March 13, 2025 – Fosun Pharma announced that its subsidiary S-INFINITY Co., Ltd. (“S-INFINITY”) recently received approval from the National Medical Products Administration (NMPA) to conduct clinical trials for XH-S003 Capsule (registration category: chemical drug Class 1; hereinafter referred to as “XH-S003”) for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). S-INFINITY plans to initiate Phase 2 clinical trials in Mainland China (excluding Hong Kong, Macao, and Taiwan regions) once conditions are met.

XH-S003 is a small molecule inhibitor of the complement system independently developed by Fosun Pharma, intended for the treatment of diseases associated with abnormal complement activation. As a specific inhibitor of the alternative complement pathway, XH-S003 acts upstream of the terminal C5 pathway. It simultaneously controls intravascular and extravascular hemolysis, demonstrating superior therapeutic efficacy compared to C5 monoclonal antibody therapies. This innovation addresses the limitations of C5 antibodies while providing patients with an oral medication option.

Xingli Wang, Executive President of Fosun Pharma and CEO of the Global R&D Center, stated, “The approval of Phase II clinical trials for XH-S003 marks another milestone in Fosun Pharma’s continuous innovation in rare diseases. PNH patients have long faced limited treatment options and severe challenges to their quality of life. Fosun Pharma remains patient-centric, addressing unmet clinical needs by advancing the development of rare disease therapies. We aim to fill gaps in treatment paradigms and enhance access to innovative therapies for rare disease patients in China, ultimately benefiting more individuals.”

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a complement-mediated chronic rare blood disorder. Mutations in the PIG-A gene in patients’ hematopoietic stem cells lead to the production of red blood cells that are prematurely destroyed by the complement system, resulting in intravascular hemolysis (destruction of red blood cells within blood vessels) and extravascular hemolysis (destruction in the spleen and liver). Clinical manifestations include anemia, paroxysmal hemoglobinuria, bone marrow failure, and thrombosis. PNH severely impacts patients’ quality of life, with an incidence rate of approximately 1-2 per million, higher in Asian populations compared to Western regions. Anti-C5 therapy has been internationally recognized as the standard treatment for PNH. However, a significant proportion of patients still experience residual anemia, fatigue, and transfusion dependency post-treatment, severely affecting their daily lives.

To date, XH-S003 has completed Phase I clinical trials in Australia and Mainland China for another indication, IgA nephropathy and other complement-driven glomerular diseases, and is currently in Phase II clinical trials in Mainland China.