Sanofi presented data from the CARDINAL Phase 3 study data (final Part A) at the Late-Breaking Abstracts Session of the 61st Annual Meeting of the American Society of Hematology in Orlando, FL. Results met the primary and secondary endpoints and Sanofi intends to file a BLA in the near future.
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. The primary efficacy outcome was a responder rate based on a composite of an increase in hemoglobin ≥2 g/dL from baseline or reaching a hemoglobin level ≥12 g/dL at the 26-week treatment assessment timepoint and the absence of transfusions from Weeks 5 to 26, further, patients were not allowed to receive other CAD-related treatments.
The pre-specified primary endpoint was met. 54% (n=13) of patients met the composite endpoint criteria, with 62.5% (n=15) of patients achieving a hemoglobin ≥ 12 g/dL or an increase of at least 2 g/dL and 71% (n=17) of patients remaining transfusion-free after week 5. The secondary efficacy measures were also met, including hemoglobin, bilirubin (a measure of red blood cell destruction in CAD), Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score (a quality of life measure of fatigue), lactate dehydrogenase (LDH), and transfusion usage.
Sutimlimab has been 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.
Sutimlimab is a potential first-in-class, investigational, humanized, monoclonal antibody that has been specifically designed to target C1s, a serine protease within the C1-complex, that is the first step in activating the classical complement pathway of the immune system. Activation of the classical complement pathway is the central mechanism of hemolysis in CAD and blocking it may potentially halt the CAD disease process. With a novel mechanism of action and high target specificity, sutimlimab is designed to selectively inhibit disease processes upstream in the classical complement pathway while leaving intact the alternative and lectin complement pathways and their immune surveillance functions.
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 million, including an estimated 12,000 people in the United States, Europe and Japan.