the academic contribution to drug development
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The Academic Contribution to Drug Development. Roger M. Perlmutter Executive Vice President, Research and Development. An Innovation Deficit in Pharmaceuticals?. - PowerPoint PPT PresentationTRANSCRIPT
The Academic Contribution to Drug Development
Roger M. PerlmutterExecutive Vice President, Research and Development
For Internal Use Only. Amgen Confidential.
An Innovation Deficit in Pharmaceuticals?
The number of new chemical entities produced by the top 50 pharmaceutical companies is too small to sustain the healthy growth of this group
New technologies (e.g. genomics, combinatorial chemistry, etc.) do not appear to have had a major impact on the provision of new drugs by the industry so far
- Jurgen Drews, DDT 3: 491, 1998
3
R&D Expenditures in the Biopharmaceutical Industry Have Soared Since 1980. . .
0
10
20
30
40
50
60
1980 1982 1984 1986 1988 1990 1992 1994 1996 1998 2000 2002 2004 2006 2008
To
tal R
&D
Sp
en
din
g (
$B
)
Source: PhRMA Annual Survey 2009
CAGR = 12%
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. . .and Fewer New Drugs are Emerging: We are Spending More and Achieving Less
35
2724
1721
31
18 18 1924
$23$26
$30
$39 $39$43
$49$50
$32$33
0
5
10
15
20
25
30
35
1999 2000 2001 2002 2003 2004 2005 2006 2007 2008
$0
$10
$20
$30
$40
$50
Approved Drugs (NMEs) and PhRMA Member R&D Spend
NMEsPhRMA R&D spend ($B)
Sources: Burrill & Company, Biotech 2008 Life Sciences: A 20/20 Vision to 2020, 22nd Annual Report on the Industry; PhRMA annual membership survey (2009)
For Internal Use Only. Amgen Confidential.
The Evolving Challenge Profile of the Therapeutics Industry
• Complexity• Challenge• Pace
1980 1990 2000 2010 2020
• Few interesting targets• Room for many drugs• Strong intellectual property
• Thousands of targets• Declining success
rates• Rapid generic
penetration• Devastating product
failures
“Glaxo’s Witty Says Nine Top Drugmakers May Fail”, Times Reports*
Nine of the top 15 drugmakers will “wither” or “get taken out” in the next five years
About half a dozen of the biggest pharmaceutical companies may survive as patents expire and business strategies are “tested to destruction”
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*Quoted in Bloomberg News, October 2, 2010
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Contemporary Estimates Paint a Grim Picture of Future Industry Profitability
R&D SpendRx Revenues New Products
Total of 15 BioPharmaceutical Companies
Source: Lazard estimates
$230B $387B
$537B $244B
’89-’98
’99-’08
’97-’04
’07-’14
The View from Investment Bankers:
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“Pharmaceuticals: Exit Research and Create Value”
- - Morgan Stanley
6353
45
27
3747
55
73
1980 1989 1999 2007
Prehuman/Preclinical Phase 1 thru Approval
Percentage of Total PhRMA R&D Expenditures
Source: PhRMA Industry Profile Reports
Since 1980, Biopharmaceutical Companies have Largely Abandoned Discovery Research. . .
. . .but are These Companies Capable of Supervising Drug Development?
Most industry investment in R&D appears to be wasted
The vast majority of industry investment is spent on clinical research (i.e. development)
A 30-year perspective suggests that industry is not solving this problem on its own
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Industry and Academia: Why We May Need Each Other
Academic investigators cannot easily pursue high-quality clinical development– Teamwork is undervalued in universities– Difficulty in maintaining a compliance focus– Careerism undermines development intent– Little penalty for failure
Industry investigators have their own problems– Inherent conservatism - - the penalty of prior success– Priorities set in the context of commercial assessments– High penalty for failure, hence novel programs suffer
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No company has proved capable of refilling its pipeline
Why Might Academic Organizations Now be Able to Pursue Drug Discovery?
Industry, burdened with P&L considerations, has need to out-license NCEs that do not fit within their central focus
Improved methods of patient stratification offer the promise of increasing efficiency in clinical trials
Skilled contract research organizations can assume the burden of maintaining GCP compliance
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But – do university physicians really want to develop drugs?
Activated Osteoclast
CFU-M
Pre-Fusion Osteoclast
MultinucleatedOsteoclast
Bone
Osteoblast
Osteoclast Formation, Activation, and Survival Inhibited
Osteoclast Activated
Colony-Forming Unit Macrophage
RANK
Denosumab
RANKL
OPG
Growth Factors HormonesCytokines
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Denosumab Blocks Bone Resorption by Binding to a Pivotal Regulator, RANKL
Can a university tenure ladder accommodatethis sustained commitment? Can staff members be retained throughoutthis period?
20101995
Discovery of target
Clinical exploration
Selection of a clinical candidate
Registration-enabling studies
Development of Denosumab Required 15 Years of Investment
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• Complexity• Pace• Expense• Uncertainty
1980 1990 2000 2010 2020
• Observational anecdotes• Low efficacy hurdle• Few targets• Little incentive for
improvement• Powerful new
research technologies• Many, better targets• High-quality clinical
data• Evolving focus on
personalized medicine
The Evolving Challenge Profile in Drug Development May Favor Academic Researchers
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The Academic Contribution to Drug Development
Roger M. PerlmutterExecutive Vice President, Research and Development