Cold Agglutinin Disease Associated with Greater Than Twofold Increased Risk of Mortality in First Five Years After Diagnosis

Inc., a Sanofi company dedicated to transforming the lives of people
with rare blood disorders, today announced results from a new,
population-based retrospective study that found a greater than twofold
increased risk of mortality in people with cold agglutinin disease (CAD)
within the first five years after diagnosis. The study, which is the
first to compare overall survival among people with CAD with that of the
general population, also showed that the increased risk of mortality and
life-threatening thromboembolic events (TEs) such as stroke and heart
attack was evident starting in the first year after diagnosis. These
data were presented at the 23rd Annual Congress of the
European Hematology Association (EHA).

“The results of our survival analysis contribute to the growing body of
evidence that indicate that CAD is a more severe disease than previously
thought,” said Sigbjørn Berentsen, MD, PhD, Consultant Hematologist,
Department of Research and Innovation, Haugesund Hospital in Norway.
“The increased mortality from CAD appears to start at disease onset, and
the possible effect of earlier treatment in reducing mortality and
complications should be explored in future studies.”

CAD is a rare, chronic, and severe blood disease that results in the
constant and premature destruction of red blood cells (hemolysis) by the
body’s immune system. People with CAD suffer from chronic hemolytic
anemia, which is associated with debilitating fatigue, poor quality of
life, and life-threatening TEs. There are currently no approved
treatments for the disease.

In the survival study, researchers conducted a population-based analysis
to identify and characterize CAD patients. Using data from the Danish
National Patient Registry, 72 patients with a diagnosis of CAD were
compared to a cohort of 720 people without CAD matched for age, gender,
and region of residence. The study found that the risk of death was 2.27
times higher in the CAD cohort than in the general population in the
first five years from diagnosis. Further, the probability of survival
was only 61% among CAD patients five years after diagnosis versus 82%
for individuals in the general population cohort.

The study also identified an increased incidence of TEs among CAD
patients. This finding is consistent with the largest retrospective
study of CAD patients, which was recently presented at the American
Society of Hematology annual meeting and found a 55% overall increased
rate of TEs such as heart attack and stroke in CAD patients versus
matched controls (31% vs. 20%), as well as a statistically significant
higher frequency of multiple TEs.

“These data show that greater understanding is required about the risks
associated with cold agglutinin disease, and they reinforce the pressing
need for an approved and targeted treatment for this life-threatening
condition,” said Jaime Morales, MD, FAAP, Executive Director, Medical,
Bioverativ. “That is why we are conducting Phase 3 trials of sutimlimab,
a novel treatment that has been designed to directly and specifically
target CAD.”

Sutimlimab (formerly BIVV009) is an investigational monoclonal antibody
designed to inhibit C1s in the classical complement pathway, a part of
the immune system that is responsible for activating hemolysis in people
with CAD. Sutimlimab has been awarded Breakthrough Therapy Designation
by the U.S. Food and Drug Administration, and Phase 3 studies are
ongoing to determine the safety and efficacy of BIVV009 in primary CAD
patients. For more information about the sutimlimab Phase 3 studies,
visit clinicaltrials.gov
(study numbers: NCT03347396 and NCT03347422).

About Cold Agglutinin Disease (CAD)CAD is a chronic
autoimmune hemolytic anemia in which a part of the body’s immune system
called the classical complement pathway mistakenly destroys a person’s
own red blood cells (hemolysis). People with CAD suffer from chronic and
severe anemia, debilitating fatigue, and have an increased risk of death
and life-threatening events such as stroke and heart attack. There are
no approved therapies for CAD, which occurs in approximately 16 people
per million and affects an estimated 10,000 people in the United States
and Europe. Current treatment options are not specific to CAD, carry
significant toxicity risks, and often leave patients dependent upon
frequent blood transfusions, which can lead to chronic iron overload.

About Sutimlimab (formerly BIVV009)Sutimlimab (formerly BIVV009)
is a breakthrough C1s inhibitor for the treatment of CAD that is
currently being studied in Phase 3 clinical trials. A humanized,
monoclonal antibody, sutimlimab is designed to target C1s, a serine
protease within the C1-complex in the classical complement pathway of
the immune system, and directly impacts the central mechanism of
hemolysis in CAD. With a unique mechanism of action and high target
specificity, sutimlimab is designed to selectively inhibit disease
processes in the classical complement pathway while maintaining activity
of the alternative and lectin complement pathways, which are important
for immune surveillance and other functions.

About Bioverativ, a Sanofi companyBioverativ, a Sanofi
company, is dedicated to transforming the lives of people with
hemophilia and other rare blood disorders through world-class research,
development, and commercialization of innovative therapies. Bioverativ
is committed to actively working with the blood disorders community, and
its hemophilia therapies when launched represented the first major
advancements in hemophilia treatment in more than two decades. For more
information, visit www.bioverativ.com
or follow @bioverativ
on Twitter.

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