Local biopharmaceutical industry is going with its gut

Genome & Company researchers conduct research at the company’s lab in Pangyo, Gyeonggi. [GENOME & COMPANY]

There is one term that has been appearing frequently in the biopharmaceutical industry recently — microbiome.

Microbiome refers to the collection of all microbes — bacteria, fungi, protozoa, and viruses — that live on and inside the human body. An adult is estimated to have 38 trillion microbes and they mainly exist inside the intestines, particularly in a small part of the large intestine called the cecum. They are referred to as the gut microbiome.

Some experts call the gut microbiome the “second genome” considering its unknown, but potentially very beneficial influence on nutrition and digestion, and even in the brain.

Microbiomes have already been widely used in the food and beauty industries such as in health supplements like probiotics. But to date, there are no drugs that have been developed using microbiomes.

The Korean government and many local biopharmaceutical companies are rushing to invest in microbiome, with the goal of becoming the first to develop treatments.

"There are trillions of microbes in a human's gut, and taking out some beneficial microorganisms and using them to develop drugs, this is the theory of microbiome-based treatments," said Lee Dong-ho, a professor specializing in Gastroenterology at Seoul National University Bundang Hospital. “There are already so many research papers and reports that cite the potentiality of the microbiome in the biopharmaceutical sector."

"Healthy microbes have the ability to heal diseases, they can be beneficial in treating and preventing cancers, autism, dementia, depression and even the coronavirus," Lee added. "So I, as do many medical professionals, call microbiome the big bang of modern medical technology and the future of next-generation treatment."

The Ministry of Science and ICT on Dec. 29 announced that it will spend around 1.2 trillion won ($1 billion) over the next 10 years to acquire the core technology and support bio ventures that invest in the microbiome.

The global microbiome market, which was valued at $81.1 billion in 2019, is expected to grow at an annual rate of 7.6 percent to reach $109 billion in 2023, according to market research firm Frost & Sullivan.

Race to be first

CJ CheilJedang on Jan. 5 launched a biopharmaceutical subsidiary CJ Bioscience, with the goal of leading the microbiome industry. The company said CJ Bioscience will be a company specializing in drugs and health supplements based on microbiomes.

The launch of the new subsidiary came as CJ CheilJedang acquired 44 percent of Chun Lab, a local microbiome research company, in July.

“Our goal is to come up with 10 pipelines using microbiome by 2025, and sign two technology transfer deals with them,” a spokesperson for CJ Bioscience said.

In terms of speed of development, Genome & Company is currently one of the front runners in the industry. The Pangyo, Gyeonggi-based biopharmaceutical company has four drug candidates that are created based on microbiome, two of which are in Phase 1 clinical trials.

The two candidates include GEN-001, an anti-cancer therapy, which is currently undergoing Phase 1 clinical trials in Korea and the United States. The company aims to make the candidate treatment for solid cancer patients with three diseases: Head and neck cancers, non-small-cell lung cancer and urothelial cancer.

GEN-004, a candidate for infertility, and GEN-501 for cancer-associated rash are currently undergoing preclinical trials.

Genome & Company also expanded its business to the contract and development manufacturing organization (CDMO) area. In September, it spent $27 million on acquiring 60 percent of Campbell, California-based List Biological Laboratories, a CDMO company specializing in the microbiome.

In October, it also established U.S. subsidiary List Biotherapeutics. List Biotherapeutics recently won $31 million in Series A funding, which will be used to build a 59,500-square-meter (640,000-square-foot) microbiome manufacturing facility in Fishers, Indiana, the company said.

The Fishers city government offered the site for free and vowed to allow a 50 percent reduction in property tax for 15 years after the completion of the factory.

“Genome & Company’s entrance into the CDMO business is meaningful in three aspects,” Pae Ji-soo, CEO of Genome & Company said. “First of all, [it is our] conviction that the microbiome industry will grow rapidly, and secondly, the expression of our confidence about our pipelines.”

“Thirdly, we will build a virtuous circle that we can report stable revenue and use the money for the research and development of new drugs.”

KoBioLabs recently started global Phase 2 clinical trials of KBLP-001, its microbiome-based treatment for psoriasis. The trials will be conducted on a total of 80 patients in 10 hospitals in the United States and Australia.

It also received the nod to start Phase 2 clinical trials of KBLP-007, a candidate to treat ulcerative colitis, from the U.S. Food and Drug Administration.

The biggest hurdle

Microbiome is a sector that requires extensive research and investment. However, Korea falls far behind compared to other countries in terms of speed. Experts pinpoint the lack of infrastructure as the biggest hurdle.

“To develop a new drug based on microbiome, which is a totally new idea for the biopharmaceutical industry, some guidelines should be presented for companies to conduct clinical trials rapidly,” said Kim Dong-hyun, a biomedical sciences professor at Seoul National University College of Medicine. “But Korea currently lacks the guidelines and enough infrastructure, which is the biggest hurdle at this time.”

“Biopharmaceuticals based on microbiomes is an unknown market that no country has yet entered. The Korean government should offer more attention and support on it so that we could become one of the leading players in the market.”

Professor Lee from Seoul National University Bundang Hospital agrees with Kim, adding that the government should ease some regulations to give companies more autonomy.

"Korea should follow the global trend and learn from other advanced countries," Lee said. "Instead of having all kinds of regulations, the government should offer some guidelines and let the industry proceed with the developments themselves. Microbiome will determine national competitiveness in the near future."

According to data from Samjong KPMG, for over 10 years starting from 2007, the U.S. government spent more than $1 billion in generating resources to characterize microbiome and elucidating the roles of microbes in health and diseases under an Integrative Human Microbiome Project, run by the U.S. National Institutes of Health. The government also invested an additional $620 million in building infrastructure for further research and development.

Seres Therapeutics recently concluded Phase 3 clinical trials of SER-109, a microbiome therapeutic candidate, for patients with clostridioides difficile, a bacterium that causes an infection of the large intestine. The Cambridge, Massachusetts-based company said it will seek approval from U.S. Food and Drug Administration in mid-2022.

Rebiotix, a Minnesota-based biopharmaceutical company, is also developing a similar drug and recently finished Phase 3 clinical trials.

BY SARAH CHEA [chea.sarah@joongang.co.kr]