A Novel Pharmaceutical Platform Focused on Trapping Aldehydes September 2014 Exhibit 99.1 |
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,” “expect,” “believe,” “anticipate,” “project,” “target,” “design,” “estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Forward-looking statements include, but are not limited to, statements about: our expectations regarding our expenses and revenue, the sufficiency of our cash resources and needs for additional financing; our anticipated growth strategies; our expectations regarding competition; the anticipated trends and challenges in our business and the market in which we operate; the timing and success of preclinical studies and clinical trials conducted by us and our development partners; the ability to obtain and maintain regulatory approval of our product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing our product candidates; the size and growth of the potential markets for our product candidates and the ability to serve those markets; the rate and degree of market acceptance of any of our product candidates; our ability to establish and maintain development partnerships; our ability to attract or retain key personnel; our expectations regarding federal, state and foreign regulatory requirements; regulatory developments in the United States and foreign countries; and our ability to obtain and maintain intellectual property protection for our product candidates. • Forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. Any forward-looking statement made by us in this presentation speaks only as of the date on which it is made. Except as required by law, we assume no obligation to update these statements publicly, or to update the reasons actual results could differ materially from those anticipated in these statements, even if new information becomes available in the future. • Although we believe that we have a reasonable basis for each forward-looking statement contained in this presentation, we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operation, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this presentation as a result of, among other factors, the factors referenced in our final prospectus filed under Rule 424(b)(4) with the Securities and Exchange Commission on May 2, 2014 and in our subsequent filings with the Securities and Exchange Commission. In addition, even if our results of operation, financial condition and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in this presentation, they may not be predictive of results or developments in future periods. • You should read carefully our filings with the Securities and Exchange Commission, including risk factors described therein, to better understand the risks and uncertainties inherent in our business and underlying any forward-looking statements. 2 |
Management and Directors • Todd Brady, M.D., Ph.D. – President, CEO, and Director – 18 years of pharmaceutical business and clinical development – Domain Associates, Phenome Sciences, (acquired by Xanthus/Antisoma), Aderis Pharmaceuticals (acquired by Schwarz/UCB) • Scott Young – Chief Operating Officer – 28 years of pharmaceutical clinical development – Genzyme, Genetics Institute, Oxigene, Repligen • Steve Tulipano, CPA – Chief Financial Officer – 27 years of financial experience – Biogen, Javelin Pharmaceuticals 3 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 Jesse Treu, Ph.D. – Domain Associates Todd Brady – CEO Aldeyra Therapeutics |
Investment Highlights 4 Unique, Innovative Platform Technology to Trap Aldehydes Orphan and mass-market diseases in which toxic aldehydes are implicated Modest Funding Required for Multiple Clinical Events Lead compound in two topical indications: one dermal and one ocular Phase II/III results for Sjögren Larsson Syndrome (SLS) and Phase II results for acute anterior uveitis in 2015 Large Markets with Significant Unmet Medical Need Markets for orphan indications alone are substantial, and positive data may suggest efficacy in a broad array of mass-market diseases Strong Patent Portfolio of Compositions, Uses, and Formulations Extend to late 2020s worldwide and to 2033 in US, assuming Hatch-Waxman extension Marquee Investors Validate Opportunity Johnson & Johnson Development Corporation, Fidelity, and Domain Associates – one of the oldest and largest healthcare venture capital funds worldwide |
Aldehydes Are Mediators of Disease • Toxic mediators of numerous diseases • Modify cellular constituents, lead to indigestible aggregates, and are pro- inflammatory • Dehydrogenases attempt to eliminate free aldehydes • High levels are implicated in autoimmune, inflammatory, neurological, cardiovascular and endocrinologic diseases 5 Oxidation, Metabolism Free Aldehydes Lipid, DNA, Protein, Carbohydrate Modification Cytokine Release NF-KB Activation |
Aldehyde Traps: A Novel Therapeutic Approach 6 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 Aldeyra’s compounds rapidly trap free aldehydes Trapped aldehydes are transported to the lysosome Drug and aldehydes are metabolized within hours |
Trapping Aldehydes Generates a Broad Anti-Inflammatory Response 7 Mice treated with NS2 or vehicle 30 minutes prior to endotoxin exposure; cytokines measured two hours after endotoxin exposure ** p<0.01 *** p<0.001 ** ** *** ** In an endotoxin model of cytokine generation in mice, NS2 administration significantly reduced levels of a broad array of pro-inflammatory cytokines. |
8 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 Administration Murine Model of Allergic Dermatitis (Oxazolone) 24.5 hours after NS2 Administration Single dose of NS2 has early and potent anti-inflammatory effect that reduces swelling in two different models of skin inflammation 150 50 0 100 150 50 0 100 |
NS2 Speeds Healing and Reduces Scarring of Lesions in Animal Models 9 Vehicle NS2 p=0.01 Day 6 21 36 p=0.1 NS 2 Vehicle None Minimal Mild Marked Moderate Severe Hamster cheek pouch radiation-induced oral mucositis NS2 speeds lesion healing and reduces scarring in a model of skin and eye disease 2 3 1 0 |
10 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 SLS Mutants SLS Mutants + NS2 p<0.01 p<0.01 NS2 prevents aldehyde-mediated damage of lipid that is critical to dermal moisture barrier and ocular tear integrity |
NS2 Traps Aldehydes Generated by Dry Conditions in Human Tissue 11 Malondialdehyde concentration in human tissues after 72 hours of NS2 Dry Tissue + NS2 Eye Drop Normal Tissue Dry Tissue p < 0.01 0 5 10 15 20 25 30 0 2 4 6 8 10 12 Dry Tissue + NS2 Dermatologic Normal Tissue Dry Tissue p < 0.05 Human Ocular Tissue Human Skin 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 Summary of Efficacy: Multiple Mechanisms of Action 12 NS2 Lipid Protection Anti-fibrotic Lesion Healing Anti- inflammatory Decreased Aldehyde Load The same biological mechanisms may apply to many orphan and prevalent diseases. |
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 treatment groups o No plasma exposure detected by LC-MS/MS (<5 ng/ml) 13 NS2 is Phase II-ready as an eye drop |
Acute Anterior Uveitis: A Rare Inflammatory Ocular Disease 14 Uveitis Acute anterior ocular inflammation Pain, photophobia, loss of vision Estimated 25,000 US patients/year Aldehydes are inflammatory mediators of ocular diseases, and can lead to degradation of tear quality |
Anticipated Clinical Trial Designs for Ocular Disease Formulation Control Total Patients Treatment Time Endpoints 15 Acute Anterior Uveitis Eye Drop Active 1:1:1 45 Patients 8 weeks Cell Counts, Symptoms |
Sjögren-Larsson Syndrome (SLS): Orphan Disease with No Therapy 16 (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 patients in Europe. 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 Estimated 0.4 births/100,000 = about 1000 patients in US and a greater number in Europe (1) Therapeutic aldehyde trap would be analogous to an enzyme replacement therapy |
Anticipated Clinical Trial Designs For Dermatologic Disease Formulation Control Total Patients Treatment Time Endpoints 17 Sjögren-Larsson Syndrome Dermal Topical Placebo 1:1 Visual Rating 12 Patients 8 weeks |
Unmet Medical Need for Our Clinical Indications 18 Market demand is substantial for a novel therapy that is safe and effective in the indications that we intend to develop There is no FDA-approved therapy for Sjögren- Larsson Syndrome Therapies for acute anterior uveitis are associated with significant side effects |
Orphan Topical: Attractive Pricing, Large Market 19 Total US SLS market: ~$200M Payer research confirms similar or higher pricing for a topical SLS therapy Estimated >$200,000 per year Twice per day treatment 25% of body surface area treated Orphan Topical Pricing for Cutaneous Lymphoma |
Intellectual Property Portfolio: Composition of Matter into the 2030s 20 *Pending in Brazil, India Formulation Composition Method Topical dosing Uses for aldehyde traps in diseases NS2 to 2033 in US, assuming Hatch- Waxman extension; Issued or allowed worldwide* |
Orphan Disease Company Valuation Comparables 21 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, pending FDA review, among other contingencies. (1) Company Stage Diseases in Phase II or III Clinical Trials Valuation Aldeyra Therapeutics (ALDX) Phase II 2 $23M Bluebird Bio (BLUE) Phase II 2 $1.1B Sarepta Therapeutics (SRPT) Phase II 1 $950M Ultragenyx (RARE) Phase II 2 $1.7B Synageva BioPharma (GEVA) Phase III 1 $2.6B Intercept Pharmaceuticals (ICPT) Phase III 4 $6.4B |
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 selected for Late Breaking and Industry Review sessions • Clinical development timelines remain on track • Team expansion completed thru 2015 22 |
Investment Highlights 23 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 Orphan and mass-market diseases in which toxic aldehydes are implicated Lead compound in two topical indications: one dermal and one ocular Phase II/III results for Sjögren Larsson Syndrome (SLS) and Phase II results for acute anterior uveitis in 2015 Markets for orphan indications alone are substantial, and positive data may suggest efficacy in a broad array of mass-market diseases Extend to late 2020s worldwide and to 2033 in US, assuming Hatch-Waxman extension Johnson & Johnson Development Corporation, Fidelity, and Domain Associates – one of the oldest and largest healthcare venture capital funds worldwide |