A Novel Pharmaceutical Platform Focused on Trapping Aldehydes March 2015 Exhibit 99.1 XXXXX X |
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 plans for its product candidates. 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," "aim," "plan" or the negative of these terms, and similar expressions intended to identify forward-looking statements. • Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Aldeyra is at an early stage of development and may not ever have any products that generate significant revenue. Important factors that could cause actual results to differ materially from those reflected in Aldeyra's forward-looking statements include, among others, the timing and success of preclinical studies and clinical trials conducted by Aldeyra and its development partners; the ability to obtain and maintain regulatory approval to conduct clinical trials and to commercialize Aldeyra's product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Aldeyra's product candidates; the size and growth of the potential markets for Aldeyra's product candidates and the ability to serve those markets; Aldeyra's expectations regarding Aldeyra's expenses and revenue, the sufficiency of Aldeyra's cash resources and needs for additional financing; Aldeyra's ability to attract or retain key personnel; and other factors that are described in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Aldeyra's Quarterly Report on Form 10-Q for the quarter ended September 30, 2014 which is on file with the Securities and Exchange Commission (SEC) and available on the SEC's website at www.sec.gov. • In addition to the risks described above and in Aldeyra's other filings with the SEC, other unknown or unpredictable factors also could affect Aldeyra's results. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information in this presentation is provided only as of the date of this release, and Aldeyra undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law. 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 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) in 2015 Phase II trial initiated for acute anterior uveitis in 2015 For lead compound, IP extends to late 2020s worldwide and to 2033 in US, assuming Hatch-Waxman extension Fidelity, Perceptive, DAFNA, Sphera, Knoll, Johnson & Johnson Development Corporation, Domain Associates, and other top-tier funds Markets for orphan indications alone are substantial, and positive data may suggest efficacy in a broad array of mass-market diseases |
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 |
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. 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 In an endotoxin model of cytokine generation in mice, NS2 administration significantly reduced levels of a broad array of pro-inflammatory cytokines. 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 ** ** *** ** Data presented at the American Academy of Asthma Allergy and Immunology 2015 Annual Meeting |
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 Data presented at the American Academy of Asthma Allergy and Immunology 2015 Annual Meeting |
NS2 Speeds Healing and Reduces Scarring of Lesions in Animal Models 9 NS2 speeds lesion healing and reduces scarring in a model of skin and eye disease 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 |
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 NS2 prevents aldehyde-mediated damage of lipid that is critical to dermal moisture barrier and ocular tear integrity p<0.01 p<0.01 Data to be presented at the Society for Inherited Metabolic Disorders 2015 Annual Meeting, March 28 |
NS2 Traps Aldehydes Generated by Dry Conditions in Human Tissue 11 NS2 may reduce aldehyde-mediated damage in diseases characterized by dry tissue 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 Data presented at the Society for Investigative Dermatology 2014 Annual Meeting |
NS2 Summary of Efficacy: Multiple Mechanisms of Action 12 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 safe and tolerable in healthy volunteers at doses up to four times per day over seven days . |
Noninfectious Anterior Uveitis: A Rare Inflammatory Ocular Disease 14 Aldehydes are inflammatory mediators of ocular diseases, and can lead to degradation of tear quality |
Anticipated Clinical Trial Design for Uveitis Formulation Control Total Patients Treatment Time Endpoints 15 Eye Drop Active 1:1:1 (NS2, Steroid, NS2 + Sub- Therapeutic Steroid) 45 Patients 6 weeks Inflammation Markers, Symptoms Noninfectious Anterior Uveitis |
Sjögren-Larsson Syndrome (SLS): Orphan Disease with No Therapy 16 Therapeutic aldehyde trap would be analogous to an 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 patients in Europe. |
Anticipated Clinical Trial Design For Sjögren-Larsson Syndrome Formulation Control Total Patients Treatment Time Endpoints 17 |
Clinical Trial Updates 18 • Noninfectious Anterior Uveitis – Prior to study initiation, FDA requires protocol amendment, which has been submitted – Pending FDA review of amended protocol, study start and data in 2015, per earlier guidance • Sjögren-Larsson Syndrome – Study initiation pending final investigational review board approval – Study start and data in 2015, per earlier guidance |
Unmet Medical Need for Our Clinical Indications 19 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 20 Total US SLS market: ~$200M Payer research confirms similar or higher pricing for a topical SLS therapy |
Intellectual Property Portfolio: Composition of Matter into the 2030s 21 *Pending in Brazil, India Formulation Composition Method |
Valuation Comparables 22 Orphan disease biotechnology and late-stage specialty pharmaceutical companies are highly valued. Company Stage Diseases in Phase II or III Clinical Trials Valuation Aldeyra Therapeutics (ALDX) Phase II 2 $64M Anacor (ANAC) Phase II/III 4 $1.9B GW Pharmaceuticals (GWPH) Phase II/III 5 $1.6B Ultragenyx (RARE) Phase II 2 $1.7B Insmed (INSM) Phase II/III 3 $921M Intercept (ICPT) Phase III 4 $4.7B Data as of 2/27/15 (1) Pending FDA review of submitted filings, among other contingencies. (1) |
2015 Medical Conferences 23 Conference Date Location Data February 20-24 Houston, TX Cytokine Reduction, Contact Dermatitis, and Allergic Dermatitis March 28-31 Salt Lake, UT SLS Lipid Protection May 3-7 Denver, CO Ocular Inflammation and Ocular Fibrosis Significant data to be presented at upcoming medical conferences which will highlight the safety and efficacy of NS2 in both ocular and dermal indications |
Investment Highlights 24 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) in 2015 Phase II trial initiated 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 For lead compound, IP extends to late 2020s worldwide and to 2033 in US, assuming Hatch-Waxman extension Fidelity, Perceptive, DAFNA, Sphera, Knoll, Johnson & Johnson Development Corporation, Domain Associates, and other top-tier funds |