Slide 1: Results of a randomized, double-blind, vehicle-controlled clinical trial of topical NS2 in Sjögren-Larsson Syndrome
Results of a randomized, double-blind, vehicle-controlled clinical trial of topical dermatologic NS2 in patients with ichthyosis due to Sjögren-Larsson Syndrome August 9, 2016 Exhibit 99.1
Slide 2: Disclaimers and Forward-Looking Statements
Disclaimers and Forward-Looking Statements This presentation contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, including statements regarding Aldeyra’s possible or assumed future results of operations and expenses, business stra
Slide 3: Sjögren-Larsson Syndrome (SLS): A Rare Disease with No Approved Therapy
Sjögren-Larsson Syndrome (SLS): A Rare Disease with No Approved Therapy An aldehyde trap could be analogous to enzyme replacement therapy *Extrapolating from a Swedish estimate in addition to a US genetic mutation analysis, it is generally assumed that there are approximately 1,000 SLS patients in t
Slide 4: Sjögren-Larsson Syndrome Clinical Trial Design
Sjögren-Larsson Syndrome Clinical Trial Design Dosing NS2 1% Topical Dermatologic Once Daily on 4x10 inch area Randomization Vehicle-Controlled 1:1 Enrollment 12 Patients with Moderate to Severe Ichthyosis Treatment Time 8 Weeks Endpoints Ichthyosis Severity Score (Investigator Exam and Masked Centr
Slide 5: Sjögren-Larsson Syndrome Ichthyosis Assessment Score
Sjögren-Larsson Syndrome Ichthyosis Assessment Score Definition 0 Clear 1 Almost Clear 2 Mild 3 Moderate 4 Severe Each measure rated separately as follows: Composite Ichthyosis Severity Score is the sum of all components (range 0-20) Ichthyosis Severity Score Global Scaling Lichenification (thickeni
Slide 6: Baseline Characteristics of Clinical Trial Subjects
NS2 (n=6) Vehicle (n=6) Age (years) ± SD 14 ± 5 17 ± 5 Gender (Male, Female) 3, 3 3, 3 Race (White, Black, Latino) 3,2,1 5,1,0 Body Surface Area (m2) ± SD 1.54 ± 0.5 1.95 ± 0.9 Baseline Exam Ichthyosis Global Assessment ± SD 3.0 ± 0 3.2 ± 0.4 Baseline Characteristics of Clinical Trial Subjects
Slide 7: NS2 Demonstrated Clinical Activity in Patients with Sjögren-Larsson Syndrome
Digital Photography Central Review NS2 Vehicle Unadjusted Chi Square p value♯ Subjects with composite Ichthyosis Severity Score of ≤ 5 at Week 8 (average rating of “almost clear”) 5/6 (83%) 1/5 (20%)* 0.04 Subjects with ≥2 point reduction from baseline in composite Ichthyosis Severity Score at Week
Slide 8: NS2 Reduced Central Reader Digital Photography Assessment
NS2 Reduced Ichthyosis Severity Score Central Reader Digital Photography Assessment Reduction from Baseline in Composite Ichthyosis Severity Score ± SEM at 8 Weeks p<0.05* *p values are subject to change based on quality control analysis
Slide 9: NS2 Reduced Central Reader Digital Photography Assessment by Component
NS2 Reduced Ichthyosis Severity Score Central Reader Digital Photography Assessment Reduction from Baseline ± SEM at 8 Weeks Global Scaling Erythema Lichenification Excoriation
Slide 10: Every NS2-Treated Subject Decreased in Ichthyosis Severity
Every NS2-Treated Subject Decreased in Ichthyosis Severity Central Reader Digital Photography Assessment of NS2-Treated Subjects Composite Ichthyosis Severity Score at 8 Weeks p<0.001* *p values are subject to change based on quality control analysis
Slide 11: NS2 Observed to be Well Tolerated
No significant adverse events, serious adverse events, or discontinuations in either group Itching was reported in two subjects (33%) in the NS2 treated group Moderate itching in one subject classified as “possibly related to drug” Mild itching in one subject classified as “unlikely related to drug”
Slide 12: Elevations in Skin Cholesterol Were Reduced with NS2 Treatment
Elevations in Skin Cholesterol Were Reduced with NS2 Treatment Percentage Change from Baseline at 8 Weeks *p values are subject to change based on quality control analysis
Slide 13: Skin Biomarkers in NS2-Treated Subjects Changed from Baseline
Skin Biomarkers in NS2-Treated (But Not Vehicle-Treated) Subjects Changed from Baseline NS2 Subjects at Week 8 NS2 Subjects at Baseline Vehicle Subjects at Baseline Vehicle Subjects at week 8 p<0.004* *p values are subject to change based on quality control analysis
Slide 14: Summary: NS2 Demonstrated Clinical Activity in Patients with Sjögren-Larsson Syndrome
In summary, this highly controlled clinical trial demonstrated: Clinically meaningful and statistically significant changes with NS2 relative to vehicle on the Ichthyosis Severity Score, as evidenced by moderate disease at baseline reduced to mild disease after 8 weeks of treatment with NS2; Clinica