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From Genomics to Drugs Kitasato University – Harvard School of Public Health Symposium Kevin Rakin President & CEO Genaissance Today Our Approach: Integrating gene variation into product development and drug marketing Our Focus: Drug response GNSC The Genaissance Drug Paradigm HAP™ Technology shifts the drug response curve Safer and more effective prescriptions Ability to move market share Number of Patients HAP™ Technology Guided Drugs Conventional Drugs Drug Response Leveraging our Platform HAP™ Technology HAP™ Database DecoGen® Informatics System Partnerships Technology DrugSpecific HAP™ Typing Facility Clinical Genetic Expertise Internal Programs STRENGTH CARING Gene Haplotypes Exons Chromosome locus of gene Promoters SNPs 0 Gene SNPs 1 0 1 0 1 0 1 0 1 0 1 0 0 1 1 0 1 1 0 Haplotypes Causative Site Haplotypes are a code for defining and tracking the isoforms of a gene Most Detailed Examination of Variability in Human Genes Vol. 293 No. 5529 20 July 2001 HAP™ Technology: The Complete Pharmacogenomic Solution HAP™ Database – >6,000 genes with 200,000 HAP™ Markers DecoGen® Informatics System – Links gene variation to drug safety and efficacy HAP™ Typing Facility – CLIA certified – >2.5M genotypes to date Clinical Genetics Expertise – High-throughput statistical analysis pipeline Connecting HAP™ Markers to Breathing Improvement Number of Individuals: 119 Mean Value: 13.0 Std. Dev.: 8.0 20 18 16 FEV1 Patient Response to Albuterol 14 HAP™ Marker Pairs Associated with Response 12 10 8 6 4 2 0 -5.0 4,4 10.0 NonResponders 4,2 6,2 25.0 2,2 40.0 55.0 Responders 6,4 First Peer Reviewed Publication Linking Gene Haplotypes and Drug Response Proceedings of the National Academy of Sciences vol. 97, no. 19, pp. 10483-10488 September 12, 2000 BDProbeTec™ ET Workflow for Genotyping from Whole Blood Blood in EDTA, Citrate or Heparin Tube Universal SDA Buffer Priming Microwells Denature and Centrifuge Potential Throughput 96 genotypes 75 min Each subsequent run 45 min Total genotypes per 8 hours = ~1000 • SDA Primers • Fluorescent Detectors • dNTPs Amplification Microwells • Bst Polymerase Enzyme • BsoBI Restriction Enzyme Results STRENGTH I (Statin Response Examined by Genetic HAP™ Markers) Prospective, multicenter, 3 parallel arms, 2 doses HAP™ Markers for ~175 genes Clinical Endpoints: – LDL-C responders and non-responders – Changes in HDL-C, LDL/HDL ratios, total C, triglycerides – C-reactive protein, apolipoproteins and other selected measures – Adverse events and baseline lipid and lipoprotein levels Differentiating Statins: Mean HDL and LDL Responses + + % Improvement LDL-C % Improvement HDL-C 0 DRUG 0 A B C DRUG A B C All patients Low Dose Differentiating Statins: HAP™ Marker L40-S1 (26% Frequency) + + % Improvement LDL-C % Improvement HDL-C 0 DRUG 0 A B C DRUG All patients L40-S1 (+) A B C Low Dose Differentiating Statins: HAP™ Marker L40-S1 (74% Frequency) + + % Improvement LDL-C % Improvement HDL-C 0 DRUG 0 A B C DRUG All patients L40-S1 (+) L40-S1 (-) A B C Low Dose Conclusions From STRENGTH HAP™ Markers: • Define populations with different response • Differentiate between drugs in the same class • Can be used to drive market share Commercialization: • Statin diagnostic (safety & efficacy) • Drug development • Drug marketing Leveraging our Platform HAP™ Technology HAP™ Database DecoGen® Informatics System Partnerships Technology DrugSpecific HAP™ Typing Facility Clinical Genetic Expertise Internal Programs STRENGTH CARING HAP™ Technology Partnerships Business Model License fees Service fees for processing clinical samples Success-based milestone payments Drug and diagnostic royalties Drug-Specific Partnership Collaboration initially focused on Amevive® – moderate to severe chronic plaque psoriasis Marketing-driven strategy – Diagnostic to predict responders & non-responders – justify expensive course of treatment – define new indications Funding and milestones for HAP™ Marker association studies Royalty on drug sales and testing fee revenues within 2 years Amevive® Product Development Timeline 2002 June March 2003 December March June December Planning Phase Define patient groups, select gene list Stage I: Hypothesis Generation Measure 25-100 genes for markers in patients; Identify 8-10 genes with key markers Stage II: Confirmatory Study Optimization and preparation for commercial launch Diagnostic Partner Validate markers in separate patient population Diagnostic Partner Assay design and development Launch of home brew diagnostic test Diagnostic Partner Product Development Timelines 2002 2nd Half Biogen: Amevive® Johnson & Johnson Statin Diagnostic STRENGTH Therapeutic CARING Therapeutic 2003 1st Half 2004 2nd Half 1st Half 2nd Half 2005 1st Half 2nd Half Confirmatory Study, HAP™ Product Marker Product Launch Discovery Development HAP™ Marker Discovery Partner Identification HAP™ Marker Discovery Confirmatory Study, Product Development, Marketing Confirmatory Study, Product Development Partner Identification HAP™ Marker Discovery Product Launch Phase I & II Partner Identification Product Launch Phase I & II Phase III Phase III 2006 2nd Half Gene Haplotypes and Drug Response • Focus on genes and haplotypes • Gene specific genome wide approach • Technology base fully validated • Correlations configured into products • Blue-chip partners • Growing product portfolio • Product launch as early as 2004 From Genomics to Drugs Gene Haplotypes and Drug Response