Cemsidomide

Cemsidomide is an investigational oral cereblon-modulating protein degrader of IKZF1/3, transcription factors foundational to multiple myeloma biology. Data from the fully enrolled Phase 1 trial show cemsidomide’s differentiated safety and tolerability profile and potentially class-leading anti-myeloma activity that supports the potential for durable outcomes.

IKZF1/3 Degradation to Treat Multiple Myeloma

Multiple myeloma cells rely on IKZF1/3 for survival. Degrading IKZF1/3 leads to myeloma cell death and drives anti-myeloma activity. IKZF1/3 also directly regulate the activity of IRF4, a transcription factor that regulates downstream immune cell activation. When IKZF1/3 are degraded, it leads to downregulation of IRF4, which promotes myeloma cell death, as well as T-cell activation.

IKZF1/3 degradation is a clinically validated mechanism of action and a foundational therapy for multiple myeloma, used across multiple treatment lines and combinations because it kills myeloma cells and promotes T-cell activation.

Scientific Presentations and Publications