- Gene therapy developer bluebird bio (NASDAQ:BLUE -11.1%) slumps out the blocks as investors head for exits after the company released preliminary data from the Phase 3 Northstar-2 study assessing its LentiGlobin drug product in patients with transfusion-dependent beta thalassemia and non-β0/β0 genotypes. The results will be presented at the European Hematology Association Annual Meeting in Madrid.
- The primary endpoint of the 15-subject trial is the proportion of patients who achieve transfusion independence as defined by total hemoglobin levels of at least 9 g/dL without any red blood cell transfusions for a continuous period of at least 12 months at any time during the study.
- As of June 2, LentiGlobin had been manufactured for six patients using an improved process aimed at increasing vector copy number (VCN) and the percentage of cells successfully transduced.
- Data are presented on the first three patients, but the hemoglobin value is only presented for patient #1 (13.3) at month 6. Follow-up of the other two patients was not sufficient to be clinically relevant.
- According to ClinicalTrials.gov, the primary completion date of the study is January 2020.
- Previously: Dosing underway in bluebird's late-stage study of LentiGlobin in beta thalassemia (Dec. 14, 2016)