Aldeyra Therapeutics, Inc. · NASDAQ:ALDX
Aldeyra Therapeutics — Investor Presentation (September 2014)
23 slides from Aldeyra Therapeutics's investor presentation, Sep 5, 2014
Slide 1: A Novel Pharmaceutical Platform Focused on Trapping Aldehydes
A Novel Pharmaceutical Platform Focused on Trapping Aldehydes September 2014 Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements • This presentation includes statements contains forward-looking statements that involve risks and uncertainties. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “
Slide 3: Management and Directors
Board of Directors Boyd Clarke – former CEO Aviron (acquired by MedImmune) Gary Phillips, M.D. – Chief Strategy Officer Mallinckrodt Pharmaceuticals Ben Bronstein, M.D. – former CEO Peptimmune (acquired by Genzyme) Neal Walker, D.O. – CEO Aclaris Therapeutics Marty Joyce – former CFO of Serono USA J
Slide 4: Investment Highlights
Unique, Innovative Platform Technology to Trap Aldehydes Modest Funding Required for Multiple Clinical Events Large Markets with Significant Unmet Medical Need Strong Patent Portfolio of Compositions, Uses, and Formulations Marquee Investors Validate Opportunity Investment Highlights Orphan and mass
Slide 5: Aldehydes Are Mediators of Disease
Oxidation, Metabolism Free Aldehydes Lipid, DNA, Protein, Carbohydrate Modification Cytokine Release NF-KB Activation Aldehydes Are Mediators of Disease • Toxic mediators of numerous diseases • Modify cellular constituents, lead to indigestible aggregates, and are pro- inflammatory • Dehydrogenases
Slide 6: Aldehyde Traps: A Novel Therapeutic Approach
Aldeyra’s lead aldehyde trap, NS2, appears to have minimal pharmacology; it does not seem to affect receptors or proteins. No similar technology believed to be available. Adduct Transport Aldehyde Binding Cellular Disposal Aldehyde Traps: A Novel Therapeutic Approach Aldeyra’s compounds rapidly trap
Slide 7: Trapping Aldehydes Generates a Broad Anti-Inflammatory Response
In an endotoxin model of cytokine generation in mice, NS2 administration significantly reduced levels of a broad array of pro-inflammatory cytokines. Trapping Aldehydes Generates a Broad Anti-Inflammatory Response Mice treated with NS2 or vehicle 30 minutes prior to endotoxin exposure; cytokines mea
Slide 8: NS2 Decreases Dermal Inflammation in Animal Models
Single dose of NS2 has early and potent anti-inflammatory effect that reduces swelling in two different models of skin inflammation NS2 Decreases Dermal Inflammation in Animal Models ** Vehicle NS2 Vehicle NS2 * * p<0.05 ** p<0.01 Murine Model of Contact Dermatitis (PMA) 6.5 hours after NS2 Administ
Slide 9: NS2 Speeds Healing and Reduces Scarring of Lesions in Animal Models
NS2 speeds lesion healing and reduces scarring in a model of skin and eye disease NS2 Speeds Healing and Reduces Scarring of Lesions in Animal Models Vehicle NS2 p=0.01 Day 21 36 p=0.1 NS Vehicle None Minimal Mild Marked Moderate Severe Hamster cheek pouch radiation-induced oral mucositis
Slide 10: NS2 Protects a Key Lipid Relevant to Skin and Eye Disease
NS2 prevents aldehyde-mediated damage of lipid that is critical to dermal moisture barrier and ocular tear integrity NS2 Protects a Key Lipid Relevant to Skin and Eye Disease in Cell Systems Aldehyde- Damaged Lipid Control Aldehyde NS2+Aldehyde Human Skin Cells Aldehyde- Damaged Lipid Normal Cells S
Slide 11: NS2 Traps Aldehydes Generated by Dry Conditions in Human Tissue
Potential to reduce aldehyde-mediated damage in diseases characterized by dry tissue (Including Sjögren-Larsson Syndrome and Ocular Rosacea with Meibomian Gland Dysfunction) NS2 Traps Aldehydes Generated by Dry Conditions in Human Tissue Malondialdehyde concentration in human tissues after 72 hours
Slide 12: NS2 Summary of Efficacy: Multiple Mechanisms of Action
NS2 Lipid Protection Anti-fibrotic Lesion Healing Anti- inflammatory Decreased Aldehyde Load The same biological mechanisms may apply to many orphan and prevalent diseases. NS2 Summary of Efficacy: Multiple Mechanisms of Action
Slide 13: NS2 is Phase II-Ready as an Eye Drop
NS2 is Phase II-ready as an eye drop Positive NS2 Eye Drop Phase I Results o 48 healthy volunteers o Double-blinded and placebo controlled o Two treatment stages for two drug concentrations: - Single day 0.25% & 0.5% bid & qid - Seven day 0.25% & 0.5% qid o Eye drops were well tolerated in all treat
Slide 14: Acute Anterior Uveitis: A Rare Inflammatory Ocular Disease
Uveitis Aldehydes are inflammatory mediators of ocular diseases, and can lead to degradation of tear quality Acute Anterior Uveitis: A Rare Inflammatory Ocular Disease Acute anterior ocular inflammation Pain, photophobia, loss of vision Estimated 25,000 US patients/year
Slide 15: Anticipated Clinical Trial Designs for Ocular Disease
Eye Drop Active 1:1:1 45 Patients 8 weeks Cell Counts, Symptoms Anticipated Clinical Trial Designs for Ocular Disease Formulation Control Total Patients Treatment Time Endpoints Acute Anterior Uveitis
Slide 16: Sjögren-Larsson Syndrome (SLS): Orphan Disease with No Therapy
Orphan disease caused by mutation in Fatty Aldehyde Dehydrogenase, leading to high levels of toxic aldehydes Symptoms include severe skin thickening (ichthyosis), retinal disease, and neurological disorders Diagnosed at birth, but no approved therapy that addresses disease; patients survive into 50s
Slide 17: Anticipated Clinical Trial Designs for Dermatologic Disease
Dermal Topical Placebo 1:1 Visual Rating 12 Patients 8 weeks Anticipated Clinical Trial Designs For Dermatologic Disease Formulation Control Total Patients Treatment Time Endpoints Sjögren-Larsson Syndrome
Slide 18: Unmet Medical Need for Our Clinical Indications
Market demand is substantial for a novel therapy that is safe and effective in the indications that we intend to develop Unmet Medical Need for Our Clinical Indications There is no FDA-approved therapy for Sjögren- Larsson Syndrome Therapies for acute anterior uveitis are associated with significant
Slide 19: Orphan Topical: Attractive Pricing, Large Market
Total US SLS market: ~$200M Estimated >$200,000 per year Twice per day treatment 25% of body surface area treated Orphan Topical Pricing for Cutaneous Lymphoma Orphan Topical: Attractive Pricing, Large Market Payer research confirms similar or higher pricing for a topical SLS therapy
Slide 20: Intellectual Property Portfolio: Composition of Matter into the 2030s
Topical dosing Uses for aldehyde traps in diseases NS2 to 2033 in US, assuming Hatch- Waxman extension; Issued or allowed worldwide* Intellectual Property Portfolio: Composition of Matter into the 2030s *Pending in Brazil, India Formulation Composition Method
Slide 21: Valuation Comparables
Company Stage Diseases in Phase II or III Clinical Trials Valuation Orphan Disease Company Valuation Comparables Orphan disease-focused biotechnology companies are highly valued, but Aldeyra has potential to expand to prevalent diseases as well. Data as of 8/26/14 (1) INDs anticipated by end of 2014
Slide 22: Post-IPO Updates
Post-IPO Updates • Closed IPO on May 7, 2014 raising gross proceeds of $12M • Proceeds from IPO to be used to complete two clinical trials and expected to provide working capital through 2015 • 2014 Society for Investigational Dermatology poster on novel treatment for dry skin and eye diseases selec
Slide 23: Investment Highlights
Marquee Investors Validate Opportunity Strong Patent Portfolio of Compositions, Uses, and Formulations Large Markets with Significant Unmet Medical Need Modest Funding Required for Multiple Clinical Events Unique, Innovative Platform Technology to Trap Aldehydes Investment Highlights Orphan and mass
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