CAMBRIDGE, Mass., Nov. 6, 2019 /PRNewswire/ -- New clinical
data from Sanofi's oncology and rare blood disorders portfolios and
pipelines will be featured, including four oral presentations and
18 posters, at the 61st American Society of Hematology
(ASH) Annual Meeting & Exposition from December 7-10 in Orlando, FL.
"Blood cancers and rare
blood disorders account for some of the most challenging diseases
to treat, and patients often have limited therapeutic options,"
said John Reed, M.D., Ph.D., Global
Head of Research and Development at Sanofi. "Drawing upon our
deep expertise in hematology, and one of the industry's more robust
research and development programs actively working to address
numerous hematologic conditions, we are excited to present new data
at ASH that we believe demonstrate our commitment to advancing
science and improving the lives of patients we serve."
Advancing the understanding of multiple myeloma in
difficult-to-treat populations
Isatuximab: In the
area of multiple myeloma, analyses from the pivotal ICARIA-MM trial
for isatuximab, an investigational anti-CD38 monoclonal antibody,
will highlight depth of response and associated long-term outcomes
(abstract #3185), health-related quality of life (abstract #1850),
and outcomes in an elderly patient population (abstract #1893). The
ICARIA-MM clinical trial serves as the basis of a Biologic License
Application for isatuximab for the treatment of relapsed/refractory
multiple myeloma, which is currently under review by the U.S. Food
and Drug Administration with a target action date for a decision of
April 30, 2020. A Marketing
Authorization Application for isatuximab was also accepted for
review by the European Medicines Agency in the second quarter of
2019. Read more about our oncology data at ASH.
Striving to address unmet needs for people with rare blood
disorders
Cold Agglutinin Disease and Immune
Thrombocytopenic Purpura: New analyses of transfusion
practices in the U.S. (abstract #3559) and mortality risks
associated with cold agglutinin disease (CAD) (abstract #4790) will
underscore the seriousness of this rare and debilitating hemolytic
anemia. Sutimlimab, an investigational monoclonal antibody designed
to inhibit C1s, is being investigated as a potential first-in-class
treatment for CAD in two pivotal Phase 3 studies. An oral
presentation on sutimlimab will also explore its potential in
immune thrombocytopenic purpura (ITP) patients without adequate
response to two or more prior therapies (abstract #898). ITP
represents a second indication being investigated for
sutimlimab.
Hemophilia: Final data from a Phase 1 study
(abstract #625) of BIVV001 (rFVIIIFc-VWF-XTEN) evaluating the
safety and pharmacokinetics of repeated dosing will be shared in an
oral presentation. BIVV001 is the first and only investigational
von Willebrand (VWF)-independent factor VIII therapy that is
designed to provide high sustained factor activity and extend
protection from bleeds with once weekly dosing for people with
hemophilia A. A Phase 3 study of BIVV001 is expected to be
initiated by year-end. BIVV001 is being developed in collaboration
with Sobi.
Additional analysis of the ongoing Phase 2 open-label extension
study (abstract #1138) of fitusiran, a potential first-in-class,
once-monthly, fixed-dose subcutaneously administered RNA
interference therapeutic targeting antithrombin (AT) will also be
shared. Fitusiran is the first and only monthly investigational
therapy in Phase 3 development for the treatment of both hemophilia
A and B, with and without inhibitors.
Hemoglobinopathies: New pre-clinical and
clinical research on our pipeline of investigational, zinc finger
nuclease ex vivo gene-edited cell therapies for sickle cell
disease (BIVV003) and beta thalassemia (ST-400) will be shared in
multiple presentations. BIVV003 and ST-400 are being developed in
collaboration with Sangamo Therapeutics, Inc.
Acquired Thrombotic Thrombocytopenic
Purpura; Additional presentations include results
from studies on Cablivi® (caplacizumab-yhdp), our
first-in-class approved treatment, in combination with plasma
exchange and immunosuppressive therapy, for adult patients with
acquired thrombotic thrombocytopenic purpura (aTTP).
Oncology Poster Presentations:
Isatuximab
· Efficacy of
Isatuximab with Pomalidomide and Dexamethasone in Elderly Patients
with Relapsed/Refractory Multiple Myeloma: ICARIA-MM Subgroup
Analysis (Dr. Fredrik Schjesvold; Saturday, December 7, 2019:
Poster Presentation, 5:30-7:30 p.m. ET)
|
· Depth of
Response and Response Kinetics in the ICARIA-MM Study of
Isatuximab/Pomalidomide/Dexamethasone in Relapsed/Refractory
Multiple Myeloma (Dr. Cyrille Hulin; Sunday, December 8, 2019:
Poster Presentation, 6:00-8:00 p.m. ET)
|
·
Health-Related Quality of Life in Patients with Relapsed/Refractory
Multiple Myeloma Treated with Isatuximab plus Pomalidomide and
Dexamethasone: ICARIA-MM Study (Katherine Houghton; Saturday,
December 7, 2019: Poster Presentation, 5:30-7:30 p.m.
ET)
|
·
Exposure-response Analyses and Disease Modeling for Selection and
Confirmation of Optimal Dosing Regimen of Isatuximab in Combination
Treatment in Patients with Multiple Myeloma (Dr. Fatiha Rachedi;
Saturday, December 7, 2019: Poster Presentation, 5:30-7:30 p.m.
ET)
|
· The
Relationship Between Baseline Biomarkers and Efficacy of Isatuximab
in Combination with Pomalidomide and Dexamethasone in RRMM:
Insights from Phase 1 and Phase 3 studies (Dr. Paul Richardson;
Sunday, December 8, 2019: Poster Presentation, 6:00-8:00 p.m.
ET)
|
· Evaluating
Isatuximab Interference with Monoclonal Protein Detection By
Immuno-Capture and Liquid Chromatography Coupled to High Resolution
Mass Spectrometry in the Pivotal Phase 3 Multiple Myeloma Trial,
ICARIAMM (Dr. Greg Finn; Sunday, December 8, 2019: Poster
Presentation, 6:00-8:00 p.m. ET)
|
Rare Blood Disorders Oral and Poster Presentations:
Cold Agglutinin Disease and Immune Thrombocytopenic
Purpura
· Inhibition of
the Classical Pathway of Complement With Sutimlimab in Chronic
Immune Thrombocytopenic Purpura Patients Without Adequate Response
to Two or More Prior Therapies - #898 - Monday, December 9,
2019, 6:15 PM – 7:45 PM (ET) – Oral Presentation - Room
W307
|
· Cold
Agglutinin Disease Transfusion Practices in the United States: An
Electronic Medical Record–Based Analysis - #3559 - Monday,
December 9, 2019, 6:00 PM – 8:00 PM (ET)
|
· Mortality
Among Patients With Cold Agglutinin Disease in the United States:
An Electronic Medical Record (EMR)–Based Analysis - #4790 (abstract
only)
|
Hemophilia
· Phase 1
Repeat Dosing with BIVV001: The First Investigational Factor VIII
Product to Break through the Von Willebrand Factor–Imposed
Half-Life Ceiling - #625 –Monday, December 9, 2019 10:30 AM – 12:00
PM (ET) - Oral presentation - Room W415A
|
· Cryo-EM
Structure of BIVV001 Reveals Coagulation Factor VIII-Von Willebrand
Factor D'D3 Interaction Mode - #94- Saturday, December 7,
2019, 9:30 AM – 11:00 AM (ET) - Oral presentation – Room
W414AB
|
· Fitusiran, an
RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic
Balance in Patients with Hemophilia A or B with or without
Inhibitors: Management of Acute Bleeding Events – #1138 -
Saturday, December 7, 2019, 5:30 PM - 7:30 PM (ET)
|
· Patients' and
Caregivers' Preferences for Different Hemophilia A Treatment
Attributes - #2122 - Saturday, December 7, 2019, 5:30 PM -
7:30 PM (ET)
|
Acquired Thrombotic Thrombocytopenic Purpura
· Safety of
Caplacizumab in Patients Without Documented Severe ADAMTS13
Deficiency During the HERCULES Study - #1093 - Saturday,
December 7, 2019, 5:30 PM – 7:30 PM (ET)
|
· Efficacy of
Caplacizumab in Patients with aTTP in the HERCULES Study According
to Baseline Disease Severity #2366 - Sunday, December 8, 2019,
6:00 PM - 8:00 PM (ET)
|
· Efficacy of
Caplacizumab in Patients with aTTP in the HERCULES Study According
to Initial Immunosuppression Regimen - #2365 - Sunday,
December 8, 2019, 6:00 PM – 8:00 PM (ET)
|
· Narratives of
Patients with Fatal Outcomes During the Phase 2 TITAN and Phase 3
HERCULES Studies - #4908 (abstract only)
|
Sickle Cell Disease and Beta Thalassemia
· Genetic
Activation of NRF2 By KEAP1 Inhibition Induces Fetal Hemoglobin
Expression and Triggers Anti-Oxidant Stress Response in Erythroid
Cells - #210 - Saturday, December 7, 2019, 2:00 PM – 3:30 PM
(ET) – Oral Presentation – Room W414B
|
· Zinc Finger
Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer
Results in Enriched Biallelic Editing, increased Fetal Hemoglobin,
and Reduced Sickling in Erythroid Cells Derived from Sickle Cell
Disease Patients - #974 - Saturday, December 7, 2019, 5:30 PM
– 7:30 PM (ET) – Joint with Sangamo
|
· MetAP2
Inhibition Modifies Hemoglobin S (HbS) to Delay Polymerization and
Improve Blood Flow in Sickle Cell Disease - #2260 - Sunday,
December 8, 2019, 6:00 PM – 8:00 PM (ET)
|
· Differential
Efficacy of Anti-Sickling and Anti-Inflammatory Mechanisms in a
Fluorescent Intravital Microscopy Dorsal Skinfold Vaso-occlusion
Model in Sickle Cell Disease Townes Mice, #2264 - Sunday, December
8, 2019, 6:00 PM – 8:00 PM (ET)
|
·
Characterization of a genetically engineered HUDEP2 cell line
harboring a sickle cell disease mutation as a potential research
tool for preclinical Sickle Cell Disease Drug Discovery -
#3559 - Monday, December 9, 2019, 6:00 PM – 8:00 PM (ET)
|
· Preliminary
Results of a Phase 1/2 Clinical Study of Zinc Finger
Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic
Stem Cells for Transfusion-Dependent Beta Thalassemia – #3544
– Monday, December 9, 2019, 6:00 – 8:00 PM (ET) – Joint with
Sangamo
|
·
Identification of Novel Variants Associated with Fetal Hemoglobin
Levels in Healthy Donors (the INTERVAL study) - #2243 - Sunday,
December 8, 201, 6:00 PM - 8:00 PM (ET)
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Rare Disease Presentations:
Gaucher Disease
· Response to
Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from
4 Completed Clinical Trials - #4859 (abstract only)
|
About isatuximab
Isatuximab, an investigational
anti-CD38 monoclonal antibody, targets a specific epitope on the
CD38 receptor and is designed to trigger multiple, mechanisms of
action that are believed to directly promote programmed tumor cell
death (apoptosis) and immunomodulatory activity. CD38 is highly and
uniformly expressed on multiple myeloma cells and cell surface
receptors, making it a potential target for antibody-based
therapeutics such as isatuximab.
Isatuximab is an investigational agent and its safety and
efficacy have not been evaluated by the U.S. FDA, the European
Medicines Agency, or any other regulatory authority.
About Sutimlimab
Sutimlimab is a C1s inhibitor that
received breakthrough therapy designation and is currently being
investigated for the treatment of CAD 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, which directly impacts the
central mechanism of hemolysis in CAD. Similarly, the classical
complement pathway has been shown to contribute to the
physiopathology of immune thrombocytopenic purpura (ITP). With a
unique mechanism of action and high target specificity, sutimlimab
is designed to selectively inhibit disease processes by upstream
blockade of the classical complement pathway while maintaining
activity of the alternative and lectin complement pathways, which
are important for immune surveillance and other functions.
Sutimlimab has not been approved by the FDA, EMA or any other
regulatory authority for any indication and no conclusions can or
should be drawn regarding the safety or effectiveness of this
investigational therapeutic.
About BIVV001
BIVV001 (rFVIIIFc-VWF-XTEN) is a novel
and investigational recombinant factor VIII therapy that is
designed to provide high sustained factor activity and extend
protection from bleeds with prophylaxis dosing of once weekly for
people with hemophilia A. BIVV001 builds on the company's
innovative Fc fusion technology by adding a region of von
Willebrand factor and XTEN polypeptides to extend its time in
circulation. BIVV001 was granted orphan drug designation by the
Food and Drug Administration in August
2017 and the European Commission in June 2019. BIVV001 is being developed in
collaboration with Sobi.
BIVV001 has not been approved by the FDA, EMA or any other
regulatory authority for any indication and no conclusions can or
should be drawn regarding the safety or effectiveness of this
investigational therapeutic.
About Fitusiran
Fitusiran is potential first-in-class
investigational, once-monthly, subcutaneously administered RNA
interference therapeutic targeting antithrombin (AT) in development
for the treatment of hemophilia A and B, with and without
inhibitors. Fitusiran also has the potential to be used for rare
bleeding disorders. Fitusiran is designed to lower levels of AT
with the goal of promoting sufficient thrombin generation to
restore hemostasis and prevent bleeding. Fitusiran utilizes
Alnylam's ESC-GalNAc conjugate technology, which enables
subcutaneous dosing with increased potency and durability. The
clinical significance of this technology is under
investigation.
Fitusiran has not been approved by the FDA, EMA or any other
regulatory authority for any indication and no conclusions can or
should be drawn regarding the safety or effectiveness of this
investigational therapeutic.
About BIVV003
BIVV003 is an investigational ex
vivo gene-edited cell therapy for the treatment of people with
sickle cell disease being developed in collaboration with Sangamo
Therapeutics, Inc. BIVV003 is a non-viral cell therapy that
involves gene editing of a patient's own hematopoietic stem cells
(HSCs) using zinc finger nuclease (ZFN) technology to address
underlying disease pathophysiology. A Phase 1/2 clinical trial to
assess the safety, tolerability, and efficacy of BIVV003 in adults
with sickle cell disease has been initiated. Sanofi and Sangamo
collaborate on a similar second program, ST-400, an investigational
ex vivo gene-edited cell therapy, for the treatment of
adults with beta-thalassemia. The safety, efficacy and tolerability
ST-400 is currently being evaluated in a Phase 1/2 clinical
trial.
BIVV003 has not been approved by the FDA, EMA or any other
regulatory authority for any indication and no conclusions can or
should be drawn regarding the safety or effectiveness of this
investigational therapeutic.
About Cablivi
Cablivi should be administered upon
initiation of plasma exchange therapy, and in combination with
immunosuppressive therapy, based on a diagnosis of aTTP.
Cablivi is first administered as an 11 mg intravenous injection
prior to plasma exchange, followed by an 11 mg subcutaneous
injection after completion of plasma exchange on day 1. During the
daily plasma exchange period and 30 days following daily plasma
exchange, patients will take daily 11 mg subcutaneous injections.
If after the initial treatment symptoms of the underlying disease
are unresolved the treatment can be further extended for a maximum
of 28 days. Subcutaneous injection can by administered by a
patient/caregiver following proper training.
Cablivi was developed by Ablynx, which was acquired by Sanofi in
2018. Cablivi was approved in the European Union in
August 2018 and in the United States in February 2019. Cablivi is part of the company's
rare blood disorders franchise within Sanofi Genzyme, the specialty
care global business unit of Sanofi.
CABLIVI IMPORTANT SAFETY INFORMATION
What is CABLIVI?
CABLIVI (caplacizumab-yhdp) is a prescription medicine used for
the treatment of adults with acquired thrombotic thrombocytopenic
purpura (aTTP), in combination with plasma exchange and
immunosuppressive therapy.
Who should not take CABLIVI?
Do not take CABLIVI if you've had an allergic reaction to
caplacizumab-yhdp or to any of the ingredients in CABLIVI.
What should I tell my healthcare team before starting
CABLIVI?
Tell your doctor if you have a medical condition including if
you have a bleeding disorder. Tell your doctor about any
medicines you take.
Talk to your doctor before scheduling any surgery, medical or
dental procedure.
What are the possible side effects of CABLIVI?
CABLIVI can cause severe bleeding. In clinical studies, severe
bleeding adverse reactions of nosebleed, bleeding from the gums,
bleeding in the stomach or intestines, and bleeding from the uterus
were each reported in 1% of subjects. Contact your doctor
immediately if excessive bleeding or bruising occur.
You may have a higher risk of bleeding if you have a bleeding
disorder (i.e Hemophilia) or if you take other medicines that
increase your risk of bleeding such as anti-coagulants.
CABLIVI should be stopped for 7 days before surgery or any
medical or dental procedure. Talk to your doctor before you stop
taking CABLIVI.
The most common side effects include nosebleed,
headache and bleeding gums.
Tell your healthcare provider if you have any side effect that
bothers you or that does not go away. These are not all the
possible side effects of CABLIVI. Call your doctor for medical
advice about side effects.
Click here for full prescribing information.
Please visit www.cablivi.com.
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
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Investor Relations
Contact
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Ashleigh
Koss
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George
Grofik
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Tel.: +1 (908)
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Tel.: +33 (0)1 53 77
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Ashleigh.Koss@sanofi.com
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ir@sanofi.com
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