On May 12, 2021 Sanofi reported that New pivotal data and clinical analyses from portfolio of investigational therapies for immune-mediated rare blood disorders will be featured at the 26th Annual European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress, June 9-17, 2021 (Press release, Sanofi, MAY 12, 2021, View Source [SID1234579803]).

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"The data being presented at EHA (Free EHA Whitepaper) 2021 demonstrate Sanofi’s commitment to providing first-in-class and potentially transformative treatments for people with immune-mediated rare blood disorders, where significant unmet needs persist," said Karin Knobe, Head of Development, Rare and Rare Blood Disorders, Sanofi. "We are excited to share pivotal data from the placebo-controlled CADENZA Phase 3 study of sutimlimab in people with CAD with no recent history of transfusion as well as interim data from our rilzabrutinib Phase 1/2 study in patients with ITP."

Working to break barriers in the treatment of cold agglutinin disease

Cold agglutinin disease (CAD) is a rare, chronic autoimmune hemolytic anemia that causes the body’s immune system to mistakenly attack healthy red blood cells and cause their destruction (hemolysis) via activation of the classical complement pathway. CAD patients may experience chronic anemia, profound fatigue, acute hemolytic crisis, and other potential complications, including an increased risk of thromboembolic events and early death.1,2,3 CAD impacts the lives of an estimated 12,000 people in the U.S., Europe, and Japan.4 Currently there are no approved therapies for CAD.

Sutimlimab is an investigational, potential first-in-class monoclonal antibody designed to selectively target C1-activated hemolysis in CAD.

New data from pivotal Phase 3 CADENZA and CARDINAL studies

Abstract #S291: C1s-Targeted Inhibition of Classical Complement Pathway by Sutimlimab in Cold Agglutinin Disease (CAD): Efficacy and Safety Results from the Randomized, Placebo (PBO)-Controlled Phase 3 CADENZA Study. Oral Presentation.
Abstract #S290: Sutimlimab, a Targeted Complement C1s Inhibitor, Improves Quality of Life (QOL) in Patients with Cold Agglutinin Disease (CAD): Results from the Randomized, Placebo-Controlled Phase 3 CADENZA Study. Oral Presentation.
Abstract #S312: Sustained Improvements in Patient-Reported Outcomes with Sutimlimab in Patients with Cold Agglutinin Disease: 1-Year Follow-Up Interim Results from the CARDINAL Study. Oral Presentation.
Abstract #EP1179: Clinically Important Change in FACIT-Fatigue Score for Patients with Cold Agglutinin Disease: An Analysis Using the Phase 3 CARDINAL and CADENZA Studies. Poster Presentation.
Abstract #EP689: Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): 1-Year Interim Results of the Phase 3 CARDINAL Study Long-term Follow-up (adaptation). Poster Presentation.

Data on the potential impact of sutimlimab on QOL and fatigue in people with CAD

Abstract #EP709: Inflammation and Fatigue in Patients with Cold Agglutinin Disease (CAD): Analysis from the Phase 3 CARDINAL Study (adaptation). Poster Presentation.

New analyses on disease burden and healthcare resource utilization by people living with CAD

Abstract #EP1180: Increased Antidepressant Use Among Newly Diagnosed Patients with Cold Agglutinin Disease Compared with Other Patients in a Large US Healthcare System. Poster Presentation.
Abstract #EP1193: Healthcare Resource Utilization Among Patients with Cold Agglutinin Disease in Denmark (adaption). Poster Presentation.
Aiming to address unmet needs in immune thrombocytopenic purpura

Immune thrombocytopenic purpura (ITP) is an acquired autoimmune blood disorder characterized by immune-mediated platelet destruction and impairment of platelet production, which leads to thrombocytopenia, a predisposition to bleeding, and altered quality of life for patients. There remains a need in relapsed/refractory ITP for patients failing to maintain their platelet counts with the current therapies.

Rilzabrutinib is an investigational peripheral Bruton’s tyrosine kinase (BTK) inhibitor with Tailored Covalency technology intended to treat ITP.

Abstract #S299: Phase I/II Updated Safety and Efficacy Results of Oral Bruton Tyrosine Kinase (BTK) Inhibitor Rilzabrutinib in Relapsed/Refractory Immune Thrombocytopenia (ITP). Oral Presentation.
Abstract #PB1733: Phase III Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of Rilzabrutinib, Oral BTK Inhibitor, in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia. Abstract-only.
Additional presentation

Abstract #EP1145: Epidemiology, Treatment Patterns, and Clinical Outcomes Among Patients with Acquired Thrombotic Thrombocytopenic Purpura (aTTP) in the United States: an Electronic Health Records Analysis

Editor’s Note:
About sutimlimab: Sutimlimab is an investigational, 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, sutimlimab inhibits the activation of the classical complement pathway with the goal of halting C1-activated hemolysis in CAD to prevent the abnormal destruction of healthy red blood cells. By selectively inhibiting the classical pathway upstream at C1s, sutimlimab does not inhibit the lectin and alternative complement pathways.

Sutimlimab has been granted Breakthrough Therapy by the U.S. Food and Drug Administration (FDA) and Orphan Drug status by the FDA, European Medicines Agency and the Pharmaceuticals and Medical Devices Agency in Japan. Sutimlimab is currently under clinical investigation and has not been approved by any regulatory authority.

About rilzabrutinib: Rilzabrutinib is an oral, peripheral Bruton’s tyrosine kinase inhibitor with Tailored Covalency technology being investigated for the treatment of immune-mediated diseases, including immune thrombocytopenic purpura. BTK is involved in innate and adaptive immune responses and is a signalling molecule in immune mediated diseases. Rilzabrutinib has the potential to target the underlying disease pathogenesis and has not been shown to alter platelet aggregation5. The clinical significance of these mechanisms is currently under investigation and have not been approved by any regulatory authority. A Phase 3 clinical trial to evaluate rilzabrutinib for the treatment of ITP is currently underway with the first patient dosed in April 2021. Rilzabrutinib has been granted orphan drug designation and fast track designation by the U.S. FDA for ITP.

Rilzabrutinib is also being investigated in a Phase 3 trial for pemphigus, an immune- mediated disease characterized by blisters in mucous membranes and skin as well as a Phase 2 study in the autoimmune condition IgG4 disease. Three additional Phase 2 studies in immunological diseases are planned to start in 2021.

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*Tailored Covalency is a registered trademark of Principia Biopharma Inc., a Sanofi company

Broome C, et al. Increased risk of thrombotic events in cold agglutinin disease: A 10-year retrospective analysis. Res Pract Thromb Haemost. 2020;00:1–8.
Quentin A. Hill, Rajeshwari Punekar, Jaime Morales Arias, Catherine M Broome, Jun Su; Mortality Among Patients with Cold Agglutinin Disease in the United States: An Electronic Health Record (EHR)-Based Analysis. Blood 2019; 134 (Supplement_1): 4790.
Lauren C. Bylsma, Anne Gulbech Ording, Adam Rosenthal, Buket Öztürk, Jon P. Fryzek, Jaime Morales Arias, Alexander Röth, Sigbjørn Berentsen; Occurrence, thromboembolic risk, and mortality in Danish patients with cold agglutinin disease. Blood Adv 2019; 3 (20): 2980–2985.
Berentsen S, et al. Haematologica. 2006;91(4):460-466
Langrish CL et al – Preclinical Efficacy and Anti-Inflammatory Mechanisms of Action of the Bruton Tyrosine Kinase Inhibitor Rilzabrutinib for Immune-Mediated Disease – J Immunol published online 5 March 2021