A Novel Therapy for SSADH: An Aldehyde Trap for an Aldehyde-Mediated Disease March 2016 Exhibit 99.1 |
Disclaimers and Forward-Looking Statements 2 • 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 strategies and plans, research and development plans or expectations, trends, market sizing, competitive position, industry environment and potential growth opportunities, among other things.. Forward-looking statements include all statements that are not historical facts and, in some cases, can be identified by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “anticipate,” “project,” “target,” “design,” “estimate,” “predict,” “potential,” “plan” or similar expressions and the negatives of those terms. • Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Aldeyra’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. 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 of commencement, enrollment and completion of Aldeyra’s clinical trials; 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 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 its expenses and revenue, the sufficiency or use of Aldeyra’s cash resources and needs for additional financing; the rate and degree of market acceptance of any of Aldeyra’s product candidates; Aldeyra’s expectations regarding competition; Aldeyra’s anticipated growth strategies; Aldeyra’s ability to attract or retain key personnel; Aldeyra’s ability to establish and maintain development partnerships; Aldeyra’s expectations regarding federal, state and foreign regulatory requirements; regulatory developments in the United States and foreign countries; Aldeyra’s ability to obtain and maintain intellectual property protection for its product candidates; the anticipated trends and challenges in Aldeyra’s business and the market in which it operates; the use or sufficiency of Aldeyra’s cash or cash equivalents; 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 Annual Report on Form 10-K for the year ended December 31, 2015, 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 March 31, 2016, and Aldeyra undertakes no obligation to update any forward-looking statements contained in this presentation on account of new information, future events, or otherwise, except as required by law. - - |
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 |