 A Novel Therapy for SSADH:
An Aldehyde Trap for an Aldehyde-Mediated Disease
March 2016 Exhibit 99.1 |
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 Aldehydes Are Toxic Destruction of fats, proteins, sugars Formation of toxic metabolites such as -hydroxybutyric acid (GHB) Inflammation Aldehydes |
 Symptoms of SSADH Deficiency
Believed to be Due to Aldehydes
Genetic Mutations in SSADH Autism, Cognitive Delay, Decreased Muscle Tone, Seizures Destruction of fats, proteins, sugars Formation of GHB, D-2- hydroxyglutarate (D-2-HG) Inflammation SSA |
 An
Aldehyde Trap Could Diminish the Symptoms of SSADH
Deficiency Genetic
Mutations in SSADH |
 Aldehyde Traps: A Novel Therapeutic Approach 6 Aldeyra’s lead aldehyde trap, NS2, appears to have minimal pharmacology, with no known
effects on receptors, enzymes and other proteins. In clinical trials to
date, NS2 has been observed to be safe and well-tolerated.
Aldeyra is not aware of any similar technology. Aldeyra’s
lead aldehyde trap, NS2, appears to have minimal pharmacology, with no known effects on receptors, enzymes and other proteins. In clinical trials to date, NS2 has been
observed to be safe and well-tolerated. Aldeyra is not aware of any similar
technology. •
Aldeyra’s compounds rapidly trap
free aldehydes
• Trapped aldehydes are transported to the lysosome • Drug-aldehyde adducts are metabolized within hours Aldehyde Binding Adduct Transport Cellular Disposal |
 An
Aldehyde Trap is Proven to Reduce Inflammation in Humans
Ocular Itching
Ocular Tearing
Vehicle NS2 Visit 4 Minutes Post-Challenge 7 * * * * * * * *p<0.05; p values are subject to change based on quality control analysis Phase IIa Allergic Conjunctivitis Clinical Trial (February, 2016) -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0 50 100 150 200 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 50 100 150 200 0 |
 Aldeyra
is Clinically Testing Aldehyde Traps in Several Diseases
Discovery Formulation
Pre-Clinical Phase I Phase II Phase III Allergic Conjunctivitis (NS2 Ocular) Noninfectious Anterior Uveitis (NS2 Ocular) Sjögren-Larsson Syndrome (NS2 Dermatologic) Sjögren-Larsson Syndrome (NS2 Systemic) SSADH Deficiency (NS2 Systemic) Autoimmune Crisis (NS2 Systemic) Various (NSX Series & Other) 8 |
 Our
Lead Candidate, NS2, Traps the Toxic Aldehyde in SSADH
Deficiency By trapping SSA, NS2 may prevent the formation of
pathologic metabolites that are thought to be responsible for the
severe neurological complications of SSADH Deficiency. By
trapping SSA, NS2 may prevent the formation of pathologic metabolites that are thought to be responsible for the severe neurological complications of SSADH Deficiency.
Data presented at the 2015 American Society of Human Genetics Annual
Meeting Ainslie, Vogel, Cullen, Young, Brady and Gibson, 2015
Washington State University and Aldeyra
Therapeutics NS2 administered as a single dose to SSADH knock-out mice |
 NS2 May Reduce GHB and D2HG
Levels in SSADH KO Mice
Ainslie, Vogel, Cullen, Young, Brady and Gibson, 2015
Washington State University and Aldeyra
Therapeutics t test (**p<0.01) |
 Next
Steps in the Development of NS2 for SSADH Deficiency
1. Generate additional preclinical data supporting the use of NS2 in SSADH
patients •
Identify endpoints to assess clinical efficacy of NS2
2. Natural History Study • Understand levels of metabolites and clinical endpoints in patients • Use data to confirm endpoints for Phase II study in SSADH 3. Healthy Volunteer Safety Study • Confirm safety and pharmacological activity of systemic NS2 in adults 4. Phase II study in SSADH patients • Metabolites and clinical endpoints • Progress to longer duration dosing evaluating disease modification |
 Hope
for the Future 12
“In 2016, we expect to build on our accomplishments from last year by initiating clinical development of aldehyde traps that are administered systemically. We are excited to continue to advance NS2 and other aldehyde traps as a novel therapeutic approach for inflammatory disease and inborn errors of aldehyde metabolism.” Aldeyra Therapeutics Reports Full Year 2015 Financial Results Press Release, March 30, 2016 |