1 dawn bell, pharmd president and ceo [email protected]

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  • Slide 1
  • 1 Dawn Bell, PharmD President and CEO [email protected]
  • Slide 2
  • Business Model Development company focused on translating innovative scientific discoveries into medicines for patients with serious or life- threatening diseases Exit/revenue strategy: Out-licensing Phase II/III ready candidates Capital efficient nearly virtual model with emphasis on non- dilutive financing and strategic outsourcing Collaborative agreements in place with Harvard, Emory and Baker Heart Institute in Australia Lead product is an antibody against platelets that is only active when cold (e.g. therapeutic hypothermia), being developed for use during hypothermic cardiopulmonary bypass 2
  • Slide 3
  • The Problem: Bleeding in Cardiopulmonary Bypass (CPB) Surgery Over 300,000 CPB procedures performed each year Frequent bleeding and use of blood products 1 60% of patients get blood transfusions 25% of patients get platelet transfusions 20% of patients get plasma transfusions Blood product use increases mortality, morbidity and costs 2 CPB outcomes publically reported and reimbursement is fixed (so hospital is liable for costs of managing complications and has to report them) 3 1. Bennett-Guerrero et al. JAMA. 2010;304(14):1568-1575 2. Murphy et al. Circulation. 2007; 116(22):2544-2552.
  • Slide 4
  • The Solution Platelet Anesthesia Platelets are the first responders of blood clotting CPB leads to destruction of platelets and impaired platelet function Put platelets to sleep during CPB - Conserve platelet number and function - Reduce bleeding and blood product use Platelet GPIIb/IIIa receptor known target - Imperfect solution: Available GPIIb/IIIa inhibitors not reversible, cause bleeding 4 Straub et al. ThrombRes (2008) 122,:383389 and Eur J CT Surg (2005) 27:617621
  • Slide 5
  • Improved Solution: Cool Platelet Inhibition (CLB100) 5 Above 35C, this portion of the protein changes shape and prevents binding to the receptor Antibody with GpIIb/IIIa binding-site in red Patients body temperature is on/off switch Reversible platelet anesthesia Unique GPIIb/IIIa fusion-protein, turned off at end of CPB by routine warming Exclusive world-wide license with strong IP position
  • Slide 6
  • ... 6 Proof of Concept: Effect at Clinically Relevant Temperatures Temperature (C) 37 282232 Anti-platelet effect demonstrated at these temps Temp range common in valve or complex cases Temp range common in US CABG cases
  • Slide 7
  • Proof of Target and in vivo Efficacy %Fibrinogen Binding Topcic D., et al., Arterioscler Thromb Vasc Biol 2011, 31:2015-2023 Antithrombotic Effects in Mice 7 Jugular Flow (% BL)
  • Slide 8
  • The Market 8 Indication#Patients (US) Revenue/ Patient BenefitsEst. Peak Sales Cardio- pulmonary Bypass 300,000$2500Decreased post- operative complications $200M (assumes ~25% market share) Acute Ischemic Stroke 700,000$4500Decreased bleeding and residual neurological deficits $400M (assumes ~15% market share) Cardiac Arrest/Other 100,000$4500Decreased myocardial ischemia $100M (assumes ~20% market share) Potential sales >$500M
  • Slide 9
  • Competitive Landscape Product Use During CPB* Reduce Bleeding Conserve Platelets Reduce ThrombosisPeak Sales CLB-100 $500M Abciximab $300M Eptifibatide $330M Tirofiban $80M Aprotinin $285M Aminocaproic Acid Generic Tranexamic acid Generic MDCO-2010 Phase II 9 *Cardiopulmonary Bypass
  • Slide 10
  • Pipeline Development Peptide sequence can be coupled to any protein to create new patented molecules through Harvard collaboration Additional antibody development for new patented molecules through Baker collaboration Active pursuit of additional compounds Exciting/Innovative science >$200M/yr projected peak revenue Well- characterized biological pathway/known drug-able target that leverages our core expertise(e.g. acute/critical care, cardiovascular or blood diseases) Proof of concept achievable with less than 50 patients Defined regulatory path 10
  • Slide 11
  • Executive Management NameFunctional Role Area of ExpertisePast Affiliations Dawn BellPresident and CEO Strategic development of anti- platelet/anti-thrombotic agents including out-licensing/M&A WVU Cor Therapeutics The Medicines Company Canyon Pharmaceuticals Michael KurzChief Scientific Officer Pre-clinical development/animal efficacy models, leading non-dilutive funding efforts Gensia Centocor/J&J Cordis/J&J Canyon Pharmaceuticals 11
  • Slide 12
  • Collaborators/Advisors 12 NameSpecialtyAffiliation Joy Cavagnaro, PhDPre-Clin Tox, Regulatory Strategy Ex-FDA Elliott Chaikof, MD, PhDInnovator of EMP, molecular engineering Harvard Karlheinz Peter, MD, PhDInnovator of GPIIb/IIIa antibodyBaker Heart (Australia) Frank Selke, MDCardiac Surgery, Animal Efficacy Models Brown Jerrold Levy, MDCardiac Anesthesia, Thrombosis Models Emory
  • Slide 13
  • Exit Strategy Partner lead product after early clinical trials Targeted partners: CV/Thrombosis Acute Care Specialty Hospital Biologics Field of ~15 identified CompanyProgram Fit BaxterHospital Biologics J&JCV/Thrombosis/Biologics PfizerCV/Thrombosis/Biologics Roche/GenentechCritical Care (Stroke)/Biologics 13 Recent Phase I Cardiac Surgery Deals CompanyPhaseUpfrontTotal Cubist/DyaxI$17.5M$214M + undisclosed royalties Medicines/CuracyteI$23.7M$70M + undisclosed royalties
  • Slide 14
  • Timeline and Cash Needs 14 $3M $2M $4M $22M 36 months IND 12 months Phase I Pre-Clin/CMC Phase IIa 12 months Out-License Phase IIb 24 months 4-7 years Exit
  • Slide 15
  • Investment Opportunity Proposed Terms Seeking $1.6M in seed funding for pre-clinical development of lead candidate (CLB100) Convertible debt 8% per annum interest 2 year term Discounted conversion at Series A 15 Current Funding $100,000 committed from founders NIH grants under review ($2.5M)
  • Slide 16
  • Use of $1.6M Seed Round: Strategic Use of Funds to Decrease Investment Risk 16 Time (months) $40,000 Temperature Response Studies $450,000 in vivo CPB Study Licensing/Legal Fees/G&A $50,000 12 1.6M 0.8M 0.4M 1.2M ex-vivo CPB Study $200,000 $10,000 Species Specificity 66 24 $610,000 36 $10,000 Protein Production GMP Production Pre-IND Meeting January 7, 2012 $300k to FDA Feedback
  • Slide 17
  • Summary Translating Cool Science into Important Medicines Known therapeutic target decreases development risk Management team with deep therapeutic, development and market experience Capital efficient model with emphasis on strategic partners and non-dilutive financing Large and rapidly growing with important unmet needs 17
  • Slide 18
  • Questions 1. What data should COOL~BIO be presenting to potential partners in order to de-risk the deal for them? 2. What should COOL~BIO be doing in addition to the pre-clinical development plan (or instead of) to increase valuation prior to the Series A round? 3. How can we better communicate the value proposition of the molecule for partners and investors? Is end-user market research needed at this stage (e.g. Cardiac surgeons)? 18