C&C New Drug Research Institute Presents Preclinical Results of 'Acute Myeloid Leukemia Treatment' at AACR

On the 28th (local time), C&C New Drug Research Institute presented a poster on the preclinical study results of CNC-01, a dual STAT5/STAT3 inhibitor candidate being developed as a treatment for acute myeloid leukemia, at AACR 2025 held in Chicago, USA. Photo by JW JoongWai Pharmaceutical

C&C New Drug Research Institute announced on April 29 that it presented a poster on the preclinical study results of 'CNC-01' (provisional code name), a dual STAT5/STAT3 inhibitor candidate being developed as a treatment for acute myeloid leukemia (AML), at the 2025 American Association for Cancer Research (AACR 2025) held in Chicago, USA.

AACR is the world's largest academic conference in the field of cancer research. Through this presentation, C&C New Drug Research Institute disclosed, for the first time, the mechanism of action and preclinical efficacy data of the dual STAT5/STAT3 inhibitor, while also exploring opportunities for global technology partnerships.

The newly unveiled CNC-01 is an oral small-molecule compound that selectively and directly inhibits both STAT5 and STAT3 proteins simultaneously. It is a first-in-class innovative drug candidate with a novel mechanism of action that can overcome resistance issues associated with existing AML therapies.

According to the preclinical study results presented at AACR 2025, CNC-01 demonstrated superior tumor growth inhibition compared to the current standard FLT3 inhibitor in various resistance models, including those mimicking the FLT3-TKD (F691L) mutation and the bone marrow microenvironment.

Additionally, in AML animal models that did not respond to the standard BCL-2 inhibitor therapy, CNC-01 alone showed strong tumor inhibition. When administered in combination with a BCL-2 inhibitor, the therapeutic effect was further enhanced. Notably, both monotherapy and combination therapy resulted in tumor responses corresponding to complete remission (CR) and partial remission (PR).

In survival analysis, the CNC-01 monotherapy group demonstrated superior survival extension compared to the BCL-2 inhibitor group. In the combination therapy group, more than half of the subjects survived until the end of the experiment, and the median survival was not reached, further confirming the outstanding anticancer efficacy of both monotherapy and combination therapy.

A representative from C&C New Drug Research Institute stated, "AML is a disease that is difficult to treat due to diverse genetic mutations and drug resistance, and there is a very high level of unmet medical need. Our STAT5/3-targeted compound has demonstrated excellent efficacy in preclinical studies based on its innovative dual inhibitory action. We plan to pursue clinical development through global partnerships and technology transfer in the future."

Meanwhile, this compound was discovered through JW's artificial intelligence (AI)-based integrated drug research and development (R&D) platform, JWave. It was selected as a national new drug development project in 2023 and is currently under development with research funding support.

C&C New Drug Research Institute is the first Korea-Japan joint bio-venture in Korea, established in 1992 by JW JoongWai Pharmaceutical and Chugai Pharmaceutical, a member of the Roche Group. In 2020, it became a wholly owned subsidiary of JW JoongWai Pharmaceutical.

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