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

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 Studies: Confirmation of Efficacy safety and of HMPL-523 in Adult Patients With wAIHA

Phase II Study: the proportion of patients with overall Hb response by Week 24 Phase III study: the proportion of patients who achieve a durable response by Week 24

Phase II: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 8 weeks and at least 16 weeks open-label treatment. Phase III: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 24 weeks and enter open-label phase in the opinion of the Investigator.

Phase II: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 8 weeks and at least 16 weeks open-label treatment. Phase III: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 24 weeks and enter open-label phase in the opinion of the Investigator.

Phase III: Durable Hb response rate: the proportion of patients who achieve a durable response by Week 24

Inclusion Criteria: Voluntarily signed the informed consent form (ICF); Males or females aged 18 to 75 years; Patients diagnosed with primary wAIHA or secondary wAIHA whose underlying diseases are stable; Organs in good function. Exclusion Criteria: Patients with other types of AIHA other than wAIHA; Patients with secondary wAIHA with unstable underlying disease; Patients with drug-induced secondary wAIHA; Patients with infections requiring systemic treatment; Patients previously treated with Syk inhibitors (e.g., fostamatinib); Patients with known allergy to the active ingredients or excipients of the study drug; Patients with serious psychological or mental disorder; Alcoholic or drug abuser; Female patients who are pregnant and lactating.

The results of this study can be published in core journals or international scientific conferences, and the primary investigators who make significant contributions to the implementation and management of this study and the personnel who makes great contributions to the design, interpretation or analysis of this study (such as the staffs or consultants of the sponsor) can have their authorship attribution. The sponsor promises to provide the manuscript to the investigator for review before publication of any result of the study. Investigators have to obtain the approval of the sponsor before submitting academic articles or abstracts. The study personnel have the right to publish results of this study, however, the requirement of protecting confidential information must be met. The confidential information is the property of the sponsor only, cannot be disclosed to others without the written approval of the sponsor, and cannot be used for other purposes.