New data in blood cancers, hemophilia, and other hematological disorders to be presented at ASH 2021
November 4, 2021
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 interference (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, looking at the safety and efficacy of sutimlimab to inhibit C1- activated 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 which evaluated sutimlimab in CAD, will report on physical and mental component summary scores and contribution to fatigue in people living with the disease.
Sutimlimab, a first-in-class investigational C1s inhibitor, 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-edited 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
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