Six oral and 14 poster presentations will be featured during the American Society of Hematology (ASH) Annual Meeting from December 11-14.
“These upcoming data presentations demonstrate our ongoing commitment to delivering meaningful innovations for patients with significant unmet needs in oncology and hematology,” said Dietmar Berger, Global Head of Clinical Development and Chief Medical Officer, Sanofi. “In our efforts to support the transformation of therapeutic landscapes, we look forward to presenting our Phase 3 results on fitusiran’s ability to provide protection from bleeds for people with hemophilia A or B with inhibitors. We’re also pleased to work with GMMG as they present investigational data evaluating Sarclisa in patients with newly diagnosed multiple myeloma, the first Phase 3 trial adding an anti-CD38 monoclonal antibody in combination with bortezomib, lenalidomide and dexamethasone.”
Understanding the potential for Sarclisa® (isatuximab-irfc) in earlier lines of therapy for multiple myeloma and other blood cancers
New data are from the Phase 3 German-Speaking Myeloma Multicenter Group (GMMG) HD7 study evaluating the effect of isatuximab in combination with bortezomib, lenalidomide and dexamethasone (VRd) on the rate of achieving minimal residual disease (MRD) negativity in transplant-eligible patients with newly diagnosed multiple myeloma (MM) after induction therapy, before transplant. These are the first results from a Phase 3 trial adding an anti-CD38 monoclonal antibody to VRd, a recommended first regimen in transplant-eligible newly diagnosed MM. GMMG-HD7 is also the first trial to evaluate MRD negativity at the end of induction as a co-primary endpoint, along with progression free survival, in transplant-eligible patients with newly diagnosed MM. The results of the study were selected as an oral presentation (abstract #463) and as part of the press program. Sanofi provided financial support to GMMG for this study.
Additionally, data from the Phase 2 ISAKIDS trial evaluating isatuximab in combination with standard salvage chemotherapies in children with relapsed or refractory leukemia in first or second relapse will be shared as an oral presentation (abstract #516).
The uses of Sarclisa in adult patients with newly diagnosed multiple myeloma and children with leukemia are currently under clinical investigation and their safety and efficacy in these settings have not been fully evaluated by any regulatory authority.
Sarclisa is approved in several geographies, in combination with pomalidomide and dexamethasone, for the treatment of certain adult patients with relapsed refractory MM who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor. This anti-CD38 monoclonal antibody medicine is also approved in several geographies in combination with carfilzomib and dexamethasone for the treatment of adult patients with relapsed or refractory MM who have received one to three prior lines of therapy.
Advancing our innovative approaches to find treatments for people with rare blood disorders
Hemophilia: The results of the ATLAS-003 Phase 3 study evaluating fitusiran prophylaxis in patients with hemophilia A or B with inhibitors dosed with the 80 mg monthly treatment will be presented in the plenary scientific session (abstract #150273).
Additionally, the Phase 1/2 study results for long-term health-related quality of life in patients with hemophilia A, with or without inhibitors, treated with fitusiran prophylaxis will be shared in a poster presentation (abstract #3197).
Fitusiran is a novel, subcutaneous small interfering (si)RNA therapy in development for people with hemophilia A or B, with or without inhibitors.
A post hoc analysis from the Phase 1/2 studies looking at the half-life and clearance of efanesoctocog alfa – previously known as BIVV001 – will be shared in a poster presentation (abstract #1035).
Efanesoctocog alfa is an investigational once-weekly factor therapy for people with hemophilia A that may provide high sustained factor activity and near-normal factor levels for the majority of the week. It represents a potential new class of factor VIII therapy. Efanesoctocog alfa is being developed in collaboration with Sobi.
Fitusiran and efanesoctocog alfa are currently under clinical investigation and their safety and efficacy have not been evaluated by any regulatory authority.
Cold Agglutinin Disease (CAD): An oral presentation (abstract #349) shares new data from the CADENZA study evaluating the safety and efficacy of sutimlimab to inhibit hemolysis in people with CAD. Additionally, two poster presentations (abstracts #4057 and #2002) provide an overview of the burden of living with CAD, including patient-reported outcome symptom measures. Also, a new analysis from the Phase 3 CARDINAL and CADENZA studies that evaluated sutimlimab in CAD will report on physical and mental component summary scores and their contributions to fatigue in people living with the disease.
Sutimlimab is a first-in-class investigational monoclonal antibody that inhibits C1s in the classical complement pathway. It has the potential to be the first approved treatment for hemolysis in adults with CAD, a serious and chronic autoimmune hemolytic anemia. Sutimlimab is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
Immune Thrombocytopenic Purpura (ITP): An oral presentation on rilzabrutinib, an investigational oral Bruton tyrosine kinase inhibitor (BTKi), reports on updated Phase 1/2 safety and efficacy results for the treatment of ITP (abstract #14) and a poster presentation (abstract #1010) on the design of the Phase 3 LUNA3 study. Rilzabrutinib is a potential first-in-class, oral BTKi in development for immune-mediated diseases. It is also being investigated for the autoimmune condition IgG4-related disease, asthma, atopic dermatitis, chronic spontaneous urticaria and warm autoimmune hemolytic anemia.
Rilzabrutinib is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
Acquired Thrombotic Thrombocytopenic Purpura (aTTP): Two poster presentations will share safety and efficacy results for Cablivi® (caplacizumab-yhdp) in aTTP: abstract #2080 will share long-term safety and efficacy from the post-HERCULES study in aTTP; additionally, abstract #1009 will show results from a Phase 2/3 study in Japanese patients with immune-mediated TTP.
Cablivi® (caplacizumab-yhdp) is approved in several geographies in combination with plasma exchange and immunosuppression for the treatment of aTTP in adults.
Sickle Cell Disease: Preliminary data from the ongoing Phase 1/2 PRECIZN-1 study evaluating the safety and efficacy of SAR445136, formerly known as BIVV003, for the treatment of patients with severe sickle cell disease will be shared in a poster presentation (abstract #2930). SAR445136, an investigational zinc finger nuclease ex vivo gene-editing cell therapy in development with Sangamo, has the potential to be a one-time treatment for sickle cell disease.
SAR445136 is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
Rare Blood Disorders Abstracts:
Cold Agglutinin Disease
Immune Thrombocytopenic Purpura
Acquired Thrombotic Thrombocytopenic Purpura
Sickle Cell Disease
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. Especially tell your healthcare provider if you have ever taken a medicine for your heart.
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
— dizziness, lightheadedness, or fainting
— rash or itching
— runny or stuffy nose
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:
These are not all the possible side effects of SARCLISA. For more information, ask your healthcare provider or pharmacist.
IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS
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 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.
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.
Please see full Prescribing Information.
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