Why biopharmaceuticals again?
The importance of biopharmaceuticals has grown!

Biopharmaceuticals are attracting attention as a new growth engine that will lead Korea's Fourth Industrial Revolution, and their importance in the therapeutics field has recently grown.
Most biopharmaceuticals are administered by injection into the bloodstream, subcutaneously, or intramuscularly, and this fact alone does not make them any different from other types of drugs.
However, biopharmaceuticals are completely different drugs from existing synthetic drugs.
Currently, biopharmaceuticals account for 25% of the total pharmaceutical market, and 7 of the top 10 best-selling drugs in 2016 were biopharmaceuticals, making them a significant part of the pharmaceutical market.
Moreover, the biopharmaceutical market is already a huge industry with over 67 trillion won invested annually in research and development worldwide, but the general public is still unfamiliar with biopharmaceuticals.
You may think that life sciences are not relevant to your life, or that it is impossible for a non-expert to stay up to date on the rapidly evolving field of life sciences.
However, even if you don't fully understand the detailed academic and technical details of biopharmaceuticals, you can still get a sense of what biopharmaceuticals are and what impact they will have on our daily lives.


This book provides an easy-to-understand introduction to the different types of biopharmaceuticals, why you should be interested in biopharmaceuticals now, and the key regulatory issues in biopharmaceutical development and approval.
In addition, we discuss the direction in which the Korean bio industry should move forward by examining why there are both bright and dark sides to biosimilars and why Korean biopharmaceutical companies have recently failed clinical trials.
It also provides helpful information and key considerations when investing in biopharmaceutical stocks.
The COVID-19 pandemic, which has upended the lives of people around the world over the past four years, has also brought about a crucial turning point from a biopharmaceutical perspective.
The successful commercialization of mRNA vaccines and the widespread adoption of COVID-19 diagnostic kits utilizing the principles of polymerase chain reaction (PCR) and rapid antigen response have significant implications within the broader category of "bio."
As technology in the biopharmaceutical field rapidly develops, a revised and expanded edition was published, supplementing some of the content included in the first edition and adding new topics.
Because it provides an overview of biopharmaceuticals and assesses their future prospects, it will be a very useful book for not only students majoring in biopharmaceuticals/pharmaceutical science, but also those working in the biopharmaceutical industry, investors focusing on the bio industry, and the general public interested in biopharmaceutical treatment.

When the general public hears the word medicine, they think of the medicines displayed in pharmacies.
Most drugs available at pharmacies are synthetic drugs, meaning they are drugs in the traditional sense of the word, produced through chemical reactions.
So, what are biopharmaceuticals? First of all, it's clear that biopharmaceuticals are also drugs.
Biopharmaceuticals, like synthetic drugs, are substances used to treat or alleviate diseases, alleviate symptoms, or prevent diseases.
So we have to ask:
What "properties" of biopharmaceuticals differ from those of synthetic drugs? To cut to the chase, biopharmaceuticals are drugs "extracted or produced from biological sources."
To properly understand this definition, we must first understand the process by which drugs are developed and approved.

--- p.25, Chapter 1.
From the “Definition of Biopharmaceuticals”

Let's say you're playing with Legos.
There are two sets of Lego blocks, the first set contains four different types of blocks.
The blocks are almost the same shape, but their colors are slightly different.
The second Lego set, on the other hand, contains 20 different types of blocks.
Moreover, the shapes of the blocks are diverse and all blocks are connected to each other.
So which of the two LEGO sets would allow you to create more diverse structures? The second set, of course.
The synthesis of nucleic acids and proteins resembles the behavior of two Lego sets. DNA and RNA each consist of only four types of bases, and even these have similar structures.
On the other hand, there are approximately 20 types of amino acids that make up proteins, and their structures are very diverse.
Naturally, proteins, which are composed of units (amino acids) of various types and shapes, have more diverse structures than DNA or RNA.

--- p.50, Chapter 2.
From “Fundamentals of Modern Biology”

Criticism has been raised that applying double-blind, randomized, placebo-controlled research methodology to clinical trials of COVID-19 vaccines that have received emergency use authorization or conditional approval is unethical.
Accordingly, in the clinical trials conducted by Moderna and Pfizer in the United States to submit additional data, blinding was lifted and all participants were vaccinated.
In addition, the difficulty of securing a sufficient number of participants was raised as a problem in a situation where vaccines that have already received emergency use approval and conditional approval are being widely administered.
As COVID-19 vaccinations roll out globally, many people have already received existing COVID-19 vaccines.
However, it is difficult to accurately evaluate the efficacy of a new vaccine if clinical trials are conducted on people who have been vaccinated with existing vaccines.
However, if the study were conducted only on people who have not been vaccinated with the existing COVID-19 vaccine, it would be difficult to secure a sufficient number of participants.
COVID-19 is still ongoing and new variants are constantly emerging.
Therefore, efforts to develop a COVID-19 vaccine must continue, and new approaches must be discussed for future COVID-19 vaccine clinical trials.
In particular, there is a growing need to establish criteria and methods for selecting suitable participants and to develop a clinical trial design that can accurately confirm the efficacy of drugs without using a placebo control condition.

--- p.141~142, Chapter 6.
Among the "vaccines"

Pharmaceutical companies have long been reluctant to develop gene therapy drugs because they have caused serious and unpredictable adverse reactions in clinical trials.
Moreover, because we do not understand the mechanisms by which adverse reactions occur or why adverse reactions (immune reactions) occur only in certain patients, patients receiving gene therapy must be closely monitored after receiving the treatment.
It is important to periodically check whether the patient's immune system is functioning properly so that adverse reactions can be dealt with quickly when they occur.
Because gene therapy requires a lot of medical resources, the burden of medical expenses on patients also increases.

--- p.153~154, 「Chapter 7.
Among “cell and gene therapy”

Despite risks hindering development and market entry, the biosimilars market is growing rapidly.
There are still expectations that the introduction of biosimilars will lower drug prices.
This is because there are many strategic options available to latecomers developing biosimilars.
In the case of biopharmaceuticals, “the process is the product,” so even if production technology is innovatively improved, it is difficult for original biopharmaceuticals to fully improve their processes, making it difficult to benefit from innovative technologies.
However, latecomers can gain an advantage over standard biopharmaceuticals by leveraging innovative technologies to increase production efficiency and reduce costs.

--- p.206, Chapter 9.
Why are biosimilars not biogenerics?

In November 2016, ShillaJen began a global phase 3 clinical trial of the anticancer drug 'Pexa-Vec'.
Pexa-Vec is an anticancer drug made by recombining the genes of the vaccinia virus, and is an anticancer virus that selectively infects only cancer cells.
Pexa-Vec, which uses a genetic recombination method to remove thymidine kinase, an enzyme that is highly expressed in cancer cells, instead steals thymidine kinase from cancer cells.
Because thymidine kinase is important for the growth and proliferation of viruses, cancer cells that are deprived of thymidine kinase are eventually destroyed and killed by the body's immune cells.
Additionally, Pexa-Vec contains the GM-CSF gene, which activates the immune system, making it more effective in attacking cancer cells.
Thanks to this unique mechanism, it was called a “dream drug” as it cured two liver cancer patients in a phase 2a clinical trial involving 30 patients with terminal liver cancer.
However, the global phase 3 clinical trial of 'Pexa-Vec', which was being conducted in 16 countries around the world targeting patients with terminal liver cancer, was halted in August 2019 at the recommendation of the Data Monitoring Committee (DMC).
This is because there was no significant difference in the survival period after drug administration between the treatment group and the control group.
This is like a death sentence for a drug under development that has no value as a standalone treatment and should not continue clinical trials.

--- p.237~238, 「Chapter 11.
Among the major events in the domestic biopharmaceutical market

If investors believe that new drugs are not a target for development but rather a treasure to be unearthed, then pharmaceutical companies' expertise can be limited to the preclinical stage.
Of course, the task of selecting substances with a high potential to become pharmaceuticals is important in increasing the overall development success rate.
But if you look at the actual development process, new drug discovery experts are just doing their best.
More importantly, successfully developing and obtaining approval for a "candidate" substance that has entered the non-clinical stage requires numerous timely decisions based on medical expertise.
Therefore, when investors are interested in biopharmaceutical stocks, the first thing they should consider is not the probability that a new drug candidate under development will become a drug.
Rather, we should examine whether the pharmaceutical company has the capacity to 'identify' and 'resolve' the problems and clinical issues that may arise at the current development stage.

--- p.253~254, 「Chapter 12.
Should I continue investing in biopharmaceutical stocks?