New clinical and health-related quality of life data in multiple myeloma and rare blood disorders to be presented at ASH 2020
Oncology: Results from an interim analysis of Sarclisa® (isatuximab-irfc) in patients with relapsed multiple myeloma, including an evaluation of depth of response, will be shared in an oral presentation
Rare Blood Disorders: Presentations across three FDA-approved therapies and three investigational candidates show the breadth and depth of Sanofi’s commitment to people living with rare blood disorders

November 5, 2020

New clinical and health-related quality of life data in Sanofi’s oncology and rare blood disorders portfolios and pipelines, representing seven oral and 27 poster presentations, will be featured during the American Society of Hematology (ASH) Annual Meeting from December 5-8.

New data advances understanding of Sarclisa to treat multiple myeloma

Sarclisa is approved in several geographies to treat adult patients with relapsed and refractory multiple myeloma (MM) who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor. Data to be presented at ASH from the ICARIA trial reinforces its use in patients with this difficult-to-treat disease (abstracts #1411, 2289).

Additional data is emerging regarding the potential use of Sarclisa in combination with carfilzomib and dexamethasone after one to three prior therapies based on interim results from the IKEMA clinical study. In an oral presentation (abstract #414), results from an interim analysis will be presented, including an evaluation of the depth of response seen in patients with relapsed myeloma treated with Sarclisa plus carfilzomib and dexamethasone (compared to carfilzomib and dexamethasone alone). Poster presentations with results from an interim analysis of the IKEMA trial (abstract #2316), as well as a subgroup analysis in patients with renal impairment (abstract #3241) will also be presented. The use of Sarclisa in combination with carfilzomib and dexamethasone in relapsed MM is investigational; regulatory submissions were recently completed, but its safety and efficacy in this combination have not been fully evaluated by regulatory authorities.

Breaking barriers with ground-breaking science aiming to help people with rare blood disorders

Cold Agglutinin Disease (CAD): Two presentations (abstracts #2484, 1631) provide an overview of the experience of living with CAD, including patient-reported disease burden and medically attended anxiety or depression in newly diagnosed people with CAD. Currently, there are no approved therapies for CAD.

A new analysis (abstract #1674) from the Phase 3 pivotal study CARDINAL for sutimlimab, an investigational C1s inhibitor, in CAD evaluated complement-mediated inflammation contribution to fatigue in people living with CAD, and report new interim results from the CARDINAL Study Long-term Follow-up. Finally, additional oral presentations (abstracts #151, 426) describe the potential impact of sutimlimab on healthcare resource utilization.

Immune Thrombocytopenic Purpura (ITP): An oral presentation on sutimlimab, an investigational therapy, reports on long-term safety and efficacy data in people living with ITP (abstract #23).

Sutimlimab is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.

Hemophilia: Several presentations (including abstracts #2693, 877) will be shared on fitusiran, a novel siRNA therapy in development for hemophilia A and B with or without inhibitors, including interim results from a Phase 2 study (abstract #511).

An overview of the ongoing BIVV001 Phase 3 trial design (the XTEND-1 study) will be shared in a poster presentation (abstract #856). BIVV001 is an investigational once-weekly factor therapy for people with hemophilia A and represents a potential new class of factor VIII therapy that has the potential to provide high sustained factor activity levels. BIVV001 is being developed in collaboration with Sobi.

Fitusiran and BIVV001 are currently under clinical investigation and their safety and efficacy have not been evaluated by any regulatory authority.

Acquired Thrombotic Thrombocytopenic Purpura (aTTP): An oral presentation (abstract #428) on the serious burden of illness for people living with aTTP will be presented. Additionally, two posters (abstracts #843, 1754) share outcomes for patients with worsening aTTP despite receiving daily plasma exchange therapy in the Phase 3 HERCULES trial, and the impact of caplacizumab on platelet response, respectively.

Oncology Abstracts:

  • Depth of Response and Response Kinetics of Isatuximab plus Carfilzomib and Dexamethasone in Relapsed Multiple Myeloma: IKEMA Interim Analysis (Dr. Thomas Martin; Oral Presentation; Number 414)
  • Isatuximab Plus Carfilzomib and Dexamethasone Versus Carfilzomib and Dexamethasone in Relapsed Multiple Myeloma Patients with Renal Impairment: IKEMA Subgroup Analysis (Dr. Marcelo Capra; Poster Presentation; Number 3241)
  • Isatuximab Plus Carfilzomib and Dexamethasone vs Carfilzomib and Dexamethasone in Relapsed/Refractory Multiple Myeloma (IKEMA): Interim Analysis of a Phase 3, Randomized, Open-label Study (Dr. Philippe Moreau; Poster Presentation; Number 2316)
  • Isatuximab Plus Pomalidomide and Dexamethasone in Frail Patients with Relapsed/Refractory Multiple Myeloma: ICARIA-MM Subgroup Analysis (Dr. Fredrik Schjesvold; Poster Presentation; Number 1411)
  • Isatuximab Plus Pomalidomide and Dexamethasone in Patients With Relapsed/Refractory Multiple Myeloma and Soft-tissue Plasmacytomas: ICARIA-MM Subgroup Analysis (Dr. Meral Beksac; Poster Presentation; Number 2289)
  • Isatuximab Short-Duration Fixed-Volume Infusion Plus Bortezomib, Lenalidomide and Dexamethasone Combined Therapy for Newly Diagnosed Multiple Myeloma (NDMM): Results from a Phase 1b Feasibility/Safety Study (Dr. Enrique Ocio; Poster Presentation; Number 1413)
  • Updates from a Phase Ib Study of Isatuximab, Bortezomib and Dexamethasone Plus Cyclophosphamide or Lenalidomide in Transplant Ineligible Newly Diagnosed Multiple Myeloma (Dr. Enrique Ocio; Poster Presentation; Number 2288)
  • Isatuximab Short-duration Fixed-Volume Infusion Combination Therapy for Relapsed/Refractory Multiple Myeloma: Final Results of a Phase 1b Feasibility/Safety Study (Dr. Saad Usmani; Poster Presentation; Number 3207)
  • Development of a Hydrasys 2/4 -Isatuximab Assay to Mitigate Interference with Monoclonal Protein Detection on Immunofixation Electrophoresis IVD Tests in Multiple Myeloma (Greg Finn; Poster Presentation; Number 1562)
  • Health-Related Quality of Life in Heavily Pre-Treated and Renally Impaired Patients with Relapsed/Refractory Multiple Myeloma Receiving Isatuximab plus Pomalidomide and Dexamethasone: ICARIA-MM Study (Dr. Meletios Dimopoulos; Poster Presentation; Number 3438)
  • Daratumumab Dose Analysis Among Patients with Multiple Myeloma in a US Community Oncology Setting: A Retrospective Observational Study in Integra Connect Network (Dr. Robert Smith; Poster Presentation; Number 3222)
  • Model Based Approach to Evaluate Isatuximab Monthly Dosing Regimen in Relapsed/Refractory Multiple Myeloma Patients (Hoai-Thu Thai; Online Publication Only; Number 4165)
  • Treatment Patterns and Healthcare Resource Utilization in Patients with Multiple Myeloma and Baseline Renal Impairment (Dr. Parameswaran Hari, Online Publication Only)
  • Real-World Renal Function Among Patients with Multiple Myeloma in the United States (Dr. Joseph Mikhael; Online Publication Only)
  • Examination of Real-World Outcomes and Health Care Resource Utilization in the Prevention and Treatment of Tumor Lysis Syndrome (Wynter Balcerski; Online Publication Only)

 

Rare Blood Disorders Abstracts:

Hemophilia

  • Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors – Results from the Phase II Study (Dr. Steven Pipe; Oral Presentation; Number 511)
  • Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients with Haemophilia A (Dr. Christoph Königs; Oral Presentation; Number 509)
  • Final Results of PUPs B-LONG Study: Evaluating Safety and Efficacy of rFIXFc in Previously Untreated Patients with Haemophilia B (Dr. Beatrice Nolan; Poster Presentation; Number 856)
  • Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results (Prof. Claude Négrier; Poster Presentation; Number 2693)
  • Mechanistic Studies of Thrombin Generation Assay to Evaluate Procoagulant Potential of Fitusiran (Dr. Sravya Kattula; Poster Presentation; Number 857)
  • Reducing antithrombin in plasma to levels observed in fitusiran-treated patients does not interfere with coagulation assays (Arjan van der Flier; Poster Presentation; Number 862)
  • Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects With Hemophilia A and B with Inhibitors assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) (Dr. Sylvia von Mackensen; Poster Presentation; Number 877)
  • Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design (Dr. Barbara Konkle; Poster Presentation; Number 856)

 

Cold Agglutinin Disease

  • Long-term Safety and Efficacy of Sutimlimab in Patients with Chronic Immune Thrombocytopenia (Dr. Catherine Broome; Oral Presentation; Number 23)
  • Longitudinal Analysis of Anemia Severity, Treatment and Healthcare Resource Utilization among Patients with Primary Cold Agglutinin Disease in a Large US Database (Dr. Huy P Pham; Oral Presentation; Number 151)
  • Effect of Sutimlimab Treatment on Healthcare Resource Utilization in Patients with Cold Agglutinin Disease (Dr. Alexander Röth; Oral Presentation; Number 426)
  • Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD):  Interim Results of the Phase 3 Cardinal Study Long-term Follow-up (Dr. Alexander Röth; Poster Presentation; Number 1674)
  • Inflammation and Fatigue in Patients with Cold Agglutinin Disease (CAD): Analysis from the Phase 3 Cardinal Study (Dr. Ilene Weitz; Poster Presentation; Number 759)
  • Medically Attended Anxiety or Depression Is Increased Among Newly Diagnosed Patients With Cold Agglutinin Disease (CAD) (Parija Patel; Poster Presentation; Number 1631)
  • Patient-Reported Disease Burden: In-depth Interviews of Patients with CAD (Jun Su; Poster Presentation; Number 2484)
  • Sutimlimab, a Complement C1s Inhibitor, Improves Quality of Life in Patients With Cold Agglutinin Disease: Patient-Reported Outcomes Results of the Phase 3 Cardinal Study (Dr. Alexander Röth; Poster Presentation; Number 765)
  • Cold Agglutinin Disease (CAD) Real World Evidence (CADENCE) Registry: Design of the First International, Prospective CAD Registry (Dr. Alexander Röth; Poster Presentation; Number 2537)

 

Immune Thrombocytopenic Purpura

  • Treatment Patterns Among Patients with Immune Thrombocytopenia (ITP) in the United States: an Electronic Medical Record (EMR)–Based Analysis (Amanda Wilson; Poster Presentation; Number 2544)
  • Understanding and measuring key symptoms and health-related quality of life in patients with chronic ITP (Prof. Florence Joly; Poster Presentation; Number 3461)

 

Acquired Thrombotic Thrombocytopenic Purpura

  • Burden of Illness Among Medicare and Non-Medicare Populations With Acquired Thrombotic Thrombocytopenic Purpura, 2010-2018 (Dr. Huy P Pham; Oral Presentation; Number 428)
  • Outcomes of patients with worsening acquired thrombotic thrombocytopenic purpura despite daily therapeutic plasma exchange in the Phase 3 HERCULES trial (Dr. Marie Scully; Poster Presentation; Number 843)
  • Caplacizumab Induces Fast and Durable Platelet Count Responses With Improved Time to Complete Remission and Recurrence-Free Survival in Patients With Acquired Thrombotic Thrombocytopenic Purpura (Dr. Paul Coppo; Poster Presentation; Number 1754)

 

Rare Disease Abstract:

  • Hematologic Malignancies and Monoclonal Gammopathy of Undetermined Significance (MGUS) in Gaucher Disease Type 1 Patients in the International Cooperative Gaucher Group Gaucher Registry (Dr. Barry E. Rosenbloom; Poster Presentation; Number 3164)

 

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.

Inform your healthcare provider before scheduling any elective surgery, dental procedure or other invasive interventions.

What are the possible side effects of CABLIVI?

CABLIVI can cause bleeding. Bruising and bleeding, including bleeding from the nose and gums, may occur more easily and take longer than usual to stop.  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.

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. 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 Instructions for Use.

IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS

What is SARCLISA?

SARCLISA is a prescription medicine used in combination with pomalidomide and dexamethasone to treat adults who have received at least 2 prior therapies, including lenalidomide and a proteasome inhibitor, to treat multiple myeloma.

It is not known if SARCLISA is safe and effective in children.

Do not receive SARCLISA if you have a history of severe allergic reaction to isatuximab-irfc or any of the ingredients in SARCLISA (see the list of ingredients in full Prescribing Information).

Before receiving SARCLISA, tell your healthcare provider about all of your medical conditions, including if you:

  • are pregnant or plan to become pregnant. SARCLISA may harm your unborn baby. You should not receive SARCLISA during pregnancy.  
    • Females who are able to become pregnant should use an effective method of birth control during treatment and for 5 months after your last dose of SARCLISA. Talk to your healthcare provider about birth control methods that you can use during this time.

Tell your healthcare provider right away if you think you are pregnant or become pregnant during treatment with SARCLISA.

  • are breastfeeding or plan to breastfeed. It is not known if SARCLISA passes into your breast milk. You should not breastfeed during treatment with SARCLISA.

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?

  • SARCLISA will be given to you by your healthcare provider by intravenous (IV) infusion into your vein.
  • SARCLISA is given in treatment cycles of 28 days (4 weeks), together with the medicines pomalidomide and dexamethasone.
    • In cycle 1, SARCLISA is usually given weekly.
    • Starting in cycle 2, SARCLISA is usually given every 2 weeks.

                          Your healthcare provider will decide how long you should receive SARCLISA.

  • If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment.
  • Your healthcare provider will give you medicines before each dose of SARCLISA to help reduce the risk of infusion reactions (make them less frequent and severe).

What are the possible side effects of SARCLISA?

SARCLISA may cause serious side effects, including:

  • Infusion reactions. Infusion reactions are common with SARCLISA and can sometimes be severe.
    • Your healthcare provider will prescribe medicines before each infusion of SARCLISA to help decrease your risk for infusion reactions or to help make any infusion reaction less severe. You will be monitored for infusion reactions during each dose of SARCLISA.
    • Your healthcare provider may slow down or stop your infusion, or completely stop treatment with SARCLISA, if you have an infusion reaction.

Tell your healthcare provider right away if you develop any of the following symptoms of infusion reaction during or within 24 hours after an infusion of SARCLISA:

  • feeling short of breath
  • cough
  • chills
  • nausea

 

  • ​​Decreased white blood cell counts. Decreased white blood cell counts are common with SARCLISA and certain white blood cells can be severely decreased. You may have an increased risk of getting certain infections, such as upper and lower respiratory infections.

    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.
     
  • Risk of new cancers. New cancers have happened in people during treatment with SARCLISA. Your healthcare provider will monitor you for new cancers during treatment with SARCLISA.
     
  • Change in blood tests. SARCLISA can affect the results of blood tests to match your blood type. Your healthcare provider will do blood tests to match your blood type before you start treatment with SARCLISA. Tell all of your healthcare providers that you are being treated with SARCLISA before receiving blood transfusions.

The most common side effects of SARCLISA include:

 

  • lung infection (pneumonia)
  • decreased red blood cell counts (anemia)
  • upper respiratory tract infection
    decreased platelet counts (thrombocytopenia)

 

  • diarrhea

 

     

These are not all the possible side effects of SARCLISA. For more information, ask your healthcare provider or pharmacist.

Please see full Prescribing Information, including Patient Information.

About Sanofi

Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

Sanofi, Empowering Life

 

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