dawn bell, pharmd president and ceo [email protected]

18
1 Dawn Bell, PharmD President and CEO [email protected] Translating cool science into important medicines

Upload: kirra

Post on 23-Feb-2016

67 views

Category:

Documents


0 download

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 Presentation

TRANSCRIPT

Page 1: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

1

Dawn Bell, PharmDPresident and CEO

[email protected]

Translating cool science into important medicines

Page 2: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

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

Page 3: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

3

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.

Page 4: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

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

Straub et al. ThrombRes (2008) 122,:383–389 and Eur J CT Surg (2005) 27:617–621

Page 5: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

5

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

Page 6: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

. .

.

6

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

Page 7: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

7

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)

Page 8: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

8

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

Page 9: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

9

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

Page 10: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

10

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

Page 11: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

11

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

Page 12: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

12

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

Page 13: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

13

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

Page 14: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

Timeline and Cash Needs

14

$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

Page 15: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

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

Current Funding • $100,000 committed from founders• NIH grants under review ($2.5M)

Page 16: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

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

12

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

Page 17: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

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

Lead Product

Team Model Market

Page 18: Dawn Bell,  PharmD President and CEO dawnbell@cool-bio.com

18

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)?