A Majority of Patients Achieve Transfusion Independence in Phase 3 Trials of LentiGlobin

June 12, 2020 – Bluebird bio has reported the findings from its Phase 3 clinical trial program of betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for β-thalassemia). These show that pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia are able to achieve and maintain transfusion independence with hemoglobin (Hgb) levels that are near-normal (≥10.5 g/dL).

The Northstar-2 trial has enrolled transfusion-dependent β-thalassemia patients who do not have the β0/β0 genotype. Patients enrolled in the Northstar-2 trial ranged in age from four to 34 years, including eight pediatric (<12 years of age) and 15 adolescent/adult (>12 years of age) patients. Eighty-nine percent of evaluable patients (17/19) achieved transfusion independence, with median weighted average total Hgb levels of 11.9 g/dL. Improved iron levels were observed and trends toward improved iron management were seen. Over half of patients have stopped chelation therapy to date, with 7 out of 23 patients using phlebotomy for iron reduction. Biomarkers of erythropoiesis continue to demonstrate a trend toward normalization in patients who achieved transfusion independence, which supports the disease-modifying potential of beti-cel in patients with transfusion-dependent thalassemia.

The Northstar-3 trial has enrolled transfusion-dependent β-thalassemia patients with β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotypes. Six of eight evaluable patients have achieved transfusion independence to date, with median weighted average total Hgb levels of 11.5 g/dL. These patients continued to maintain transfusion independence as of the data cutoff.

Further information is available in the press release at: http://investor.bluebirdbio.com/news-releases/news-release-details/majority-evaluable-patients-across-genotypes-achieve-transfusion

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