bluebird bio: Why This Firm’s Stock Gained $6.37 Yesterday

Why has bluebird bio Been Hammered in the Market Recently?

Investors should be asking why bluebird bio (BLUE) has been hammered in the market recently before asking why the stock gained $6.37 in trading yesterday. Why was it subjected to a selloff a few days ago? Why? When you consider the announcement of conditional marketing authorization, granted from the European Commission (EC), for the firm’s gene therapy ZYNTEGLO™, for a specific group of patients with transfusion-dependent β-thalassemia (TDT).  If they had asked they would have learned that there were no reasons what so ever for the selling. Except, of course, to satisfy the rule of thumb; selling on good news and taking a profit immediately following stocks’ gains; regardless of the good news that originated the stock’s rally.

Yesterday’s Rallying of bluebird bio

The reasons for investors’ enthusiasm were the promising data announced regarding patients in Group C of the ongoing Phase 1/2 HGB-206 study of LentiGlobin® gene therapy for sickle cell disease (SCD). This announcement was made at the 24th European Hematology Association (EHA) Congress in Amsterdam, the Netherlands.

About Sickle Cell Disease (SCD)

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the β-globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile. Resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children with SCD this means unpredictable episodes of excruciating pain due to vaso-occlusion. As well as other acute complications, such as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD is an investigational gene therapy being studied as a potential treatment for SCDbluebird bio’s clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.

HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for SCD that includes three treatment cohorts: Groups A, B, and C.

We learned from the announcement, at the EHA Congress in Amsterdam, that the latest Group C data, from the Phase 1/2 study, showed an increase in total hemoglobin and substantial reduction in vaso-occlusive crises relative to baseline.  As of March 7, 2019 25 patients were enrolled. A total of 13 patients had been treated with LentiGlobin in Group C, with a median post-treatment follow-up of nine months. Group C, of Phase 1/2 HGB-206 study of LentiGlobin, now includes multiple patients with at least one year of follow-up. Among these patients many had a history of vaso-occlusive crises (VOC).

Study Results:

These patients’ symptoms appear to be resolving. There have been no incidents of ACS or serious VOC reported, and many of their lab tests are approaching normal.

Eight of the 13 treated patients in Group C had at least six months of follow-up, at the time of the data cutoff. In these patients, production of gene therapy-derived hemoglobin (HbAT87Q) ranged from 4.5–8.8 g/dL and total unsupported hemoglobin (Hb) levels ranged from 10.2–15.0 g/dL, at the last study visit. The median concentration of HbAT87Q continued to increase accounting for ≥50 percent of total Hb in patients with at least 12 months of follow up.

Results demonstrate that no ACS or VOC were reported in patients in Group C at up to 15 months post-treatment with LentiGlobin.

In an exploratory analysis, key markers of hemolysis trended toward normal levels. There have been no serious adverse events (SAEs) related to LentiGlobin for SCD. One mild, non-serious event of hot flush, reported by the investigator, was considered related to the LentiGlobin for SCD; it occurred and resolved on the day of drug product infusion and required no treatment.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.  The U.S. FDA granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

Prohost Observations

There were many reasons behind each and every bluebird bio stock gain; while no valid reasons have been observed to be behind the bluebird bio stock selloffs. We found no reasons to abandon the BLUE ship while it sails towards a celebrated destination. Indeed, the best is yet to come.

Those interested in more details about the announced news can read the press release here

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