HMPL-523 (Sovleplenib) in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

A Randomized, Double-Blind, Placebo-Controlled Phase II/III Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of HMPL-523 in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Phase II Study: To evaluate the safety and preliminary efficacy of HMPL-523 in adult patients with wAIHA.

Phase III Study(Part A): Confirmation of Efficacy safety and of HMPL-523 in adult patients with wAIHA.

Phase III Study (Part B): To further evaluate the long-term safety and tolerability of HMPL-523 in adult patients with wAIHA.

Phase II Study: An eight-week randomized, placebo-controlled, double-blind phase followed by at least a 16-week open-label HMPL-523 treatment to evaluate the safety and preliminary efficacy of HMPL-523. The primary endpoint was the proportion of patients with overall Hb response by week 24.

Phase III study (Part A):A 24-week randomized, placebo-controlled double-blind phase to evaluate efficacy and safety of HMPL-523. The primary endpoint was the proportion of patients who achieve a durable response during weeks 5 to 24.

Phase III Study (Part B): An open-label Phase evaluating long-term safety and efficacy of HMPL-523 treatment. Eligible patients include those with lack of efficacy during the 20-week Phase III Part A treatment, completion of the Phase II study, or completion of 24-week Phase III Part A treatment with investigators assessment of potential benefit from open-label treatment with HMPL-523.

Inclusion Criteria:

1. Voluntarily signed the informed consent form (ICF);
2. Males or females aged 18 to 75 years;
3. Patients diagnosed with primary wAIHA or secondary wAIHA whose underlying diseases are stable;
4. Organs in good function.

Exclusion Criteria:

1. Patients with other types of AIHA other than wAIHA;
2. Patients with secondary wAIHA with unstable underlying disease;
3. Patients with drug-induced secondary wAIHA;
4. Patients with infections requiring systemic treatment;
5. Patients previously treated with Syk inhibitors (e.g., fostamatinib);
6. Patients with known allergy to the active ingredients or excipients of the study drug;
7. Patients with serious psychological or mental disorder;
8. Alcoholic or drug abuser;
9. Female patients who are pregnant and lactating.