EHA Updates: Companies Share Positive Data in Different Blood Disorders


The 2021 European Hematology Association is in full swing and data presentations are showing promises for people diagnosed with blood-based diseases. BioSpace is rounding up some of the presentations from the past two days.

Bristol Myers Squibb and Acceleron

Bristol Myers Squibb and Acceleron Pharma announced a first look at data from the Phase II BEYOND study assessing Reblozyl (luspatercept-aamt) in patients with non-transfusion dependent (NTD) beta thalassemia. Data presented at the conference showed that 77.7% of patients treated with Reblozyl achieved a hemoglobin increase than 0% of patients in the placebo arm. 

Changes in patient-reported outcomes also correlated with increases in hemoglobin. Reblozyl is the only erythroid maturation agent approved in the European Union and the United States that addresses anemia-associated beta thalassemia and lower-risk myelodysplastic syndromes. Patients with non-transfusion-dependent beta thalassemia experience chronic anemia and iron overload.

In a key secondary endpoint of the study, during weeks 13-24, 52.1% of patients in the Reblozyl arm achieved a mean hemoglobin increase of ≥1.5 g/dL compared to baseline versus 0 patients in the placebo arm. Also, 89.6% of patients in the Reblozyl arm remained transfusion free at weeks 1-24 versus 67.3% of patients in the placebo arm. 

Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. 

“Results from the BEYOND study show the clinical potential of luspatercept to sustain the elevation of hemoglobin levels in a majority of patients regardless of their baseline hemoglobin status, and improvements were noted in quality of life outcomes in adults with non-transfusion dependent beta thalassemia,” Ali Taher, a physician with the American University of Beirut and BEYOND study investigator said in a statement. 


Sanofi presented Phase III data from the CADENZA trial investigating sutimlimab in the treatment of cold agglutinin disease (CAD) that reinforces the understanding of sutimlimab as a first-in-class investigational C1s inhibitor with the potential to be the first approved treatment for hemolysis in people with CAD. Cold agglutinin disease is a chronic autoimmune hemolytic anemia. 

The disease causes the body’s immune system to mistakenly destroy its healthy red blood cells. Data from the study showed that in CAD patients who do not have a recent history of blood transfusions saw clinically significant hemolysis improvements, anemia, and fatigue following treatment with sutimlimab. 

CADENZA is a second pivotal Phase III study investigating sutimlimab in the treatment of CAD. The CARDINAL study completed in 2019 also assessed sutimlimab in this indication. Karen Knobe, head of Development for Rare Blood Disorders at Sanofi said the data from the two studies would be the basis of a New Drug Application in the European Union. 

“Together, the studies highlight the promising potential of sutimlimab to have a meaningful impact for people living with CAD. Based on the robust clinical evidence we have to-date, sutimlimab significantly inhibits hemolysis and has the potential to be an important new treatment for CAD,” Knobe said in a statement. 

Equillium, Inc.

California-based Equillium presented data from the Phase Ib EQUATE study assessing itolizumab alongside standard of care in first-line acute graft-versus-host-disease (aGVHD). The study results showed that treatment with itolizumab resulted in complete response and an overall response rate of 55% and 70%, respectively. 

Overall survival at month six across all dosing cohorts was 67%, the company said. Most patients were able to maintain response until at least day 169. Additionally, the patients also were able to reduce steroid use significantly.

Itolizumab is a first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM pathway. The pathway plays a central role in modulating the activity and trafficking of T cells that drive several immuno-inflammatory diseases. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited. 

Dolca Thomas, executive vice president of research and development and chief medical officer of Equillium, touted the topline results from the EQUATE study, calling the data “promising” in first-line treatment of acute graft-versus-host disease. 

“The clinical responses achieved were rapid and durable. The complete response rates are particularly compelling given all patients in the study presented with high-risk aGVHD. These data support clinical advancement into pivotal studies in this severely ill patient population where no drugs are approved, and standard of care remains high-dose corticosteroid treatment,” Thomas said in a statement. 

bluebird bio

Massachusetts-based bluebird presented data from multiple studies assessing Zyntelgo (betibeglogene autotemcel, beti-cel), the company’s gene therapy for transfusion-dependent β-thalassemia (TDT), a severe genetic disease caused by mutations in the β-globin gene that result in reduced or significantly reduced hemoglobin (Hb). Beti-cel is a one-time gene therapy that adds functional copies of a modified form of the β-globin gene into a patient’s own blood stem cell. 

Richard Colvin, bluebird’s interim CMO, said the company’s maturing Zyntelgo data continues to show that patients who have received the therapy can be free of the need for transfusion and maintain strong Hb levels over multiple years. Transfusion independence is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

“TDT is usually diagnosed in the first two years of life, and patients with this disease will require regular blood transfusions for the rest of their lives – most as often as every few weeks. It is truly transformative for these patients and their families that they no longer need ongoing blood transfusions,” Colvin said in a statement. 


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