bluebird bio sharply higher on its sickle cell drug

bluebird bio presents updated data from HGB-205 study of LentiGlobin

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bluebird bio presents updated data from HGB-205 study of LentiGlobin

bluebird bio (BLUE) announced updated data from the ongoing HGB-205 clinical study of its LentiGlobin gene therapy product candidate in patients with severe sickle cell disease and transfusion-dependent beta-thalassemia.

HGB-205 is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of patients with severe SCD and TDT.

The study enrolled three patients with severe SCD and four patients with TDT, who have undergone infusion with LentiGlobin DP. Results as of September 20 include: SCD: All three treated patients showed rising HbAT87Q levels in the first six months.

Patient 1204 was 13 years old at study enrollment. At last follow-up, this patient had a total hemoglobin of 12.4 g/dL, of which 6.1 g/dL was HbAT87Q. HbAT87Q concentration in this patient has remained stable since approximately nine months post-infusion.

The patient continues to show marked clinical improvement.

Patient 1207 was 16 years old at study enrollment. At last follow-up, this patient had a total hemoglobin of 10.0 g/dl, of which 0.7 g/dl was HbAT87Q.

This patient had a pre-treatment history of frequent episodes of vaso-occlusive crisis and acute chest syndrome despite hydroxyurea prior to beginning regular transfusions.

Patient 1207 had episodes of ACS and hospitalization at six and eight months post-treatment, and received three transfusions. Patient 1208 was 21 years old at study enrollment.

At last follow-up, this patient had a total hemoglobin of 10.6 g/dL, of which 2.7 g/dL was HbAT87Q.

This patient had a pre-treatment history of frequent episodes of VOCs and ACS prior to beginning regular transfusions, and was still symptomatic while receiving regular transfusions.

Following LentiGlobin treatment, Patient 1208 has had no episodes of VOCs or ACS. TDT: All four patients with TDT have remained free of chronic transfusions since shortly after receiving LentiGlobin DP. Patient 1201 has been free of transfusions for 45.2 months with total hemoglobin of 10.1 g/dL, of which 6.7 g/dL was HbAT87Q. Patient 1202 has been free of transfusions for 40.1 months with total hemoglobin of 12.9 g/dL, of which 10.1 g/dL was HbAT87Q.

Patient 1206 has been free of transfusions for 23.8 months with total hemoglobin of 11.1 g/dL, of which 8.0 g/dL was HbAT87Q. Patient 1203, who is homozygous for the severe beta+ mutation IVS1-110, has been free of transfusions for 20.9 months with total hemoglobin of 8.7 g/dL, of which 6.7 g/dL was HbAT87Q.

Three of four patients were able to begin therapeutic phlebotomy. Patient 1202 subsequently discontinued iron chelation and phlebotomy. The safety profile of LentiGlobin DP continues to be consistent with myeloablative conditioning with single-agent busulfan.

No DP-related adverse events have been observed, and there is no evidence of clonal dominance.

BLUE closed at $171.15. It last traded at $223.40.


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