Aldeyra Therapeutics, Inc. · NASDAQ:ALDX

Aldeyra Therapeutics — Technical Presentation (April 2018)

16 slides from Aldeyra Therapeutics's technical presentation, Apr 30, 2018

  1. Slide 1: Phase 2a Dry Eye Disease Clinical Trial of Reproxalap (ADX-102)

    A Randomized, Double-Masked, Parallel-Group, Phase 2a Dry Eye Disease Clinical Trial to Evaluate the Safety and Efficacy of Topical Ocular ADX-102 (Reproxalap), a Novel Aldehyde Sequestering Agent David Clark, MD Chief Medical Officer Aldeyra Therapeutics, Lexington, MA ARVO: April 30th, 2018

  2. Slide 2: Aldehydes Are Mediators of Inflammation

    Aldehydes Are Mediators of Inflammation Thiol/Amine • Aldehydes covalently bind thiol Adduct (Michael addition) and amine Formation (Schiff base) residues on proteins • Direct protein binding leads to conformational changes in Scavenger proteins, resulting in Receptor/kinase, Receptor A dysfunctiona

  3. Slide 3: The Scientific Literature Supports the Toxicity of Pro-Inflammatory Reactive Aldehyde Species (RASP)

    The Scientific Literature Supports the Toxicity of Pro-Inflammatory Reactive Aldehyde Species (RASP) Cardiovasc Res. 2010 Nov 1;88(2):352-9. HNE-induced 5-LO expression is regulated by NF-kB/ERK and Sp1/p38 MAPK pathways via EGF receptor in murine macrophages. Biofactors. 2005;24(1-4):229-36. Role o

  4. 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

  5. Slide 5: RASP Scavengers Represent A Novel Therapeutic Approach

    RASP Scavengers Represent A Novel Therapeutic Approach RASP • Covalent reaction between drug and RASP that is Binding essentially irreversible in vivo Adduct • Scavenged RASP are transported inside the cell Transport Cellular • Drug-RASP adducts are Degradation metabolized within hours 5

  6. Slide 6: Dry Eye Disease Phase 2a Clinical Design

    Dry Eye Disease Phase 2a Clinical Design Design Randomized, Double-Masked, Clinical Trial Topical Ocular Reproxalap (ADX-102) Formulations: • Current Formulation 0.5% Dosing • Current Formulation 0.1% • Novel Lipid Formulation 0.5% Enrollment 51 Dry Eye Patients with Active Disease Treatment Time 4

  7. Slide 7: Inclusion Criteria: Active Disease

    Inclusion Criteria: Active Disease • History of dry eye for at least 6 months • History of use or desire to use eye drops for dry eye symptoms within 6 months • Score of ≥ 2 on the Ora Calibra® Ocular Discomfort & 4- symptom questionnaire in at least one symptom • Schirmer’s Test score of ≤ 10 mm an

  8. Slide 8: Reproxalap Improved Numerous Dry Eye Disease Signs and Symptoms in Phase 2a

    Reproxalap Improved Numerous Dry Eye Disease Signs and Symptoms in Phase 2a Clinical Trial Endpoint (Pooled Data) Pre-Treatment Post-Treatment (Day 28) p value* Symptom Assessment in Dry Eye (SANDE) Score 61 52 p = 0.003 Ocular Discomfort Score 2.3 1.5 p = 0.00002 Overall 4-Symptom Score 2.6 2.0 p =

  9. Slide 9: Improvement Effect Sizes Were Robust and Statistically Significant

    Improvement Effect Sizes Were Robust and Statistically Significant in Phase 2a Clinical Trial Effect size = Mean difference from Day 0 to Day 28 / Standard Deviation of Day 0. 9

  10. Slide 10: A Dose Response Was Observed in Phase 2a Clinical Trial

    A Dose Response Was Observed in Phase 2a Dry Eye Disease Clinical Trial Effect size = Mean difference from Day 0 to Day 28 / Standard Deviation of Day 0. 10

  11. Slide 11: Drug Activity Supported by Biomarker Reduction and Increasing Efficacy

    Drug Activity in Phase 2a Clinical Trial Supported by Biomarker Reduction and Increasing Efficacy over Time Pre-Treatment = Day 0, Post-Treatment = Day 28. 11

  12. Slide 12: Aldehyde Reduction Correlated with Sign Improvement Within Individual Patients

    Aldehyde Reduction Correlated with Sign Improvement Within Individual Patients Pooled data from Phase 2a clinical trial. 12

  13. Slide 13: Aldehyde Reduction Correlated with Sign Improvement Within Individual Patients

    Aldehyde Reduction Correlated with Sign Improvement Within Individual Patients Pooled data from Phase 2a clinical trial. 13

  14. Slide 14: Phase 2a Clinical Trial Safety Summary

    Phase 2a Clinical Trial Safety Summary • No observed safety concerns • Transient stinging consistent with other eye drops and prior reproxalap clinical experience • Tolerability of 0.1% reproxalap consistent with standard of care in the dry eye disease population 14

  15. Slide 15: Conclusions

    Conclusions • Reproxalap demonstrated statistically significant and clinically relevant improvement in a broad range of dry eye disease signs and symptoms: • Rapid onset of activity within one week of dosing • Improvement increased over time, and a modest dose-response was observed • Reproxalap acti

  16. Slide 16: Dry Eye Disease Phase 2b Clinical Design

    Dry Eye Disease Phase 2b Clinical Design Initiated Q1 2018; Results expected H2 2018 • 0.1% Reproxalap Groups • 0.25% Reproxalap • Vehicle Randomization Double-Masked 1:1:1 Treatment 12 Weeks, Topical Ocular Enrollment 300 Patients with Dry Eye Disease Endpoints Standard Dry Eye Disease Signs and Sy

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