Media Spotlight | Dr. Wang Tong: Embarking on the "Stellar Voyage" of Drug Discovery, Starting from a Fragment-Molecule Library

In recent years, despite the continuous emergence of novel molecular-entity drugs, small-molecule therapies remain an indispensable topic in pharmaceutical R&D—and they continue to be the primary battleground for developing innovative new medications. As a company dedicated to the research, development, and commercialization of breakthrough small-molecule therapies, Shengshi Taikang has recently been celebrating one success after another. Just after successfully advancing its first China-U.S. dual-regulatory submission for a new drug (the CXCR4 antagonist CGT-1881) and embarking on its international journey, the company’s next-generation ALK inhibitor has also received U.S. approval to begin clinical trials.

So, what are the key factors that determine whether a drug gets approved for clinical use? And in the field of anticancer small molecules, what are the winning strategies to stand out and secure a prominent position? Recently, the Mingxin Talk interview featured Dr. Tong Wang, Chief Scientific Officer of Shengshi Taikang, who shared insights into the research and development strategies—and emerging trends—of precision-targeted small-molecule anticancer drugs.

Mingxin Hub: Congratulations to Shengshi Taikang on completing the first China-U.S. dual-regulatory New Drug Application for its CXCR4 antagonist product CGT-1881—and embarking on its global journey! Could you share what were the key factors that led to the approval of this drug for clinical trials?

Dr. Wang Tong ： The three main factors that contributed to the successful approval of the CGT-1881 project for clinical trials are as follows:

First, we Identify suitable small-molecule compounds early in the research and development phase—they exhibit excellent activity, favorable pharmacokinetic properties, and a high safety profile. This product is currently the world’s first orally administered CXCR4 antagonist approved for clinical use, offering superior convenience and accessibility compared to existing injectable medications on the market. Additionally, it boasts significant advantages in terms of oral bioavailability and its ability to cross the blood-brain barrier.

Second, we The preclinical R&D work was thoroughly conducted, successfully completing CMC, pharmacological and efficacy studies, as well as safety experiments. Part of this work was carried out with the assistance of our partner, WuXi AppTec. We are deeply grateful for WuXi AppTec’s invaluable support throughout the drug development process.

Finally, Communication with regulatory authorities is also crucial. We would like to extend our heartfelt thanks to our regulatory and clinical teams, who played a pivotal role in preparing the Phase 1 clinical application. Throughout the submission process, they maintained seamless communication and coordination with experts from both the CDE and the FDA, proactively addressing challenges, making continuous revisions, and fine-tuning the application. Their dedication and collaborative efforts have truly set a remarkable example for our first-ever China-U.S. dual-regulatory project, helping us gain invaluable experience along the way. With this solid foundation, we’re confident that we’ll deliver even stronger results in future new drug applications.

Mingxin Hub: Shengshi Taikе boasts a domestically leading, integrated drug research and development technology platform. Could you share what technical approaches and strategic advantages Shengshi Taikе leverages in the process of small-molecule drug design and development?

Dr. Wang Tong: Shengshi Taikе's biggest advantage is its exclusive fragment molecule library. This was first conceived by our CEO, Dr. Yu Qiang, over 20 years ago and has since been meticulously developed over nearly two decades by numerous R&D scientists, including members of our core team. Our fragment molecule library is neither a commercial library nor a virtual one—it’s a real, tangible collection of compound structures. , it was discovered, screened, and identified from the literature, and further optimized through multifaceted synthetic efforts, Enable it to have excellent ductility. 。

In addition, we’ve put significant effort into understanding the biophysical properties of these structures. We aim to ensure that the small-molecule fragments exhibit excellent drug-like characteristics as well as favorable biophysical properties. In the development of small-molecule new drugs, in addition to achieving excellent activity through structural binding, their biophysical properties significantly influence pharmacokinetic characteristics as well as safety profiles. Many new drug development projects are often forced to halt in later stages due to unfavorable biophysical properties. Our small-molecule fragment library addresses this challenge early on, prioritizing the optimization of pharmacokinetic profiles and safety from the very beginning. This approach helps us avoid the common scenario where a compound shows strong initial activity but later runs into issues—such as poor pharmacokinetics or safety concerns—stemming from its biophysical characteristics. As a result, we ensure from the outset that these promising small molecules or lead compounds possess exceptional attributes across all critical parameters, enabling them to swiftly advance into the PCC stage. This is one of our key distinguishing features.

Currently, we're also exploring the integration of AI and computer technologies to further screen small-molecule drugs. This effort is just getting started, and we’re hopeful that in the future, we’ll see this technology unlock its true potential.

Mingxin Hub: What are the key aspects of precision cancer drug development?

Dr. Wang Tong: Precision drug development is a key component of our innovative R&D platform, rooted in an important medical or biomedical research principle: all tumors arise from abnormal and uncontrolled cell growth driven by one or more genetic mutations—rather than being caused solely by a single enzyme target or isolated cellular dysfunction. Therefore, Genetic mutations are actually the masterminds behind tumors. "Ultimately, we must catch the mastermind behind this to have any real chance of successfully advancing an anti-cancer drug into clinical trials or even market launch. Precision drug development is essentially gene-variation driven—everything we do, from research and development through late-stage efficacy studies to clinical trials, revolves around this central principle."

First, during the early stages of research and development—specifically when selecting targets and initiating projects—we’ve focused on precision. This is achieved by carefully screening and identifying targets and R&D directions through thorough research on genetic mutations. This approach is critical because many cancers are driven by genetic alterations. For instance, the well-known KRAS mutation promotes tumor growth in up to 95% of pancreatic cancer patients. Moreover, KRAS gene mutations are also found in patients with various other cancers, including skin and lung cancers. Similarly, MYC-driven and CCND3-driven mutations occur in tumors such as Burkitt lymphoma. Therefore, It is crucial to first clarify the characteristics and direction of genetic variations before determining research and development priorities. 。

Second, during the new drug optimization process, once we identify a lead compound, we will Conduct multi-faceted, precision-based activity tests of the compound against tumor cells to identify its most effective and highly sensitive variant. ; On the other hand, it is being carried out during research and development Multiple mechanism compound screening, combined with the identification of mutated genes, helps determine the research and development direction for this compound. 。

Third, Precision at the clinical stage can help us identify biomarkers. (Biomarker)—specific biomarkers are clinically crucial and serve as a key indicator for assessing the success of clinical trials. Typically, once precise, targeted genes are identified, Starting with the mutated gene allows us to quickly identify biomarkers in the clinical stage that can be used to assess whether a compound is effective. We had already identified our direction early on, which helped us avoid unexpected clinical challenges while significantly boosting our clinical success rate.

Mingxin Hub: Shengshi Taikang has built a robust pipeline of innovative drugs, covering multiple therapeutic areas including diabetes management, cancer treatment, and rare diseases. What are the key considerations behind this strategic layout, and what are the company’s future plans?

Dr. Wang Tong: Shengshi Taikе started with a fragment-based molecule library and has since specialized in synthesis and medicinal chemistry—making us a chemistry-driven company at the forefront of innovative drug discovery. When evaluating and advancing new drug research projects, we strive to leverage our strengths while minimizing weaknesses, ensuring we fully capitalize on our unique expertise.

Shengshi Taikе initially focused on the diabetes and metabolic field, partly because the market for these areas is relatively large. Another key consideration was that, at the time when we were developing sengliflozin, we happened to have a small-molecule fragment library that served as an excellent alternative to lead or reference compounds—enabling us to carve out our own proprietary patent space while also enhancing the compound’s potency and pharmacokinetic properties. This story underscores the importance of fully leveraging our fragment molecule library, as well as our strengths in both medicinal chemistry and synthetic chemistry.

We’ve continued to deepen our expertise and expand our strategic positioning in the sugar metabolism field—though this area has relatively limited target options. To address this, we’ve already positioned ourselves around key targets like SGLT2 and GLP-1. While the diabetes market remains vast, it’s also becoming increasingly crowded. This makes it essential for us to explore new clinical directions and broaden our therapeutic applications. On the other hand, the oncology space is incredibly diverse, with growing opportunities driven by the rise of precision medicine and increasingly segmented markets. From this perspective, our fragmented molecular library, combined with our strengths in medicinal chemistry and synthetic chemistry, positions us uniquely to capitalize on these trends. Additionally, having spent many years conducting cancer drug research in the U.S., I’m well-equipped to leverage my extensive experience and accelerate the development of innovative oncology therapies at Shengshitaike. These two key considerations have enabled us to move beyond our initial focus on sugar metabolism disorders and boldly expand into the dynamic and promising field of oncology.

In the field of rare disease research and development, we consider ourselves opportunists. Whenever we spot an existing or well-understood area where a potential therapeutic approach could benefit a specific rare disease, we seize the moment to conduct pharmacological and efficacy studies. One particularly intriguing example from our work involves the CGT-1881 project, which targets a chemokine receptor 4 inhibitor. Interestingly, there’s a rare disease caused precisely by mutations in the CXCR4 chemokine receptor—specifically, a defect linked to immune system dysfunction in infants. This is a highly distinctive CXCR4-related disorder, and coincidentally, we happen to have a CXCR4 inhibitor in development. It’s truly a case of perfect timing, and naturally, we’re not letting this golden opportunity slip away!

Additionally, the company continuously updates its development strategies based on the latest research advancements in the biopharmaceutical field. We are actively involved in cutting-edge initiatives within the small-molecule space, such as PROTACs (Proteolysis-Targeting Chimeras) and molecular glues—two of the most promising new directions in drug discovery. Beyond this, we’re also leveraging artificial intelligence to accelerate our R&D efforts. For instance, we’re harnessing AI’s powerful data-analysis capabilities to scrutinize clinical data, identify optimal therapeutic indications, and even explore innovative approaches for interpreting and evaluating drugs more effectively. At its core, the company’s drug-development process draws inspiration from the latest biomedical breakthroughs, empowering our small-molecule drug programs to soar—flying faster and reaching greater heights than ever before.