Scientific Discoveries Allow Development of Targeted Therapeutics for Individuals with Autism Spectrum Disorders

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1 Scientific Discoveries Allow Development of Targeted Therapeutics for Individuals with Autism Spectrum Disorders Randall L. Carpenter, M.D. Co-Founder, President and CEO August 9, 2012 Research Supported by: NIMH, NICHD, NINDS Best Pharmaceuticals for Children Act FRAXA & Autism Speaks

2 Outline Our approach Challenges in translating scientific discoveries into novel therapeutics Our case history Lessons learned

3 Vision and Strategic Focus Translate Scientific Discoveries into Therapeutics that Improve the Lives of Individuals with Autism Understand Causes of Autism Single gene disorders pathogenesis druggable targets Prioritize Disease-modifying Therapeutics Small, Smart POC Trials in Single Gene Disorders Bio-markers/signatures Personalized Medicine Establish Broad Strategic Collaborations

4 Fragile X Syndrome Most common known genetic cause of autism and intellectual disability Results from mutation in a single gene that prevents expression of a single protein FMRP Function of FMRP conserved from fly fish mouse

5 Seaside s Approach to Translational Medicine in FXS 5

6 The Promise of Molecular Medicine Human with Autism Phase 2/3 Trials (2012) STX209 Novel therapeutics (2007) Gene discovery Target ID and drug development Disease models Disease pathophysiology Basic neurobiology 6

7 STX209 Study in Individuals with ASD Study will complete this month, 150 subjects Double-blind, placebo-controlled, parallel group 12 weeks treatment duration Age 5-21 years Biomarker investigations Study results by end of year 7

8

9 Costs to Discover and Develop a New Drug Basic Research $19mm, 5.5 years $281mm Traditional Philanthropy Government & Universities Pharma / Biotech NIH Financing Gap Foundations Venture Philanthropy Angels Venture Capital Private Equity Public Markets Paul SM, Nature Rev Drug Discov,

10

11 11

12 Phase 2/3 Clinical Trials Ongoing in FXS and Autism Glutamate (and GABA) receptors at excitatory synapses Presynaptic axon terminal Postsynaptic dendritic spine GABA R B STX STX107 - mglur5

13 Pharmacologic Rescue with mglur5 Inhibitors Efficacy in multiple animal models of fragile X suggests evolutionary conservation As of April 1, distinct phenotypes - 31 papers 13

14 14

15 Fragile X phenotype: Corrected by mglur5 inhibition? Corrected by STX209? Excess protein synthesis Excess spine density Excess AMPAR turnover Excess excitability (AGS) Excess marble burying Collaborator Peter Vanderklish Scripps Peter Kind Edinburgh Steve Warren Emory Richard Paylor Baylor Richard Paylor Baylor 15 15

16 Diverse genetic mutations converge on brain signaling pathways that regulate synaptic function Auerbach, Osterweil, Bear. Nature

17 Mutations Causing Syndromic Autism Define an Axis of Synaptic Pathophysiology Auerbach, Osterweil, Bear. Nature

18 Translational Medicine / Research Requires deep expertise and coordinated efforts in multiple disciplines Large Pharma / Biotech Collaborations between Academics-Industry- NIH-Foundations-Venture Philanthropy

19 Seaside s Collaborative Business Model Foundations FRAXA, Autism Speaks/NAAR/CAN, NFXF Merck & Co, Inc. Merck-Seaside mglur5 NAM collaboration NIH UO1 Translational Research Cooperative Agreement NIMH, NICHD/BPCA, NINDS, FRAXA, Autism Speaks Baylor Medical Center Richard Paylor, PhD Behavioral Research Collaboration (plus Autism Speaks and NIH) Vanderbilt University P. Jeff Conn, PhD Vanderbilt Institute of Chemical Biology Program in Drug Discovery Academic Scientists and Clinicians Family Members and other Concerned Individuals F. Hoffman-La Roche Ltd Roche-Seaside Alliance

20 Lessons Learned Fundraising is the biggest challenge We secured venture philanthropy seed funding Grant applications can augment funding Intellectual property is critical File patents for basic research discoveries use of Group I mglur antagonists to treat disorders of brain development (fragile X, autism) Our patents facilitated licensing mglur5 antagonists from Merck & Co Inc. GABA-B patents enable financing of STX209 development Collaborative business model is a necessity

21 Recommendations Focus on the ultimate goal - successful development of novel therapeutics Resist incentives to over-value your contribution Align shareholder interests Share upside: 100% of nothing = nothing Consider open-source initiatives

22 Translating breakthrough discoveries in neurobiology into innovative drug treatments that improve the lives of patients and families with fragile X syndrome, autism and other neurodevelopmental disorders

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