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Translating cool science into important medicines. Dawn Bell, PharmD President and CEO dawnbell@cool-bio.com. Business Model. Development company focused on translating innovative scientific discoveries into medicines for patients with serious or life-threatening diseases
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Translating cool science into important medicines Dawn Bell, PharmD President and CEO dawnbell@cool-bio.com
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
The Problem:Bleeding in Cardiopulmonary Bypass (CPB) Surgery • Over 300,000 CPB procedures performed each year • Frequent bleeding and use of blood products1 • 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 costs2 • CPB outcomes publically reported and reimbursement is fixed (so hospital is liable for costs of managing complications and has to report them) 1. Bennett-Guerrero et al. JAMA. 2010;304(14):1568-1575 2. Murphy et al. Circulation. 2007; 116(22):2544-2552.
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 Straub et al. ThrombRes (2008) 122,:383–389 and Eur J CT Surg(2005) 27:617–621
Improved Solution:Cool Platelet Inhibition (CLB100) Patient’s 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 Above 35°C, this portion of the protein changes shape and prevents binding to the receptor Antibody with GpIIb/IIIa binding-site in red
Proof of Concept: Effect at Clinically Relevant Temperatures Anti-platelet effect demonstrated at these temps Temp range common in valve or complex cases . . Temp range common in US CABG cases . 22 28 32 37 Temperature (°C)
Proof of Target and in vivo Efficacy Antithrombotic Effects in Mice %Fibrinogen Binding Jugular Flow (% BL) Topcic D., et al., ArteriosclerThrombVascBiol2011, 31:2015-2023
The Market Potential sales >$500M
Competitive Landscape *Cardiopulmonary Bypass
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
Exit Strategy • Partner lead product after early clinical trials • Targeted partners: • CV/Thrombosis • Acute Care Specialty • Hospital Biologics • Field of ~15 identified
Timeline and Cash Needs IND Pre-Clin/CMC 36 months Phase I Exit Phase IIa 12 months Exit 12 months Phase IIb Exit 24 months Out-License 4-7 years $3M $2M $4M $22M
Investment Opportunity 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 Proposed Terms • $100,000 committed from founders • NIH grants under review ($2.5M) Current Funding
Use of $1.6M Seed Round:Strategic Use of Funds to Decrease Investment Risk 1.6M Protein Production $300k to FDA Feedback $10,000 Temperature Response Studies 1.2M $40,000 ex-vivo CPB Study $200,000 Pre-IND Meeting 0.8M Species Specificity $10,000 GMP Production $610,000 0.4M in vivo CPB Study Licensing/Legal Fees/G&A $50,000 $450,000 36 24 12 6 6 Time (months) January 7, 2012
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 Model Market Team Lead Product
Questions • What data should COOL~BIO be presenting to potential partners in order to de-risk the deal for them? • 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? • 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)?