Transcript Cardinal SPS Customer Meeting and Event 2005

Bio- and Nanotechnology:
Emerging Medical Miracles on
the Horizon
Marsha K. Millonig, MBA, RPh
President & CEO
Catalyst Enterprises, LCC
Today’s Objectives
• Understand biotechnology and
nanotechnology
• Describe the biotechnology market size
• Explain what fields are parts of the emerging
business of life science
• List a number of new therapies resulting from
bio and nanotechnology
• Discuss the implications of these new
therapies on pharmacy and its technology
partners
Why is it Important?
Global Population Growth
• Earth’s capacity is estimated at 12 billion people
• It took 100 million years before Earth had 1 billion
people in 1830
• Yet only 170 years for the population to reach 6
billion plus
• 2005: 6,436,562,930
• 2009: 6,752,560,109
• ½ the people who have ever lived are on the
planet today
• Less than 100 years before we reach capacity
Biotechnology is needed to…
• Create better fuels that don’t harm
environment
• Create tools to clean environment, feed
a burgeoning global population, cure
untold human suffering
Biotechnology Definition
• Use of cellular and biomolecular processes to solve
problems or make useful products. Life
sciences…biology/chemistry technology affecting
discovery and development of products for:
–Healthcare (therapeutics, diagnostics, drug delivery, cell and
gene therapy, devices, drug/device combinations)
–Agriculture (food, feed, fibers, transgenics)
–Industrial and Environment (reduce pollution, clean energy)
• All driven by a new set of enabling technology
(genomics, combinatorial chemistry, SNPs, proteomics,
sequencing…)
The firsts……
Development is evolutionary…
4000-2000 BC: biotech
used to leaven bread &
ferment beer
1830: Proteins discovered
1833: First enzyme
discovered
1865: Genetic science
begins-Mendel discovers
laws of heredity
1879: Chromosomes
discovered
Development is evolutionary…
1906: The term genetics introduced
1919: The word biotechnology is first
used in print
1938: The term molecular biology coined
1941: The term genetic engineering is
first used
Development is evolutionary…
1953: Watson and Crick DNA
Structure
1958: DNA made in test tube
Sickle cell caused by AA change
1960: Messenger RNA discovered
1967: First automatic protein
sequencer is perfected
1969: Enzyme synthesized in
vitro the first time
1970: First enzyme discovered to
cut DNA molecules at a
specific site
Development is evolutionary…
1971: First complete synthesis of a gene
1973: First time DNA fragments linked
1975: First monoclonal antibodies made
1976: First NIH research guidelines
Boyer co-founds Genentech, 1st bio co.
1978: Recombinant insulin first produced
1980: Oil-eating microbes patented by Exxon
1982: First recombinant DNA vaccine for livestock
1983: First whole plant grown from biotechnology
Development is evolutionary…
1980: First gene-synthesizers
developed
1981: First transgenic animals
1982: First biotech drug: insulin
1983: First artificial
chromosome synthesized
First genetic markers for
inherited disease found
Development is evolutionary…
1984: DNA fingerprinting developed
1985: Genetic fingerprinting entered
as evidence in courtroom
1986: Interferon first anti-cancer drug
from biotech
First genetically engineered vaccine
for humans: Hepatitis B
Microbes used to clean oil spill
…but speeding up.
1988: First US patent for genetically altered
animal—a transgenic mouse
1989: First DNA exoneration now 216 (4/08)
1990: First food product from biotech
approved: modified yeast
1994: First FDA approval for first whole food
product: FLAVRSAVR™ tomato
1997: First weed & insect resistant crops
developed
First cloned animal: Hello Dolly!
…but speeding up.
1998: Human embryonic stem cells lines
established
Herceptin approved-considered first
pharmacogenomic (personalized) medicine
First complete animal genome: roundworm
2000: First complete map of a plant genome
First draft human genome
…but speeding up.
2004: First genetically modified pet: the
GloFish
FDA clears genotyping test to aid in
medication selection
First cloned pet, a kitten
2005: Skin cells converted to embryonic stem
cells
1 billionth acre biotech seed planted
First complete dog genome: boxer
2006: FDA approves Gardasil-first vaccine for
cancer-causing virus
…but speeding up.
2007: Successful reprogramming human skin cells
to create cells indistinguishable from embryonic
stem cells
Biotech cattle that cannot develop prions=no mad
cow disease
2008: draft corn genome
10 disease bearing stem cell lines created
Mature human embryos created from adult skin
cells: personalized stem cells for disease
treatment
First synthetic genome: the road to creating life
First complete map of cancer patient genome
First US FDA application for gene-therapy/cancer
2005 and Today
• 2005: Biotechnology will transform industries, including
health care
• Today: Biotechnology IS transforming industries
• Industrial convergence of farmers, doctors, drugmakers,
chemical processors, computer and communication
companies, energy companies and many others into the
business of life science.
• Is revolutionizing healthcare and transforming economics
of the Rx business. Will need to craft ways of dealing with
industry’s new economic landscape.
A single herd of goats may
soon replace a $150 million
drug factory…HBR 4/2000
2006: ATryn approved for DVT
The Human Genome & Biotechnology
• “A milestone in biology unlike any other.”
• “We’ve called the human genome the book of life, but
it’s really 3 books: It’s a history book. It’s a shop
manual and parts list. And, it’s a textbook of medicine
more profoundly detailed than ever.”
» --Francis Collins, former director NHRI director
• A short 50 years after the discovery of DNA by Watson
and Crick in 1953
• A complete list of coded instructions to make a person
• Would fill a stack of paperbacks 200 feet high
• 50 years to type at 8 hours/day, 60 wpm
The Human Genome: Fun Facts
• 30,000-40,000 genes not the 100-120,000 thought earlier
• Five times as many as in baker’s yeast
• About twice as many as that needed to grow a worm or
fly!
• Bananas share about ½ our genome while
mice share 90%!
• BUT, each single human gene can make 10 proteins vs. a
worm or fly’s genes making just one or two.
• “We have the Cuisinart vs. the paring knife
--Francis Collins
• 5 million strands of DNA can fit through the eye of a
needle
• All our DNA laid end to end would go to the sun and back
600 times!
• The genetic instructions for making a person take up less
than 1” of the 6-ft long strand of DNA in each cell
Since HGP…
• ENCODE: the Encyclopedia Of
DNA Elements, in 9/2003, to ID
all functional elements in the
human genome sequence
• HAPMAP: haplotype map, will
allow researchers to find
genes/genetic variations that
affect health and disease: 100
ID’d so far
• 1000 Genomes: extends HAPMAP
through global collaboration to
map 1,000 genomes in 3 years
Since HPG
• NIH Roadmap Project on genotype tissue
expression: are variants of disease risk
associated in relevant tissue
• Knock-Out Mouse Project (KOMP)
• Mammalian gene collection
• Cancer Genome Atlas: feasibility of full-scale
effort to systematically explore entire spectrum
of genomic changes involved in human cancer
• Molecular manual of disease created: 12/2008
• Proteinpedia being created: largest free
resource of experimental info human proteins
Goal
• Diagnostics to prevention
• Pharmacogenomic knowledge transfer
to therapeutics for gene therapy, drug
therapy
• Personalize genome + family
history=personalized medicine
• Other non-profit commercial efforts
underway
Other Efforts
• Bringing the genome into the light, Church says, is
the great project of our day.
• “To Church, who built his first computer at age 9 and
taught himself three programming languages by 15,
all of this is unfolding according to the same laws of
exponential progress that have propelled digital
technologies, from computer memory to the Internet
itself, over the past 40 years: Moore's law for circuits
and Metcalfe's law for networks. These principles are
now at play in genetics, he argues, particularly in
DNA sequencing and DNA synthesis.”
» ---Wired
Personal Genome Mapping
• Polonator G.007 $150K machine using
open architecture like IBM in 80’s
fueling PC revolution
• DeCodeMe
• 23andMe
• Navigenics
• KNOME
• Price range $400 to $350,000
Gene Map Becomes a Luxury Item…
• 3/2008
• “I’d rather spend my money on my genome than a
Bentley or an airplane.”
– Dan Stoicescu, millionaire retired Biotech exec
• X Prize Foundation: $10 million to first group to
sequence 100 human genomes in 10 days for $10K
or less
• Commercial goal: get price to $1K or less
• Has dropped by 4 orders of magnitude in 5 years
• Scientists call for better government regulation of
genetic tests
• DNA databases blocked from public by NIH after
study show a new type of DNA analysis could confirm
ID of individual in a pool of masked data if that
person’s gene profile was already known
Future Environment
Personalization of medicine
Present
Future
Disease by symptoms–diagnosis
Disease by mechanics–prognosis
Disease uniformity–guidelines,
formularies
Disease heterogeneity–
customization, targeted Rx
Patient uniformity–care standards
Patient variability–tailored care
Blockbusters–Universal Rx
Multibusters–pharmacogenetics
Consumer ignorance–acute care
Consumer empowerment–preventive
care
“Personalized Medicine Poised for
Progress in 2009”
• 12/24/2008
• “poised for dramatic progress in 2009”
in the clinic and laboratory
• “personal, predictive, preventive”
• “We’ve finally gotten to the point where
we can tell people about how their DNA
impacts their health,” Elissa Levin,
23andMe
Promises and Implications BIG*
Promise:
• Rapid technological innovation and vast number of new targets
identified and predictive tests
• Today 483 targets account for all Rx drugs marketed, tomorrow 510,000 targets
• Rapid acceleration in pace of new therapeutic introductions
• Drugs and treatments that are more tailored to specific patients
Implications:
• Development becomes a bottleneck
• Shorter product life cycles
• Market fragmentation
• Blurring distinction between “product” and “service”
* Gary Pisano, HBS 3/2000, adapted
Specialty Pharmaceuticals
• Created when many products moved from
“medical” side to the “pharmacy” side of the
budget=PBM control
• No specific criteria, but general attributes:
– Expense with annual therapy costs between
$20,000 and $250,000
– Treatment for chronic condition, possible lifetime
therapy
– Special handling, route of administration, patient
support
What Does It Mean to the Market?
• IMS Health estimates
specialty pharmacy sales of
$85 billion in 2008
• Expected to reach $100
billion in 2010
• 18% growth next 2 years
• Global biotech Rx sales grew
12.5% 2007 to $75 billion
• Biotech growing 2x pharma
(6.4%)
• US sales 56% of total ($42
billion)
Market Trends
• 22 biotech products generated
>$1 billion sales compared to 6
in 2002
• Biotech products represent
25% of the Rx pipeline
• http://www.phrma.org/files/Bio
tech%202008.pdf
Biotech Product Trends
• Development of biotech compounds is
explosive
• 633 biotech medicines in US pipeline
• 2001: Most compounds focused in the
oncology arena, followed by infectious
disease, CV and neuroscience
• 2007: Cancer 254, infectious disease 162,
autoimmune disease 59, HIV/AIDS 34, CV
25, diabetes 19
• Breakthrough treatments may provide
new hope of some diseases
Major Diseases
• Autoimmune: rheumatoid arthritis, MS,
lupus, chronic fatigue syndrome
• CV: CHF, MI, PAD, hypercholesterimia
• Gene disorders: CF, Gaucher’s
• ID: Hep A, B, C, tuberculosis, anthrax,
bird flu
• Neurodisorders: AD, PD, muscular
dystrophy, spinal cord injuries
Impact on People
•
•
•
•
•
•
•
•
Cancer
CVD
Alzheimer’s
Parkinson’s
MS
Diabetes
HIV
Hep A B C
1.5 million US
71 million US
5 million US
1 million US
400,000 US
24 million US
1.2 million US
>5 million US
$219 B
$403 B
$148 B
$35.5 B
$10 B
$132 B
$37 B
$3 B
Therapeutic Technologies
Pharming
• FDA guidelines
governing genetic
engineering of
animals for food,
drugs, or medical
devices
• Released 9/2008
. . . Contact us for the rest of
the presentation
• www.catalystenterprises.net
• [email protected]
• Tel: 651-905-9002