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320 Preliminary Results of a Phase 2 Study of IMM01 Combined with Azacitidine (AZA) As the First-Line Treatment in Adult Patients with Higher Risk Myelodysplastic Syndromes (MDS)

Program: Oral and Poster Abstracts
Type: Oral
Session: 637. Myelodysplastic Syndromes – Clinical and Epidemiological: Treatments and Outcome Predictions in High Risk MDS
Hematology Disease Topics & Pathways:
Research, clinical trials, MDS, Clinical Research, Chronic Myeloid Malignancies, Diseases, Myeloid Malignancies
Saturday, December 9, 2023: 4:15 PM

Wei Yang1*, Sujun Gao2*, Xiaojing Yan3*, Rong Guo4*, Lijie Han4*, Fei Li5*, Yafei Wang6*, Junmin Li7*, Chunkang Chang, MD8*, Haiping Yang9*, Ronghua Hu10*, Hongyan Tong11*, Xingli Zhao12*, Qiubai Li13*, Jingdong Zhang14*, Xin Du, MD15*, Sanfang Tu16*, Cheng Zhang17*, Congmeng Lin18*, Xin Du19*, Zhenling Li20*, Ligen Liu21*, Zhenyu Li22*, Zheng Dong23*, Yixuan Yang24*, Qiying Lu24*, Wenzhi Tian24* and Zhijian Xiao25*

1Department of Hematology, Shengjing Hospital of China Medical University, Shenyang, China
2The First Hospital of Jilin University, Changchun, China
3The First Hospital of China Medical University, Shenyang, China
4The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China
5The First Affiliated Hospital of Nanchang University, Nanchang, China
6Tianjin Medical University Cancer Institute and Hospital, Tianjin, China
7Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China
8Shanghai Sixth People's Hospital, Shanghai, China
9The First Affiliated Hospital of Henan University of Science and Technology, Luoyang, China
10Xuanwu Hospital of Capital Medical University, Beijing, China
11First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, China
12Tianjin People's Hospital, Tianjin, China
13Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
14Ganzhou People's Hospital, Ganzhou, China
15Division of Hematology, Shenzhen Second People’s Hospital, The First Affiliated Hospital of Shenzhen University, Shenzhen, China
16Department of Hematology, Zhujiang Hospital of Southern Medical University, Guangzhou, China
17Xinqiao Hospital, Army Medical University, Chongqing, China
18Zhangzhou Municipal Hospital of Fujian Province, Zhangzhou, China
19Guangdong Provincial People's Hospital, Guangzhou, China
20China-Japan Friendship Hospital, Beijing, China
21Tongren Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China
22Affiliated Hospital of Xuzhou Medical University, Xuzhou, China
23ImmuneOnco Biopharmaceuticals (Shanghai) Inc., Beijing, China
24ImmuneOnco Biopharmaceuticals (Shanghai) Inc., Shanghai, China
25Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China


Patients diagnosed with higher-risk myelodysplastic syndrome (MDS) have poor prognosis. IMM01 is a recombinant signal regulatory protein α (SIRPα) IgG1 fusion protein that exerts anti-tumor activities via blocking "Don't eat me" signal and activating the "Eat me" signal to induce strong antibody-dependent cellular phagocytosis (ADCP). Furthermore, IMM01 activated macrophages could process and present tumor antigen to T cells and elicit long-last tumor-specific T cell immune response.


This is an open-label, multi-center, phase 2 study (NCT05140811) that evaluated safety and efficacy of IMM01 in combination with AZA as the first-line treatment for patients with untreated higher-risk MDS. Enrolled patients were aged ≥18 years with intermediate to very high risk MDS (IPSS-R >3.5) who were not eligible for stem cell transplant or intensive chemotherapy. Patients with treatment-naive MDS were administered intravenous IMM01 at a dosage of 2.0mg/kg/week and subcutaneous AZA at a dosage of 75 mg/m2 on D1-7 per 28-day cycle. Adverse events (AEs) were reported according to CTCAE v5.0. Efficacy was assessed by IWG 2006 (MDS) criteria.


A total of 54 patients were enrolled from June 29, 2022 to June 11, 2023. The median age was 64 (30-83) years, with 39 (72.2%) being male, and 52 (96.3%) having an ECOG of ≥1. Based on risk classification per IPSS-R, 13 patients (24.1%) were intermediate risk (IR), 25 (46.3%) high risk (HR), and 16 (29.6%) very high risk (vHR). At baseline, the median levels of blood counts were 69 (35-95)g/L for hemoglobin, 39.5 (2-409)×109/L for platelets and 0.8 (0.1-8.6)×109/L for neutrophils. By the data cut of June 11th, 2023, the median duration of follow-up was 5.6 months (95%CI:3.6-7.9). Among the 22 efficacy evaluable patients who received initial treatment of ≥4 months, overall response rate (ORR) was 81.8% (18/22), including 36.4%(8/22) complete response (CR) rate, 22.7%(5/22) marrow CR (mCR) with hematologic improvement (HI), 9.1% (2/22) HI and 13.6% (3/22) mCR alone. Among the 17 efficacy evaluable patients who received initial treatment of ≥6 months, ORR was 88.2%(15/17), including 41.2% (7/17) CR, 29.4% (5/17) mCR with HI, 5.9% (1/17) HI and 11.8% (2/17) mCR alone. The median duration of response (DoR) was not reached. Biomarker data showed mutation burdens of several markers, including TP53, DNMT3A, ASXL1, U2AF1, were dramatically reduced on study treatment. The most frequent treatment related adverse events (TRAEs) (≥20%) were leukopenia (85.2%), thrombocytopenia (72.2%), neutropenia (66.7%), lymphopenia (57.4%), anemia (44.4%), vomiting (44.4%), pyrexia (33.3%), infusion related reaction (33.3%), constipation (29.6%), nausea (25.9%), hypoalbuminemia (22.2%) and infection (20.4%). The most common ≥G3 TRAEs (≥10%) included leukopenia (81.5%), thrombocytopenia (68.5%), neutropenia (66.7%), lymphopenia (57.4%), anemia (44.4%) and infection(16.7%). These AEs were consistent with the AE profile of the AZA monotherapy in treatment-naive MDS as commonly reported in China. Without using of a low priming dose, no Grade ≥3 hemolysis occurred. The study is ongoing.


Preliminary data from IMM01 (without low-dose priming) combined with AZA were well tolerated and showed exciting efficacy results in patients with treatment-naive higher-risk MDS.

Disclosures: Dong: ImmuneOnco Biopharmaceuticals (Shanghai) Inc.: Current Employment. Yang: ImmuneOnco Biopharmaceuticals(Shanghai) Inc.: Current Employment. Lu: ImmuneOnco Biopharmaceuticals(Shanghai) Inc.: Current Employment. Tian: ImmuneOnco Biopharmaceuticals (Shanghai) Inc: Current Employment, Current equity holder in private company.

*signifies non-member of ASH