Gene therapy leader bluebird bio (BLUE) is up 23% trading at a new all-time high in the premarket after the company gave encouraging updates to its clinical pipeline at the annual American Society of Hematology (ASH) meeting in Atlanta over the weekend.
The biggest news came from blue bird's anti-B-cell maturation antigen (BCMA) CAR T cell therapy that is partnered with oncology giant Celgene (CELG). The company updated results from the ongoing CRB-401 Phase 1 clinical study of bb2121, an investigational anti-B-cell maturation antigen (BCMA) CAR T cell therapy, in 21 patients with late-stage relapsed/refractory multiple myeloma. The overall and complete response rate was very strong and would represent huge progress in a difficult to treat disease. Celgene hopes to start a larger pivotal trial (that would enable FDA approval) for this program next year.
Blue bird also announced data from two studies of its LentiGlobin gene therapy product candidate in patients with transfusion-dependent ß-thalassemia (TDT). The majority of patients in the Phase 3 Northstar-2 (HGB-207) study show higher in vivo vector copy numbers and hemoglobin levels; 5/6 patients producing >6g HbAT87Q at 3 months. 90% of patients with non-ß0/ß0 genotypes free of transfusions in Phase 1/2 Northstar (HGB-204) study after up to three-year follow up, demonstrating durable treatment effect in TDT.
Blue bird also updated HGB-206 -- its LentiGlobin gene therapy in Severe Sickle Cell Disease. The company noted promising early results from two patients treated under amended study protocol and with refined manufacturing process show 51% and 28% anti-sickling HbAT87Q at six and nine months, respectively, exceeding levels seen previously in the HGB-206 study. "These early results with the revised study protocol indicate that gene therapy with LentiGlobin may allow people with SCD to produce substantial levels of normal, anti-sickling, adult hemoglobin. We are hopeful about the possibility that this could substantially reduce the painful and damaging crises that are a hallmark of this disease, potentially allowing patients to live longer, healthier lives."
Jefferies upgraded the stock to Buy this morning while Goldman raised its price target to $309.
Blue bird's stock has had a wild ride. The stock briefly traded above $200 in 2015 when there was hype that its sickle cell gene therapy could be a ‘cure'. Disappointment around that program and in beta thalassemia sent the stock back to $40 in early 2016 but the company has reported encouraging data for its gene therapy programs since then as sentiment around biotech stocks and gene therapy has improved.
Above $200/share, blue bird's market cap is nearly $10 billion.