Cevidoplenib
Cevidoplenib (SKI-O-703) is an oral, highly selective SYK (Spleen tyrosine kinase) inhibitor that inhibits SYK-dependent Fcγ receptor signaling in macrophages, thereby reducing autoantibody-mediated platelet destruction in ITP (Immune thrombocytopenia).
DEVELOPMENT STAGE
Phase 2 — Completed in adults with persistent or chronic ITP
Commercialized
AT A GLANCE
Target
SYK
A cytoplasmic non-receptor tyrosine
kinase that plays a key role in
immune cell signaling
INDICATION
ITP
Persistent or chronic primary ITP
MODALITY
Small molecule
Oral, highly selective
The limitations of existing treatments for ITP and other autoimmune diseases supported development of a more selective SYK inhibitor
Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder in which available treatments, including corticosteroids, intravenous immunoglobulin (IVIG), thrombopoietin receptor agonists (TPO-RAs), rituximab, and splenectomy, can improve platelet counts but are often limited by variable or non-durable responses, relapse, treatment-related toxicities, and the need for prolonged therapy. These limitations created a need for mechanism-based therapies capable of providing sustained disease control with improved long-term tolerability.
SYK is a central signaling kinase downstream of Fcγ receptors and B-cell receptors and plays a key role in antibody-mediated platelet clearance as well as broader immune-cell activation. Its biology therefore provided a strong rationale for therapeutic intervention not only in ITP, but also in other autoimmune and hematologic diseases driven by aberrant Fc receptor- or B-cell receptor signaling. Although first-generation SYK inhibitors validated the target clinically, their broader kinase activity and tolerability limitations supported the development of more selective next-generation SYK inhibitors.
Selective SYK inhibition to reduce autoantibody-mediated platelet destruction
Cevidoplenib is an oral, highly selective SYK inhibitor designed to reduce autoantibody-mediated platelet destruction in ITP. Autoantibody-coated platelets engage Fcγ receptors on macrophages, activating SYK-dependent signaling that promotes phagocytic platelet clearance. By inhibiting SYK, cevidoplenib is designed to reduce platelet destruction and help restore platelet counts. In a global Phase 2 study, cevidoplenib demonstrated dose-dependent platelet responses and was generally well tolerated. Although the primary endpoint did not reach statistical significance, clinically meaningful platelet responses were observed across secondary endpoints. Cevidoplenib also received U.S. FDA Orphan Drug Designation for primary ITP in 2024.
Publications & Presentations
Clinical
A novel selective spleen tyrosine kinase inhibitor SKI-O-703 (cevidoplenib) ameliorates lupus nephritis and serum-induced arthritis in murine models
Cho S et al. · Clin Exp Immunol · 2023
Licensed to Agios
Exclusive global license · June 2026
Oscotec retains an option for exclusive development and commercialization rights in South Korea following Phase 3 results.
