• First-in-Class oral dual inhibitor targeting STAT5/STAT3
• Discovered through JW’s AI-based integrated R&D platform, JWave
• Demonstrated potential to overcome resistance to FLT3 and BCL-2 inhibitors in AML models
April 29, 2025
C&C Research Laboratories announced that it presented preclinical results for its investigational therapy CNC-01, a dual STAT5/STAT3 inhibitor, at the American Association for Cancer Research Annual Meeting 2025 (AACR 2025) on April 28 in Chicago, USA.
AACR is the world's largest academic conference dedicated to cancer research. At this year’s meeting, C&C Research Laboratories unveiled the mechanism of action and preclinical efficacy data of CNC-01 for the first time, while also exploring potential global licensing opportunities.
CNC-01 is an oral, small-molecule compound that selectively and directly inhibits both STAT5 and STAT3 proteins. It represents a First-in-Class therapeutic approach designed to address resistance mechanisms seen with existing acute myeloid leukemia (AML) treatments.
According to the preclinical data presented at AACR 2025, CNC-01 demonstrated superior tumor suppression compared to current FLT3 inhibitors in resistant models, including FLT3-TKD (F691L) mutations and bone marrow microenvironment-mimicking models.
Moreover, CNC-01 exhibited robust antitumor efficacy as a monotherapy in animal models unresponsive to BCL-2 inhibitors. Combination treatment with BCL-2 inhibitors further enhanced therapeutic outcomes. Notably, both complete remission (CR) and partial remission (PR) responses were observed in monotherapy and combination settings.
In survival analyses, CNC-01 monotherapy significantly prolonged survival compared to BCL-2 inhibitors alone. In combination therapy, more than half of the subjects remained alive at the study’s end, with the median survival not yet reached, underscoring the strong anticancer potential of CNC-01.
A representative from C&C Research Laboratories commented,
"AML remains a challenging disease with high unmet medical needs due to diverse genetic mutations and treatment resistance. Through its innovative dual inhibition mechanism, our STAT5/STAT3-targeted candidate has demonstrated outstanding preclinical efficacy. We aim to advance clinical development through global partnerships and licensing opportunities."
CNC-01 was discovered via JW’s proprietary AI-driven integrated R&D platform, JWave, and is currently under development with funding support as part of the Korea Drug Development Fund (KDDF) initiative, selected in 2023.
[Photo Caption]
C&C Research Laboratories presented preclinical results of CNC-01, an investigational dual STAT5/STAT3 inhibitor for AML, at AACR 2025 in Chicago on April 28 (local time).
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About C&C Research Laboratories
Founded in 1992 as Korea’s first joint biotech venture between JW Pharmaceutical and Chugai Pharmaceutical (a member of Roche Group), C&C Research Laboratories became a wholly owned subsidiary of JW Pharmaceutical in 2020. Since 2006, the company has been pursuing innovative drug discovery by integrating chemical and biological big data with AI and deep learning technologies.
C&C Research Laboratories currently manages a pipeline of eight novel drug candidates across oncology and immunology indications, with a strategic focus on targeting STAT (Signal Transducer and Activator of Transcription) proteins involved in cell growth, mutation, and proliferation.
About Acute Myeloid Leukemia (AML) and Market Outlook
AML is the most common type of leukemia in adults but remains difficult to treat due to high relapse rates and drug resistance. Given its rapid progression and potential for systemic spread, AML presents significant clinical challenges. Current standard therapies include cytarabine-based chemotherapy and venetoclax regimens, while relapsed/refractory AML cases are treated with targeted agents like gilteritinib (FLT3 inhibitor).
According to DRG Reports, the global AML therapeutics market, valued at USD 1.355 billion in 2021, is projected to grow at a CAGR exceeding 10%, reaching approximately USD 3.323 billion by 2031.