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bluebird (BLUE) Reports Updated Data on Lenti-D Gene Therapy

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bluebird bio, Inc. BLUE announced updated results from phase II/III Starbeam study (ALD-102) of its investigational Lenti-D gene therapy in boys aged 17 years or below with cerebral adrenoleukodystrophy (CALD). The company also announced initial results from the ongoing observational ALD-103 study of outcomes from allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys aged 17 years or below with CALD.

Year to date, shares of bluebird decreased 9.4% compared with the industry’s decline of 3%.

CALD is a genetic disease that mainly affects young boys causing progressive damage to the brain, leading to the permanent loss of physical and cognitive function.


The phase II/III Starbeam study completed its targeted enrollment and had a total study population of 31 patients as of Apr 25, 2018. Of these 31 patients, 29 have received Lenti-D and the median follow-up for all treated patients was 34 months. The primary efficacy endpoint of the study was the proportion of patients who are alive and free of major functional disabilities (MFD) at month 24.

According to the previous data from the study reported in October 2017, of the 17 patients treated with Lenti-D who completed 24 months of follow-up, 15 patients (88%) were alive and MFD-free. One patient withdrew from the study, and one died following rapid development of MFDs post-treatment. Patients who were MFD-free at the end of 24 months (n=15) continued to be free.

As per the updated data from April 2018, 12 more patients received Lenti-D in the phase II/III Starbeam study. Though these patients did not reach the primary endpoint of 24-month follow-up, there were no MFDs reported as of April 25, 2018.

Moreover, bluebird also entered into an agreement with the FDA and the EMA on the clinical development program to support future marketing applications for Lenti-D in the treatment of CALD. Data from the phase II/III Starbeam study suggest that Lenti-D, which utilizes a child’s own cells and does not require a matched donor, may be a potential treatment for CALD.

Further, the company also announced results from the ongoing observational study, ALD-103. As of April 25, 2018, 41 pediatric patients were enrolled in the ALD-103 study and had undergone allo-HSCT. The results were consistent with safety and efficacy outcomes for allo-HSCT in patients with CALD, where 31 patients received cells from unrelated donors and 10 received cells from a related donors.

Allo-HSCT has been successfully used to treat CALD but it may be associated with adverse immunologic complications.

Zacks Rank & Stocks to Consider

bluebird is a Zacks Rank #3 (Hold) stock.

Some better-ranked stocks in the biotech sector are Gilead Sciences Inc. GILD, Ligand Pharmaceuticals Incorporated LGND and Celgene Corp. CELG. While Gilead and Ligand sport a Zacks Rank #1 (Strong Buy), Celgene carries a Zacks Rank #2 (Buy).You can see the complete list of today’s Zacks #1 Rank stocks here.

Gilead’s earnings per share estimates have increased from $6.11 to $6.58 for 2018 and from $6.36 to $6.48 for 2019 over the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 6.43%. The stock has rallied 4% so far this year.

Ligand’s earnings per share estimates have moved up from $4.93 to $5.64 for 2018 and from $5.39 to $5.59 for 2019 in the last 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 59.54%. Share price of the company has increased 85.7% year to date.

Celgene’s earnings per share estimates have increased from $8.52 to $8.76 for 2018 and from $10.18 to $10.65 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 2.38%.

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