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Partnering with Patients Howard Fillit, MD Executive Director , Alzheimer’s Drug Discovery Foundation Richard Insel, MD Executive Vice President, Research , Juvenile Diabetes Research Foundation James C. O’Leary Chief Innovation Officer , Genetic Alliance Louise Perkins, PhD Chief Scientific Officer, Multiple Myeloma Research Foundation Steven Perrin, PhD Chief Executive Officer and Chief Scientific Officer , ALS Therapy Development Institute Kristin Schneeman - Chair Program Director, FasterCures

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nonprofit think tank and catalyst for action that works across sectors and diseases to improve the effectiveness and efficiency of the medical research system

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Nonprofits as “De-Riskers” <ul><li>Developing pre-clinical tools </li></ul><ul><li>Targeting research to support translation </li></ul><ul><li>Funding mechanisms that bring in industry </li></ul><ul><li>Managing academic science </li></ul><ul><li>Access to patient community </li></ul><ul><li>New indications for existing drugs </li></ul><ul><li>High-throughput screening of promising compounds </li></ul><ul><li>Access to scientific expertise </li></ul><ul><li>Advocating with FDA </li></ul>

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TRAIN Central Station www.fastercures.org/train

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An effort designed to facilitate cross-sector collaborations needed to turn a scientific discovery into an accessible therapy. Be a part of an effort that brings together people with the expertise, experience, and creativity needed to transform the medical research system.

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The MMRF and MMRC are sister organizations contributing to all phases of drug development. <ul><li>Funds worldwide research </li></ul><ul><li>Builds myeloma community </li></ul><ul><li>Partners with MMRC </li></ul><ul><li>Supports 13 institutions </li></ul><ul><li>Conducts tissue banking </li></ul><ul><li>Facilitates Phase I/II trials </li></ul>

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With over $140M raised to date, the MMRF serves as an end-to-end solution for drug development.

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Implementation of business solutions to trial barriers led to MMRC trials opening 35% faster and robust trial enrollment. Trial Activation Enrollment 361 247 74 120

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The MMRF Patient Navigator program aims to drive clinical trial accrual with specific benefits for late-stage trials. Program Highlights Up-to-date searchable database Trained clinical specialists Late-stage trials prioritized Site support Marketing Targeted communications to stage of disease and geography MMRF Newsletter articles, ads Ads in MMRC, cancer publications Social networking/communities

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Accelerating Drug Discovery for Alzheimer’s Disease: The Alzheimer’s Drug Discovery Foundation

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Accelerating Drug Discovery for Alzheimer’s Disease: ADDF Venture Philanthropy

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Some Potential Advantages to Early Stage Biotechs of Venture Philanthropy Funding

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Biotechnology Founders Program: CoMentis Leading Anti-Amyloid Therapy

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Biotechnology Founders Program: CoMentis Leading Anti-Amyloid Therapy

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Biotechnology Founders Program: Allon Therapeutics Leading Anti-tangle Therapy

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ADDF Biotechnology Founders Program: Allon Therapeutics, Leading Anti-tangle Therapy Listed on the Toronto Exchange (NPC.TSX) 2005, current market capitalization around $50M Received return of note at time of IPO

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<ul><ul><li>Start-up biotech company focused on development of small molecule mimetics of neurotrophins (NGF, BDNF) to treat AD, PD, and other neurodegenerative diseases </li></ul></ul><ul><ul><ul><li>“ holy grail” of neuroprotection </li></ul></ul></ul><ul><ul><li>Founder is Professor Frank Longo, Chairman, Neurology and Neurosciences at Stanford </li></ul></ul><ul><ul><li>PTX chose the ADDF Venture Philanthropy Fund as preferred founding investor </li></ul></ul>ADDF Venture Philanthropy Fund Investments

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ADDF Venture Philanthropy Fund Investments

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Juvenile Diabetes Research Foundation <ul><li>Mission : To find a cure for type 1 diabetes and its complications </li></ul><ul><li>Strategy : Proactively accelerate the discovery, development, and delivery of drugs and devices to cure, better treat, or prevent type 1 diabetes and its complications </li></ul><ul><li>Core Principles : P eople with T1DM, P ipeline, P riorities, P artners </li></ul>

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Beta Cell Therapies Immune Therapies JDRF’s Industry Partners DIABECELL® Encapsulated Cell Replacement Therapy

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JDRF’s Industry Partners Glucose Control Complications Therapies

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JDRF’s Clinical Trial Awareness Activities: JDRF Type 1 Diabetes Clinical Trials Connection (www.trials.jdrf.org)

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<ul><li>Biobank of pancreata and related tissues from cadaveric organ donors with: </li></ul><ul><ul><li>Recent onset type 1 diabetes </li></ul></ul><ul><ul><li>Established diabetes including long-standing (>50yrs) </li></ul></ul><ul><ul><li>At-risk, islet autoantibody positive </li></ul></ul><ul><li>Tissues being used by > 50 groups to address key immunological, histological, viral, and metabolic questions to better understand type 1 diabetes </li></ul><ul><li>www.jdrfnpod.org </li></ul>JDRF’s BioBank: nPOD

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JDRF’s FDA Activities <ul><li>FDA-NIH-JDRF Workshops: </li></ul><ul><ul><li>How Good Does a Beta Cell Have to Be: November 2009 </li></ul></ul><ul><ul><li>Towards An Artificial Pancreas: July 2008 </li></ul></ul><ul><ul><li>Obstacles and Opportunities on the Road to Artificial Pancreas: Closing the Loop: December 2005 </li></ul></ul><ul><li>Artificial Pancreas placed on Critical Path list in 2006; Artificial Pancreas Review team received FDA “Leveraging/Collaboration Award” in 2007 </li></ul><ul><li>FDA acceptance of in silico model to test algorithms </li></ul><ul><ul><li>Algorithms can be extensively tested on in silico ‘patients’ prior to clinical study and allows elimination of animal testing </li></ul></ul>

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JDRF’s Reimbursement Activities <ul><li>JDRF funded a multicenter phase IV clinical trial of effectiveness of Continuous Glucose Monitors in Type 1 Diabetes -> Change in reimbursement policy ( United Healthcare, Aetna, Cigna, Wellpoint, Kaiser Permanente) and clinician and patient adoption </li></ul>

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Why Does ALS TDI Exist? <ul><li>Nature Drug Discovery, February 2010 </li></ul><ul><li>Nature Drug Discovery, March 2010 </li></ul>Drug Discovery Pre Clinical Development Phase I Phase II Phase III 3.1 yrs 2.5 yrs 1.5 yrs 2.5yrs 3.0 yrs Prototypical Pharmaceutical Timelines * Average cost to commercialization: $1.8B

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R&D Productivity Model Target ID Hit Lead Optimization Preclinical Validation Phase I Phase II Phase III Submission Launch 80% 24.3 $1 1.0 3% $94 75% 19.4 $2.5 1.5 6% $166 85% 14.6 $10 2.0 17% $414 69% 12.4 $5 1.0 7% $150 54% 8.6 $15 1.5 15% $273 34% 4.6 $40 2.5 21% $319 70% 1.6 $150 2.5 27% $314 91% 1.1 $40 1.5 5% $48 P(TS) WIP per Launch Cost per WIP per Phase Cycle Time (Years) Cost per Launch % Total Cost per NME <ul><li>P(TS) : probability of technical success </li></ul><ul><li>WIP : work in progress </li></ul><ul><li>V : value </li></ul><ul><li>CT : cycle time </li></ul><ul><li>C : cost </li></ul><ul><li>Nature Drug Discovery March 2010 </li></ul>Preclinical Cost per NME Average: $18.5M Capitalized: $824M Cycle time: 5.5 years Clinical Cost per NME Average: $245M Capitalized: $954M Cycle time: 8 years P(TS) x WIP x V CT x C R&D Productivity =

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ALS TDI Investment in ALS TDI 00846 Drug Discovery Pre Clinical Development Phase I Phase II Phase III 3.1 yrs ($10M) 1.4 yrs ($20M) 1.7 yrs ($10M) 2.7 yrs ($30M) 1.9 yrs ($100M) Discovery Tools Pk Studies Efficacy Studies PD Studies Tolerability Studies Disease Models Clinical Samples Therapeutic Development Prototypical Pharmaceutical Cost & Timelines 0.75 yrs ($0.6M) 1 yr ($0.8M) 1.5 yrs ($1.2M) ALS TDI Cost & Timelines <ul><li>ALS TDI 00846 preclinical development: $2.5M </li></ul><ul><li>40% of investment is in therapeutic development, pk, and tolerability </li></ul>

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ALS-TDI Facilitates Clinical Translation De-Risk the Opportunity & Broker the Deal Clinical Development Study Disease  Pre-Clinical Development   Build Drug  Pre-Clinical Development Clinical Basic Research Drug Development Gap Find a Drug Target

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<ul><li>Largest Online Discussion of ALS Therapeutics </li></ul><ul><li>3,000+ Members, 40,000 posts </li></ul><ul><li>Topics include Therapeutics & Clinical Trials to Living With ALS and Advocacy </li></ul><ul><li>Free, anonymous and open to the public </li></ul>Merit Cudkowicz, M.D. Mass General Hospital Harvard Medical School Jonathan Katz, M.D California Pacific Medical Center (Forbes Norris) Stanley Appel, M.D. Methodist Neuro. Institute Cornell Medical School ALS TDI Patient Forum Preclinical Data Clinical Trials Experimental Therapies Patient Care Carey Goldberg Boston Globe NY- Times, LA- Times, AP ALS Forum Build and Empower a Community

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Transforming Health through Genetics Promoting an Environment of Openness

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Survey of Disease Advocacy Organizations <ul><li>37 organizations responded to the survey </li></ul><ul><li>13 organizations (35%) were involved with a biorepository, and 15 organizations (41%) were involved with a registry </li></ul><ul><li>Half of biorepositories and 73% of registries have been in operation for more than 5 years </li></ul><ul><li>Of those organizations without biorepositories or registries, 65% were interested in establishing a biorepository, and 86% were interested in establishing a registry </li></ul><ul><li>Cost was the major reason for not establishing a biorepository or registry </li></ul>

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DAO Demographics Characteristic N (%) Organization Age <5 years 2 (6) 5-10 years 13 (36) 11-20 years 15 (42) >20 years 6 (17) Paid Employees Yes 28 (76) Volunteer Network Yes 29 (81) Annual Budget <$100K 17 (49) $100K-$499K 11 (31) $500K-$999K 6 (17) $1M-$5M 1 (3) >$5M 1 (3)

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DAO Involvement Types of Engagement N (%) Established Biorepository/registry 12 (67) Own Biorepository/registry 9 (53) Provide Financial Support 10 (56) Help Obtain Financial Support 10 (56) Recruit Participants 17 (94) Obtain Informed Consent 11 (61)

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Sample Types Biorepository Collects N (%) Blood 13 (72) Tissue (Frozen) 10 (56) Tissue (Formalin) 9 (50) Cell Blocks 5 (28) Cell Lines 7 (39) Other Biological Material 3 (17) Desire to Collect other Types 13 (81)

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Sample Use and Storage Characteristic N (%) Experiments DNA/genomics Analysis 9 (64) RNA Expression 1 (7) Protein/immunohistochemistry 2 (14) Other 5 (36) Users Research Consortium 5 (31) Any Qualified Researcher 12 (75) Other 5 (31) Costs Passed on to Researcher 9 (50) Location Nonprofit Organization 3 (21) Commercial Lab 3 (21) University/research Lab 8 (57) Government Lab 1 (7) Other 3 (21)

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Disease Specific Organization Data and Sample Management Informed Consent Robust Clinical Information Sample Collection & Tracking Data and Sample Query Data Management Electronic & Paper Records Security & Access Control Patient Enrollment Disease Sample and Clinical Banking Biological Samples Clinical Information Web based Boot camps Manuals Workbooks Wikis Webinars

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Demonstrating Private Access’ Technology to Transform Patient Recruitment

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Thank you <ul><li>James C. O'Leary Chief Innovation Officer </li></ul><ul><li>Genetic Alliance 4301 Connecticut Ave. NW Suite 404 Washington, DC 20008-2369 </li></ul><ul><li>(202) 966-5557 x206 [email_address] www.geneticalliance.org </li></ul>