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 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 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
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1 Berentsen S, et al. Haematologica.
2006;91(4):460-466
Source: Sanofi (EURONEXT: SAN) (NASDAQ: SNY)
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