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Sanofi, a leader in immune-mediated rare blood disorders, to present latest data at EHA 2022

June 10, 2022. The latest clinical research across Sanofi’s portfolio of investigational and approved therapies for immune-mediated rare blood disorders will be presented at the 27th Annual European Hematology Association (EHA) Congress. Sanofi’s goal is to fundamentally redefine the management of these rare and often chronic blood disorders and the company has launched innovative treatments for patients with limited options in cold agglutinin disease (CAD) and acquired thrombotic thrombocytopenic purpura (aTTP).

At EHA, five presentations on CAD will be shared, including two-year follow-up data from the Phase 3 pivotal CARDINAL study that demonstrated the safety and efficacy of Enjaymo™ (sutimlimab-jome). Additional presentations include long-term data from an extension trial of rilzabrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor for immune thrombocytopenia (ITP), and three-year safety and efficacy findings for use of Cablivi® (caplacizumab-yhdp) in aTTP.

Karin Knobe, MD, PhD
Global Head of Development, Rare Diseases and Rare Blood Disorders at Sanofi
Sanofi’s commitment to finding solutions where there is significant unmet need is unwavering. Following the FDA’s recent approval of Enjaymo (sutimlimab) as the first-and-only approved treatment indicated to decrease the need for red blood cell transfusion due to hemolysis in adults with CAD, we are excited to share our latest research in the area in addition to updates regarding our work in ITP and aTTP. The data we’re presenting at EHA 22 represent a robust portfolio of potential first-in-class treatments that are providing hope for people living with immune-mediated rare blood disorders.

Cold Agglutinin Disease (CAD) Data Presentations

Enjaymo™ is a first-in-class humanized monoclonal antibody that selectively inhibits the C1 complex of the classical complement pathway, preventing its CAD-associated activation and targeted destruction of healthy red blood cells while leaving the alternative and lectin complement pathways intact. Oral presentations will report two-year follow-up results from the pivotal Phase 3 CARDINAL study, an open-label, single-arm study with a 26-week primary treatment period (Part A), and a 2-year extension (Part B).

Follow-up data support the CARDINAL Part A findings, showing that Enjaymo™ maintained mean hemoglobin >11g/dL and achieved sustained normalization of mean bilirubin in patients after two years, demonstrating the efficacy and safety profile for the therapy in CAD (abstract #S285). Additionally, Enjaymo™ was shown to produce rapid and sustained improvements in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scores and have meaningful impact on patient quality of life based on other patient reported outcomes after two years of treatment (abstract #304). No new safety concerns were identified throughout two years of treatment, and sutimlimab was shown to improve and stabilize critical bloodstream components that are typically altered in patients with CAD.

Immune Thrombocytopenia (ITP) Data Presentation

An oral presentation (abstract #S291) on rilzabrutinib, a potential first-in-class investigational oral Bruton’s tyrosine kinase (BTK) inhibitor in development for immune-mediated diseases, including ITP, will report long-term extension (LTE) findings for a global phase 1/2 trial (NCT03395210) for the treatment of ITP, a rare blood disorder in which platelets are targeted for destruction by the immune system, leading to high bleeding risk and poor quality of life.

Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Data Presentation

Long-term safety and efficacy findings from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study for patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial (NCT02553317), will also be presented in an oral presentation at EHA 2022 (abstract #S294).

aTTP is a rare, life-threatening autoimmune blood disorder causing excessive blood clot formation and impaired oxygen transport to vital organs.

All Rare Blood Disorders Abstracts:

Cold Agglutinin Disease (CAD)

Inhibition of Complement C1s With Sutimlimab in Patients With Cold Agglutinin Disease (CAD): 2-Year Follow-Up From The CARDINAL Study. Oral Presentation.Oral Presentation
Abstract #S285
Friday, June 10
11:30 – 12:45 CEST
Inhibition of Complement C1s With Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results Following 9-Week Washout Period In Phase 3 CARDINAL Study (NCT03347396). Poster Presentation.Poster Presentation
Abstract # P1538
Friday, June 10
16:30 – 17:45 CEST
Sutimlimab After Prior Rituximab Use in Patients with Cold Agglutinin Disease (CAD): Pooled Post-hoc Analyses from the CARDINAL and CADENZA Trials. Poster Presentation.Poster Presentation
Abstract # P1532
Friday, June 10
16:30 – 17:45 CEST
Seasonality of Healthcare Resource Utilization Among Cold Agglutinin Disease Among Patients in Denmark: A Retrospective Analysis. Poster Presentation.

 
Poster Presentation
Abstract # P1539
Friday, June 10
16:30 – 17:45 CEST
Sutimlimab, A Complement C1s Inhibitor, Provides Sustained Improvements in Patient-Reported Outcomes in Patients With Cold Agglutinin Disease (CAD): 2 Year Follow-Up From The CARDINAL Study. Oral Presentation.Oral Presentation
Abstract #S304
Saturday, June 11
11:30 – 12:45 CEST

Immune Thrombocytopenia (ITP)

Phase I/II Study of Rilzabrutinib, An Oral Bruton Tyrosine Kinase Inhibitor, in Patients with Immune Thrombocytopenia: Long-Term Follow-Up. Oral Presentation.Oral Presentation
Abstract #S291
Saturday, June 11
11:30 – 12:45 CEST

Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Long-term Safety and Efficacy of Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura (aTTP): The Post-HERCULES Study. Oral Presentation.Oral Presentation
Abstract #S294
Saturday, June 11
11:30 – 12:45 CEST

About Enjaymo™ (sutimlimab)

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 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 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 and Sanofi expects an opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) in Europe by the end of 2022.

About rilzabrutinib

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 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.

About Cablivi® (caplacizumab)

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.

About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

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