Pivotal data from phase III study of sutimlimab in cold agglutinin disease to be presented at ASH 2019 meeting.- Sanofi

Full data from the pivotal Phase III 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 III data. The late-breaking abstract �Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results from the Phase III Cardinal Study� will be presented by lead author, Alexander R�th, 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.

The open-label CARDINAL study looked at sutimlimab in 24 adults with primary CAD who received a recent blood transfusion who were given a fixed weight-based dose of sutimlimab (6.5g or 7.5g) of sutimlimab via intravenous infusion on day zero, day seven and then once every other week up to week 26. Two patients withdrew, but the estimated mean Hb increase at treatment assessment time point was 2.6g/dL. Hemoglobin improved rapidly after the first dose of sutimlimab with 1.2 g/dL and 2.3 g/dL increases by weeks one and three, respectively. Mean overall hemoglobin was maintained above 11g/dL after week 3. 20 patients had a mean hemoglobin increase greater than 1 g/dL. The primary efficacy outcome was response rate based on a composite of an increase in hemoglobin greater than 2g/dL from baseline or reaching a hemoglobin level greater than 12g/dL at treatment assessment endpoint and the absence of transfusions between weeks five to 26. The prespecified primary endpoint was met (13 [54.2%] patients).

Hemoglobin, bilirubin, and quality of life (QOL, measured by the functional assessment of chronic illness therapy fatigue (FACIT-F) scale) improvements correlated with rapid normalization of complement C4 and near-complete inhibition of CP activity. The researchers said the results showed that targeting the CP represents �a novel, effective therapeutic approach for the management of CAD and indicate that sutimlimab has the potential to change treatment practices for patients with this condition.�

The second Phase III sutimlimab trial, CADENZA, is investigating patients who do not have a recent history of blood transfusions.