New data from gene therapy betibeglogene autotemcel (beti-cel), for transfusion-dependent beta-thalassemia is presented at 64th ASH annual meeting.- bluebird bio
New and updated data from bluebird bio inc.’s gene therapy programs in beta-thalassemia were presented at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition.
The updated data demonstrate sustained treatment effect in patients treated with betibeglogene autotemcel (beti-cel) gene therapy through additional follow-up. The ASH Annual Meeting is taking place December 10-13, 2022, in New Orleans and virtually.
Richard Colvin, M.D., Ph.D., chief medical officer, bluebird bio commented. “Long-term results presented at ASH 2022 show durable transfusion independence in beta-thalassemia patients treated with beti-cel through eight years of follow-up".
Updated data continue to demonstrate sustained treatment effect and quality of life improvements in patients with beta-thalassemia who require regular red blood cell transfusions following treatment with beti-cel. Long-term follow-up data presented at ASH include follow-up for patients living with beta-thalassemia who require regular red blood cell (RBC) transfusions up to 8 years post-treatment (n=3), across ages and genotypes.
As of July 2022, 63 patients received beti-cel across four clinical studies and were followed for a median of 52.0 (20.1–101.7) months. These include two Phase III studies (n=41) which led to the FDA approval of Zynteglo the first and only gene therapy for patients with beta-thalassemia who require regular RBC transfusions in August 2022. In those studies, 90.2% (37/41) of patients achieved transfusion independence (TI). Patients who achieved TI produced normal or near normal levels of total hemoglobin and demonstrated improvements in markers of iron overload and markers of ineffective erythropoiesis.
Patients who achieved TI also showed continued improvement in quality-of-life measures through 3 years following treatment in long-term follow up study, LTF-303. Based on patient testimonials collected at Month 36, the ability to seek employment or be employed increased to 93% of patients (13/14) from 67% (10/15) at baseline. There was also a reduction in school absences compared with baseline (from 95% [18/19] of impacted patients to 50% [5/10]). In addition, 81% (17/21) reported improvement in physical activity at three years, and 100% (20/20) reported an overall benefit from undergoing treatment with beti-cel.
No hematologic malignancies, insertional oncogenesis, vector-derived replication competent lentivirus, or clonal predominance was observed and overall, the safety of the beti-cel treatment regimen largely reflected the known side effects the busulfan conditioning regimen. Nineteen percent (12/63) of patients experienced greater than 1 adverse event (AE) considered related or possibly related to beti-cel; the most common beti-cel related AEs were abdominal pain (5/63 [8%]) and thrombocytopenia (3/63 [5%]). Veno-occlusive liver disease, observed in 11% (7/63) of patients, resolved after treatment. One patient who achieved TI required packed red blood cell (pRBC) transfusions for acute events (for surgery, Phase III, n=1).
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