Cemsidomide is an orally bioavailable MonoDAC™ degrader (or monofunctional degradation activating compound) designed to be highly potent and selective against its intended targets of Ikaros (IKZF1) and Aiolos (IKZF3) and overcome shortcomings of currently approved therapies to treat multiple myeloma and non-Hodgkin’s lymphoma. Initial clinical data show cemsidomide is well tolerated, demonstrates anti-myeloma activity and displays evidence of immunomodulatory effects.

Opportunity for Targeted Protein Degradation

IKZF1/3 are transcription factors required for cancer cell growth and survival in multiple myeloma and non-Hodgkin’s lymphomas. However, transcription factors do not have an active site binding pocket; this means inhibitors cannot target these important proteins. Rather, degraders are the only therapeutic approach. Today, approved IKZF1/3 degraders (lenalidomide and pomalidomide) are widely used in multiple myeloma treatment, but there remains a high unmet medical need for therapies to treat relapsed/refractory multiple myeloma.

Scientific Presentations and Publications