Genuine Biotech's Independent R&D Achievements Listed as "Late-Breaking Research" in AACR Annual Meeting 2025

The 116th Annual Meeting of the American Association for Cancer Research (AACR) 2025 will take place 25–30 April in Chicago. The next-generation "small molecule drug" + "novel toxin" dual-platform project independently developed by Genuine Biotech will be presented at the AACR annual meeting as "Late-Breaking Research".

The AACR annual meeting is one of the world's oldest and largest scientific meetings dedicated to high-quality cancer research and innovations of all types. As one of the most authoritative academic conferences in the field of cancer, the AACR annual meeting has always been the focus of attention in the medical and pharmaceutical industries globally, attracting tens of thousands of cancer scientists, clinicians, and medical professionals from all over the world to attend each year. More than 20,000 people attended the AACR Annual Meeting 2024.

The dual-platform project of next-generation "small molecule drug" + "novel toxin" (project code: ZS-1003) is independently developed by Genuine Biotech. Utilizing an AI-Computer Assisted Drug Design (CADD) approach, the research team break through the structural limitations of traditional camptothecin drugs, modifying the core nucleus of camptothecin, which led to discover hundreds of novel TOPO1 inhibitor molecules with new structures and multiple candidate compounds with the properties of a new generation of payloads with strong global IP protection positions.

Currently, the discovery of the first small-molecule PCC compound under ZS-1003 project has been completed, namely ZSSW-136. This PCC molecule demonstrates broad-spectrum antitumor activity and has unique advantages in overcoming resistance to common anti-tumor drugs. Preclinical studies have demonstrated that it showed significant anti-tumor effects, with an inhibitory activity against irinotecan-resistant tumor cells 400 times as high as that of irinotecan. It is also expected to been applied in various XDC (e.g., ADC, PDC, and SMDC) R&D projects to provide effective solutions for various drug-resistant tumors.

Over the past few decades, the most widely used TOPO1 inhibitor has been irinotecan, a camptothecin derivative, which has become the backbone of various anti-tumor combination therapies. Camptothecin and its derivatives(e.g., irinotecan) have been used as standard chemotherapeutic agents for more than 60 years. However, the modification of camptothecin-based drugs is challenging and the existing drugs mostly share the same type of molecule. Camptothecin-based drugs face the problems of primary and post-treatment drug resistance, which is a shared challenge faced by the industry.

The new generation of payloads under the company's research and development can overcome drug resistance, and are comparable in anti-tumor activity to commonly used ADC payloads (e.g. SN-38, DXd, and other camptothecin-based compounds), but with much improved druggability (e.g. membrane permeability, nonexocytosis, etc.). The company has discovered numerous payload candidates with more than 50-fold higher potency compared to DXD in the DXD-resistant cancer cell lines.

For an antibody-drug conjugate (ADC), the toxin is a core component, mainly playing a role in killing tumor cells. ADCs have become a focus of global drug R&D in recent years, and the market is keeping booming. In 2024, the global market size of ADCs reached $13 billion, with a year-on-year growth of 24%. China, with its innovative strength and policy support in the field of biomedicine, has become one of first-tier players in global ADC R&D. It has become a popular hot spot for global ADC sales, and has been securing multiple overseas licensing deals. According to statistics, at least 14 ADCs in China secured overseas licensing deals in the whole year of 2024, of which the total transaction amount of nearly half of these drugs individually exceeded $1 billion.

Being listed as "Late-Breaking Research" by AACR this time is another proof of the innovative strength and R&D achievements of Genuine Biotech. In the future, Genuine Biotech will accelerate project progress and intensify core technology research to benefit patients worldwide with new and better treatment options.