A Tale of Two(?) IBDs CYMMBiosis for Cure Colleen Feriod, PhD candidate Yujie Wang, MBA candidate Matthew Fleming, PhD candidate Michael Coggins, PhD Brian Cope, MBA/MEM candidate
Executive Summary License GTX-001 “Gastrolux” Pass on GTX-002
IBD Landscape Ulcerative Colitis • Disease mechanism unknown: genetic, environment, pathogenic factors • Highest incidence and prevalence in age group 15-30 Crohn’s Disease Disease mechanism unknown: genetic, environment, pathogenic factors Highest incidence and prevalence in age group 20-29
Target product profile Target product profile GastroluxGTX-002 Criteria Is it medically plausible? Is there a penetrable market? Is this financially viable? Is it safe? Does it perform better than SOC?
Pathophysiology of IBD Crohn’s Disease Ulcerative Colitis Unknown (genetic, environment, pathogenic) Initiation Visible and invisible symptoms Immune response Diagnosis of severity: Mild, moderate, severe Treatment depends on: Incident vs. recurrent Severity of diagnosis Unknown (genetic, environment, pathogenic) Initiation11111 Visible and invisible symptoms Immune 1111 Response1111 Diagnosis of severity: Mild, moderate,111 severe111 Treatment depends on: Incident vs. recurrent111 Severity of diagnosis111
Market Analysis Crohn’s Disease Ulcerative Colitis GTX-002 Gastrolux Competitors
Financial Analysis • NPV most sensitive to changes in discount rate and price growth • The breakeven values: discount rate = 13.9%; price = $7,554 • Expected Value of Average Scenario = $46,793, 384
Critical Signals in Development Pre-Clinical Phase I Specifics -In vitro and in vivo studies on animal IBD models -Dosage -Gastrolux vs. SOC -Safety assessment -Single ascending dose and/or multiple ascending dose studies -Food effect study Phase III Phase II -IIA: Assess dosing requirements -IIB: Study drug efficacy -Gastrolux efficacy vs. SOC -Submit for approval to FDA and EMEA *Efficacy is defined as decrease in symptom severity, increase in time between flares, reduction in disease severity progression, and/or inducing remission during acute flares
Evaluation Signal Risks Reward > Risk? Medical Market Financial Safety Improved treatment Legislative Long-term surveillance • Gastrolux is a viable target product • High unmet need • High revenue potential • Barren competitive landscape GTX-002 is not viable • Greater market size but higher consumer elasticity • Breakeven price exceeds market potential
Biological Risk Gastrolux • Able to safely and effectively block VLA-1 in humans and in appropriate patient subset? • Is VLA-1 present on T2 cells allowing UC as viable market? GTX-002 • Able to safely and effectively block NFκB in appropriate T cells only? General • Recent science suggests T17 cells are a point of convergence for CD and UC. • Is VLA-1 present on T17 cells? • Can NFκB be targeted in this T cell subset?