System
Targeting RASP: Mediators of Inflammation and Activators of the Immune System Thiol / Amine Adduct Formation Receptor / kinase, lipid modification Inflammasome, NFkB activation; cytokine release Scavenger Receptor A binding Auto-antibody formation RASP Activated Immune Cell Inflammation Scientific L
Slide 14: ADX-629: Novel Pre-Clinical RASP Inhibitor for Systemic Immune-Mediated Disorders
ADX-629: A Novel Pre-Clinical RASP Inhibitor for Treatment of Systemic Immune-Mediated Disorders Highly prevalent disease characterized by liver inflammation, fibrosis, cirrhosis, and hepatocellular carcinoma No FDA-approved therapy RASP end-products observed in NASH Over one million patients in the
Slide 15: NASH Pathogenesis: Chronic Progression of Inflammation and Fibrosis
Normal hepatocyte Fatty hepatocyte Fatty & injured hepatocyte Apoptotic hepatocyte NASH Pathogenesis: Chronic Progression of Inflammation and Fibrosis Adapted from Diehl and Day NEMJ 377:2062-2072, 2017; RASP activity as shown based on published literature and Aldeyra data on file. RASP-Mediated RAS
Slide 16: IBD Pathogenesis: Chronic Relapsing Intestinal Inflammation
Environmental factors Microorganisms Diet Infections Stress NSAIDs Appendectomy Smoking Antibiotics Genetic factors Antimicrobial peptides Autophagy Handling of bacteria Chemokines Cytokines Initiating triggers Phase I: Pre-disease stage Phase II: Acute intestinal inflammation Phase III: Recurrent i
Slide 17: ADX-629: A Pre-Clinical Novel RASP Inhibitor
ADX-629: A Pre-Clinical Novel RASP Inhibitor ADX-629 is an analog of reproxalap Reproxalap has demonstrated activity in immune-mediated diseases Pre-Clinical Models Sepsis Inflammatory pain Oral mucositis Allergic dermatitis Contact dermatitis Acute lung injury Corneal fibrosis Clinical Trials Dry e
Slide 18: ADX-629 Decreased LPS-Induced Pro-inflammatory Cytokine Levels in Animal Models
ADX-629 Decreased LPS-Induced Pro-inflammatory Cytokine Levels and Increased Levels of an Anti-inflammatory Cytokine in Animal Models Cytokine Decrease RANTES 93.8% MIP-1α 93.1% IL-12(p40) 92.4% G-CSF 91.1% LIF 85.8% MIG 83.3% IL-5 82.3% IL-17 77.4% M-CSF 75.1% GM-CSF 73.7% IL-13 73.6% IL-15 72.1% C
Slide 19: Potential ADX-629 Development Overview
Planning to initiate clinical testing in 2019 Potential ADX-629 Development Overview 2018 2020 Today 144 days Jul 17 - Feb 1 106 days Feb 1 - Jun 30 155 days Jul 1 - Jan 31 155 days Jul 1 - Jan 31 Contingent on pre-clinical studies, clinical trials, funding, regulatory review, and other factors. Pha
Slide 20: Targeting RASP for Inflammatory Retinal Disease ADX-103
Targeting RASP for Inflammatory Retinal Disease ADX-103
Slide 21: ADX-103: A Structurally Distinct Pre-Clinical RASP Inhibitor
ADX-103: A Structurally Distinct Pre-Clinical RASP Inhibitor Potential product candidate for treatment of retinal disease RASP observed in retinal disease Efficacy in several preclinical models of ocular inflammation Diabetic macular edema (DME) Dry age-related macular degeneration (AMD) / Stargardt
Slide 22: Diabetic Macular Edema: ADX-103 Blocked Diabetes-Induced Retinal Changes
Diabetic Macular Edema: ADX-103 Blocked Diabetes-Induced Retinal Changes in Animal Models Male Norway rats were administered streptozotocin (STZ), 55 mg/kg IP, on Day 0 to induce diabetes Two single doses of ADX-103 (17.5 µg each) were administered intravitreally, after induction of diabetes (Days 4
Slide 23: Endotoxin-Induced Uveitis: ADX-103 Decreased Ocular Inflammation
Endotoxin-Induced Uveitis: ADX-103 Decreased Ocular Inflammation in Animal Models Ocular inflammation in rats induced by footpad injection of a bacterial endotoxin (LPS) Severe model Peaks at 24 hours A single intravitreal dose of ADX-103 (25 µg/eye) was administered at hour 1 post-LPS administratio
Slide 24: Potential ADX-103 Retina Program Overview
Planning to initiate clinical testing in 2019 Potential ADX-103 Retina Program Overview 2018 2020 Today 144 days Jul 17 - Feb 1 106 days Feb 1 - Jun 30 155 days Jul 1 - Jan 31 155 days Jul 1 - Jan 31 Phase 2 Clinical Trial IND-Enabling Toxicology Phase 1/2 Clinical Trial Contingent on pre-clinical s
Slide 25: Targeting Hsp90 for Lymphoproliferative Immune Disease and Cancer ADX-1612
Targeting Hsp90 for Lymphoproliferative Immune Disease and Cancer ADX-1612
Slide 26: ADX-1612: Clinically Advanced Asset With Extensive Data
ADX-1612: Clinically Advanced Asset With Extensive Preclinical, Nonclinical, and Clinical Data In-licensed for its potential in immune-mediated disease ADX-1612 clinically-tested in oncology as ganetespib Preclinical efficacy in immune disorders Unregulated proliferation of immune cells Lymphoprolif
Slide 27: ADX-1612: Expanding the Potential Repertoire for Immune-Mediated Diseases
ADX-1612: Expanding The Potential Repertoire for Treatment of Immune-Mediated Diseases Adapted from Tukaj and Wegrzyn Cell Stress and Chaperones 21:213 – 218, 2016. Cytoplasm Nucleus ADX-1612 ADX-1612 Hsp90 Hsp90 Hsp70 NFkB Inflammation HSF1 HSE mRNA hsp70 ADX-1612 Hsp90 inhibitor Hsp90 Upregulated
Slide 28: ADX-1612: Observed Effects on Vasculitis in Phase 1 Patient
ADX-1612: Observed Effects on Vasculitis in a Patient With Leukemia in Phase 1 Clinical Trial Pre-Treatment Post-Treatment Vasculitis: Inflammation of blood vessel walls Fever, headache, fatigue, weight loss, aches and pains, night sweats, rash, ulcers, numbness or weakness Clearing of limb rash aft
Slide 29: ADX-1612: Inhibition of Immune Cell Proliferation in Lupus Model
ADX-1612: Inhibition of Immune Cell Proliferation Observed in an Animal Model of Lupus vehicle 2X/wk 1X/wk Every other wk ADX-1612 Treatment was initiated at 7 weeks of age and continued through 22 weeks of age Dosing: 50 mg/kg, IV twice weekly once weekly every other week Systemic autoimmunity (MRL
Slide 30: ADX-1612: Skin Lesions and Lymphoadenopathy Decreased in Lupus Model
ADX-1612: Skin Lesions and Lymphoadenopathy Decreased in an Animal Model of Lupus Skin Lesion Score at 22 Weeks Lymphoadenopathy Score at 22 Weeks ADX-1612 vehicle 2X/wk 1X/wk every other wk vehicle 2X/wk 1X/wk every other wk ADX-1612 Source: Data on file
Slide 31: Proposed Indication: Post-Transplant Lymphoproliferative Disorder
Proposed Indication: Post-Transplant Lymphoproliferative Disorder (PTLD) Lymphomas occurring after stem cell transplant or organ transplant Most serious complication of transplantation, resulting from immunosuppression Uncontrolled proliferation of lymphocytes Medication-induced reduction in immune
Slide 32: Rationale for Synergism of Hsp90 Inhibitor and Platins
DNA repair complex Rationale for Synergism of Hsp90 Inhibitor and Platins for The Treatment of Cancer Inhibition of Hsp90 prevents repair and inhibits proteins involved in tumor cell survival Platins induce DNA damage… Adapted from Noll et al., Front. Biosci. 9:421-437, 2004 Hsp90 …But damaged DNA c
Slide 33: ADX-1612: A Promising Asset for Oncology
ADX-1612: A Promising Asset for Oncology Investigator-sponsored trials (IST) in cancer ongoing Mesothelioma: ADX-1612 + pemextred (antimetabolite) / platinum (DNA damage inducer) therapy Expected data readout 2H 2018 Ovarian cancer (EUDARIO): ADX-1612 + carboplatin + niraparib (PARP inhibitor) Initi
Slide 34: ADX-1615: Oral Pro-Drug of ADX-1612
ADX-1615: Oral Pro-Drug of ADX-1612 Orally administered Oral administration may be better suited to treatment of chronic immune-mediated disorders May also be useful in oncology setting Has shown activity in mast cell tumors in dogs (monotherapy) Manuscript submitted Next Steps Manufacturing IND-ena
Slide 35: Partnership Update
YALDEYRA THERAPEUTICS™ Partnership Update
Slide 36: Johnson & Johnson Innovation: Collaborative Research Agreement
Partnership Update Johnson & Johnson Innovation: Collaborative research agreement Focus: RASP inhibitors (not including reproxalap) Indications: immune-mediated diseases characterized by systemic inflammation Governed by Joint Scientific Review Committee Limited option to negotiate exclusive license
Slide 37: Research Day 2018 Update on Research Programs
Research Day 2018 Update on Research Programs June 26, 2018 New York NASDAQ: ALDX
Slide 38: 2018 Progress and Near-Term Development Catalysts
2018 Progress and Near-Term Development Catalysts Support Path to Commercialization Initiated reproxalap Phase 2b clinical trial in dry eye disease First patient enrolled in reproxalap Phase 3, Part 1 clinical trial in Sjögren-Larsson Syndrome Q3 2018 Reproxalap dry eye disease Phase 2b clinical tri
Slide 39: Early-Stage Development Expected Milestones
Early-Stage Development Expected Milestones: Novel Approaches to Address Immune-Mediated Disease ADX-1612 mesothelioma clinical trial results (investigator sponsored trial) H2-2018 ADX-1612 ovarian cancer clinical trial initiation (investigator sponsored trial) H2-2018 ADX-629 Phase 1 clinical trial
Slide 40: Deep and Innovative Pipeline
Deep and Innovative Pipeline Not Disclosed Reproxalap Ocular Reproxalap Dermal ADX-629 Systemic ADX-103 ADX-1612 Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis Sjögren-Larsson Syndrome Autoimmune Disease Retinal Disease Lymphoproliferative Immune Disease Phase 2b results H2-2
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