Pivotal data from Phase 3 study of sutimlimab in cold agglutinin disease to be presented at ASH 2019 late-breaking session

Cambridge, MA--(BUSINESS WIRE)--Full data from the pivotal Phase 3 CARDINAL trial evaluating the safety and efficacy of sutimlimab in people with primary cold agglutinin disease (CAD) with a history of recent transfusions will be presented as part of the Late-Breaking Abstracts Session at the upcoming 61st Annual Meeting of the American Society of Hematology (ASH), December 10 at 7:45AM (ET). Sutimlimab met the primary endpoint of the CARDINAL study.

CAD is a rare, chronic and serious blood disease that results in the constant and premature destruction of red blood cells (hemolysis) by the body’s immune system. There are currently no approved therapies for CAD. People with CAD suffer from chronic hemolytic anemia and debilitating fatigue. Sutimlimab is the first investigational complement inhibitor for the treatment of CAD to present Phase 3 data.

“People with cold agglutinin disease currently have no approved therapies and experience serious disease burden,” said John Reed, M.D., Ph.D., Global Head of Research and Development at Sanofi. “These pivotal data demonstrate the potential of sutimlimab as a first-in-class targeted treatment for this chronic and debilitating rare blood disorder.”

Sutimlimab, a potential targeted C1s therapy

Sutimlimab is an investigational monoclonal antibody designed to selectively target and inhibit C1s in the classical complement pathway, a part of the immune system that is responsible for activating the mechanism of hemolysis in CAD. Sutimlimab aims to selectively inhibit disease processes related to the classical complement pathway while maintaining activity of the alternative and lectin complement pathways, which are important for immune surveillance.

The late-breaking abstract “Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results from the Phase 3 Cardinal Study” will be presented by lead author, Alexander Röth, M.D., Department of Hematology, University Hospital, University of Duisburg-Essen, Germany. These data will serve as the basis for marketing authorization applications for sutimlimab.

Sutimlimab was granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) and Orphan Drug status by the FDA, European Medicines Agency and the Pharmaceuticals and Medical Devices Agency in Japan. The efficacy and safety of sutimlimab has not been reviewed by any regulatory authority.

About the pivotal Phase 3 CARDINAL Trial

Sutimlimab is being evaluated in two Phase 3 clinical trials. The CARDINAL trial is a pivotal, open-label, single-arm study to assess the efficacy and safety of sutimlimab in adult patients with primary CAD who received a recent blood transfusion. Patients received a fixed weight-based dose (6.5g or 7.5g) of sutimlimab via intravenous infusion on Day 0, Day 7 and then once every other week up to Week 26. A total of 24 patients were enrolled; two patients withdrew from the study for non-drug related reasons. All 22 patients that completed Part A of the CARDINAL study have enrolled in an ongoing long-term follow-up study (Part B).

The primary efficacy outcome was response rate based on a composite of an increase in hemoglobin ≥ 2 g/dL from baseline or reaching a hemoglobin level ≥ 12g/dL at treatment assessment endpoint and the absence of transfusions between Weeks 5 to 26. Further, patients were not allowed to receive other CAD-related treatments. Secondary endpoints included mean change from baseline in hemoglobin, bilirubin, lactate dehydrogenase (LDH) and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score as well as number of transfusions and blood units transfused after the first five weeks of drug administration.

For more information, please visit www.clinicaltrials.gov, study identifier number NCT03347396.

About Cold Agglutinin Disease (CAD)

CAD is a serious, chronic rare blood disease in which a part of the body’s immune system called the complement system mistakenly attacks a person’s own healthy red blood cells. People with CAD suffer from chronic anemia, debilitating fatigue, acute hemolytic crisis and a poor quality of life. Retrospective analyses have also demonstrated other potential complications for CAD patients including an increased risk of thromboembolic events and early mortality. CAD occurs in approximately 16 people per millioni, including an estimated 12,000 people in the United States, Europe and Japan.

i Berentsen S, et al. Haematologica. 2006;91(4):460-466

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