ASCAT 2023 | Results of the BELIEVE study: luspatercept for the treatment of beta-thalassemia
Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, presents the results of the Phase III BELIEVE trial (NCT02604433) of luspatercept to treat patients with transfusion-dependent beta-thalassemia, which were the basis of its approval by the FDA. This interview was recorded at the 18th Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT) 2023, held in London, UK.
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Transcript (edited for clarity)
The BELIEVE study is a randomized, double-blind, placebo Phase III study using luspatercept. Luspatercept is a first in class agent which corrects or reduces the ineffective erythropoiesis, which is the driver of the pathophysiology of thalassemia, through interacting with activating receptor trap. So it is impacting on the pathway of SMAD2 and SMAD3, allowing the erythropoiesis to go through maturation...
The BELIEVE study is a randomized, double-blind, placebo Phase III study using luspatercept. Luspatercept is a first in class agent which corrects or reduces the ineffective erythropoiesis, which is the driver of the pathophysiology of thalassemia, through interacting with activating receptor trap. So it is impacting on the pathway of SMAD2 and SMAD3, allowing the erythropoiesis to go through maturation. BELIEVE is the Phase III in transfusion-dependent thalassemia patients, [it is] already concluded and data have been published, and the data allowed prompted registration of the drug, either by EMA and FDA, for the treatment of anemia in transfusion-dependent thalassemia patients. What did the results show? The primary endpoint of the study was to achieve a reduction equal or more than 33% compared to baseline in 24 weeks of treatment, in a core period, and the primary endpoint was achieved. Also, the secondary endpoint was achieved, which was a reduction of equal or more than 50% in a specific period of time. So now the drug is available, REBLOZYL® is available in real life, and quite a lot of patients in Europe are already on treatment and I believe that it will be extremely important to collect real life data. You know, clinical trials always have some limitations due to the selection of patients, but in real life and in my hand now, the results are very good.
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