Bluebird bio gene therapy shows high response rate in beta thalassemia

Bluebird bio (NASDAQ:BLUE) announces new data from ongoing Phase 3 clinical trials evaluating gene therapy Zynteglo (betibeglogene autotemcel) (formerly LentiGlobin) in adolescent and adult patients with a range of genotypes of transfusion-dependent beta-thalassemia (TDT).

All 23 patients in the Phase 3 Northstar-2 study have been treated and followed for a median of 19.4 months. In 19 evaluable subjects, 89.5% (n=17/19) achieved transfusion independence. Previously, the 17 required a median of 17.5 transfusions each year.

In Northstar-3, 15 patients (genotypes: 9 β⁰/β⁰, 3 β⁰/ β+IVS1-110, 3 homozygous IVS-1-110 mutation) were treated and followed for a median of 14.4 months. 75.0% (n=6/8) achieved transfusion independence while 84.6% (n=11/13) with at least seven months’ follow-up had not received a transfusion in more than seven months. Previously, the 11 required a median of 18.5 transfusions each year.

On the safety front, there were three serious events of veno-occlusive liver disease and two serious events of thrombocytopenia in Northstar-2 and two serious events of pyrexia (elevated body temperature) in Northstar-3. No deaths, graft failures, vector-mediated replication competent lentivirus or clonal dominance, leukemia or lymphoma were reported.

https://seekingalpha.com/news/3582576-bluebird-bio-gene-therapy-shows-high-response-rate-in-beta-thalassemia