dawn bell, pharmd president and ceo [email protected]
DESCRIPTION
Translating cool science into important medicines. Dawn Bell, PharmD President and CEO [email protected]. Business Model. Development company focused on translating innovative scientific discoveries into medicines for patients with serious or life-threatening diseases - PowerPoint PPT PresentationTRANSCRIPT
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Dawn Bell, PharmDPresident and CEO
Translating cool science into important medicines
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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
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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):1568-15752. Murphy et al. Circulation. 2007; 116(22):2544-2552.
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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
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Improved Solution:Cool Platelet Inhibition (CLB100)
Above 35°C, this portion of the protein changes
shape and prevents binding to the receptor
Antibody with GpIIb/IIIa binding-site in red
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
. .
.
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Proof of Concept: Effect at Clinically Relevant Temperatures
Temperature (°C)372822 32
Anti-platelet effect demonstrated at these tempsTemp range
common in valve or complex cases
Temp range common in US CABG cases
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Proof of Target and in vivo Efficacy
ReoPro CLB100 15 μg/mL CLB100 50 μg/mL0102030405060708090
100Binding of Human Platelets
32°C 37°C
%Fib
rinog
en B
indi
ng
Topcic D., et al., Arterioscler Thromb Vasc Biol 2011, 31:2015-2023
Antithrombotic Effects in Mice
Control Integrilin CLB1000102030405060708090
100
28°C 37°C
Jugu
lar F
low
(% B
L)
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The Market
Indication #Patients (US)
Revenue/Patient
Benefits Est. Peak Sales
Cardio-pulmonary Bypass
300,000 $2500 Decreased post-operative complications
$200M(assumes
~25% market share)
Acute Ischemic Stroke
700,000 $4500 Decreased bleeding and residual neurological deficits
$400M(assumes
~15% market share)
Cardiac Arrest/Other
100,000 $4500 Decreased myocardial ischemia
$100M(assumes
~20% market share)
Potential sales >$500M
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Competitive LandscapeProduct
Use During CPB*
ReduceBleeding
Conserve Platelets
ReduceThrombos
isPeak Sales
CLB-100 ✔ ✔ ✔ ✔ $500MAbciximab ✔ ✔ $300MEptifibatide ✔ ✔ $330MTirofiban ✔ ✔ $80MAprotinin ✔ ✔ $285MAminocaproic Acid ✔ ✔ GenericTranexamic acid ✔ ✔ GenericMDCO-2010 ✔ ✔ Phase II*Cardiopulmonary Bypass
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Pipeline DevelopmentPeptide sequence can be coupled to any protein to create
new patented molecules through Harvard collaborationAdditional antibody development for new patented
molecules through Baker collaborationActive 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
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Executive ManagementName Function
al RoleArea of Expertise Past Affiliations
Dawn Bell President and CEO
Strategic development of anti-platelet/anti-thrombotic agents including out-licensing/M&A
• WVU• Cor Therapeutics• The Medicines Company• Canyon Pharmaceuticals
Michael Kurz Chief Scientific Officer
Pre-clinical development/animal efficacy models, leading non-dilutive funding efforts
• Gensia• Centocor/J&J• Cordis/J&J• Canyon Pharmaceuticals
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Collaborators/AdvisorsName Specialty AffiliationJoy Cavagnaro, PhD Pre-Clin Tox, Regulatory
StrategyEx-FDA
Elliott Chaikof, MD, PhD Innovator of EMP, molecular engineering
Harvard
Karlheinz Peter, MD, PhD
Innovator of GPIIb/IIIa antibody Baker Heart (Australia)
Frank Selke, MD Cardiac Surgery, Animal Efficacy Models
Brown
Jerrold Levy, MD Cardiac Anesthesia, Thrombosis Models
Emory
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Exit StrategyPartner lead product after early clinical trialsTargeted partners:
CV/ThrombosisAcute Care SpecialtyHospital Biologics
Field of ~15 identified
Company Program FitBaxter Hospital BiologicsJ&J CV/Thrombosis/BiologicsPfizer CV/Thrombosis/BiologicsRoche/Genentech Critical Care (Stroke)/Biologics
Recent Phase I Cardiac Surgery DealsCompany Phase Upfront TotalCubist/Dyax I $17.5M $214M + undisclosed
royaltiesMedicines/Curacyte I $23.7M $70M + undisclosed royalties
Timeline and Cash Needs
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$3M$2M
$4M $22M
36 monthsIND
12 months
Phase I
Pre-Clin/CMC
Phase IIa12 months
Out-License
Phase IIb24 months
4-7 years
Exit
Exit
Exit
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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
Current Funding • $100,000 committed from founders• NIH grants under review ($2.5M)
Use of $1.6M Seed Round:Strategic Use of Funds to Decrease Investment Risk
16Time (months)
$40,000
Temperature Response Studies
$450,000
in vivo CPB StudyLicensing/Legal Fees/G&A
$50,000
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1.6M
0.8M
0.4M
1.2M
ex-vivo CPB Study
$200,000
$10,000
Species Specificit
y
66 24
$610,000
36
$10,000
Protein Production
GMP Production
Pre-IND Meeting
January 7, 2012
$300k to FDA Feedback
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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
Lead Product
Team Model Market
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Questions1. 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)?