It’s a very rewarding trading day for investors in bluebird bio Inc (NASDAQ:BLUE) with shares up nearly 25%, making the stock Wall Street’s bull of the day. The reason? On Sunday at ASH 2017, bluebird and Celgene (NASDAQ:CELG) updated results of the ongoing study for bb2121 in the treatment of Multiple Myeloma patients. Fortunately for investors, the new data suggested more patients convert to complete responses over time, a very positive development. Importantly, bb2121 showed a 56% complete response rate vs. 23% complete response/stringent complete response previously. Overall response rate also increased to 94% from 83%.
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J.P. Morgan Cory Kasimov commented, “More than living up to the high expectations, bb2121 continued to demonstrate a remarkable clinical profile with the majority of these very sick patients showing durable responses to treatment (9 mos PFS of 71%; 9/10 evaluable pts MRD-). We continue to see CELG/BLUE as clearly leading the BCMA race but also suspect that competition (many earlier-stage approaches showcased at ASH) could come into focus over the next 12-18 months. We note that both CELG and BLUE are hosting investor events tonight at ASH.”
Bluebird bio CMO Dave Davidson stated, “Addressing the underlying genetic cause of TDT to restore production of functional hemoglobin can potentially eliminate or reduce the need for chronic blood transfusions in people with this disease, which we expect will reduce the risk of iron overload and associated long-term complications of TDT, and may allow cessation of chelation therapy […] Northstar-2 is the first clinical trial to use our refined manufacturing process for LentiGlobin drug product. Early data from this study demonstrates consistently higher in vivo vector copy numbers and HbAT87Q hemoglobin levels, potentially enabling patients to consistently achieve near-normal or normal total hemoglobin levels. It is important to demonstrate the long-term benefit of gene therapy, and follow-up data of up to three years from the first Northstar study show that nearly all patients with non-β0/β0 genotypes were transfusion-free. We are engaged with the regulatory authorities in the context of the Breakthrough Designation from FDA, and PRIME and Adaptive Pathways from EMA, and look forward to submitting these data to seek marketing approval for LentiGlobin in TDT.”
bluebird bio, Inc. is a clinical-stage biotechnology company. Its integrated product platform includes gene therapy, cancer immunotherapy, and gene editing.