Experimental Drug Shows Promise against Rare Blood Disorder


Initial human clinical trials for Sutimlimab revealed that the drug has a significant effect in treating cold agglutinin disease, a rare chronic blood disorder for which there are currently no approved treatments. Until now, the only option for patients was Rituximab — a treatment for certain blood cancers, sometimes combined with chemotherapy, which produced limited results.

The disease usually appears due to an underlying condition such as an infection (Epstein – Barr virus, mycoplasma), another autoimmune disease, or certain cancers such as lymphoma. It causes antibodies to latch onto and kill red blood cells, leading to hemolytic anemia as bone marrow can’t produce red blood cells as quickly as they are destroyed.

Sutimlimab is a first-in-class monoclonal antibody that acts by selectively inhibiting one of the three complement pathways in the immune system leaving the other two pathways intact.

The drug was well tolerated, produced clinically meaningful increases in hemoglobin levels, and precluded the need for transfusions, even in patients for whom multiple prior therapies had failed”, noted senior author Bernd Jilma, MD, of the Medical University of Vienna.

Cold agglutinin disease generally affects patients 55 and older and symptoms include severe pain in the back and legs, headache, vomiting, diarrhea, dark urine and hepatosplenomegaly. A cold environment or a concurrent infection may trigger or exacerbate the condition.

Trial participants typically responded within a week of treatment with a full dose of Sutimlimab as hemoglobin levels significantly increased.

The drug clearly addresses an unmet medical need, as we have seen rapid, strong responses in patients for whom multiple prior therapies have failed”, reported Dr. Jilma.