Here's The Beef With Bluebird's Starbeam Study Results

Bluebird bio IncBLUEreported on Monday top-line results from a study called Starbeam.

Starbeam explores the company's LentiD investigational gene therapy for the treatment of boys under the age of 18 with cerebral adrenoleukodystrophy. Top-line data from the ongoing Phase 2/3 study showed that as of June 13, 88 percent of patients with CALD who completed two years of follow-up post-Lenti-D treatment remain free of major functional disabilities, the company said in a press release. This marks the primary endpoint of the trial and exceeded the predefined interim efficacy benchmark for the study of MFD-free survival of 76 percent.

BTIG: Limited Data

Data provided in Bluebird's press release was "relatively limited," and the company is unable to provide additional commentary due to a pending capital raise, BTIG's Dane Leone commented in a research report.

While the analyst acknowledged that the study did meet the primary endpoint there are still some areas of concern:

1. "At the last data update there were patients with diffuse contrast enhancement reemergence post 12-months. Although the measure can be volatile as a biomarker of inflammation, it is correlative to disease progression."
2. "One patient died due to complications with allogeneic HSCT following withdrawal from the study, and another patient experienced rapid neurological deterioration."
3. "Safety profile has been consistent with myeloablative conditioning. There have been no signs of graft rejection or clonal dominance."

Bottom line, Bluebird's study was light on details, and the Leone's revenue projection of $156 million by 2021 remains unchanged following the data release. As such, the analyst maintains a Buy rating and $112 price target, which values the stock at around 5x EV/2021 sales.

At last check, shares of Bluebird were down 3.02 percent at $107.81.

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