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)

作者：Wei Yang, Sujun Gao, Xiaojing Yan, Rong Guo, Lijie Han, Fei Li, Yafei Wang, Junmin Li, Chunkang Chang, Haiping Yang, Rong‐Hua Hu, Hongyan Tong, Xingli Zhao, Qiubai Li, Jingdong Zhang, Xin Du, Sanfang Tu, Cheng Zhang, Congmeng Lin, Xin Du, Zhenling Li, Ligen Liu, Zhenyu Li, Zheng Dong, Yixuan Yang, Qiying Lu, Wenzhi Tian, Zhijian Xiao · 发表于：Blood · 年份：2023 · DOI：10.1182/blood-2023-174420 · 被引用次数：9 · 研究领域：Acute Myeloid Leukemia Research、Acute Lymphoblastic Leukemia research、Immunodeficiency and Autoimmune Disorders

Background: 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. Methods: 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/m 2 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. Results: 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 ris...