Cambridge, M.A., November 7, 2023. New data from across Sanofi’s oncology and rare blood disorders portfolios will be featured in 25 clinical research presentations, including seven oral presentations, at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition taking place December 9-12, 2023 in San Diego. These data demonstrate our commitment to scientific innovation and the potential of our investigational and approved therapies to address unmet needs across a spectrum of hematologic conditions with limited treatment options.
Brian Foard
Head, Specialty Care North America
“The spectrum of data Sanofi will present at ASH has the potential to reframe the possibilities for millions of patients living with hematologic conditions. New data for ALTUVIIIO, a first-in-class, high-sustained factor VIII replacement therapy, demonstrate our ambition to change treatment expectations for adults and children living with hemophilia A. Additionally, we continue to explore Sarclisa in combination with standard-of-care therapies in earlier lines of treatment for patients with newly diagnosed multiple myeloma.”
New data from the Phase 3 IsKia clinical trial, in partnership with the European Myeloma Network (EMN), evaluating the investigational combination of Sarclisa® (isatuximab-irfc) added to the standard-of-care regimen carfilzomib, lenalidomide and dexamethasone (KRd) in newly-diagnosed multiple myeloma (NDMM) are being presented. IsKia has the potential to provide insights in an area of unmet need in NDMM patients.
Presentations of the Sanofi oncology pipeline at ASH include updated efficacy and safety results from an open-label, first-in-human, dose-escalation study of an investigational CD123 targeting Natural Killer Cell Engager (NKCE). Results investigating SAR443579 as a monotherapy for the treatment of blood cancers with high unmet needs, including relapsed or refractory acute myeloid leukemia, B-cell acute lymphoblastic leukemia and high-risk myelodysplasia show data across all dose levels tested. Observed clinical remissions will also be presented.
Paul Rowe
Head of Medical Affairs, Specialty Care North America
“Our research highlights advances for our innovative treatments in hematology and our ambition in immunology. We are excited to share findings from our robust pipeline and investigational assets, including new data for our CD123 targeting natural killer cell engager in several hematological malignancies, and our BTK inhibitor in immune thrombocytopenia with a new mechanism of action for this condition. We are committed to pursuing scientific innovation and for difficult-to-treat hematologic diseases to transform care and improve outcomes.”
In hemophilia, our focus is to evolve treatment expectations for patients. Data from the Phase 3 XTEND-Kids trial evaluating factor activity levels in children younger than 12 years of age receiving once-weekly ALTUVIIIO® [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] will be presented. Additional qualitative insights from this trial based on the caregiver perspective will detail the potential impact of treatment with ALTUVIIIO for both children less than 12 years of age and their caregivers.
New results from Part B of the Phase 1/2 LUNA study for rilzabrutinib, a new and investigational Bruton’s tyrosine kinase (BTK) inhibition therapy for immune thrombocytopenia (ITP), will show encouraging data regarding the effect on platelet levels, safety profile, and quality of life measures in patients with relapsed ITP. ITP is a serious, acquired autoimmune blood disorder characterized by autoantibody-mediated platelet destruction and impaired platelet production, leading to thrombocytopenia and an increased risk of life-threatening bleeding episodes (like intracranial hemorrhage). Patients with ITP can experience quality-of-life impairments such as fatigue and cognitive impairment, and despite current treatment options, unmet needs remain for these patients. Rilzabrutinib is currently under clinical investigation and has not been evaluated by any regulatory authority.
An abstract evaluating the potential cost effectiveness of the U.S. Sanofi Promise Warranty Program for Cablivi® (caplacizumab-yhdp) will be shared. Cablivi is the first and only treatment for adults with acquired thrombotic thrombocytopenia purpura (aTTP) in combination with plasma exchange and immunosuppression.
A combined safety analysis from the two Phase 3 studies, CARDINAL and CADENZA, of Enjaymo® (sutimilab-jome) in adults with CAD will be presented. CAD is a rare autoimmune hemolytic anemia, with over 90% of patients suffering from uncontrolled complement-mediated hemolysis where part of the body’s immune system mistakenly destroys healthy red blood cells. Enjaymo is the first approved treatment for people with CAD in the U.S., EU, Switzerland, South Korea and Japan.
A full list of abstracts and presentations across the portfolios is included below.
Multiple Myeloma
Oncology Pipeline
Hemophilia A
Hemophilia A and B
Immune Thrombocytopenia
Acquired Thrombotic Thrombocytopenic Purpura
Cold Agglutinin Disease
About Sarclisa
Sarclisa is a monoclonal antibody that binds to a specific epitope on the CD38 receptor on multiple myeloma (MM) cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a potential target for antibody-based therapeutics such as Sarclisa.
Based on the Phase 3 ICARIA-MM study, Sarclisa is approved in 52 countries, including the U.S. and EU, in combination with pomalidomide and dexamethasone for the treatment of certain patients with relapsed refractory MM (RRMM) who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor. Based on the Phase 3 IKEMA study, Sarclisa is also approved in multiple countries in combination with carfilzomib and dexamethasone, including in the U.S. for the treatment of patients with RRMM who have received 1–3 prior lines of therapy and in the European Union for patients with MM who have received at least 1 prior therapy. In the U.S., the generic name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA).
Sarclisa continues to be evaluated in multiple ongoing Phase 3 clinical trials in combination with current standard treatments across the MM treatment continuum. It is also under investigation for the treatment of other hematologic malignancies, and its safety and efficacy have not been evaluated by any regulatory authority outside of its approved indication.
For more information on Sarclisa clinical trials, please visit www.clinicaltrials.gov.
IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS
What is SARCLISA?
SARCLISA is a prescription medicine used in combination with:
It is not known if SARCLISA is safe and effective in children.
Important Safety Information
Do not receive SARCLISA if you have a history of a severe allergic reaction to isatuximab-irfc or any of the ingredients in SARCLISA (see the list of ingredients in the full Prescribing Information).
Before receiving SARCLISA, tell your healthcare provider about all of your medical conditions, including if you:
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
How will I receive SARCLISA?
What are the possible side effects of SARCLISA?
SARCLISA may cause serious side effects, including:
Get medical help right away if you develop any of the following symptoms of infusion reaction during or after an infusion of SARCLISA:
— shortness of breath, wheezing, or trouble breathing
— swelling of the face, mouth, throat, or tongue
— throat tightness
— palpitations
— dizziness, lightheadedness, or fainting
— headache
— cough
— rash or itching
— nausea
— runny or stuffy nose
— chills
Your healthcare provider will check your blood cell counts during treatment with SARCLISA. Your healthcare provider may prescribe an antibiotic or antiviral medicine to help prevent infection, or a medicine to help increase your white blood cell counts during treatment with SARCLISA.
Tell your healthcare provider right away if you develop any fever or symptoms of infection during treatment with SARCLISA.
The most common side effects of SARCLISA in combination with pomalidomide and dexamethasone include:
The most common side effects of SARCLISA in combination with carfilzomib and dexamethasone include:
Heart failure can happen during treatment with SARCLISA in combination with carfilzomib and dexamethasone. Tell your healthcare provider right away if you develop any of the following symptoms:
These are not all the possible side effects of SARCLISA. For more information, ask your healthcare provider or pharmacist.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see full Prescribing Information, including Patient Information.
About ALTUVIIIO®
ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] is a first-in-class high-sustained factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for adults and children with hemophilia A. In adults and adolescents, ALTUVIIIO has a 3 to 4-fold longer half-life relative to standard and extended half-life factor VIII products. Once-weekly treatment with ALTUVIIIO provides high-sustained factor activity levels within the normal to near-normal range for most of the week in adults and for approximately three days in children. ALTUVIIIO is the first factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on other factor VIII therapies. ALTUVIIIO builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to extend its time in circulation.
Sobi and Sanofi collaborate on the development and commercialization of efanesoctocog alfa, or ALTUVIIIO in the US.
IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS
INDICATION
ALTUVIIIO® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl] is an injectable medicine that is used to control and reduce the number of bleeding episodes in people with hemophilia A (congenital Factor VIII deficiency).
Your healthcare provider may give you ALTUVIIIO when you have surgery.
IMPORTANT SAFETY INFORMATION
What is the most important information I need to know about ALTUVIIIO?
Do not attempt to give yourself an injection unless you have been taught how by your healthcare provider or hemophilia center. You must carefully follow your healthcare provider's instructions regarding the dose and schedule for injecting ALTUVIIIO so that your treatment will work best for you.
Who should not use ALTUVIIIO?
You should not use ALTUVIIIO if you have had an allergic reaction to it in the past.
What should I tell my healthcare provider before using ALTUVIIIO?
Tell your healthcare provider if you have had any medical problems, take any medications, including prescription and non-prescription medicines, supplements, or herbal medicines, are breastfeeding, or are pregnant or planning to become pregnant.
What are the possible side effects of ALTUVIIIO?
You can have an allergic reaction to ALTUVIIIO. Call your healthcare provider or emergency department right away if you have any of the following symptoms: difficulty breathing, chest tightness, swelling of the face, rash, or hives.
Your body can also make antibodies called “inhibitors” against ALTUVIIIO. This can stop ALTUVIIIO from working properly. Your healthcare provider may give you blood tests to check for inhibitors.
The common side effects of ALTUVIIIO are headache, joint pain, and back pain.
These are not the only possible side effects of ALTUVIIIO. Tell your healthcare provider about any side effect that bothers you or does not go away.
Please see full Prescribing Information.
About the Sanofi and Sobi collaboration
Sobi and Sanofi collaborate on the development and commercialization of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialization of efanesoctocog alfa, or ALTUVIIIO in the US. Sobi has final development and commercialization rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory.
About fitusiran
Fitusiran is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors. Fitusiran is designed to lower antithrombin, a protein that inhibits blood clotting, with the goal of promoting thrombin generation to rebalance hemostasis and prevent bleeds. Fitusiran utilizes Alnylam Pharmaceutical Inc.’s ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability.
Fitusiran is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
About rilzabrutinib
Rilzabrutinib is an oral Bruton’s tyrosine kinase (BTK) inhibitor incorporating Sanofi’s TAILORED COVALENCY® technology being investigated for the treatment of immune-mediated diseases, including immune thrombocytopenia (ITP). BTK is an intracellular signaling molecule involved in innate and adaptive immune responses related to certain immune-mediated diseases. By inhibiting BTK, rilzabrutinib has the potential to target the underlying disease pathogenesis.
Rilzabrutinib is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
About Enjaymo® (sutimlimab-jome)
Enjaymo is currently the only approved treatment for hemolysis in adults with CAD. It is a first-in-class humanized monoclonal antibody that is designed to selectively target and inhibit the classical complement pathway specific serine protease, C1s. By blocking C1s of the classical complement pathway, Enjaymo inhibits C1-activated hemolysis in CAD to prevent hemolysis, while leaving the lectin and alternative pathways intact.
IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS
INDICATION
ENJAYMO is a prescription medicine used to decrease the need for red blood cell transfusion due to the breakdown of red blood cells (hemolysis) in adults with cold agglutinin disease (CAD). It is not known if ENJAYMO is safe and effective in children.
IMPORTANT SAFETY INFORMATION
Do not receive ENJAYMO if you are allergic to sutimlimab-jome or any of the ingredients in ENJAYMO.
ENJAYMO can cause serious side effects, including:
The most common side effects of ENJAYMO include:
These are not all the possible side effects of ENJAYMO. Call your doctor for medical advice about side effects.
Before receiving ENJAYMO, tell your healthcare provider about all of your medical conditions, including if you:
Tell your healthcare provider about all the medicines you take, including prescription and over-the counter medicines, vitamins, and herbal supplements.
Please see accompanying full Prescribing Information.
About Cablivi® (caplacizumab-yhdp)
Cablivi is a von Willebrand Factor (vWF) antibody fragment, which inhibits the interaction between ultra-large vWF multimers and platelets and, therefore, stops the formation of the micro-clots that can form during an acute episode of acquired thrombotic thrombocytopenia purpura. Cablivi was approved in the European Union in August 2018 and in the United States in February 2019.
IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS
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, including medicines that increase your risk of bleeding such as anti-coagulants and anti-platelet agents.
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. In the post-marketing setting, cases of life-threatening and fatal bleeding were reported in patients receiving CABLIVI. Contact your doctor immediately if symptoms of excessive bruising, excessive bleeding, or major bleeding occur. Signs and symptoms of bleeding include: pain, swelling or discomfort, prolonged bleeding from cuts, increased menstrual flow or vaginal bleeding, nosebleeds, bleeding of gums from brushing, unusual bleeding or bruising, red or dark brown urine, red or tar black stools, headache, dizziness, or weakness.
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 and anti-platelet agents. 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 doctor 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.
Please see accompanying full Prescribing Information.
About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
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