Timdarpacept (IMM01) combined with azacitidine (AZA) as first-line therapy in adults with higher risk chronic myelomonocytic leukemia (CMML): results from a prospective, multicenter, phase II study.
15 affiliations
- 1The First Affiliated Hospital of Zhejiang University School of Medicine, 58# Chengzhan Road, Hangzhou, Zhejiang, China.
- 2Shengjing Hospital of Chinese Medical University, Shenyang, China.
- 3The First Hospital of Jilin University, Changchun, China.
- 4Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China.
- 5Henan Cancer Hospital, Affiliated Cancer Hospital of Zhengzhou University, Zhengzhou, China.
- 6Tianjin People's Hospital, Tianjin, China.
- 7The First Hospital of Chinese Medical University, Shenyang, China.
- 8Zhujiang Hospital of Southern Medical University, Guangzhou, China.
- 9The First Affiliated Hospital of Nanchang University, Nanchang, China.
- 10The First Affiliated Hospital of Henan University of Science and Technology, Luoyang, China.
- 11Xuanwu Hospital of Capital Medical University, Beijing, China.
- 12Fujian Medical University Union Hospital, Fuzhou, China.
- 13ImmuneOnco Biopharmaceuticals (Shanghai) Inc., Shanghai, China.
- 14The First Affiliated Hospital of Zhejiang University School of Medicine, 58# Chengzhan Road, Hangzhou, Zhejiang, China. [email protected].
- 15Blood Diseases Hospital, Institute of Hematology, Chinese Academy of Medical Sciences and Peking Union Medical College, #288 Nanjing Road, Tianjin, China. [email protected].
Abstract
Background
Timdarpacept (IMM01) is a recombinant signal regulatory protein α-IgG1 fusion protein that exerts antitumor activity by blocking the "don't eat me" signal while simultaneously activating the "eat me" signal, inducing robust antibody-dependent cellular phagocytosis. We report the final phase II results of treatment-naïve higher risk chronic myelomonocytic leukemia (CMML) patients treated with IMM01 in combination with azacitidine.Patients and methods
We assessed the safety and efficacy of IMM01 in combination with azacitidine (AZA) as first-line therapy for newly diagnosed CMML patients. IMM01 was administered intravenously at 2.0 mg/kg weekly, and AZA was administered subcutaneously at 75 mg/m² on days 1-7 of each 28-day cycle.Results
As of the data cut-off on May 13, 2025, 24 patients were enrolled. Of 22 efficacy-evaluable patients, the overall response rate (ORR) was 72.7% (16/22; 95% CI 49.8-89.3), including 27.3% complete response (CR), 13.6% marrow CR (mCR) with hematologic improvement (HI), 4.5% HI alone, and 27.3% mCR alone. The median time to response was 1.8 months (range, 0.9-3.6), and the median duration of response was 16.9 months (95% CI 5.1-NE). Median overall survival and progression-free survival were 22.2 months (95% CI 9.0-NE) and 17.8 months (95% CI 5.3-NE), respectively. The most common grade ≥ 3 treatment-related adverse events (≥ 10%) were lymphopenia (66.7%), leukopenia (62.5%), neutropenia (62.5%), thrombocytopenia (58.3%), anemia (33.3%), and pneumonia (16.7%).Conclusion
IMM01 in combination with AZA was generally well tolerated and effective in treatment-naïve higher risk CMML patients. Trial registration ClinicalTrials.gov identifier: NCT05140811.
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BibTeX
@article{ma2026timdarpacept,
title = {{Timdarpacept (IMM01) combined with azacitidine (AZA) as first-line therapy in adults with higher risk chronic myelomonocytic leukemia (CMML): results from a prospective, multicenter, phase II study.}},
author = {Liya Ma and Wei Yang and Sujun Gao and Junmin Li and Qingsong Yin and Xingli Zhao and Xiaojing Yan and Sanfang Tu and Fei Li and Haiping Yang and Ronghua Hu and Shaoyuan Wang and Zheng Dong and Xiwen Zhao and Guodong Lu and Zhuli Wu and Wenzhi Tian and Hongyan Tong and Zhijian Xiao},
journal = {Experimental hematology \& oncology},
year = {2026},
month = oct,
doi = {10.1186/s40164-026-00851-8},
url = {https://europepmc.org/article/MED/42958448},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - Timdarpacept (IMM01) combined with azacitidine (AZA) as first-line therapy in adults with higher risk chronic myelomonocytic leukemia (CMML): results from a prospective, multicenter, phase II study.
AU - Liya Ma
AU - Wei Yang
AU - Sujun Gao
AU - Junmin Li
AU - Qingsong Yin
AU - Xingli Zhao
AU - Xiaojing Yan
AU - Sanfang Tu
AU - Fei Li
AU - Haiping Yang
AU - Ronghua Hu
AU - Shaoyuan Wang
AU - Zheng Dong
AU - Xiwen Zhao
AU - Guodong Lu
AU - Zhuli Wu
AU - Wenzhi Tian
AU - Hongyan Tong
AU - Zhijian Xiao
PY - 2026
DA - 2026/10/28
JO - Experimental hematology & oncology
DO - 10.1186/s40164-026-00851-8
UR - https://europepmc.org/article/MED/42958448
AB - Background Timdarpacept (IMM01) is a recombinant signal regulatory protein α-IgG1 fusion protein that exerts antitumor activity by blocking the "don't eat me" signal while simultaneously activating the "eat me" signal, inducing robust antibody-dependent cellular phagocytosis. We report the final phase II results of treatment-naïve higher risk chronic myelomonocytic leukemia (CMML) patients treated with IMM01 in combination with azacitidine.Patients and methods We assessed the safety and efficacy of IMM01 in combination with azacitidine (AZA) as first-line therapy for newly diagnosed CMML patients. IMM01 was administered intravenously at 2.0 mg/kg weekly, and AZA was administered subcutaneously at 75 mg/m² on days 1-7 of each 28-day cycle.Results As of the data cut-off on May 13, 2025, 24 patients were enrolled. Of 22 efficacy-evaluable patients, the overall response rate (ORR) was 72.7% (16/22; 95% CI 49.8-89.3), including 27.3% complete response (CR), 13.6% marrow CR (mCR) with hematologic improvement (HI), 4.5% HI alone, and 27.3% mCR alone. The median time to response was 1.8 months (range, 0.9-3.6), and the median duration of response was 16.9 months (95% CI 5.1-NE). Median overall survival and progression-free survival were 22.2 months (95% CI 9.0-NE) and 17.8 months (95% CI 5.3-NE), respectively. The most common grade ≥ 3 treatment-related adverse events (≥ 10%) were lymphopenia (66.7%), leukopenia (62.5%), neutropenia (62.5%), thrombocytopenia (58.3%), anemia (33.3%), and pneumonia (16.7%).Conclusion IMM01 in combination with AZA was generally well tolerated and effective in treatment-naïve higher risk CMML patients. Trial registration ClinicalTrials.gov identifier: NCT05140811.
SN - 2162-3619
ER -