A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia

A total of 40 subjects diagnosed with non-transfusion-dependent β-thalassemia will be enrolled in this study and assigned into four dosage cohorts. In each cohort, subjects will be randomized in a 4:1 ratio to receive 9MW3011 or placebo via intravenous infusion.

Key Inclusion Criteria:

• Male and female subjects aged 18 to 65 years (inclusive)
• Subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
• Subjects must meet the criteria for non-transfusion-dependent thalassemia
• Subjects must have a baseline hemoglobin level between 70-100 g/L(inclusive), based on 2 consecutive measurements taken at least 1 week apart within 4 weeks before randomization
• Subjects must have evidence of iron overload during screening
• Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1
• Subjects must fully understand the study procedures and methods, voluntarily participate in the trial, and sign an informed consent form

Key Exclusion Criteria:

• Subjects diagnosed with alpha-thalassemia
• Subjects diagnosed with HbS/beta-thalassemia or transfusion-dependent beta-thalassemia
• Subjects exhibit severe iron overload at the time of screening
• In addition to thalassemia, subjects have any other forms of anemia and hematological disorders that the investigator assesses may compromise safety or influence study outcomes
• Combined with any significant systemic diseases or psychiatric disorders
• Subjects have New York Heart Association (NYHA) Class III-IV heart failure and other cardiovascular diseases within 6 months prior to screening or currently present
• During the screening or baseline period, subjects exhibiting a QTcF interval of ≥450ms for males and ≥470ms for females on a 12-lead electrocardiogram (ECG), or presenting an abnormal 12-lead ECG with clinical significance
• Uncontrolled hypertension before screening
• A history of malignant neoplasm occurring within the last five years
• Severe infection requiring hospitalization or intravenous antimicrobial therapy, or uncontrolled systemic bacterial, fungal, or viral active infection
• Subject have received concomitant treatment that was not permitted by the protocol
• Subjects whose hematological parameters did not meet the inclusion criteria during screening
• Subjects with a history of substance abuse, as well as those who yield positive results on substance abuse screening
• Subjects who are unable to undergo MRI scans
• Pregnant or lactating women
• Subjects presenting any other factors deemed unsuitable for participation assessed by the investigator