Aldeyra Therapeutics, Inc. · NASDAQ:ALDX
Aldeyra Therapeutics — Investor Presentation (March 2016)
31 slides from Aldeyra Therapeutics's investor presentation, Mar 9, 2016
Slide 1: A Novel Pharmaceutical Platform Focused on Inflammation and Inborn Errors of Aldehyde Metabolism
A Novel Pharmaceutical Platform Focused on Inflammation and Inborn Errors of Aldehyde Metabolism March 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: Corporate Highlights
Corporate Highlights Clinical Proof of Concept in Inflammation Achieved for Lead Candidate Two Additional Clinical Events Expected in 2016 Aldehydes are toxic and pro-inflammatory, and are implicated in inflammatory disease and inborn errors of aldehyde metabolism Innovative Small Molecule Platform
Slide 4: Management and Directors
Management and Directors Todd Brady, M.D., Ph.D. – President, CEO, & Director 20 years of pharmaceutical business and clinical development Domain Associates, Phenome Sciences, (acquired by Xanthus/Antisoma), Aderis Pharmaceuticals (acquired by Schwarz/UCB) Steve Tulipano, CPA – Chief Financial Offic
Slide 5: The Aldehyde Trap Platform
OY aLDEYRA THERAPEUTICS” [ The Aldehyde Trap Platform
Slide 6: Aldehydes Are Mediators of Disease
Aldehydes Are Mediators of Disease Toxic mediators of numerous diseases Modify cellular constituents, lead to indigestible aggregates, and are pro-inflammatory High levels are implicated in inflammatory, neurological, cardiovascular and endocrinologic diseases Enzymes called aldehyde dehydrogenases
Slide 7: Aldehyde Traps: A Novel Therapeutic Approach
Aldehyde Traps: A Novel Therapeutic Approach Aldeyra’s lead aldehyde trap, NS2, appears to have minimal pharmacology, with no known effects on receptors, enzymes and other proteins. Aldeyra is not aware of any similar technology. Aldehyde Binding Aldeyra’s compounds rapidly trap free aldehydes Adduc
Slide 8: Overview of Clinical Programs
Overview of Clinical Programs
Slide 9: Platform and Pipeline
Platform and Pipeline 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 (
Slide 10: Allergic Conjunctivitis: A Common Unmet Medical Need
Allergic Conjunctivitis: A Common Unmet Medical Need Aldeyra’s lead aldehyde trap mitigated the ocular allergic response at levels that are statistically and clinically significant in a Phase IIa clinical trial Q1 2016. Allergic conjunctivitis affects 20% or more of worldwide population Symptoms inc
Slide 11: Noninfectious Anterior Uveitis: Inflammation Proof of Concept
Noninfectious Anterior Uveitis: Inflammation Proof of Concept Aldehydes are inflammatory mediators of ocular diseases, and may also mediate pain. Uveitis Acute anterior ocular inflammation Pain, photophobia, loss of vision Estimated 25,000 US patients/year Currently treated with steroids
Slide 12: Sjögren-Larsson Syndrome (SLS): A Rare Disease with No Therapy
Sjögren-Larsson Syndrome (SLS): A Rare Disease with No Therapy An aldehyde trap could be analogous to enzyme replacement therapy (1) Extrapolating from a Swedish estimate, it is generally assumed that there are approximately 1,000 SLS patients in the United States and a greater number of SLS patient
Slide 13: Unmet Medical Need for Our Current Clinical Indications
Unmet Medical Need for Our Current Clinical Indications We believe that there is significant market demand for a novel therapy that is safe and effective in the indications that we intend to develop. For allergic conjunctivitis, long-term activity of anti-histamines and adrenergic agonists is variab
Slide 14: Inflammatory Disease: Allergic Conjunctivitis, Noninfectious Anterior Uveitis
Inflammatory Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis
Slide 15: Phase IIa Clinical Trial Design for Allergic Conjunctivitis
Phase IIa Clinical Trial Design for Allergic Conjunctivitis Dosing Control Enrollment Treatment Time Endpoints Further information can be found on www.clinicaltrials.gov: Trial #NCT02578914. 4 Challenge Visits 5 6 7 1 Day 14 15 16 Allergic Conjunctivitis NS2 Topical Ocular QID Vehicle 1:1, Double-Ma
Slide 16: Clinically Meaningful Effects Observed in Allergic Conjunctivitis Phase IIa
Clinically Meaningful Effects Observed in Allergic Conjunctivitis Phase IIa NS2 achieved reductions in ocular itching and tearing that were clinically relevant and statistically greater than vehicle. Change in Score from Baseline Ocular Itching Ocular Tearing Vehicle NS2 Visit 4 Minutes Post-Challen
Slide 17: Clinically Meaningful Effects Observed in Allergic Conjunctivitis Phase IIa
Clinically Meaningful Effects Observed in Allergic Conjunctivitis Phase IIa NS2 achieved reductions in ocular itching and tearing that were clinically relevant and statistically greater than vehicle. Change in Score from Baseline Ocular Itching Ocular Tearing Vehicle NS2 *p<0.05; p values are subjec
Slide 18: Sustained Clinical Effects Observed in Allergic Conjunctivitis Phase IIa
Sustained Clinical Effects Observed in Allergic Conjunctivitis Phase IIa NS2 achieved reductions in ocular itching and tearing that were sustained across visits and consistently greater than vehicle. Change in Score (AUC) from Baseline Ocular Itching Ocular Tearing Vehicle NS2 Of the 76 assessments
Slide 19: Phase IIa Allergic Conjunctivitis Clinical Summary
Phase IIa Allergic Conjunctivitis Clinical Summary The current Phase II in noninfectious anterior uveitis is testing the same concentration of topical ocular NS2 at the same dosing frequency. Topical ocular NS2 generally well-tolerated with no safety issues Two drop-outs (4%) in the NS2 treatment ar
Slide 20: Phase II Clinical Trial Design for Uveitis
Phase II Clinical Trial Design for Uveitis Dosing Control Enrollment Treatment Time Endpoints Further information can be found on www.clinicaltrials.gov: Trial #NCT02406209. Clinical data expected to be released in Q2 2016. Noninfectious Anterior Uveitis NS2 Topical Ocular QID Active 1:1:1, Double-M
Slide 21: Trapping Aldehydes Generates a Broad Anti-Inflammatory Response
Trapping Aldehydes Generates a Broad Anti-Inflammatory Response In a murine model of cytokine storm, NS2 administration significantly reduced levels of pro-inflammatory cytokines while up-regulating an anti-inflammatory cytokine. Mice treated with NS2 or vehicle prior to endotoxin exposure; cytokine
Slide 22: NS2 Decreases Dermal Inflammation in Animal Models
NS2 Decreases Dermal Inflammation in Animal Models Mouse Ear Thickness (% Change) ** Vehicle NS2 Vehicle NS2 * *p<0.05 **p<0.01 Murine Model of Contact Dermatitis (PMA) 6.5 hours after NS2 Administration Murine Model of Allergic Dermatitis (Oxazolone) 24.5 hours after NS2 Administration A single dos
Slide 23: Topical NS2 Decreases Ocular Inflammation in an Animal Model
Topical NS2 Decreases Ocular Inflammation in an Animal Model pg/mg tissue * *p<0.05 ****p<0.00001 Rat LPS-Induced Ocular Inflammation at 24 Hours Post-Stimulus, NS2 dosed topically QID Topically administered NS2 has clinically significant effects in a widely used animal model of ocular inflammation
Slide 24: Inborn Errors of Aldehyde Metabolism
Inborn Errors of Aldehyde Metabolism Sjögren-Larsson Syndrome Succinic Semi-aldehyde Dehydrogenase Deficiency
Slide 25: NS2 Protects a Key Lipid Target in Sjögren-Larsson Syndrome
NS2 Protects a Key Lipid Target in Sjögren-Larsson Syndrome Aldehyde-Damaged Lipid Control Aldehyde NS2+Aldehyde Human Skin Cells Aldehyde-Damaged Lipid Normal Cells SLS Mutants SLS Mutants + NS2 NS2 prevents aldehyde-mediated damage of a lipid that is critical to dermal moisture barrier pathology i
Slide 26: Phase II Clinical Trial Design For Sjögren-Larsson Syndrome
Phase II Clinical Trial Design For Sjögren-Larsson Syndrome Dosing Control Enrollment Treatment Time Endpoint Further information can be found on www.clinicaltrials.gov: Trial #NCT02402309. Enrollment expected to be completed in March 2016. Sjögren -Larsson Syndrome NS2 Topical Dermal QD Vehicle 1:1
Slide 27: Succinic Semi-Aldehyde Dehydrogenase Deficiency
Succinic Semi-Aldehyde Dehydrogenase Deficiency An aldehyde trap could be analogous to enzyme replacement therapy Rare disease caused by mutation in succinic semi-aldehyde dehydrogenase, leading to high levels of toxic aldehyde Symptoms include seizures, cognitive delay, hypotonia , ataxia Third par
Slide 28: NS2 Traps the Toxic Aldehyde in SSADH Deficiency
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. NS2 administered as a single dose to SSADH knock-out mice. Brain Liver Kidney 0.
Slide 29: IP and Investment Thesis
OY aLDEYRA THERAPEUTICS” IP and Investment Thesis
Slide 30: Intellectual Property Portfolio: Composition of Matter into the 2030s
Intellectual Property Portfolio: Composition of Matter into the 2030s *Pending in Brazil Formulation Composition Method NS2 to 2033 in US, assuming Hatch-Waxman extension; Issued or allowed worldwide* Uses for aldehyde traps in diseases Topical dosing
Slide 31: Corporate Highlights
Corporate Highlights Clinical Proof of Concept in Inflammation Achieved for Lead Candidate Two Additional Clinical Events Expected in 2016 Aldehydes are toxic and pro-inflammatory, and are implicated in inflammatory disease and inborn errors of aldehyde metabolism Innovative Small Molecule Platform
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