Complement-Focused Biotech Kangjing Pharma Advances Global FIC Drug CG001 with Dual IND Approvals in US and China

In the global market for innovative drugs, complement therapeutics represent a critical sector—one of the most challenging frontiers to conquer, yet one with immense application potential.

Professor Hu Weiguo of Fudan University has been engaged in complement research for nearly 20 years. In 2017, he formally entered the industry by founding Shanghai ComGen Biopharmaceutical Co., Ltd. (ComGen), focusing on the innovative drug development targeting the complement system.ComGen has currently established more than ten first-in-class (FIC) complement drug pipelines. Its complement inhibitor CG001, which targets a novel global target, has received Investigational New Drug (IND) approvals in both China and the United States. The company has also garnered favor from numerous renowned investment institutions, including Sequoia China and Yahui Investment.

In early 2005, Hu Weiguo arrived at Harvard Medical School, where the vibrant atmosphere of biomedical innovation and entrepreneurship in Boston left an indelible mark on him. Now, he is in his 12th year as a professor at Fudan University. He represents a cohort of Chinese scientists who aspire to establish companies to translate their technologies into practical applications that benefit patients. While these scientists possess solid foundations in basic research and have products or technologies with significant development potential, they also face numerous challenges in entrepreneurship.

Recently, VCBeat New Medicine conducted an exclusive interview with Professor Hu Weiguo to explore, from his perspective, how to achieve innovative breakthroughs in the global field of complement therapeutics, spanning from scientific research to clinical translation.

Before founding ComGen, Hu Weiguo was engaged in scientific research, but the seed of entrepreneurship had long been sown in his heart. In 2005, he began his postdoctoral work at Harvard Medical School. The Lab for Translational Research, where he worked, housed more than 20 postdoctoral fellows and specialized in translating basic research findings into practical applications, with several original innovative drugs and diagnostic reagents having entered clinical use. “I used to think that drug development was an extremely difficult endeavor, but seeing many of my colleagues successfully achieve translational outcomes gave me great confidence.” During his more than five years at Harvard University, he remained committed to translational research and successfully advanced multiple projects toward commercialization.

In 2010, Hu Weiguo returned to Shanghai from Boston and became a professor at the Institutes of Biomedical Sciences (IBS) of Fudan University. Shanghai was the city where he had spent his entire student life, having studied at the Second Military Medical University and the Shanghai Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences. After returning to China, he embarked on a new chapter of his career there. When asked why he chose Fudan University, he said, “At that time, most of the institutions to which I applied offered me positions. I ultimately chose the Institutes of Biomedical Sciences at Fudan University primarily because it strongly supports translational medicine research, which aligns closely with my philosophy.”

Over the following seven years, Hu Weiguo devoted himself daily to laboratory work, focusing on the research and development of complement therapeutics, during which he developed and advanced more than ten first-in-class (FIC) complement drugs.With the continuous accumulation of research achievements, Hu Weiguo ultimately founded ComGen in Zhangjiang Pharma Valley in May 2017.

Hu Weiguo is highly confident in his research, but the transition from academia to industry is no easy feat, with many hurdles to overcome. One such challenge is scaling up the process from laboratory-scale trials to pilot-scale production. “In academia, handling volumes of 1 or 2 liters poses no problem, but it is difficult to scale up to an industrial volume of 200 liters,” said Hu Weiguo. However, thanks to the extensive industrialization experience of co-founder Dr. Xu Ting, ComGen has successfully resolved this challenge.

The company has established partnerships with enterprises such as Alphamab Oncology and Sherpa Biosciences, as well as research institutions including Fudan University, the Chinese Academy of Sciences, and Shanghai Jiao Tong University. This October, ComGen launched its new 1,500-square-meter facility, and the company’s team has gradually expanded to approximately 30 members.

Complement refers to a group of proteins present in the blood, tissue fluids, and on cell membranes of humans and animals that exhibit enzyme-like activity upon activation. It is a crucial component of innate immunity and serves as a bridge connecting innate and adaptive immunity. Abnormal activation of the complement system can lead to cellular and tissue damage, thereby causing autoimmune diseases.

However, due to the complex theoretical framework and highly challenging technical operations, complement drug development faces significant barriers. As a result, newly launched drugs in the complement field are few and far between; currently, only five complement inhibitors have been approved for marketing worldwide, and no complement therapies are yet available in China.

Complement drugs targeting different sites face numerous challenges, including unsatisfactory therapeutic efficacy, elevated infection risks, high production costs, or exorbitant treatment expenses.To this end, leveraging its extensive long-term research and development advantages, ComGen has developed CG001, a Class 1.1 cell-targeted complement inhibitor aimed at C3b, a novel and potentially optimal global target in the complement system, which is expected to overcome the aforementioned limitations.

Phase Ia clinical trial results demonstrated that ultra-low doses of CG001 achieved nearly 30% complement inhibition, with a favorable safety and tolerability profile and no immunogenicity. “CG001 targets C3b,All three complement activation pathways are blocked, all pathogenic active products generated by activation are also blocked or inhibited,Optimal Complement Inhibition Efficacy.” Hu Weiguo stated that CG001 efficiently binds to C3b,It specifically binds to the surface of cells under complement attack, thereby exhibiting specific cell-targeting properties that enable lower clinical dosages, reduced side effects, and extended dosing intervals.

Although the C3b target holds immense R&D value, its development is extremely challenging. Currently, very few companies worldwide are developing complement drugs targeting C3b, and ComGen is poised to fill this gap in C3b-targeted complement therapeutics.

It is reported that CG001 received FDA Investigational New Drug (IND) approval in September this year. Multi-center Phase Ib patient enrollment has been completed at Peking Union Medical College Hospital, Tianjin Medical University General Hospital, and Huashan Hospital of Fudan University, with the first patient dosing expected to be completed by the end of November. As research and development progress, CG001 may provide patients with an improved treatment option.

By building technological platforms to pave the way for drug development, ComGen has established multiple proprietary complement technology platforms,including a comprehensive platform for complement gene knockout mice, a platform for animal models of complement-related diseases, and a platform for complement activity assays.Based on the company's proprietary technology platform, ComGen'sThe product pipeline covers more than ten complement inhibitors and complement activators targeting novel mechanisms., the pipeline development is both robust and rich with imaginative potential.

Regarding the indications for complement therapeutics, Hu Weiguo emphasized, “Although the currently approved indications for marketed complement drugs are all rare diseases, these agents actually hold greater market potential in the treatment of prevalent and common conditions.” Consequently, ComGen has adopted a strategy that begins with orphan drugs for rare diseases and expands to cover major disease areas. The company aims to progressively broaden the therapeutic indications of its drug candidates, thereby providing safer and more effective clinical treatment options for a larger patient population.

In fact, the complement system is closely associated with autoimmune diseases. Although approved indications such as atypical hemolytic uremic syndrome (aHUS) and neuromyelitis optica spectrum disorder (NMOSD) are rare autoimmune conditions, there are hundreds of types of autoimmune diseases, including many prevalent disorders. Therefore, the therapeutic area for autoimmune diseases offers substantial market potential. After validating technologies and products through rare diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and aHUS,ComGen will further expand the indications of its complement inhibitors into a broader range of common and prevalent autoimmune diseases in the future,such as mesangial proliferative glomerulonephritis, spontaneous lupus nephritis, renal ischemia-reperfusion injury, ankylosing spondylitis, and age-related macular degeneration.Furthermore, the company has also developed complement agonist products for the treatment of tumors.

Hu Weiguo stated that although the development of novel targets often faces significant challenges, it also offers immense potential. With an eye toward the future development of China’s biopharmaceutical industry, we must ultimately take the step toward original innovation and develop innovative drugs targeting novel targets discovered by Chinese researchers.

“New targets and new technologies constitute true innovation.” Benefiting from national policies that encourage and support scientists in launching startups, and leveraging the power of capital—particularly the solid foundation laid by seasoned professionals who have returned to China from major multinational pharmaceutical companies with extensive experience in drug R&D and management—China’s pharmaceutical industry is undergoing a transformation toward source-driven innovation. Accordingly, capital investment is increasingly shifting toward early-stage, original innovation.

ComGen secured clinical trial approval from China’s Center for Drug Evaluation (CDE) for its novel first-in-class drug CG001, targeting a new mechanism of action, with an initial angel round of RMB 10 million. After completing a Series A financing round of nearly RMB 100 million, co-led by Sequoia Capital China and Yahui Investment, the company obtained FDA clinical trial approval this year, with clinical development progressing smoothly. Hu Weiguo stated that, in addition to his long-term scientific accumulation in complement research, these achievements were made possible by national policy support and encouragement, as well as trust and assistance from the capital market.

The company currently plans to initiate its Series A+ financing round. The proceeds from this round will be allocated to the clinical development of existing pipeline assets, primarily to fund R&D for pipeline reserves, and to further strengthen team building.

During his time at Harvard University, Hu Weiguo experienced Boston’s vibrant atmosphere for biopharmaceutical innovation and entrepreneurship. He introduced to VBInsight the technology transfer system at Harvard University and recalled how many of his patents were successfully commercialized under this framework.

“To help researchers gain market recognition for their scientific achievements as quickly as possible, Harvard University’s Technology Transfer Office carefully selects ten core patents each year, compiles them into a brochure, and hosts a dinner the week before Christmas, inviting investment institutions from across the United States to publicly recommend these patents. ‘My patent was also selected at that time, ranked second, and was successfully commercialized later,’ introduced Professor Hu. He further explained that Harvard University has well-established institutional regulations governing equity distribution following the successful licensing or assignment of patents.”

There is much to consider regarding how to establish a robust support system for entrepreneurship, foster a vibrant culture of innovation and entrepreneurship, accelerate the market entry and commercialization of scientists’ research achievements, and ultimately benefit patients. Meanwhile, the overall ecosystem of China’s innovative drug industry is undergoing continuous reform and improvement, with an increasing number of scientists like Hu Weiguo choosing to join the industry, hoping to contribute their share to the development of China’s innovative drug sector.