- Presentations include findings on hemophilia and acquired thrombotic thrombocytopenic purpura portfolio therapies and pipeline candidates
Cambridge, MA – June 29, 2020 -- Clinical data from marketed and investigational therapies for hemophilia and acquired thrombotic thrombocytopenic purpura (aTTP) will be presented at the XXVII Congress of the International Society on Thrombosis and Haemostatis (ISTH) virtually July 12-14, 2020.
“Every day we are working to improve the standard of care for people living with rare blood disorders. Our aim isn’t incremental improvements, but breakthrough science that can deliver a better tomorrow for patients,” said Bill Sibold, Executive Vice President and Head of Sanofi Genzyme. “At ISTH 2020, we look forward to sharing updates from our hemophilia pipeline that have the potential to redefine what patients and their physicians can expect from hemophilia treatment in the future, as well as data on portfolio products suggesting their potential to address treatment gaps and improve patient outcomes.”
Breaking barriers in hemophilia treatment
Hemophilia is a rare, genetic bleeding disorder in which a person’s blood does not clot properly. This can result in people with hemophilia A or B experiencing internal bleeding episodes caused by trauma or spontaneous internal bleeding that can cause pain, irreversible joint damage and life-threatening hemorrhages. As the treatment landscape continues to evolve, Sanofi’s goal is to advance novel therapies with the potential to challenge the limitations of current therapies so that patients may no longer need to make tradeoffs between protection, treatment burden and living the active life they choose.
Factor replacement therapy remains a cornerstone of hemophilia management. Data shared in five presentations add to a growing body of clinical evidence for Eloctate® Antihemophilic Factor (Recombinant), Fc Fusion Protein and Alprolix® Coagulation Factor IX (Recombinant), Fc Fusion Protein, leading extended half-life factor therapies for hemophilia A and B, respectively. The company with Sobi will present final results from the PUPs A-LONG and PUPs B-LONG studies, which evaluate the efficacy and safety of Eloctate and Alprolix in previously untreated patients (PUPs) with hemophilia A and B, respectively.
- Oral Communication Session on Sunday, July 12, 2020 from 10:15-11:30 a.m. ET: Final results of the PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients with Haemophilia A. (Joint with Sobi)
- Abstract PB0956: Final Results of PUPs B-LONG Study: Evaluating Safety and Efficacy of rFIXFc in Previously Untreated Patients with Haemophilia B. Poster Presentation (Joint with Sobi)
- Abstract PB1774: The Fc and C1/C2 domains of recombinant factor VIII Fc fusion protein engage monocytes in an FcgammaRII-dependent manner to reduce their differentiation potential into osteoclasts. Poster Presentation
- Abstract PB0879: Real-world effectiveness evaluation of the recombinant factor VIII Fc fusion protein in adolescents and children with hemophilia A in Japan: “Fc Adolescent and Children Treatment” study (FACTs) Part 1 interim analysis. Poster Presentation
- Abstract PB0948: Real-world data of first-time and rescue immune tolerance induction with recombinant factor VIII Fc fusion protein in patients with hemophilia A with inhibitors in Japan: “Fc Adolescent and Children Treatment” study (FACTs) Part 2 interim analysis. Poster Presentation
With its pipeline of investigational hemophilia therapies, Sanofi seeks to break barriers by pursuing treatments that may provide improved bleed protection for longer periods of time, so patients with hemophilia A and B can lead a more active life.
Pharmacokinetic data will be presented on BIVV001, the first investigational von Willebrand (VWF)-independent factor VIII therapy designed to provide high sustained factor levels in a once-weekly dose for patients with hemophilia A. BIVV001 is being developed in collaboration with Sobi and its unique molecular design offers the potential of near-normal factor levels for the majority of the week.
- Abstract PB0949: Population pharmacokinetic (PK) analysis of BIVV001 (rFVIIIFc-VWF-XTEN), a new class of factor VIII (FVIII) replacement. Poster Presentation (Joint with Sobi)
An analysis of perioperative management of patients with hemophilia receiving fitusiran will also be presented. Fitusiran is a novel investigational RNA interference (RNAi) therapy that has the potential to transform the management of hemophilia as the first true once monthly, subcutaneous treatment in a single dose and aims to provide consistent bleed protection for people with hemophilia A and B, with or without inhibitors.
- Abstract PB1142: Perioperative Management of Patients With Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia. Poster Presentation
ISTH provides recommendations for rare autoimmune blood clotting disorder
Acquired TTP (aTTP) is a rare, life-threatening, autoimmune blood clotting disorder and is considered a medical emergency. Outcome data will also be presented from the Phase 3 study of Cablivi® (caplacizumab-yhdp), a von Willebrand Factor-directed antibody fragment treatment indicated for use in combination with plasma exchange and immunosuppressive therapy for adult patients with aTTP.
- Abstract PB1361: Outcomes of patients with worsening acquired thrombotic thrombocytopenic purpura (aTTP) despite daily therapeutic plasma exchange (TPE) in the Phase 3 HERCULES trial. Poster Presentation
Alprolix® Coagulation Factor IX (Recombinant), Fc Fusion Protein is a recombinant clotting factor therapy developed for hemophilia B using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Alprolix to use a naturally occurring pathway to extend the time the therapy remains in the body.
Alprolix is marketed by Sanofi for the treatment of hemophilia B in the United States, Japan and Canada. It is also approved in Australia, New Zealand, Brazil and other countries, and Sanofi has marketing rights in these regions. It is also authorized in the European Union, Iceland, Liechtenstein, Norway and other countries, where it is marketed by Sobi.
Eloctate® Antihemophilic Factor (Recombinant), Fc Fusion Protein is a recombinant clotting factor therapy developed for hemophilia A using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Eloctate to use a naturally occurring pathway to extend the time the therapy remains in the body.
Eloctate is marketed by Sanofi in the United States, Japan and Canada. It is also approved in Australia, New Zealand, Brazil and other countries, and Sanofi has marketing rights in these regions. It is also approved as Elocta® in the European Union, Switzerland, Iceland, Liechtenstein, Norway and other countries where it is marketed by Sobi.
BIVV001 (rFVIIIFc-VWF-XTEN) 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. BIVV001 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 only therapy that has been shown to break through the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. BIVV001 is being developed and commercialized in collaboration with Sobi. BIVV001 was granted orphan drug designation by the US Food and Drug Administration in August 2017 and the European Commission in June 2019. BIVV001 is currently under clinical investigation and has not been evaluated by any regulatory authority.
Fitusiran is an investigational, once-monthly, subcutaneously administered RNA interference therapeutic in development for the treatment of people with hemophilia A and B, with or without inhibitors. Fitusiran is designed to target antithrombin, a protein that inhibits blood clotting, with the goal of promoting sufficient thrombin generation to naturally rebalance hemostasis and prevent bleeds. Fitusiran utilizes Alnylam'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.
Cablivi is a vWF antibody fragment, which inhibits the interaction between ultra-large von Willebrand Factor (vWF) multimers and platelets and, therefore, stops the formation of the micro-clots that can form during an acute episode of aTTP.
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.
ALPROLIX INDICATIONS AND IMPORTANT SAFETY INFORMATION
ALPROLIX® Coagulation Factor IX (Recombinant), Fc Fusion Protein is an injectable medicine that is used to help control and prevent bleeding in people with hemophilia B. Hemophilia B is also called congenital Factor IX deficiency. Your healthcare provider may give you ALPROLIX when you have surgery.
IMPORTANT SAFETY INFORMATION
Do not use ALPROLIX if you are allergic to ALPROLIX or any of the other ingredients in ALPROLIX.
Tell your healthcare provider if you have or have had any medical problems, take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have any allergies and all your medical conditions, including if you are pregnant or planning to become pregnant, are breastfeeding, or have been told you have inhibitors (antibodies) to Factor IX.
Common side effects of ALPROLIX include headache, abnormal sensation in the mouth, and pain in your side with blood in your urine, which may be a sign of clot formation in the urinary collecting system. These are not all of the possible side effects of ALPROLIX. Talk to your healthcare provider right away about any side effect that bothers you or does not go away, and if bleeding is not controlled using ALPROLIX.
Allergic reactions may occur with ALPROLIX. Call your healthcare provider or get emergency treatment right away if you have any of the following symptoms: hives, chest tightness, wheezing, difficulty breathing, or swelling of the face.
Your body can also make antibodies called "inhibitors" against ALPROLIX, which may stop ALPROLIX from working properly.
ALPROLIX may increase the risk of formation of abnormal blood clots in your body, especially if you have risk factors for developing blood clots. Call your healthcare provider or seek emergency care if you have symptoms of a possible abnormal blood clot, which may include: chest pain, difficulty breathing, unexpected swelling of an arm or leg with or without pain or tenderness.
ELOCTATE INDICATIONS AND IMPORTANT SAFETY INFORMATION
ELOCTATE® Antihemophilic Factor (Recombinant), Fc Fusion Protein is an injectable medicine that is used to help control and prevent bleeding in people with Hemophilia A (congenital Factor VIII deficiency). Your healthcare provider may give you ELOCTATE when you have surgery.
IMPORTANT SAFETY INFORMATION
Do not use ELOCTATE if you have had an allergic reaction to it in the past.
Tell your healthcare provider if you have or have had any medical problems, take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have any allergies, are breastfeeding, are pregnant or planning to become pregnant, or have been told you have inhibitors (antibodies) to Factor VIII.
Allergic reactions may occur with ELOCTATE. Call your healthcare provider or get emergency treatment 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 ELOCTATE, which may stop ELOCTATE from working properly.
The most frequently occurring side effects of ELOCTATE are headache, rash, joint pain, muscle pain and general discomfort. These are not all the possible side effects of ELOCTATE. Talk to your healthcare provider right away about any side effect that bothers you or that does not go away, and if bleeding is not controlled after using ELOCTATE.
CABLIVI INDICATION AND 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.
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