A model collaboration using the Pool Susanne Hollinger, Ph.D., J.D. Chief Intellectual Property Officer Emory University

Overview How did Emory get involved? Our experience – The Patent Pool New therapeutics/research directions – The Knowledge Pool Problem solving – the iThemba collaboration Educational mission

Emory Background Strong history in drug development for infectious diseases commercial products include emtricitabine, lamivudine, telbivudine >90% of HIV patients take drugs developed at Emory >80 issued US patents on small molecule infectious disease treatments August 2009: Emory establishes Emory Institute for Drug Discovery (EIDD) – Focus on drug discovery for commercially neglected diseases promoting multidisciplinary research fostering global health partnerships

Emory Institute for Drug Discovery (EIDD) Mission - Drug discovery and preclinical research Medicinal Chemistry Process Development Cellular and Protein Biology Drug Metabolism Pharmacokinetics In Vivo Pharmacology In Vivo Toxicology High Throughput Compound Screening Computational Modeling & Drug Design Multidisciplinary environment Experienced investigators Fully equipped chemistry and biology labs Fully staffed rodent and non-human primate vivaria

Emory Institute for Drug Discovery (EIDD) Mission - Drug discovery and preclinical research Multidisciplinary environment Experienced investigators Fully equipped chemistry and biology labs State of the art computational models/drug design High throughput compound screening facility Fully staffed rodent and non-human primate vivaria

Knowledge Pool Timeline February 2009 –“patent pool” announced January 2010 – – Emory is first University to join pool – iThemba is first company to join as user – Tres Cantos facility established – BVGH takes over administration of pool

Why did Emory join pool? Goals: – Access new therapeutics specifically targeted at neglected diseases – Expand drug development capacity at University – Enhance drug development expertise for scientists

Original ‘user’ model – Emory given list of available patents – Emory to provide a list of projects of interest – GSK will provide access to company scientists to provide information about the history and current development of selected compounds – Once Emory identifies technologies it wishes to pursue, Emory and GSK will develop a license agreement and additional collaborative research Implementation

Our Experiences – The Patent Pool Initial concept of “patent pool” user difficult to implement – Access to thousands of patents, but how does one direct interests? – Initially, Emory scientists sifted through patents to identify small molecules that could be polymerase inhibitors, then met with GSK to gain insights on status of projects Six groups of patent families identified as attractive; Emory provided review of our understanding to GSK and GSK provided updates to supplement the summaries

Results Concept morphed to “Knowledge Pool” then “Open Innovation Pool” – Access to knowledge, not just patents – Allows problem solving by building alliances – Enhances education of next generation scientists, especially in developing world

The Knowledge Pool – Case study August 2007: Emory enters into agreement with iThemba Pharmaceuticals – Development of small molecules to target infectious diseases (HIV, Malaria, TB) iThemba – Only small molecule chemistry company in South Africa (and Africa) – Commenced operations June 2008 (raised $6.5M from gvt and collaborators) – Offers Contract synthetic organic or medicinal chemistry Produce novel chemical intermediates Hit validation service

The iThemba-Emory TB Knowledge Pool Emory The TB collaboration is focused on inhibition of two enzymatic targets (isocitrate lyase and malate synthase) associated with latency stage of TB. BVGH Glaxo SmithKline iThemba Pharm. Alnylam

Problem Solving: The iThemba Collaboration Emory-iThemba collaboration already yielded: – Highly active isocitrate lyase drug candidates Best validated target for persistence – Identified compound with low nm activity against isolated enzyme – Limited in vivo screening Problem presented to pool: – Can you help us profile our leads?

Knowledge Pool Solutions Enhanced screening facilities – In vivo screening of identified candidates – Prodrug optimization for relevant species to decrease dosing needed Access to IP – In-licensed synthetic granuloma technology to identify compounds with permeability characteristics

Emory/EIDD – Goal to enhance drug development in academia – Emory in discussions for researcher exchange at Tres Cantos – train in drug development and new screening techniques – Emory in discussions to export educational expertise for industry researchers iThemba – Goal to bring drug development to South Africa – iThemba finalized researcher exchange to allow knowledge transfer in drug development – first scientist January 2011 – 6 months at Tres Cantos, 6 months at iThemba Educational Mission - Training Next Generation

Why is it working? All parties have same goal