China-based Staidson (Beijing) Pharmaceutical Co., Ltd (SHE: 300204) has announced the first subject dosing in a Phase II clinical study for its Category 1 biologic STSP-0601. The study aims to evaluate the drug as an on-demand treatment for bleeding in patients with hemophilia A or B without inhibitors. This marks a significant step forward in the development of innovative treatments for hemophilia.
Previous Clinical Studies and Breakthrough Designation
STSP-0601 received approval for a clinical study in China in July 2019. In August 2021, the drug concluded a Phase I clinical study as an on-demand treatment for bleeding in patients with hemophilia A or B with inhibitors, demonstrating a good safety and tolerability profile. The drug was awarded breakthrough therapy designation (BTD) in August of last year, underscoring its potential to address significant unmet needs in hemophilia treatment.
Hemophilia Background
Hemophilia is a recessive hereditary hemorrhagic disease linked to chromosome X, divided into hemophilia A and hemophilia B. Hemophilia A is characterized by a deficiency of coagulation factor VIII (F VIII), while hemophilia B is characterized by a deficiency of coagulation factor IX (F IX). Both conditions are caused by mutations in the corresponding coagulation factor genes. The lack or abnormal function of F VIII or F IX in circulating blood leads to deficiencies in activated coagulation factor X (F Xa) and thrombin, affecting normal blood coagulation and causing bleeding. Clinical features include a bleeding tendency, primarily in joints, muscles, and deep tissues. Repeated bleeding, if untreated, can lead to joint deformity and pseudotumor formation, and severe cases may be life-threatening.
Market Landscape
Currently, treatments available in China include Roche’s Hemlibra (emicizumab), China Biologic Products’ human coagulation factor IX, and Sanofi’s Alprolix (eftrenonacog alfa). Staidson’s STSP-0601 aims to offer a new therapeutic option for patients with hemophilia A and B, enhancing the treatment landscape for this challenging condition.-Fineline Info & Tech
