In target identification, the source of new drug R&D, more than 2,000 therapeutic targets for various diseases have been identified, but only 400 new targets can be accessed by the new drugs which are approved by the FDA for marketing. In recent years, with the increasing amount of human genome data, the progress of modern molecular biology, and the introduction of metabonomics analysis method, more and more new targets have been put into clinical research. However, it's a long journey from the discovery of new targets to the real drugs for sale.

 

For today's Big Name Talk, we are honored to have invited Dr. Liu Xiaoyu, founder, Chairman and President of Chengdu FANXI Biopharma Co., Ltd., to interpret the target identification of new drug R&D and the product layout of FANXI.

 

Dr. Liu Xiaoyu

Founder of Chengdu FANXI Biopharma Co., Ltd.

In 1997, Dr. Liu graduated from the Medical School of the University of Alberta, Canada. He worked in GlaxoSmithKline (GSK) and Eli Lilly and Company after graduation, and returned to China in 2005, serving successively as the Chief Scientist of Zhejiang Hisun Pharmaceutical Co., Ltd. and Senior Vice President as well as President of the Pharmaceutical Research Institute of Waterstone Pharmaceuticals Group. In 2017, he started his own business, focusing on the development of new original drugs for anti-tumor, anti-virus and liver diseases.

 

Target selection plays an important role in the whole process of R & D of drugs. In modern pharmaceutical research, the establishment of new targets is often the premise and guarantee of new drug innovation, Dr. Liu said.

 

Up to now, researches has proved that there are more than one million genes in human individual, while only three or four hundred targets have been identified for drugs. Therefore, it's of great significance to identify and verify new targets.

 

According to Dr. Liu, a promising target must have three properties: firstly, the pathophysiological changes caused by the target itself need to be closely related to the disease; secondly, the target itself does not cause obvious toxic and side effects; thirdly, the three-dimensional structure of the target is easy to be combined for screening and developing drugs.

China's hardware and software strength are already in place, but why is it rare for China to identify and innovate targets independently?

 

Dr. Liu Xiaoyu said:

Firstly, in domestic scientific research environment, many fundamental researchers are unable to focus on projects with rare outcomes, because they are constrained by title evaluation and research task indicators.

Secondly, some emerging targets can't be fully studied because of the researchers' lack of knowledge and interdisciplinary research ability.

Thirdly, in China, with limited interdisciplinary communication among scientists in different disciplines due to many obstacles, the in-depth exploration of major scientific research issues is to some extent restrained.

 

In addition, the R&D of innovative drugs is often accompanied with large investment and high risks, which is a practical problem that domestic enterprises have been facing for a long time. Therefore, domestic SMEs used to stay away from the R&D of original drugs.

 

At present, many large pharmaceutical companies start to track and monitor the cutting-edge developments of new drug target research, and create opportunities for cooperation and sharing of achievement transformation, which have greatly shortened the cycle and reduced the cost of new drug R&D, warming up domestic target research.

 

If target identification is the premise and guarantee of drug innovation in the process of new drug development, the results of preclinical and clinical trials play a key role in the approval of drugs.

 

It's a common sense that the R&D of a new drug requires sufficient preclinical research, and we need to prove its safety and effectiveness in animal experiments before applying it to clinical trials on the human body.

 

Which is more important, animal experiment or clinical trial?

 

Dr. Liu Xiaoyu believed that clinical trials and preclinical experiments complement each other, both of which are indispensable in the R&D of new drugs.

 

The difference between preclinical animal experiment and human trial may cause big problems, but because of the huge cost of clinical experiment, if the effects of drugs discovered in the animal experiment cannot be confirmed in the clinical trial, it means a great loss of manpower and material resources; in short, animal experiment and clinical trial are both necessary for new drug R&D, both of which are of equal importance.

 

As an expert of innovative drug R&D, Dr. Liu is especially good at the R&D of innovative drugs for liver disease treatment. Liver disease, a generalized disease with great potential harm to human body, has diversified targets, so it's hard to develop drugs for liver disease.

 

For this social need, FANXI focuses on drugs for liver disease, and make layouts of the R&D pipelines for antiviral and anticancer drugs.

 

 

At present, the R&D of a multi-target tyrosine kinase inhibitor innovative drug has achieved phased results; an anticancer drug for the treatment of triple-negative breast cancer and a kind of malignant metastatic melanoma is about to apply for new drug clinical trial at the Center for Drug Evaluation, NMPA; a drug for the treatment of myelodysplastic syndrome has achieved simultaneous treatment of myelogenous leukemia and the fusion of tumors through targets aimed at expanding the range.

 

FANXI R&D Pipeline

 

FANXI owns independent intellectual property rights and operates flexibly in the asset-light mode. Its future development layout can be divided into three aspects:

First, the successfully developed drugs may realize the process from R&D to marketing all by themselves.

Secondly, it will work with domestic listed companies with weak ability yet willing to develop drugs, so as to share R&D achievements and accelerate the marketing of new drugs.

Thirdly, it will consider transferring overseas rights and interests to large foreign enterprises when the innovation pipeline reaches a certain stage, so as to form a virtuous circle of capital chain and achievement transformation.

Since returning to China in 2005, Dr. Liu Xiaoyu has successively served as a senior executive in Zhejiang Hisun Pharmaceutical Co., Ltd. and Waterstone Pharmaceuticals Group. By 2017, he has become an entrepreneur with a global perspective and a deep understanding of the domestic biomedical industry.  Driven by the wave of "mass entrepreneurship and innovation", he established FANXI in Tianfu Life Science Park.

 

He said that there are two reasons for FANXI's positioning of innovative drug R&D: firstly, stick to his interest and do what he is best at; secondly, comply with national policies and undertake social responsibilities.

 

Talking about his understanding of Chengdu's biomedical environment, Dr. Liu noted, Chengdu's biomedical environment is less developed than that in the coastal areas of Jiangsu and Zhejiang, but the gap has been narrowing year by year. Moreover, the government pays great attention to the development of biomedical industry and attaches great importance to it. The construction of industrial bases such as Tianfu Life Science Park bears Chengdu's dream of biomedical industry development and becoming an international medical consumption center, which shows Chengdu's determination to build a global biomedical innovation and creation center.

 

He also believes that Chengdu should have more public service platforms such as the National (Chengdu) New Drug Safety Evaluation Center, because many start-ups like FANXI hope that the government could invest in or take the lead in building public service platforms, including inspection and testing platforms, drug check platforms, etc.

 

At the end of the interview, Dr. Liu told us the implied meaning of FANXI-- "extraordinary promise to the west", and said that FANXI's social responsibility is to develop drugs aiming at the global market but original in China, and carry out international cooperation for the benefit of the world.