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GTX-001: A Risk Worth Taking

GTX-001: A Risk Worth Taking. 2009 YBPS Case Competition November 20, 2009. Victoria Clark, Kelly Fitzgerald Minji Kim, Frederick Wang Juliana Mastronunzio. Licensing Proposal from Gastrex. Two drug candidates for inflammatory bowel disease: GTX-001 and GTX-002.

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GTX-001: A Risk Worth Taking

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  1. GTX-001: A Risk Worth Taking 2009 YBPS Case Competition November 20, 2009 Victoria Clark, Kelly Fitzgerald Minji Kim, Frederick Wang Juliana Mastronunzio

  2. Licensing Proposal from Gastrex Two drug candidates for inflammatory bowel disease: GTX-001 and GTX-002 RECOMMENDATION: License only GTX-001 • Targets relevant inflammatory pathways in Crohn’s disease and ulcerative colitis • Addresses unmet needs of patients with moderate and severe disease • Larger potential market share than GTX-002 • Positive net present value even when accounting for long-term risk

  3. Inflammatory Bowel Disease (IBD) Symptoms: Abdominal pain, diarrhea, rectal bleeding, loss of appetite, growth, extraintestinal inflammatory conditions Crohn’s Disease Ulcerative Colitis • Colon and rectum • Inflammation and ulceration of mucosa and submucosa • Small intestine and colon • Transmural inflammation and ulceration, fistulae Severe Target patients for GTX-001 and GTX-002 Refractory Biologics Moderate Steroids + Immunomodulator Steroids + 5’ASA Mild 5’ASA No treatment Prevalence of IBD (US and EU 2008)

  4. Current Therapies for Moderate/Severe Cases Peyrin-Biroulet L, et al. Lancet 2008;372:67-81.

  5. GTX-001: Anti-VLA-1 Antibody Collagen VLA-1 GTX-001 Janeway's Immunobiology . Seventh Edition. pp 469 2008

  6. GTX-002: IKK Inhibitor TLR Interleukins Cytokines Chemokines Survival IL-1R TNFR IKKs GTX-002 NF-κB Podolsky DK. N Engl J Med 2002;347:417-29. Eckmann L et al. Proc Natl Acad Sci U S A 2008;105:15058-63.

  7. Modeling the Risk of Clinical Trials Biologics: 74% NCE: 58% Marketing $140 milestone Biologics: 41% NCE: 45% Phase 3 -$171 Per drug per disease R&D cost: $300MM Milestones: $200MM $45 milestone Failure Biologics: 80% NCE: 73% Phase 2 -$99 $15 milestone Failure Phase I -$30 Failure Biologics overall success rate: 24% New Chemical Entity (NCE) overall success rate: 19%

  8. IBD Market Analysis and Projections $8.3 bn Higher efficacy & willingness to pay drive higher sales revenue for biologics

  9. Positive Risk-Adjusted Valuations GTX-001 GTX-002 Total rNPV Total rNPV Mean (rNPV) = $139MM Mean (rNPV) = $590MM • 10,000 Monte Carlo simulations performed • Total rNPV = valuation of CD market and UC market • Prices adjusted with 3% annual inflation rate • 10% discount rate Conditions Chance of success is 29% for GTX-001 and 22% for GTX-002

  10. GTX-001: A Risk Worth Taking

  11. Acknowledgements Yale Biotechnology and Pharmaceutical Society BoehringerIngelheim Covidien Saul Ewing LLP Vion Pharmaceuticals Taro Pharmaceuticals McKinsey & Company Yale Center for Customer Insights Victoria Clark, Kelly Fitzgerald Minji Kim, Frederick Wang Juliana Mastronunzio

  12. Currently Recruiting IBD Clinical Trials Ulcerative Colitis Crohn’s Disease Phase I: rhuMAb Beta7 – anti-integrin α4β7 and αEβ7 Phase II: Golimumab – anti-TNFα Mab Rituximab – anti-CD20 Mab SC12267 – inhibits pyrimidine synthesis HMPL004 - multiple cellular targets Phase III: Budesonide-MMX – corticosteroid Golimumab – anti-TNFα human Mab Vedolizumab – anti-integrin α4β7 Abatacept – binds B7 T cell receptor Phase I: ZP1848 – GLP-2 peptide analogue KLH-TNFα kinoid – vaccine C326 – IL-6 Inhibitory Avimer protein Phase II: AIN457 – anti IL-17 Mab KLH-TNFα kinoid – vaccine rhIGF – recombinant IGF OPC-6535 – superoxide anion production inhibitor Ustekinumab – anti-IL-12, IL-23 human Mab HMPL004 – multiple cellular targets SC12267 – inhibits pyrimidine synthesis Phase III: Vedolizumab – anti-integrin α4β7 http://www.clinicaltrials.gov

  13. Monte Carlo Simulations • IF the drug is more effective, the model incorporates a higher market capture and price charged • Probabilities for market size and price modeled with beta-PERT distributions • Manufacturing costs not included due to incomplete data; the higher cost of producing monoclonal antibodies will lower the predicted rNPV Determination of relative effectiveness to current drugs was based on historical FDA classification of drugs by therapeutic value; system stopped in 1992

  14. Goal for new therapy: significantly increase time between flares, minimize side effects, use for induction and maintenance

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