Paris, November 30, 2022. Data from across Sanofi’s rare blood disorders franchise will be featured in 16 presentations at the 64th American Society of Hematology (ASH) Annual Meeting & Exposition from December 10-13, 2022.
Karin Knobe, MD, PhD
Global Head of Clinical Development for Rare Diseases and Rare Blood Disorders at Sanofi
“The Phase 3 results presented at ASH across hemophilia and cold agglutinin disease underscore our ambition to launch four rare blood disorder therapies in four years. We started this year strong and look forward to carrying this momentum into 2023 and beyond, with potential treatment advancements we hope will help transform the lives of people living with rare blood disorders.”
Quality of life data from the pivotal XTEND-1 Phase 3 study will be presented at ASH evaluating the effect of investigational therapy efanesoctocog alfa on pain and physical functioning for people with hemophilia A. These findings build on key results that were first presented at the 30th International Society of Thrombosis and Haemostasis (ISTH) Congress. Data from the XTEND-1 study supported the biologics licence application (BLA) for efanesoctocog alfa, which is currently under FDA priority review with a target action date of February 28, 2023.
New data to be presented from the ATLAS program for fitusiran, an investigational therapy for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors, will showcase for the first time the hemostatic equivalency of antithrombin lowering in hemophilia A. Hemostatic equivalency is used to better understand how non-factor therapies, such as fitusiran, can help correct the clotting defect in hemophilia. Fitusiran is designed to work by targeting antithrombin, a protein that helps maintain hemostatic balance. Additional fitusiran data will exhibit improvements in health-related quality of life with reduced treatment burden. Qualitative data will outline treatment expectations for people with hemophilia to live an active lifestyle.
In an oral presentation, results from the CADENZA phase 3 study, which supported a supplemental BLA currently under FDA priority review for Enjaymo® (sutimlimab-jome), will review the effect of Enjaymo on patient-reported outcomes and quality of life. Presentations will also report long-term clinical data from the CADENZA study as well as information on the COVID-19 vaccine response among people living with cold agglutinin disease (CAD) being treated with Enjaymo. Data from the pivotal CADENZA and CARDINAL Phase 3 studies recently supported the European Commission approval of Enjaymo.
Additional presentations at ASH will highlight other rare blood disorders. Two posters will explore the prevalence and burden of immune thrombocytopenia (ITP), including cognitive impairment. Data evaluating the response to rilzabrutinib, an investigational therapy, when used as an early treatment option for ITP patients will also be shown. Presentations on acquired thrombotic thrombocytopenic purpura (aTTP) include an analysis of the HERCULES Phase 3 study and a presentation of the MAYARI study design that will assess the efficacy and safety profile of Cablivi® (caplacizumab-yhdp) without plasma exchange in an investigational setting. Cablivi is approved in several geographies in combination with plasma exchange and immunosuppression for the treatment of aTTP in adults.
Hemophilia A and B
Cold Agglutinin Disease
Acquired Thrombotic Thrombocytopenic Purpura
About efanesoctocog alfa
Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A. It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the first investigational factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. Altuviiio™ is the intended trade name of efanesoctocog alfa in the US. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority. Efanesoctocog alfa is currently under FDA review with a target action date of February 28, 2023. The FDA also granted efanesoctocog alfa Breakthrough Therapy designation in May 2022, – the first factor VIII therapy to receive this recognition – Fast Track designation in February 2021, and Orphan Drug designation in August 2017. Sanofi and Sobi® collaborate on the development of efanesoctocog alfa.
Fitusiran is an investigational, subcutaneously administered small interference RNA (siRNA) 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 has not been evaluated by any regulatory authority.
About Enjaymo® (sutimlimab-jome)
Enjaymo is a humanized monoclonal antibody that is designed to selectively target and inhibit C1s in the classical complement pathway, which is part of the innate immune system. By blocking C1s, Enjaymo inhibits the activation of the complement cascade in the immune system and inhibits C1-activated hemolysis in cold agglutinin disease (CAD) to prevent the abnormal destruction of healthy red blood cells. Enjaymo does not inhibit the lectin and alternative pathways.
Enjaymo was approved by the U.S. Food and Drug Administration (FDA) in February 2022 as the first and only treatment indicated to decrease the need for red blood cell transfusion due to hemolysis in adults with CAD. Sanofi has filed for an sBLA to add CADENZA (BIVV009-04) clinical data into the label and was granted priority review by the FDA in September 2022.
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. Use of Cablivi without plasma exchange is currently investigational and has not been evaluated by any regulatory authority.
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.
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