Gene therapy developer bluebird bio (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)
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