From good biotech to great biotech
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Description
Book Introduction
Vertex Pharmaceuticals and Regeneron Pharmaceuticals, once the targets of sarcasm about "geek scientists just making money."
However, at some point, the two biotechs became big biotechs with market capitalizations of $100 billion and are churning out new drugs.
We analyze how they went from good biotech to great biotech.
However, at some point, the two biotechs became big biotechs with market capitalizations of $100 billion and are churning out new drugs.
We analyze how they went from good biotech to great biotech.
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index
I.
There are many good biotechs, but great biotechs are rare.
Chapter 1: Good and Great Are Different 09
II.
Vertex
Chapter 2: Everyone Experiences Ordinary Failure 23
Chapter 3: The Only Certainty is Science 45
Chapter 4: Doing What You Need to Do 81
Chapter 5: Money, Time, and Science 95
III.
Regeneron
Chapter 6: Scientists Seem Like Cranky Guys 115
Chapter 7: A New Drug for Technology? Technology for New Drugs! 151
Chapter 8: Scientific Contracting 197
IV.
Great Biotech
Chapter 9: How Desperate Is It? 213
Chapter 10: Words Have Power 223
Chapter 11: It Must Be Science, It Must Be Scientists 233
From Good Biotech to Great Biotech 243
Reference 257
There are many good biotechs, but great biotechs are rare.
Chapter 1: Good and Great Are Different 09
II.
Vertex
Chapter 2: Everyone Experiences Ordinary Failure 23
Chapter 3: The Only Certainty is Science 45
Chapter 4: Doing What You Need to Do 81
Chapter 5: Money, Time, and Science 95
III.
Regeneron
Chapter 6: Scientists Seem Like Cranky Guys 115
Chapter 7: A New Drug for Technology? Technology for New Drugs! 151
Chapter 8: Scientific Contracting 197
IV.
Great Biotech
Chapter 9: How Desperate Is It? 213
Chapter 10: Words Have Power 223
Chapter 11: It Must Be Science, It Must Be Scientists 233
From Good Biotech to Great Biotech 243
Reference 257
Into the book
Joshua Boger envisioned a research organization that could create innovative new drugs.
The new research organization had to do away with the volume-centric screening method and the multi-layered decision-making structure that managed it, and put researchers directly in charge of new drug development.
Boger was thinking about how all researchers at Vertex could manage all new drug development projects together.
Researchers each carry out their own development projects, but share their project progress with other researchers.
I thought that researchers would help each other in these kinds of routine seminars or conferences.
It's a method that looks very similar to a graduate school research lab that studies basic science.
Boger also eliminated research positions, a move so radical that it was said to have created difficulties when recruiting researchers from outside the university because they could not offer positions.
--- p.34
Vertex was focused on causal relationships on an extremely small scale, but with such precision and certainty.
And we even developed a verification model to verify this more accurately.
It was scientific, although it seemed to take 'commerciality' out of consideration.
What's interesting is that once we identified a small but definite causal relationship, it began to expand.
Although it started out treating only about 4% of all cystic fibrosis (CF) patients, the identified causal relationships began to expand, leading to the development of new drugs.
About 10% of CF patients are not yet targeted by Vertex's new drug.
And if we can treat this 10% of patients, we can treat all CF patients.
--- pp.90-91
Developing a new drug takes at least 10 years, no matter how short the timeline.
It costs astronomical amounts of money and carries significant risks.
And Vertex CEO Joshua Boger knew exactly what this meant.
The important thing was to secure the money.
Vertex has consistently raised funds through large-scale funding.
And to spread the risk, I always had about 10 projects going on at the same time.
After all, not all new drug development projects can be successful.
Therefore, to quickly determine the likelihood of success of projects, discard those with low probability, and focus on those with high probability, you need to experience multiple failures simultaneously.
Working on multiple projects simultaneously also helps save time.
When it takes at least 10 years to determine the success of a project, it is too risky to move on to another project after completing one and checking the results.
Therefore, it is important to do multiple things simultaneously and to identify failures quickly.
It's a way to buy failure with money.
--- pp.97-98
Was Regeneron really just a bunch of eccentric researchers who couldn't make drugs and only gobbled up money? By tracking 220 marketed drugs as of 2013, we calculated the cost of each new drug for companies worldwide that had launched three or more new drugs over a 10-year period. The average R&D cost was $4.3 billion.
However, Regeneron only raised $736 million.
Even Pfizer spent an average of $7.8 billion to develop one new drug.
After developing a new drug and calculating it, Regeneron was developing the new drug in the most economical way.
Regeneron is a difficult biotech to explain.
--- pp.118-119
Vazelos's arrival at Regeneron sent shockwaves through the pharmaceutical industry.
When the legendary leader who led Merck announced that he would be leaving for a small biotech company run by eccentric scientists who were experiencing a series of failures, the media was in an uproar.
News was reported that it was joining a 'tiny biotech company' and a 'struggling biotech firm'.
Regeneron's stock price soared over 33% the day the news of the merger broke, sparking a flurry of speculation, but Vagelos's response was simple.
"I decided to join them not because of their recent failures, but because of their scientific credentials," said Vagelos. "I've been doing science my whole life, and I still miss it so much, and it gave me the opportunity to focus on science again."
He added that Regeneron's basic research is led by outstanding scientists, some of whom are performing research that is comparable to that of anyone else in the world, and that Regeneron is working in a rapidly growing field.
--- pp.138-139
How can biotech companies overcome the constraints of limited resources, low odds of success, and all-in on a small number of new drug development projects? Schleifer, Vazelos, and Yancopoulos agreed that the only way out of this predicament was through science.
Biotechnology is not a field where everything is staking its bets on a brilliant idea and a project derived from that idea, but rather a field that desperately needs logical new drug development based on science to increase the odds.
The reason Regeneron is failing repeatedly is because it does not yet have a logical and scientific new drug development system in place.
And this system was the platform.
Yancopoulos has a system to help investors determine which new drug development projects to pursue.
They said they would build (the technology), but investors asked back.
'So where is the product?'
--- p.157
In 2014, Regeneron established the Regeneron Genetic Center (RGC), a human genome sequencing big data research center.
RGC, created by Regeneron, is an independent subsidiary that operates like a small biotech startup.
As recently as 2014, the cost of sequencing a single human genome was in the thousands of dollars.
Therefore, biotech companies that do genome sequencing are often created for profit.
But Regeneron's approach was different.
The goal was to build a large-scale database for drug discovery by providing the most efficient sequencing capabilities and providing sequencing at cost.
And this data is used in Regeneron's new drug development process.
Nevertheless, the concept of RGC was one that set goals that a public research institute would aspire to.
--- p.171
Typically, 50:50 terms are seen in contracts between large pharmaceutical companies.
The agreement is made to accelerate development and divide the areas in which sales will be focused.
In other words, it is something that happens at the marketing strategy level when new drug development is nearing completion.
Two factors contributed to Regeneron's ability to partner with global pharmaceutical companies on nearly equal footing and favorable terms.
One is Regeneron's confidence in the science.
If you are precisely attacking the weak links in disease with "a science as solid as genetics," rather than a "case-testing game" of confirming ideas through countless experiments, you will be able to be confident in your science.
And if you have confidence in the science, you can say 50:50 at the negotiating table.
--- p.200
Science can fail at any time and in any way.
And this makes people behave conservatively.
The time it takes to develop a new drug is 10, 20, or 30 years.
In terms of personal time, this is the time it takes for a new researcher to start working at a biotech company until they reach retirement age.
In other words, conducting research on new drug development in biotechnology can be a lifelong adventure.
So while this long adventure unfolds,
It is also very difficult for Guza and his colleagues to maintain an objective attitude toward their project.
I feel attached to the project, and I have a feeling that it will be successful.
Maybe when a project stops, you don't feel like starting something again.
Researchers who fall into the trap will conduct clinical trials that only produce the results they want to see, and will avoid clinical trials that are likely to produce negative results.
There is a strange thing that happens where you don't do experiments that could kill the project, but do all the other experiments.
But if you're a biotech, and if you're a scientist, you have to do experiments that can kill your project as quickly as possible.
As Regeneron's Schleifer put it, "if you can catch failures early, like in Phase 1, you don't have to have a big messy funeral in the final Phase 3 trial."
--- pp.217-218
Joshua Boger believes that clinical development decisions should be based solely on facts, but that making decisions based solely on facts requires money, and that money should come from the revenue generated by the biotech, not the money received from investments.
Even though it might take 10 years after starting a biotech business to make money, I thought that we needed to create a culture that thinks about sales from the day we started biotech.
In fact, Vertex has spent nearly $3.4 billion since its founding as of 2024, but has earned nearly $1.7 billion.
Such sales would have given Vertex the strength to continue developing new drugs based solely on facts, without being swayed by other positions.
It might have been possible to decide to conduct an experiment that would kill the project.
--- p.220
'If I hesitate before making an investment, it is not because the investment is too risky.
Because the value of what you are trying to invest in is small.
If the value is high, you can take the risk, but if the value is low, you will always be anxious about when to get out.
In new drug development, the value is science.
If there was science, we would take risks and invest, but if there was no science, we would be anxious all the time.
Both Vertex and Regeneron are biotechs that have taken risks with science.
If this is called greatness, then humanity owes a debt to it.
To those who have taken a great step forward in the face of the value of science that truly saves people.
And I am willing to invest in this greatness.'
The new research organization had to do away with the volume-centric screening method and the multi-layered decision-making structure that managed it, and put researchers directly in charge of new drug development.
Boger was thinking about how all researchers at Vertex could manage all new drug development projects together.
Researchers each carry out their own development projects, but share their project progress with other researchers.
I thought that researchers would help each other in these kinds of routine seminars or conferences.
It's a method that looks very similar to a graduate school research lab that studies basic science.
Boger also eliminated research positions, a move so radical that it was said to have created difficulties when recruiting researchers from outside the university because they could not offer positions.
--- p.34
Vertex was focused on causal relationships on an extremely small scale, but with such precision and certainty.
And we even developed a verification model to verify this more accurately.
It was scientific, although it seemed to take 'commerciality' out of consideration.
What's interesting is that once we identified a small but definite causal relationship, it began to expand.
Although it started out treating only about 4% of all cystic fibrosis (CF) patients, the identified causal relationships began to expand, leading to the development of new drugs.
About 10% of CF patients are not yet targeted by Vertex's new drug.
And if we can treat this 10% of patients, we can treat all CF patients.
--- pp.90-91
Developing a new drug takes at least 10 years, no matter how short the timeline.
It costs astronomical amounts of money and carries significant risks.
And Vertex CEO Joshua Boger knew exactly what this meant.
The important thing was to secure the money.
Vertex has consistently raised funds through large-scale funding.
And to spread the risk, I always had about 10 projects going on at the same time.
After all, not all new drug development projects can be successful.
Therefore, to quickly determine the likelihood of success of projects, discard those with low probability, and focus on those with high probability, you need to experience multiple failures simultaneously.
Working on multiple projects simultaneously also helps save time.
When it takes at least 10 years to determine the success of a project, it is too risky to move on to another project after completing one and checking the results.
Therefore, it is important to do multiple things simultaneously and to identify failures quickly.
It's a way to buy failure with money.
--- pp.97-98
Was Regeneron really just a bunch of eccentric researchers who couldn't make drugs and only gobbled up money? By tracking 220 marketed drugs as of 2013, we calculated the cost of each new drug for companies worldwide that had launched three or more new drugs over a 10-year period. The average R&D cost was $4.3 billion.
However, Regeneron only raised $736 million.
Even Pfizer spent an average of $7.8 billion to develop one new drug.
After developing a new drug and calculating it, Regeneron was developing the new drug in the most economical way.
Regeneron is a difficult biotech to explain.
--- pp.118-119
Vazelos's arrival at Regeneron sent shockwaves through the pharmaceutical industry.
When the legendary leader who led Merck announced that he would be leaving for a small biotech company run by eccentric scientists who were experiencing a series of failures, the media was in an uproar.
News was reported that it was joining a 'tiny biotech company' and a 'struggling biotech firm'.
Regeneron's stock price soared over 33% the day the news of the merger broke, sparking a flurry of speculation, but Vagelos's response was simple.
"I decided to join them not because of their recent failures, but because of their scientific credentials," said Vagelos. "I've been doing science my whole life, and I still miss it so much, and it gave me the opportunity to focus on science again."
He added that Regeneron's basic research is led by outstanding scientists, some of whom are performing research that is comparable to that of anyone else in the world, and that Regeneron is working in a rapidly growing field.
--- pp.138-139
How can biotech companies overcome the constraints of limited resources, low odds of success, and all-in on a small number of new drug development projects? Schleifer, Vazelos, and Yancopoulos agreed that the only way out of this predicament was through science.
Biotechnology is not a field where everything is staking its bets on a brilliant idea and a project derived from that idea, but rather a field that desperately needs logical new drug development based on science to increase the odds.
The reason Regeneron is failing repeatedly is because it does not yet have a logical and scientific new drug development system in place.
And this system was the platform.
Yancopoulos has a system to help investors determine which new drug development projects to pursue.
They said they would build (the technology), but investors asked back.
'So where is the product?'
--- p.157
In 2014, Regeneron established the Regeneron Genetic Center (RGC), a human genome sequencing big data research center.
RGC, created by Regeneron, is an independent subsidiary that operates like a small biotech startup.
As recently as 2014, the cost of sequencing a single human genome was in the thousands of dollars.
Therefore, biotech companies that do genome sequencing are often created for profit.
But Regeneron's approach was different.
The goal was to build a large-scale database for drug discovery by providing the most efficient sequencing capabilities and providing sequencing at cost.
And this data is used in Regeneron's new drug development process.
Nevertheless, the concept of RGC was one that set goals that a public research institute would aspire to.
--- p.171
Typically, 50:50 terms are seen in contracts between large pharmaceutical companies.
The agreement is made to accelerate development and divide the areas in which sales will be focused.
In other words, it is something that happens at the marketing strategy level when new drug development is nearing completion.
Two factors contributed to Regeneron's ability to partner with global pharmaceutical companies on nearly equal footing and favorable terms.
One is Regeneron's confidence in the science.
If you are precisely attacking the weak links in disease with "a science as solid as genetics," rather than a "case-testing game" of confirming ideas through countless experiments, you will be able to be confident in your science.
And if you have confidence in the science, you can say 50:50 at the negotiating table.
--- p.200
Science can fail at any time and in any way.
And this makes people behave conservatively.
The time it takes to develop a new drug is 10, 20, or 30 years.
In terms of personal time, this is the time it takes for a new researcher to start working at a biotech company until they reach retirement age.
In other words, conducting research on new drug development in biotechnology can be a lifelong adventure.
So while this long adventure unfolds,
It is also very difficult for Guza and his colleagues to maintain an objective attitude toward their project.
I feel attached to the project, and I have a feeling that it will be successful.
Maybe when a project stops, you don't feel like starting something again.
Researchers who fall into the trap will conduct clinical trials that only produce the results they want to see, and will avoid clinical trials that are likely to produce negative results.
There is a strange thing that happens where you don't do experiments that could kill the project, but do all the other experiments.
But if you're a biotech, and if you're a scientist, you have to do experiments that can kill your project as quickly as possible.
As Regeneron's Schleifer put it, "if you can catch failures early, like in Phase 1, you don't have to have a big messy funeral in the final Phase 3 trial."
--- pp.217-218
Joshua Boger believes that clinical development decisions should be based solely on facts, but that making decisions based solely on facts requires money, and that money should come from the revenue generated by the biotech, not the money received from investments.
Even though it might take 10 years after starting a biotech business to make money, I thought that we needed to create a culture that thinks about sales from the day we started biotech.
In fact, Vertex has spent nearly $3.4 billion since its founding as of 2024, but has earned nearly $1.7 billion.
Such sales would have given Vertex the strength to continue developing new drugs based solely on facts, without being swayed by other positions.
It might have been possible to decide to conduct an experiment that would kill the project.
--- p.220
'If I hesitate before making an investment, it is not because the investment is too risky.
Because the value of what you are trying to invest in is small.
If the value is high, you can take the risk, but if the value is low, you will always be anxious about when to get out.
In new drug development, the value is science.
If there was science, we would take risks and invest, but if there was no science, we would be anxious all the time.
Both Vertex and Regeneron are biotechs that have taken risks with science.
If this is called greatness, then humanity owes a debt to it.
To those who have taken a great step forward in the face of the value of science that truly saves people.
And I am willing to invest in this greatness.'
--- pp.255-256
Publisher's Review
Life, Health, and Biotechnology
Cystic fibrosis (CF) is a rare genetic disease.
People with cystic fibrosis experience a buildup of sticky mucus in their lungs, bronchial tubes, and digestive tract.
The sticky mucus interferes with the patient's normal breathing and nutrient absorption, which limits their life expectancy to around 20 years.
However, American biotech company Vertex Pharmaceuticals (hereinafter referred to as Vertex) developed 'Kalideco', a treatment for cystic fibrosis.
There is a photo related to this hanging in the Vertex cafeteria.
This is a photo of the instep with an unknown chemical structure drawn on it. (From Good Biotech to Great Biotech, p. 238) The subject of the photo was a patient suffering from cystic fibrosis.
He was able to save his life with Kalydeco, developed by Vertex.
He had the chemical formula for Kalydeco, which saved his life, tattooed on the top of his foot and took a picture.
And around the photo, the new drug development team that participated in the development of Kalydeco left their signatures, and Vertex proudly framed them and hung them.
This scene makes us rethink what it means to develop a new drug.
Biotech companies developing new drugs are for-profit companies driven by the desire to make money, but the value of their work cannot be measured solely by sales, operating profit, and market capitalization.
Saving and restoring life is a priceless value to patients and their families.
However, although all biotechs challenge the precious value of new drug development, not all succeed.
In the United States, where biotech new drug development is most active, there are approximately 5,000 biotech companies, but only about 10 new drugs are released each year.
The development of new biotech drugs can save patients' lives and health, and even bring them immense wealth and fame.
But why do some biotechs succeed in developing new drugs, while others don't? "From Good to Great Biotech" begins with this question.
Can you tell the difference between bubble and value?
Author Kim Seong-min is a journalist who specializes in covering trends in biopharmaceutical companies and the current state of new drug development around the world.
Given the nature of the pharmaceutical industry and new drug development, which relies heavily on cutting-edge science and technology, the author has focused on analyzing papers and clinical trial data published by biotech and pharmaceutical companies.
But at some point, I began to think that the content I analyzed to write articles every day could evaporate and disappear.
And before the content, which might one day be helpful to someone's new drug development, is scattered, I started the work of collecting and fixing it into a book.
Commentary on cutting-edge science and technology used in new drug development (『Understanding Bioscience』 1st edition [2017], revised 2nd edition [2023]), an overview of challenging research situations such as the development of new drugs for Alzheimer's disease (??How to Fix the Brain - Focusing on the Development of New Drugs for Alzheimer's Disease?? [2019]), an introduction to fields where Korea can quickly achieve results such as diagnosis and medical AI (『Diagnosis as a New Drug - Early Diagnosis, Companion Diagnosis, Metastatic Cancer Diagnosis, Imaging Markers』 [2020]), and the development story of Keytruda, an immune checkpoint inhibitor that is evaluated to be changing the concept of cancer treatment (『Keytruda Story - How Merck Succeeded in Immune Oncology』 [2022]) were works that fixed into a book the analysis content that the author had produced every day for the past 7 years.
As the author completed his four-volume work, he wanted to find a way to determine whether biotech, which is challenging itself with new drug development, is a bubble or contains value.
People who develop new drugs, people who invest in new drug development, and almost everyone I meet through reporting have wondered about the value of biotechnology.
This curiosity has repeatedly led to biobooms and bioinvestment ice ages.
Is there any way to determine whether biotech is a bubble or harboring hidden value? It shouldn't be done by a wizard-like method of picking promising biotechs or providing a checklist of key factors.
Just as previous work had been a scientific approach based on papers and clinical trial analyses, the work of distinguishing between bubbles and value also had to be scientific.
The author decided to first select biotechs that are valuable from a current perspective and will remain valuable for the foreseeable future.
From good biotech to great biotech
Money doesn't tell everything, but it does tell a lot.
Vertex and Regeneron Pharmaceuticals (hereafter referred to as Regeneron) are biotech companies that opened around the same time in the late 1980s.
These two biotechs are expected to surpass $100 billion in market capitalization in early 2024.
A market capitalization of $100 billion is comparable to global pharmaceutical companies like Bristol-Myers Squibb (BMS) and Pfizer. BMS and Pfizer are global pharmaceutical companies, several times, or even dozens of times, larger than Vertex and Regeneron in terms of size, revenue, and history.
However, Vertex and Regeneron are valued similarly in the market to these large pharmaceutical companies.
In other words, the market's assessment of Vertex and Regeneron was that they were 'valuable.'
What exactly happened at Vertex and Regeneron that allowed them to create this much value?
"From Good Biotech to Great Biotech" examines the poor decisions Vertex and Regeneron made and how they led to their failure.
The two biotechs would certainly have been similar to ordinary biotechs.
Therefore, it was important to identify the mistakes that Vertex and Regeneron made and examine how they overcame them.
For example, Vertex and Regeneron also fell into the trap of confirmation bias, a type of bias that other good biotechs have fallen into.
The process of developing ideas and hypotheses based on cutting-edge science and cutting-edge technology and proving them is an adventure of embarking on a path no one has ever traveled before.
Biotechnology, which takes on uncharted paths, requires confidence in one's own science and technology, and confidence in one's ideas and hypotheses.
However, if this kind of confidence is distorted, it is easy to fall into confirmation bias, seeing only what you want to see and hearing only what you want to hear.
Especially, the better biotech companies that work hard at science are more likely to fall into this trap.
The hard work, diligent research, the expectation that one's hypotheses and ideas will lead to the development of new drugs, and the regret over the money and time already invested, all lead biotech companies to act in a biased manner.
It is to avoid experiments that can confirm that hypotheses and ideas are wrong, and to focus on experiments and research other than those.
Both Vertex and Regeneron fell into this trap, but after a long period of trial and error, they were finally able to escape.
What exactly was the secret to the escape of these two biotech companies? "From Good to Great" specifically confirms and proves that the secret to Vertex and Regeneron's escape was "transforming good science into great science."
In fact, from good science to great science
And the people who walk that path
Both new drug development and biotechnology are ultimately done by people.
For this reason, the author also focuses on observations of people.
By following the thoughts, judgments, and actions of Joshua Boger, the physician and scientist who founded Vertex, and Jeffrey Leiden, who succeeded him in designing and implementing today's Vertex, you will understand why Vertex is jumping into the development of treatments for rare and intractable genetic diseases that are often thought to have little marketability due to the small number of patients.
The people at Vertex developed new drugs to fight human immunodeficiency virus (HIV) and hepatitis C virus (HCV) infections, but failed to commercialize them.
In this way, Vertex went through a period of mediocre success and mediocre failure, but it entered the path of great biotech by moving into the development of a new drug for cystic fibrosis, a rare and incurable genetic disease.
The story of those who led Regeneron unfolds in a more exciting way.
Genentech was the first biotech to genetically engineer an insulin-secreting E. coli strain.
Leonard Schleifer, a scientist and physician who had tried to develop a treatment for Lou Gehrig's disease by founding a biotech company like Genentech, started Regeneron despite opposition from those around him.
Leonard Schleifer recruits a young, science-crazed researcher, George Yancopoulos, and hatches an ambitious plan to develop a cure for Lou Gehrig's disease, Alzheimer's disease, and Parkinson's disease, but it fails miserably.
And to properly develop new drugs, they recruit Roy Vagelos, a scientist-turned-manager who had led a large pharmaceutical company.
These three people are establishing their own new drug development process by changing the problems of Regeneron's new drug development one by one.
"From Good Biotech to Great Biotech" follows a group of ordinary scientists as they encounter the mistakes and errors typical of biotech, but ultimately develop their own formula for new drug development.
And we reaffirm that the turning point that enabled ordinary, good biotech to become great biotech was when they accepted the very obvious proposition of 'doing science properly and with passion.'
Discovered in great biotech
Scientific Leadership
This book was set out to explore what it takes to successfully develop a new drug, a task often considered more difficult than a camel passing through the eye of a needle.
It was intended to be helpful to the numerous members of the biotech industry who are currently working on new drug development in laboratories and clinical trial sites, as well as to future researchers who will develop new drugs in the future.
But apart from the good intentions of developing new drugs to save patients from disease, the efforts of those who strive for scientific achievement, and the risky and arduous process that requires a considerable amount of time and resources, all of this becomes valuable only when a new drug is actually developed.
And value can only be created by being a good biotech, not a great biotech like Vertex or Regeneron.
The author concludes the book by defining the scientific leadership necessary to become a great biotech and listing the conditions for acquiring scientific leadership.
This work also helps those who wish to participate in the valuable work of new drug development through biotechnology investment in their decision-making.
When Vertex and Regeneron started, famous biotechs including Genentech were already active(?).
And the gap between Vertex, Regeneron and Genentech was about 20 years.
It's similar to the 20-year gap between the development of biopharmaceuticals in Korea and the development of biopharmaceuticals in the United States.
These realistic conditions make those who participate in new drug development hesitant to invest.
"Can new drugs be developed in Korea?" "Is my investment in Korean biotech companies worthwhile?" "Which biotech companies should I invest in, and which should I avoid?" Those about to invest face deepening concerns, and biotech companies that cannot develop new drugs without investment face difficulties.
The rules of new drug development that the author discovered at Vertex and Regeneron were the answers to questions such as, "Does biotech have scientific leadership?", "Does biotech specifically declare its vision and mission to develop new drugs through science, and does it strongly share and uphold this vision and mission with internal and external members?", and "Does biotech wholeheartedly and passionately pursue only science and innovation that can change patients' lives?"
Any biotech that can answer this question specifically is either already a great biotech or will become one, like Vertex and Regeneron.
Readers will be able to remember the conditions for great biotechnology revealed throughout this book and apply them one by one when making investment decisions.
Cystic fibrosis (CF) is a rare genetic disease.
People with cystic fibrosis experience a buildup of sticky mucus in their lungs, bronchial tubes, and digestive tract.
The sticky mucus interferes with the patient's normal breathing and nutrient absorption, which limits their life expectancy to around 20 years.
However, American biotech company Vertex Pharmaceuticals (hereinafter referred to as Vertex) developed 'Kalideco', a treatment for cystic fibrosis.
There is a photo related to this hanging in the Vertex cafeteria.
This is a photo of the instep with an unknown chemical structure drawn on it. (From Good Biotech to Great Biotech, p. 238) The subject of the photo was a patient suffering from cystic fibrosis.
He was able to save his life with Kalydeco, developed by Vertex.
He had the chemical formula for Kalydeco, which saved his life, tattooed on the top of his foot and took a picture.
And around the photo, the new drug development team that participated in the development of Kalydeco left their signatures, and Vertex proudly framed them and hung them.
This scene makes us rethink what it means to develop a new drug.
Biotech companies developing new drugs are for-profit companies driven by the desire to make money, but the value of their work cannot be measured solely by sales, operating profit, and market capitalization.
Saving and restoring life is a priceless value to patients and their families.
However, although all biotechs challenge the precious value of new drug development, not all succeed.
In the United States, where biotech new drug development is most active, there are approximately 5,000 biotech companies, but only about 10 new drugs are released each year.
The development of new biotech drugs can save patients' lives and health, and even bring them immense wealth and fame.
But why do some biotechs succeed in developing new drugs, while others don't? "From Good to Great Biotech" begins with this question.
Can you tell the difference between bubble and value?
Author Kim Seong-min is a journalist who specializes in covering trends in biopharmaceutical companies and the current state of new drug development around the world.
Given the nature of the pharmaceutical industry and new drug development, which relies heavily on cutting-edge science and technology, the author has focused on analyzing papers and clinical trial data published by biotech and pharmaceutical companies.
But at some point, I began to think that the content I analyzed to write articles every day could evaporate and disappear.
And before the content, which might one day be helpful to someone's new drug development, is scattered, I started the work of collecting and fixing it into a book.
Commentary on cutting-edge science and technology used in new drug development (『Understanding Bioscience』 1st edition [2017], revised 2nd edition [2023]), an overview of challenging research situations such as the development of new drugs for Alzheimer's disease (??How to Fix the Brain - Focusing on the Development of New Drugs for Alzheimer's Disease?? [2019]), an introduction to fields where Korea can quickly achieve results such as diagnosis and medical AI (『Diagnosis as a New Drug - Early Diagnosis, Companion Diagnosis, Metastatic Cancer Diagnosis, Imaging Markers』 [2020]), and the development story of Keytruda, an immune checkpoint inhibitor that is evaluated to be changing the concept of cancer treatment (『Keytruda Story - How Merck Succeeded in Immune Oncology』 [2022]) were works that fixed into a book the analysis content that the author had produced every day for the past 7 years.
As the author completed his four-volume work, he wanted to find a way to determine whether biotech, which is challenging itself with new drug development, is a bubble or contains value.
People who develop new drugs, people who invest in new drug development, and almost everyone I meet through reporting have wondered about the value of biotechnology.
This curiosity has repeatedly led to biobooms and bioinvestment ice ages.
Is there any way to determine whether biotech is a bubble or harboring hidden value? It shouldn't be done by a wizard-like method of picking promising biotechs or providing a checklist of key factors.
Just as previous work had been a scientific approach based on papers and clinical trial analyses, the work of distinguishing between bubbles and value also had to be scientific.
The author decided to first select biotechs that are valuable from a current perspective and will remain valuable for the foreseeable future.
From good biotech to great biotech
Money doesn't tell everything, but it does tell a lot.
Vertex and Regeneron Pharmaceuticals (hereafter referred to as Regeneron) are biotech companies that opened around the same time in the late 1980s.
These two biotechs are expected to surpass $100 billion in market capitalization in early 2024.
A market capitalization of $100 billion is comparable to global pharmaceutical companies like Bristol-Myers Squibb (BMS) and Pfizer. BMS and Pfizer are global pharmaceutical companies, several times, or even dozens of times, larger than Vertex and Regeneron in terms of size, revenue, and history.
However, Vertex and Regeneron are valued similarly in the market to these large pharmaceutical companies.
In other words, the market's assessment of Vertex and Regeneron was that they were 'valuable.'
What exactly happened at Vertex and Regeneron that allowed them to create this much value?
"From Good Biotech to Great Biotech" examines the poor decisions Vertex and Regeneron made and how they led to their failure.
The two biotechs would certainly have been similar to ordinary biotechs.
Therefore, it was important to identify the mistakes that Vertex and Regeneron made and examine how they overcame them.
For example, Vertex and Regeneron also fell into the trap of confirmation bias, a type of bias that other good biotechs have fallen into.
The process of developing ideas and hypotheses based on cutting-edge science and cutting-edge technology and proving them is an adventure of embarking on a path no one has ever traveled before.
Biotechnology, which takes on uncharted paths, requires confidence in one's own science and technology, and confidence in one's ideas and hypotheses.
However, if this kind of confidence is distorted, it is easy to fall into confirmation bias, seeing only what you want to see and hearing only what you want to hear.
Especially, the better biotech companies that work hard at science are more likely to fall into this trap.
The hard work, diligent research, the expectation that one's hypotheses and ideas will lead to the development of new drugs, and the regret over the money and time already invested, all lead biotech companies to act in a biased manner.
It is to avoid experiments that can confirm that hypotheses and ideas are wrong, and to focus on experiments and research other than those.
Both Vertex and Regeneron fell into this trap, but after a long period of trial and error, they were finally able to escape.
What exactly was the secret to the escape of these two biotech companies? "From Good to Great" specifically confirms and proves that the secret to Vertex and Regeneron's escape was "transforming good science into great science."
In fact, from good science to great science
And the people who walk that path
Both new drug development and biotechnology are ultimately done by people.
For this reason, the author also focuses on observations of people.
By following the thoughts, judgments, and actions of Joshua Boger, the physician and scientist who founded Vertex, and Jeffrey Leiden, who succeeded him in designing and implementing today's Vertex, you will understand why Vertex is jumping into the development of treatments for rare and intractable genetic diseases that are often thought to have little marketability due to the small number of patients.
The people at Vertex developed new drugs to fight human immunodeficiency virus (HIV) and hepatitis C virus (HCV) infections, but failed to commercialize them.
In this way, Vertex went through a period of mediocre success and mediocre failure, but it entered the path of great biotech by moving into the development of a new drug for cystic fibrosis, a rare and incurable genetic disease.
The story of those who led Regeneron unfolds in a more exciting way.
Genentech was the first biotech to genetically engineer an insulin-secreting E. coli strain.
Leonard Schleifer, a scientist and physician who had tried to develop a treatment for Lou Gehrig's disease by founding a biotech company like Genentech, started Regeneron despite opposition from those around him.
Leonard Schleifer recruits a young, science-crazed researcher, George Yancopoulos, and hatches an ambitious plan to develop a cure for Lou Gehrig's disease, Alzheimer's disease, and Parkinson's disease, but it fails miserably.
And to properly develop new drugs, they recruit Roy Vagelos, a scientist-turned-manager who had led a large pharmaceutical company.
These three people are establishing their own new drug development process by changing the problems of Regeneron's new drug development one by one.
"From Good Biotech to Great Biotech" follows a group of ordinary scientists as they encounter the mistakes and errors typical of biotech, but ultimately develop their own formula for new drug development.
And we reaffirm that the turning point that enabled ordinary, good biotech to become great biotech was when they accepted the very obvious proposition of 'doing science properly and with passion.'
Discovered in great biotech
Scientific Leadership
This book was set out to explore what it takes to successfully develop a new drug, a task often considered more difficult than a camel passing through the eye of a needle.
It was intended to be helpful to the numerous members of the biotech industry who are currently working on new drug development in laboratories and clinical trial sites, as well as to future researchers who will develop new drugs in the future.
But apart from the good intentions of developing new drugs to save patients from disease, the efforts of those who strive for scientific achievement, and the risky and arduous process that requires a considerable amount of time and resources, all of this becomes valuable only when a new drug is actually developed.
And value can only be created by being a good biotech, not a great biotech like Vertex or Regeneron.
The author concludes the book by defining the scientific leadership necessary to become a great biotech and listing the conditions for acquiring scientific leadership.
This work also helps those who wish to participate in the valuable work of new drug development through biotechnology investment in their decision-making.
When Vertex and Regeneron started, famous biotechs including Genentech were already active(?).
And the gap between Vertex, Regeneron and Genentech was about 20 years.
It's similar to the 20-year gap between the development of biopharmaceuticals in Korea and the development of biopharmaceuticals in the United States.
These realistic conditions make those who participate in new drug development hesitant to invest.
"Can new drugs be developed in Korea?" "Is my investment in Korean biotech companies worthwhile?" "Which biotech companies should I invest in, and which should I avoid?" Those about to invest face deepening concerns, and biotech companies that cannot develop new drugs without investment face difficulties.
The rules of new drug development that the author discovered at Vertex and Regeneron were the answers to questions such as, "Does biotech have scientific leadership?", "Does biotech specifically declare its vision and mission to develop new drugs through science, and does it strongly share and uphold this vision and mission with internal and external members?", and "Does biotech wholeheartedly and passionately pursue only science and innovation that can change patients' lives?"
Any biotech that can answer this question specifically is either already a great biotech or will become one, like Vertex and Regeneron.
Readers will be able to remember the conditions for great biotechnology revealed throughout this book and apply them one by one when making investment decisions.
GOODS SPECIFICS
- Date of issue: October 20, 2024
- Page count, weight, size: 268 pages | 140*215*20mm
- ISBN13: 9791191768091
- ISBN10: 1191768090
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