American Univesity of Beirut

Dr. Ali Taher leads groundbreaking study to help patients with NTD beta thalassemia

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Professor of clinical hematology oncology and director of the Naef K. Basile Cancer Institute at the American University of Beirut Medical Center, Dr. Ali T​aher, leads Phase 2 BEYOND study of Reblozyl® (luspatercept-aamt) in adults with non-transfusion dependent (NTD) beta thalassemia.

Bristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) announced on June 11, 2021, the first data from the Phase 2 BEYOND study evaluating Reblozyl® (luspatercept-aamt), a first-in-class erythroid maturation agent, plus best supportive care in adult patients with non-transfusion dependent (NTD) beta thalassemia, were presented at the European Hematology Association (EHA) 2021 Virtual Congress as part of its Presidential Symposium (Abstract #S101). Results demonstrated that 77.7% of patients treated with Reblozyl achieved a hemoglobin increase (≥1.0 gram/deciliter) compared to 0% of patients in the placebo arm. Changes in patient-reported outcomes also correlated with increases in hemoglobin. NTD beta thalassemia is a term used to describe patients who do not require lifelong regular red blood cell (RBC) transfusions for survival, although they may require occasional or even frequent transfusions, usually for defined periods of time. The results of this study were featured in the Presidential Symposium of European Hematology Association's Virtual Congress as one of top six abstracts submitted.

“Patients with non-transfusion dependent beta thalassemia experience chronic anemia and iron overload, which may lead to a range of clinical complications, and treatment options are greatly needed," said Ali Taher, MD, PhD, FRCP, of American University of Beirut and BEYOND study investigator. “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 NTD beta thalassemia."

Reblozyl is the first and only erythroid maturation agent approved in the European Union, United States, and Canada to address anemia-associated beta thalassemia and lower-risk myelodysplastic syndromes, representing an important class of therapy for eligible patients.

“We are very encouraged by the magnitude of improvement seen among Reblozyl-treated patients in the BEYOND trial," said Habib Dable, president and chief executive officer of Acceleron. “These data further strengthen our confidence in Reblozyl's potential to become a meaningful option for this important, underserved patient population around the world."

“The results we are presenting at EHA continue to highlight multiple benefits observed with Reblozyl to treat anemia and achieve transfusion independence, and show its potential for patients with non-transfusion dependent disease who face a range of serious, often lifelong health complications," said Noah Berkowitz, MD, PhD, senior vice president, Hematology Development, Bristol Myers Squibb. “Along with our partners at Acceleron, we are committed to advancing our clinical program for Reblozyl for patients living with anemia-associated blood disorders." 

BEYOND Study Results

BEYOND is a Phase 2, randomized, double-blind, placebo-controlled multi-center study to determine the efficacy and safety of Reblozyl versus placebo in adults with non-transfusion dependent (NTD) beta thalassemia. Eligible patients were ≥18 years with beta thalassemia or hemoglobin (Hb) E beta thalassemia and received ≤5 red blood cell (RBC) units in the 24 weeks prior to randomization, with mean baseline Hb ≤10.0 gram/deciliter (g/dL).4

In the study, 145 patients were randomized 2:1 to receive Reblozyl, 1 milligram/kilogram (titration up to 1.25 mg/kg) or placebo subcutaneously every 3 weeks for ≥48 weeks. Patients in both arms continued to receive best supportive care, including RBC transfusions as indicated and iron chelation therapy. The primary endpoint was achievement of ≥1.0 g/dL mean Hb increase from baseline over a continuous 12-week interval from weeks 13–24 in the absence of RBC transfusions. Secondary endpoints included proportion of patients who remained transfusion free over weeks 1–24, who achieved mean Hb increase of ≥1.5 g/dL from baseline to weeks 13–24, and mean change in NTD beta thalassemia patient-reported outcome tiredness and weakness (NTDT-PRO T/W) domain scores (higher scores reflect worse quality of life; QoL).4

Over a continuous 12-week interval from weeks 13-24 in the absence of RBC transfusions, 74 of 96 (77.1%) patients in the Reblozyl treatment arm achieved the study's primary endpoint, ≥1.0 g/dL mean Hb increase from baseline, versus 0 of 49 (0%) patients in the placebo arm (P<0.0001). The primary endpoint was achieved by 40 of 55 (72.7%) patients in the Reblozyl arm with mean baseline Hb of <8.5 g/dL versus 0 (0%) of patients in the placebo arm (P<0.0001), and 34 of 41 patients (82.9%) with mean baseline Hb of ≥8.5 g/dL versus 0 patients (0%) in the placebo arm (P<0.0001). In a key secondary endpoint of the study, during weeks 13-24, 50 of 96 patients (52.1%) in the Reblozyl arm achieved mean Hb increase of ≥1.5 g/dL compared to baseline versus 0 patients (0%) in the placebo arm (P<0.0001). 89.6% of patients in the Reblozyl arm remained transfusion free at weeks 1–24 versus 67.3% of patients in the placebo arm (P=0.0013). Improvements in patient-reported QoL outcomes (tiredness and weakness) were also observed to correlate with Hb increases.

The most common treatment-emergent adverse events of any grade occurring in ≥5% of patients were bone pain (36.5% Reblozyl versus 6.1% placebo), headache (30.2% versus 20.4%), and arthralgia (29.2% versus 14.3%) No malignancies or thromboembolic events were reported in patients treated with Reblozyl.

To read the full study, press on the link:

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