New data presented at the Annual Congress of the European Hematology Association suggests the underlying genetic cause of transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes may be addressed by a one-time treatment with bluebird bio’s experimental gene therapy, LentiGlobin, in patients, and bluebird aim to file for E.U. approval later this year.
LentiGlobin, is an experimental treatment for TDT, a rare and debilitating blood disorder and sickle cell disease (SCD). These results come from two clinical trials, Northstar (HGB-204), and Northstar-2 (HGB-207). The phase 1/2 Northstar trial, was conducted in the US, Australia and Thailand with 18 adults and adolescents treated and monitored for the evalutation of safety and efficacy post-LentiGlobin infusion. Northstar-2, a phase 3 clinical trial, is being conducted on 11 patients, with the aim to enrol 15 adults and 8 adolescents in total, and to meet transfusion independence (where total hemoglobin levels of minimum 9g/dL without red blood cell transfusions for at least 12 months).
Bluebird bio announced this data ahead of the conference, which was delivered in a presentation by Franco Locatelli, M.D., Ph.D., of the IRCCS Ospedale Pediatrico Bambino Gesù of Rome, Italy. Of the 18 patients in the Phase 1/2 Northstar study, 8 out of 10 went without blood transfusions for up to 3 years. The safety profile remains consistent myeloablative conditioning with single-agent busulfan, with no reported cases of graft-versus-host disease. In the second Phase 3 Northstar-2 study, data shows that of the 11 patients, 7 out of 8 are able to produce normal/near normal hemoglobin amounts after 6 months follow up, and are transfusion independent, with a similar safety profile seen in the Northstar study, with No grade 3 or higher drug product-related adverse events (AE) have been observed..
Lead Investigator Professor Locatelli said of the results “we are now seeing more than three years of data from the Northstar study indicating that LentiGlobin therapy may enable long-term transfusion independence … these results hold the promise to change the natural history of many patients with this severe genetic disorder of hemoglobin production.”
Bluebird bio seeks to file LentiGlobin for regulatory approval from the EMA in the second half of 2018. David Davidson, M.D., Bluebird’s chief medical officer, said “We are on track to submit a marketing authorization application in the E.U. later this year, and we continue to work closely with clinical investigators and regulatory authorities to complete our ongoing clinical trials and bring this important treatment option to patients as soon as possible,”.
Source:bluebird bio Presents New Data from Northstar (HGB-204) and Northstar-2 (HGB-207) Studies of LentiGlobin™ Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia at Annual Congress of the European Hematology Association; Press Release