Frost & Sullivan interview -Zehui Biotechnology — A Leader in Stem Cell Drugs

Introduction

Beijing Zehui Chenxing Biotechnology Co., Ltd., established in 2018 (hereinafter referred to as 'Zehui Biotech'), is a pharmaceutical company focusing on the research and development and production of stem cell drugs. The cell drug (CAStem cell injection) derived from pluripotent stem cells developed by Zehui Biotech is undergoing phase II clinical trials for acute respiratory distress syndrome (ARDS) approved by the National Medical Products Administration, as well as phase I/II clinical trials for meniscus injury.

Stem cell therapy is one of the most cutting-edge and popular areas of medical research today, with related clinical studies advancing steadily at a rapid pace. Currently, therapeutic stem cell products that have entered clinical trials mainly come from two sources: adult stem cells and pluripotent stem cells.Zehui Biotechis the first pharmaceutical company in China to conduct clinical trials on pluripotent stem cell drugs. Why does Zehui Biotech focus on pluripotent stem cell drugs? What advantages do pluripotent stem cells have in terms of drug development? With this series of questions, the Frost & Sullivan (Frost & Sullivan, hereinafter referred to as 'Frost & Sullivan') Healthcare Business Unit conducted an exclusive interview with Dr. Jia Yi, Chief Medical Officer (CMO) of Zehui Biotech.

Guests for the Interview

Pluripotent stem cells have outstanding potential for drug development

Zehui Biotech is steadily advancing clinical trials

Whether globally or domestically, currently, stem cell drugs entering the clinical research and development stage can be mainly divided into adult stem cells and pluripotent stem cells. Adult stem cells refer to mesenchymal stem cells derived from different human tissues such as umbilical cord, umbilical blood, fat, and bone marrow. Pluripotent stem cell drugs mainly include functional cell drugs derived from human embryonic stem cells (hESC) and induced pluripotent stem cells (iPSC).

Compared to traditional small molecule and large molecule drugs, the advantage of stem cells lies in their special nature as living cell drugs.Since stem cells are living cells themselves, after entering the human body, they can secrete corresponding cytokines based on different diseases and pathophysiological environments within the patient's body. For example, mesenchymal stem cells can play functions such as inflammation suppression, immune regulation, or promoting the repair of damaged tissues. In addition, pluripotent stem cells can differentiate into different functional cells such as nerve cells or islet cells to replace aging or damaged cells in the human body and perform corresponding functions.Therefore, current clinical application research on stem cell drugs mainly targets acute and chronic inflammatory diseases, autoimmune diseases, diseases caused by tissue damage, or functional degeneration of cells and tissues.

Firstly, the raw materials for cell drug production are consistent, ensuring that the biological characteristics of different batches of pluripotent stem cell preparations are consistent. The preparation of cell drugs derived from pluripotent stem cells all come from a stable pluripotent stem cell line. Whether it is a human embryonic stem cell line based on fertilized eggs or an iPSC cell line formed after artificial induction of adult cells, the establishment of the pluripotent stem cell line is based on cell cloning technology, that is, all cells come from a common ancestral cell, and the cell genotype and phenotype are highly consistent, ensuring the homogeneity of starting materials from the source and ensuring that the biological characteristics and clinical efficacy of different batches of cell preparations obtained are consistent.

Secondly, pluripotent stem cells have the characteristics of self-renewal and unlimited expansion in vitro. After establishing a pluripotent stem cell bank, sufficient seed cells with consistent biological characteristics and controllable quality can be continuously provided. Coupled with mature in vitro expansion culture and induction differentiation technology, as well as standardized production processes and quality control, large-scale production of pluripotent stem cell drugs can be achieved to meet the commercial application needs for the number of preparations after drug marketing in the future.

In addition, human embryonic stem cells can differentiate into different functional cells in the three germ layers (inner, middle, and outer) in a targeted manner. Theoretically, they can be differentiated into all functional cells of the human body under suitable conditions. Therefore, it provides an all-round and controllable seed bank platform for the subsequent continuous research and development of functional cell preparations.

In 2020, the cell drug (CAStem cell injection) derived from pluripotent stem cells developed by Zehui Biotech was approved by the National Medical Products Administration and launched for phase I/II trials for acute respiratory distress syndrome caused by COVID-19 and phase II clinical trials for COVID-19-related pulmonary fibrosis. Currently, both clinical trials have been completed. According to the data of two years of follow-up after treatment, CAStem cell injection has good safety and some efficacy indicators of the subjects have been improved.

In 2022, Zehui Biotech was approved by the National Medical Products Administration to conduct clinical trials for acute respiratory distress syndrome caused by different reasons including the novel coronavirus, bacteria, trauma, etc. Currently, phase II clinical trials are underway.

In addition, in 2021, CAStem cell injection was also approved by the drug administration to conduct clinical trials for meniscus injury. Currently, more than two-thirds of the subjects recruited for this phase I/II clinical trial have been completed. The subjects who have received the experimental drug are receiving regular follow-ups to evaluate safety and efficacy. Relying on the advantage of human embryonic stem cells being able to differentiate into different germ layer functional cells, Zehui Biotech has also carried out preclinical and clinical studies on retinal pigment epithelial cell preparations for the treatment of fundus degenerative diseases and dopamine neuron precursor cell preparations for the treatment of Parkinson's disease. Subsequently, relevant new drug development work will continue.

Zehui Biotech consolidates R&D technology

Product pipeline continues to expand and upgrade

My interest in stem cell research originated during my doctoral research project when I conducted a project on using adipose-derived stem cells to differentiate into vascular endothelium and construct three-dimensional vascular structures in vitro. Another important reason I joined Zehui Biotech in 2018 to engage in stem cell drug research and development was that national regulations clearly defined stem cell drug research and development as a type of biopharmaceutical, which would be officially supervised and managed by the drug administration. This will greatly facilitate the development of the domestic stem cell drug R&D industry.

There are many types of human diseases with complex pathogenesis. Therefore, disease treatment requires the continuous emergence of new therapeutic mechanism drugs. Chemical and large molecule drugs play an important role in treating many diseases. However, there are currently also many diseases in clinical practice that lack effective treatment methods. For example, some degenerative diseases, severely damaged diseases, abnormal immune and inflammatory diseases. I believe that stem cell drugs can play an important role in these disease areas based on their special therapeutic advantages.

In terms of product research and development, firstly, Zehui Biotech will continue to explore the feasibility of developed functional cells such as CAStem cell injection in new disease treatment areas; secondly, Zehui Biotech will also develop new functional cells, including midbrain dopamine neuron precursor cells differentiated from human embryonic stem cells and retinal pigment epithelial cells differentiated from human embryonic stem cells, for the treatment of central nervous system diseases and fundus diseases. In addition, it will also explore products combining stem cells with different matrix materials to meet the medical needs of specific diseases.

Zehui Biotech is currently conducting Series B financing, with the planned financing amount reaching hundreds of millions. The funds will mainly be used to promote existing R&D projects and add new R&D projects, thereby enriching Zehui Biotech's product pipeline of pluripotent stem cells and advancing stem cell drug development.