Bristol Myers Squibb (BMS) and Acceleron have revealed the first data from a phase 2 study of its erythroid maturation agent Reblozyl in adults with non-transfusion dependent (NTD) beta thalassemia.
The data, presented at the European Hematology Association (EHA) 2021 virtual congress, demonstrated that 77% of patients treated with Reblozyl (luspatercept-aamt) achieved a haemoglobin increase compared to 0% in the placebo arm.
On a key secondary endpoint of the study, patients in the Reblozyl arm achieved a mean haemoglobin increase of ≥1.5 g/dL compared to baseline versus 0% of patients in the placebo arm during weeks 13-24.
In addition, 89.6% of Reblozyl-treated patients remained transfusion free at week one to 24 versus 67.3% of patients in the placebo arm.
The data also revealed improvements in patient-reported quality of life outcomes – such as tiredness and weakness – that appeared to correlate with haemoglobin increases.
“The results we are presenting at EHA continue to highlight multiple benefits observed with Reblozyl to treat anaemia and achieve transfusion independence, as well as show its potential for patients with non-transfusion dependent disease who face a range of serious, often lifelong health complications,” said Noah Berkowitz, senior vice president, Hematology Development, Bristol Myers Squibb.
“Along with our partners at Acceleron, we are committed to advancing our clinical programme for Reblozyl for patients living with anaemia-associated blood disorders,” he added.
Rebloyzl is the first erythroid maturation agent to be approved in the EU, US and Canada to treat anaemia in adults with beta thalassemia who require regular red blood cell transfusions.
The drug is also approved to treat anaemia in certain patients with very low- to intermediate-risk myelodysplastic syndromes.
“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.
“This data further strengthens our confidence in Reblozyl’s potential to become a meaningful option for this important, underserved patient population around the world,” he added.