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AI technology-based system applied in rare disease treatment leads to the development of new treatments

Under a ‘pilot project to build bio-big data for all ministries,’ promoted by three ministries including the Ministry of Science and ICT, big data on genomic information, such as rare diseases, of 20,000 people is being established. With this, there is a growing interest in developing medicines for rare diseases. Dr. Noa Biotech, a drug development company that uses AI technology, continues to develop new drugs with focus on rare disease treatment through its own platform.

◆  Development of Rare Disease Treatment Using an Independent Platform

DR stands for doctor, as well as "drug repositioning," which means creating new drugs from existing drugs. Noah is a biblical figure who made an ark based on his faith in the Bible to help save the world. As such, the mission of Dr. Noah Biotech is conveyed in its name. Lee Ji-hyun, the CEO of Dr. Noah Biotech, explains that the company is determined to develop treatments for rare diseases more quickly and efficiently by exploring the hidden effects of existing drugs that are confirmed to be safe and using them to compose new compounds.

"People laughed at Noah for making his own ark in the Bible. But during the great flood, all life was destroyed, but the life on Noah's ark. Dr. Noah Biotech strives for the same. It may seem reckless to use AI to develop treatments for rare diseases, but we are confident that our technology and strategy are right. We hope that many patients can benefit from our technology."

Dr. Noah Biotech recently signed an agreement with Kainos Medicine to develop new drugs using the ARK platform. CEO Lee said, "From predicting and validating the effects to optimizing them to make final products for clinical tests, each company will lead and take on responsibilities for the parts it is best at. I am looking forward to the synergy effect during this joint effort."

The ARK platform was developed by Dr. Noah Biotech to predict new drugs and optimal combinations. The platform is composed of three databases, including NOTE-R, which contains research information on drugs (literature, clinical, and side effects), and NOTE-P, a drug genomics DB.

In addition, artificial intelligence prediction systems that utilize this data include CombiNet, which predicts compounds using all data; AI Target Finder, which seeks new disease target genes; AI Virtual Compound Screening System, which seeks new drug structures; and AI Virtual Target Screening system, which can find off-targets for drugs. CEO Lee explained that they are using various DBs and systems because each rare disease requires different strategies for developing new drugs. In addition, various methods of analysis are introduced to overcome the limitations of relatively limited data available for rare diseases, compared to cancer or metabolic diseases that have been studied a lot.

“The ARK platform can be used to develop treatments for a variety of diseases as well as rare diseases. Internally, we are focusing on developing treatments for rare diseases, and in joint development with external researchers, we are studying a variety of diseases, including cancer."

◆ Accelerating the development of new drugs through partnership

Companies working on developing new drug are very interested in predicting the structures of new compounds. Dr. Noah Biotech presents Hit Compound using AI Virtual Compound Screening system for disease target proteins. This is a mandatory process in drug development because we need to find out what side effects can appear. AI Virtual Target Screening System explores the possibility of unexpected side effects.

While numerous drugs entering the clinical trial stage are suffering from insufficient data on medicinal effects, Dr. Noah Biotech presents alternatives with its original technology.  CEO Lee remarked, "Even though sufficient research has been conducted during R&D stage, clinical trials on actual patients often do not show enough effectiveness to prove statistical significance.” "From the perspective of pharmaceutical companies, they are forced to abandon their research results." On that note, Dr. Noah Biotech offers two options to overcome this crisis. First is to develop a new drug with higher efficacy, to the extent it could mark a new turning point. Second is to re-develop a drug to target the same disease, but recommend a compound partner drug to increase the effectiveness. Lee added, "If we can find a new drug that has enhanced effectiveness of existing drugs, we can proceed with the development more easily."

Based on these technologies, Dr. Noah Biotech is currently discussing and proceeding on various projects with Korean new drug developers to predict the structure of new compounds, predict side effects, and develop treatments for rare diseases. Lee emphasized the significance of these joint development projects, explaining that the collaboration with experts in various fields is essential in the long and complex new drug development process.

◆ Targeting 5 or more clinical entries within 3 years

The key component in the AI system is data. Fortunately, due to the nature of the bio industry, a lot of research data has been already gathered as it is recommended practice to disclose data used in a research when publishing papers. In an attempt to secure meaningful data, Dr. Noah Biotech created Data Management Team to perform manual curation of data and exclude unsuitable data. CEO Lee Ji-hyun said, "It may seem like nothing from the outside, but when we develop an AI system based on carefully selected data, the results will show a big difference."

Lee also said that Dr. Noah Biotech will produce data on their own in the future, even though it is selecting from public data right now. This is because limited data is available for use in developing drugs for rare diseases. On that note, Dr. Noah Biotech is planning to form partnerships with global companies that specialize in diagnosing rare diseases. By doing so, it is expected that the stem cells and genetic information of patients with rare diseases will be obtained, and data and samples of the same patients will be used in drug prediction and verification. She added, "There have been regulations in Korea so far on obtaining blood samples and genetic data, but we are anticipating and preparing for the possible change in the regulatory conditions in the future."

As a result of its steady efforts, Dr. Noah Biotech's first pipeline is now cruising. Initially, it was a drug that targeted rare diseases that had problems with neuronal differentiation. However, the target disease group has expanded to include post-stroke recovery and TBI (Traumatic Brain Injury). Lee added, "We have shown good results in animal testing, so we are working on preclinical development with the aim of proceeding with clinical trial by next year." Their second pipeline is treatment for Lou Gehrig's disease. The drug has been tested on the stem cells of Lou Gehrig patients and now being tested on animals. CEO Lee explained that two other rare disease pipelines are under development, and that she is hoping to make five or more pipelines to enter clinical trials within three years. Furthermore, she hopes to make the company specialized in developing and producing neurological drugs on its own.

◆ A company that endeavors to make its employees happy and bring hopes to the patients.

"From the beginning, Dr. Noah Biotech was founded with the goal of developing new drugs based on data analysis. At the time of its establishment, there were companies that were interested in diagnosis and application development, but none of them wanted to develop drugs directly from system development. I started a business with a single goal of expanding my research to develop a new medicine."

For CEO Lee Ji-hyun, starting a business was a challenge to a new world. She was a research professor at Seoul National University when she started the business. At the time, she did not have any experience working in a company either. Lee recalled the time, saying that it was difficult getting investment and she was not familiar with the process. There was no one willing to invest in a small company that did not have products with verified medicinal effects. She was looking for a place where drug efficacy can be verified, and she was able to get the verification through the support task of the Korea Research Institute of Chemical Technology. Using this as a stepping stone, she was able to attract investment. Lee said she was able to attract the first investment with the help of people, and this enabled her to jump into the business. In its fourth year, Dr. Noah Biotech is still close to a start-up company, but Lee's eagerness to develop treatments and the technical competence of the company are giving an assurance to many people. She said that developing treatment is her calling, and it is not just about making money. Rather, it is about life and the process of moving toward the final goal. This sense of calling is also reflected in Dr. Noah Biotech's endeavors, one of which is organizing a campaign for Rare Disease Day to improve awareness. Last year, three start-ups gathered to form a "start-up alliance." They agreed to help patients suffering from rare and incurable diseases by sharing their strengths in diagnosing those conditions, managing and distributing data, and developing treatments. Lee said that she wants to open a YouTube channel to share useful information about rare and incurable diseases with people. Dr. Noah Biotech will be continuing its campaign.

When it comes to running a company, Lee places most importance on employee’s satisfaction. She is trying to achieve work life balance of employees by eliminating overtime work, having the company get-together during lunch, and discouraging people from communicating through Kakao Talk. Lee expressed that she would like to see all employees focus on work during the work time and spend the evening after work with their family and friends. This is based on her belief that only when the employees get to refresh outside work that they can concentrate on their job and are able to come up with more creative ideas.

As Lee runs a venture company, her philosophy is "to be careful in decision making and be prompt in execution." She believes that the sense of speed is the biggest strength of a venture company. Since not all of the company's projects can be successful, she aims to launch as many projects as possible in a short period of time and minimize risks by quickly deciding which ones to pursue further, based on the initial performance and progress. The plan is to quickly determine a few viable projects for clinical tests.

Finally, she envisions nurturing a great pharmaceutical company based on technology. She has a dream of developing treatment to help people, and she believes that having a platform could make that dream come true in a smart way. As Dr. Noah Biotech Co., Ltd. continues to develop treatments for the world through its own platform, it is confident that it will bring a new hope to patients with rare diseases around the world.

Monthly People May 2020 p76-79