- CMS-D017 is a novel selective small-molecule inhibitor of complement factor B（CFB）.By targeting and inhibiting CFB, it blocks abnormal activation of the alternative complement pathway, which reduces damage to target tissues and organs caused by the membrane attack complex, and effectively slows the progression of a range of complement dysregulation-related inflammatory responses and autoimmune diseases.
- CMS-D017 has demonstrated excellent efficacy and safety in preclinical studiesand is being developed for clinical use in the treatment of paroxysmal nocturnal hemoglobinuria, providing a potentially better therapeutic alternative for patients.
- Upon approval for marketing, CMS-D017 will synergize with the marketed innovative drug Velphoro (hyperphosphatemia of Chronic Kidney Disease) and the new drug currently under NDA review, Oxemia (renal anemia), in expert networks and market resources, collectively elevating the Group’scapabilities in the field of nephrology.
China Medical System Holdings Limited ( “CMS” or the “Group”) is pleased to announce that innovative drug CMS-D017 capsules (“CMS-D017” or the “Product”) self-developed by the Group, has obtained the Drug Clinical Trial Approval Notice issued by National Medical Products Administration (“NMPA”) on 30 January 2026. The NMPA has approved the Group to conduct clinical trials in healthy participants in China to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic characteristics of CMS-D017.
About CMS-D017
CMS-D017 is a novel selective small-molecule inhibitor of complement factor B. The complement system, a vital component of the innate immune system, exerts its biological functions through activation via the classical, lectin, or alternative pathways[1]. Complement factor B, a specific serine protease primarily synthesized in the liver, acts as the “core switch” and “amplifier” of the alternative complement pathway, with its activity directly determining the intensity of complement responses. Excessive activation of the complement system can attack the body’s own cells and tissues, leading to a range of inflammatory responses and autoimmune diseases.
By targeting and inhibiting complement factor B, CMS-D017 blocks abnormal activation of the alternative complement pathway, which reduces damage to target tissues and organs caused by the membrane attack complex, and effectively slows the progression of complement dysregulation-related diseases[2]. CMS-D017 has demonstrated excellent efficacy and safety in preclinical studies. It is being developed for clinical use in the treatment of paroxysmal nocturnal hemoglobinuria. Future development plans for CMS-D017 also include the treatment of complement-mediated kidney diseases, age-related macular degeneration, myasthenia gravis and other conditions.
A potentially better therapeutic option for Paroxysmal nocturnal hemoglobinuria (PNH)
A rare disease, paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder of hematopoietic stem cells caused by mutations in the PIG-A gene located on the X chromosome. Patients typically present with clinical manifestations such as hemoglobinuria, thrombosis, and pancytopenia. Complement C5 inhibitors currently serve as the first-line treatment for PNH; however, they are effective only against intravascular hemolysis and do not address extravascular hemolysis[3]. Moreover, since complement C5 functions as a common downstream component in all three complement pathways, its inhibition carries a significant risk of infection. Existing C5 inhibitors also require intravenous or subcutaneous administration, which poses challenges for long-term treatment convenience.
CMS-D017 inhibits C3 convertase formation by binding to complement factor B upstream in the alternative complement pathway. It thereby targets both extravascular and intravascular hemolysis, while sparing the classical and lectin pathways—a mechanism that may lower infection risk[4]. Its oral formulation further enhances patient compliance, making it a potentially better therapeutic alternative for PNH patients.
Upon approval for marketing, CMS-D017 will significantly enhance the Group’s capabilities in the field of nephrology. It will synergize with the marketed innovative drug Velphoro (Sucroferric Oxyhydroxide Chewable Tablets, indicated for hyperphosphatemia of Chronic Kidney Disease) and the new drug Oxemia (Desidustat Tablets, indicated for renal anemia, currently undergoing regulatory review for market approval) in expert networks and market resources, collectively elevating the Group’s competitiveness and market position in this field.
The Group is actively preparing to initiate relevant clinical trials and strives to launch the Product as soon as possible.
Reference:
- McMurray JC., et al. Immunodeficiency: Complement disorders. Allergy Asthma Proc. 2024;45(5):305-309.
- Zhu Min, He Chenglu, Li Ya. Research Progress of Complement Factor B [J]. Journal of Qiqihar Medical University, 2025, 46(10): 979-984.
- Risitano, A.M., et al. Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT. Front Immunol, 2019. 10: p. 1157.
- Le Xi, Du Yali, Huang Yangyu, et al. Clinical Application of Complement Inhibitors in Rare Diseases [J]. Rare Disease Research.2022, 1(04): 391-399.
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