Press Releases

Pivotal data from Phase 3 study of sutimlimab in cold agglutinin disease to be presented at ASH 2019 late-breaking session
- Sutimlimab, an investigational targeted C1s inhibitor, met its primary efficacy endpoint in Phase 3 trial for patients with cold agglutinin disease (CAD)
- CAD is a severe and chronic rare blood disorder in which the immune system mistakenly attacks a person's own healthy red blood cells. There are currently no approved treatments.

CAMBRIDGE, Mass., Nov. 21, 2019 /PRNewswire/ -- 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, 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 million1, including an estimated 12,000 people in the United States, Europe and Japan.

About Sanofi

Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

Sanofi, Empowering Life

Media Relations Contact

Investor Relations Contact

Ashleigh Koss

George Grofik

Tel.: +1 (908) 981-8745

Tel.: +33 (0)1 53 77 45 45 

Sanofi Forward-Looking Statements 
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi's ability to benefit from external growth opportunities and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the  ultimate outcome of such litigation,  trends in exchange rates and prevailing interest rates, volatile economic conditions, the impact of cost containment initiatives and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2018. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements

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

Source: Sanofi (EURONEXT: SAN) (NASDAQ: SNY)