A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia

Inclusion Criteria:

1. Documented diagnosis of β-thalassemia or hemoglobin E/β-thalassemia, HbS/ β-thalassemia (β-thalassemia with α-bead mutation and/or multiplication is notallowed).

2. TDT subjects: receive regular blood transfusions, defined as 6-20 RBC units (including threshold) in the 24 weeks prior to screening assessment, and notransfusion-free period of ≥ 5 weeks during this period.

3. NTDT cohort: having transfused <6 RBC units in the 24 weeks prior to the screeningassessment, no regular transfusion schedule, and no transfusion for 4 weeks prior tothe screening assessment.

4. Subject transfusion records should be obtained within 24 weeks prior to thescreening assessment, containing the date of transfusion, transfused RBC units, andpre-transfusion hemoglobin values.

5. ECOG score 0-1.

6. NTDT subjects with Hb ≤ 10.0 g/dL at screening test and one follow-up test (twotests more than one week apart) and difference in values between the two tests ≤ 1.0g/dL.

7. Adequate liver function: Total bilirubin < 1.5 x upper limit of normal (ULN) (subjects with Gilbert syndrome, i.e., unconjugated hyperbilirubinemia, have a totalbilirubin < 3 x ULN), aspartate aminotransferase

Exclusion Criteria:

1. Other causes of anemia (e.g., hemolytic anemia, history of pure red blood cellaplastic anemia, myelodysplastic syndrome, or multiple myeloma)

2. Presence of active infection or inflammatory disease requiring systemicanti-infective therapy, including concomitant autoimmune diseases with inflammatorysymptoms (e.g. generalized erythema, ankylosing spondylitis, rheumatoid arthritis,psoriatic arthritis, dry syndrome, etc.)

3. Complicated retinal neovascularization requiring treatment (diabetic proliferativeretinopathy, age-related exudative macular degeneration, retinal vein occlusion,macular edema, etc.)

4. Inability to take oral medications, conditions with a history of gastrectomy/bowelresection that may have an effect on the absorption of gastrointestinal medications (excluding gastric polyps or colonic polypectomy), or gastroparesis that remainssymptomatic on current therapy

5. Clinically significant bleeding (requiring emergency blood transfusion within 12 hor a decrease in hemoglobin ≥ 2 g/dL within one week) within 4 weeks prior to thefirst dose, or a tendency to bleed or risk of bleeding that has not been medicallyor surgically corrected

6. Uncontrolled hypertension, defined as a diastolic blood pressure value >95 mmHg or asystolic blood pressure >160 mmHg on 2 or more of 3 repeated blood pressure tests (each at least 5 minutes apart) during the screening period

7. Complicated congestive heart failure (New York Heart Association [NYHA] class III orhigher).

8. history of stroke, transient ischemic attack (TIA), myocardial infarction,thromboembolic event (deep vein thrombosis, DVT), pulmonary embolism, or pulmonaryinfarction within 24 weeks prior to screening evaluation

9. history of significant coagulation abnormalities, or platelet count >600 x 109/L or <80 x 109/L

10. History of epilepsy or any past seizures.