Post on 12-Feb-2016
description
1Confidential
Science of Stem Cells and Potential Applications
Andrea HuntFebruary 22, 2008
2Confidential
Baxter International Inc.
At Baxter, one of our primary goals is to save and sustain lives worldwide.Every day, our products and services help treat thousands of people around the world with some of the most complex conditions — from hemophilia to cancer and immune disorders to kidney disease.
3Confidential
Company Snapshot
More than 75 years of market leadership driven by a well-recognized global brand
$10.4 billion global market leader
Over 50% of sales outside the United States
Approximately 70% of sales from products with leading market positions
More than $1.5 million invested in R&D every day
More than 3000 R&D employees
4Confidential
We are a global, diversified healthcare
company applying innovative science to
develop specialty therapeutics and
medical products that save and sustain
patients’ lives.
Baxter’s Mission
Our Missio
n
5Confidential
Baxter’s Business Portfolio
• Hemodialysis Products/Services
• Peritoneal Dialysis Products/Services
• Pharmaceuticals
MedicationMedicationDeliveryDelivery BioScienceBioScienceRenalRenal
• Premixed liquid and frozen drugs
• Infusion Systems• Anesthesia• Nutritional Products
• Therapeutic Proteins– Recombinant– Plasma Based
• Vaccines• Regenerative Medicine - Cellular Therapies - BioSurgery
6Confidential
Baxter’s Business Portfolio
• Hemodialysis Products/Services
• Peritoneal Dialysis Products/Services
• Pharmaceuticals
MedicationMedicationDeliveryDelivery BioScienceBioScienceRenalRenal
• Premixed liquid and frozen drugs
• Infusion Systems• Anesthesia• Nutritional Products• Isolex Business
• Therapeutic Proteins– Recombinant– Plasma Based
• Vaccines• Regenerative Medicine - Cellular Therapies - BioSurgery
7Confidential
BioSurgery Product Portfolio
8Confidential
Secondary cell Secondary cell processingprocessing
Cell storageCell storage
Cell Cell isolationisolation
Cell expansion/Cell expansion/cultureculture
ISOLEXMagnetic Cell
Selector LIFECELLCulture Bags
CYTOMATECell Washer CRYOCYTE
Freezing Containers
Cellular Therapies’ Product Portfolio:Used in many cell processing steps
9Confidential
Definition of Regenerative Medicine for Baxter
OtherRegenerative
Medicine
“Regenerative Medicine”The regeneration of damaged cells, tissues or organs through the use of cells, scaffolds and/or biomolecules via tissue engineering
Cells Scaffolds
Bioactive Agents
Cells Scaffolds
Bioactive Agents
approaches
10Confidential
Regenerative Medicine offers a new era in medicine …
Minimally invasive surgery
Invasive Surgery
RegenerativeMedicine
11Confidential
Isolex: selection
CD 34 + Stem Cell
Differentiation and/or stimulation
Blood Vessel
Heart Muscle
Blood Stream
Current Baxter CD34+ Phase II Stem Cell TrialPotential to reverse the effects of heart disease
12Confidential
13Confidential
Bone Programs: Fibrin Plus (PTH)Indication Current Status
Bone cysts
Long bone fractures
Vertebral augmentation
Mixing device
Mixing device
Application devicePrototype
14Confidential
Tissue engineered autologous bladder
Current Regenerative Medicine Application in Development
15Confidential
A. 1 – 5 years
B. 5 - 10 years
C. 10 -15 years
D. 15+ years
How long does it take for an idea to turninto a therapy for a patient?
16Confidential
Typically where Baxterinitially gets involved
Most Drugs and Stem Cell Therapies will take 10-15 years to develop
Product Launch
Basic Research
1 – 20+ Years
Pre-Clinical Research
1 – 3+ Years
Clinical Research
5 – 15+ Years
There must also be a market for your therapy
17Confidential
Now let’s add in chance of success
A typical drug costs $800 Million to develop
1 out of 1000+
Basic Research
Pre-Clinical Research
Clinical Research
Product Launch
1 – 20+ Years 1 – 3+ Years 5 – 15+ YearsTim
eSu
cces
sRa
te 1 out of 100+ 1 out of 10
18Confidential
Why does it take so long and cost so much?
Initial research focused on finding compounds that have the potential to work
Animal research is then conducted Clinical research is conducted in sequential stages
- Phase 1- Phase 2- Phase 3
Product is then launched into the marketplace
Demonstration of Safety and Efficacy
Regulatory agencies are involved – FDA in US, EMEA in Europe and PMDA in Japan
19Confidential
What is the unmet medical need?- target patient population
What is the scientific rationale that suggests a stem cell therapy will meet this need?- pre-clinical and clinical data
What is the viable business model?
A Business Model must also be createdfrom the start of development
A viable business model must be in place for a company to pursue development
20Confidential
Business Model Elements
Freedom to Operate Financials - Funding - Operations Partnerships Product Portfolio Manufacturing Clinical Plan
Pricing Reimbursement Strategic Plan Geographic Scope Marketing Plan Competitive Environment Distribution
Many skillsets are required to form this plan, a science background is always beneficial.
21Confidential
Business Model Viability Critical to Product Launch
It takes good science, time, dollarsand luck to launch!
1 out of 1000+
Basic Research
Pre-Clinical Research
Clinical Research
Product Launch
1 – 20+ Years 1 – 3+ Years 5 – 15+ YearsTim
eTe
chni
cal
Succ
ess
Rate 1 out of 1000+ 1 out of 10
22Confidential
Summary
Regenerative Medicine/Stem Cell Therapies could be next wave in medicine
Novel scientific breakthroughs will be achieved over the next decade and beyond
Adult stem cell therapies look promising and are the closest to being approved
Exciting new therapies are in the pipeline that will ultimately benefit patients
23Confidential
Saving and Sustaining Lives Worldwide
Thank you