Data Reinforce Sutimlimab as First-in-Class Investigational C1s Inhibitor for Hemolysis in Patients With Cold Agglutinin Disease


Results from part A of a phase 3 trial showed sutimlimab caused patients to experience a rapid and sustained inhibition of C1-activated hemolysis in patients with cold agglutinin disease.

Results from part A of the phase 3 CADENZA trial evaluating the safety and efficacy of sutimlimab in people with cold agglutinin disease (CAD) who have not received a blood transfusion within the previous 6 months were presented at the European Hematology Association 2021 Congress.

During the trial, investigators randomized patients 1:1 to receive a fixed weight-based dose of 6.5 g or 7.5 g of sutimlimab or placebo via intravenous infusion on day 0, day 7, and then once every other week until week 26. Currently, the open-label part B portion of the study is still ongoing, with a focus on assessing long-term safety and durability of response to sutimlimab among all participants with CAD.

With a mean age of 66.7 years, 42 patients were enrolled and randomized to receive either sutimlimab (N=22) or placebo (N=20). Additionally, 86% of patients in the sutimlimab arm and 100% in the placebo arms completed part A and continued onto part B. Among these patients, 14% from the sutimlimab group discontinued at part A due to the occurrence of adverse events.

Data showed sutimlimab caused patients to experience a rapid and sustained inhibition of C1-activated hemolysis in patients with CAD. This observation was noted by investigators as occurring within the first week of treatment, showing patients experienced clinically significant improvements in both hemoglobin and fatigue compared to a placebo.

“Cold agglutinin disease causes the body’s immune system to mistakenly destroy its healthy red blood cells. People living with cold agglutinin disease experience the crippling impact of chronic hemolysis that can cause severe anemia, profound fatigue and can have acute hemolytic crisis,” said principal investigator and presenting author professor Alexander Röth, MD, Department of Hematology and Stem Cell Transplantation, University Hospital, University of Duisburg-Essen, Germany, in the press release. “The positive evidence from the CADENZA trial demonstrate significant improvements in hemolysis and meaningful impact on key measures of anemia and fatigue.”

CADENZA is the first double-blind, placebo-controlled phase 3 trial investigating sutimlimab, with the primary efficacy outcome set at the proportion of patients who met 3 components of assessment. The components included improvement in hemoglobin ≥1.5 g/dL from baseline at treatment assessment timepoint, avoidance of transfusions from week 5 through week 26, and avoidance of other CAD-related therapies outside of what was permitted from week 5 through week 26.

During the trial, the secondary efficacy measures assessed the improvement from baseline in key indicators of the disease process, which included hemoglobin, bilirubin, lactate dehydrogenase (LDH) levels, and quality of life as measured by Functional Assessment of Chronic Illness Therapy-Fatigue Score.

“The results from CADENZA and data from the phase 3 CARDINAL study, presented as a late-breaker at the American Society of Hematology congress in 2019, will be the basis of our filing with the European Medicines Agency. Together, the studies highlight the promising potential of sutimlimab to have a meaningful impact for people living with CAD,” said Karin Knobe, MD, PhD, head of development, rare and rare blood disorders, Sanofi, in the press release. “Based on the robust clinical evidence we have to-date, sutimlimab significantly inhibits hemolysis and has the potential to be an important new treatment for CAD.”

Following the phase 3 trial results, sutimlimab was granted breakthrough therapy by the FDA and orphan drug status by the FDA, European Medicines Agency, and the Pharmaceuticals and Medical Devices Agency in Japan. To date, sutimlimab has not yet been approved by any regulatory authority, although the company currently has plans to resubmit its biologics license application with the FDA in the second half of 2021.


New pivotal data at EHA 2021 reinforces sutimlimab as a first-in-class investigational C1s inhibitor with the potential to be the first approved treatment for hemolysis in people with CAD, a serious and chronic autoimmune hemolytic anemia. Paris, France: Sanofi; June 11, 2021. Accessed June 14, 2021.

Updated June 17, 2021 at 10:20 AM EST

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