Bluebird Bio reports interim data from Phase 1 HGB-206 study of LentiGlobin
bluebird bio (BLUE) announced new interim data from the ongoing HGB-206 Phase 1 multicenter clinical study of LentiGlobin investigational gene therapy in patients with severe sickle cell disease will be presented in an oral presentation on Saturday, June 16 at the 23rd Congress of the European Hematology Association by Julie Kanter, M.D., Medical University of South Carolina, Charleston, South Carolina.
“The consistent production of increased amounts of anti-sickling HbAT87Q in the Group C patients reflects the substantial positive impact of the changes introduced with the amended HGB-206 study protocol and refined manufacturing process.
All four Group C patients with greater than or equal to three months follow-up are making over 30 percent anti-sickling HbAT87Q.
The first patient treated, now with six months of follow-up, is producing over 60 percent anti-sickling HbAT87Q with a normal total hemoglobin level of 14.2 g/dL,” said David Davidson, M.D., chief medical officer, bluebird bio.
“The upward trajectory in Group C at these early time points suggests the potential for these patients to exceed the initially proposed therapeutic target of 30 percent anti-sickling HbAT87Q. We continue to define the development plan with regulatory authorities, and with further follow-up, we hope to see even higher levels of HbAT87Q, as well as sustained clinical benefit for patients.”
“The early data from Group C patients are very exciting and provide increasing confidence that LentiGlobin has the potential to deliver transformative benefit to patients. The longer-term data from patients treated earlier in the study show that levels of anti-sickling HbAT87Q in patients with SCD treated with LentiGlobin remain stable for at least two years,” said Dr. Kanter, a lead investigator of the HGB-206 study.
“Treatment options that can address the underlying cause of sickle cell disease are limited and LentiGlobin gene therapy has the potential to prevent or substantially reduce damaging symptoms associated with this debilitating disease.”
Separately, bluebird bio announced that new data from the completed Phase 1/2 Northstar (HGB-204) study in adolescents and adults with transfusion-dependent beta-thalassemia and any genotype, and its ongoing, Phase 3 Northstar-2 (HGB-207) multicenter clinical study of LentiGlobin investigational gene therapy in patients with TDT and non-beta0/beta0 genotypes, will be presented in an oral session on June 16 at the 23rd Annual Congress of the European Hematology Association by Franco Locatelli, M.D., Ph.D., of the IRCCS Ospedale Pediatrico Bambino Gesu of Rome, Italy.
“The maturing data from HGB-204 and HGB-207 suggest that one-time treatment with LentiGlobin may address the underlying genetic cause of TDT.
With our refined manufacturing process, the majority of patients with TDT and non-beta0/beta0 genotypes are transfusion-free and producing total hemoglobin at normal or near-normal levels,” said David Davidson, M.D., chief medical officer, bluebird bio.
“We are on track to submit a marketing authorization application in the European Union 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.”
“Consistently higher in vivo vector copy numbers and HbAT87Q hemoglobin levels in patients indicate that LentiGlobin manufacturing refinements have resulted in improved gene therapy characteristics and may enable sustained transfusion independence for a great majority of patients,” said Professor Locatelli, the lead investigator of the Northstar-2 study.
“Further, we are now seeing more than three years of data from the Northstar study indicating that LentiGlobin therapy may enable long-term transfusion independence in the majority of patients with non-beta0/beta0 genotypes.
These results hold the promise to change the natural history of many patients with this severe genetic disorder of hemoglobin production.”
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