Sutimlimab Shows Promise for Hard-to-Treat, Rare Blood Disorder
Improves Severe Hemolytic Anemia in Cold Agglutinin Disease
Published on: December 17, 2018
December 17, 2018) – In a first-in-human clinical trial reported today in Blood, the investigational drug
sutimlimab appeared to be effective in treating cold agglutinin disease, a rare
chronic blood disorder for which there are currently no approved treatments.
agglutinin disease is caused by a malfunction in the immune system that causes antibodies––components
of the immune system that are produced in the blood and help the body fight off
disease–– to mistakenly latch onto and kill red blood cells. The disease is a
type of hemolytic anemia, a condition that occurs when the bone marrow can’t
produce red blood cells as quickly as they are destroyed.
the study, sutimlimab, a specific C1s inhibitor, rapidly halted the destruction
of red blood cells, increased hemoglobin levels, eliminated patients’ need for
blood transfusions, and caused no serious adverse effects.
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,” said senior author Bernd Jilma, MD, of the
Medical University of Vienna.
agglutinin disease is thought to affect about 10,000 people in the United
States and Europe. Most patients with the disease are over 50 years old.
date, there are no U.S. Food and Drug Administration (FDA)-approved treatments
for cold agglutinin disease, though rituximab— a treatment for certain blood
cancers — has been used with or without chemotherapy with limited success.
study included 10 patients 56-76 years old. Patients had cold agglutinin
disease for a median of five years, and many had received multiple prior
treatments that had been unsuccessful. At enrollment, all patients had
below-normal levels of hemoglobin, a component of red blood cells that is
responsible for transporting oxygen to cells and organs throughout the body.
Six patients were receiving regular blood transfusions to control their
those patients who responded, within the first week of treatment with a full
dose of sutimlimab, destruction of red blood cells stopped and patients’
hemoglobin levels significantly increased, said Dr. Jilma. Seven patients responded
and had a median increase in hemoglobin levels of 4 grams per deciliter (g/dL)
over a baseline of 7.5 g/dL within six weeks. Four patients had their
hemoglobin levels return to normal.
sutimlimab treatment was discontinued and the drug had left the patients’
blood, hemoglobin levels dropped and destruction of red blood cells began
again. However, when treatment resumed, these effects were once again reversed.
The six patients who had been sustained by regular blood transfusions remained
transfusion-free for up to 18 months while receiving sutimlimab treatment.
that safety results remain positive, sutimlimab could become the first approved
treatment for cold agglutinin disease,” Dr. Jilma said. “The drug clearly
addresses an unmet medical need, as we have seen rapid, strong responses in
patients for whom multiple prior therapies have failed.”
has been given Breakthrough Therapy Designation by the U.S. Food and Drug
Administration. Phase III studies are ongoing to determine its safety and
efficacy in primary cold agglutinin disease patients.
This study was
supported by True North Therapeutics, Inc. (now part of Bioverativ Inc., a
Sanofi company). For more information about the sutimlimab phase III studies,
visit clinicaltrials.gov (study numbers: NCT03347396 and NCT03347422).
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is a journal of the American Society of Hematology (ASH) (www.hematology.org),
the world’s largest professional society concerned with the causes and
treatment of blood disorders.
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the understanding, diagnosis, treatment, and prevention of disorders affecting
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