Shares of bluebird bio BLUE gained 8.12% after it announced updated findings from the reported case of acute myeloid leukemia (AML) in LentiGlobin for sickle cell disease (SCD) gene therapy program.
Shares of the company have plunged 24.9% in the year-to-date period compared with the industry’s decline of 0.7%.
Last month, shares had plunged significantly after the company announced a temporary suspension of its phase I/II (HGB-206) and phase III (HGB-210) studies of LentiGlobin gene therapy for SCD (bb1111) due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of AML.
LentiGlobin gene therapy for SCD is an investigational treatment being studied as a potential treatment for the same. bluebird bio’s clinical development program for LentiGlobin for SCD includes the completed phase I/II HGB-205 study, the phase I/II HGB-206 study and the phase III HGB-210 study.
The FDA had placed a clinical hold on the HGB-206 and HGB-210 studies of LentiGlobin for SCD and the HGB-207 and HGB-212 studies of betibeglogene autotemcel for β-thalassemia.
A patient who was treated more than five years ago in Group A of HGB-206 was diagnosed with AML. Consequently, the company started investigating the cause of this patient’s AML in order to determine if there is any relationship with the use of BB305 lentiviral vector in the manufacture of LentiGlobin gene therapy for SCD.
bluebird had also decided to temporarily suspend the marketing of Zynteglo (betibeglogene autotemcel) while the AML case is assessed even though no case of hematologic malignancy has been reported in any patient who has received treatment with Zynteglo for transfusion-dependent β-thalassemia. This is because it is also manufactured using the same BB305 lentiviral vector used in LentiGlobin gene therapy for SCD.
Nevertheless, based on the analyses completed to date by bluebird, it is very unlikely that the SUSAR of AML reported in its HGB-206 study of LentiGlobin gene therapy for SCD was related to the BB305 lentiviral vector (LVV).
Per the company, multiple independent analyses have confirmed that vector insertion in the AML cells from this patient took place in the VAMP4 gene, or vesicle-associated membrane protein 4. VAMP4 itself has no known role in the development of AML or with any cellular process related to cancer.
The company is now in talks with the FDA in order to resume all clinical studies currently on clinical hold, as data from its assessments provide important evidence, demonstrating that it was unlikely that BB305 lentiviral vector played a role in this case.
Other companies developing gene therapy options for SCD include CRISPR Therapeutics CRSP in collaboration with Vertex Pharmaceuticals Incorporated VRTX, conducting an ongoing phase I/II study of the former’s CTX-001. The FDA has also approved the start of Editas Medicine, Inc.’s EDIT phase I/II RUBY study in SCD on EDIT-301.
bluebird bio currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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