bluebird bio (NASDAQ:BLUE) released third-quarter earnings complete with a conference call, which the biotech doesn't usually hold for quarterly results, but this quarter was different with earnings coinciding with the release of abstracts for the American Society of Hematology (ASH) meeting next month.

Bluebird results: The only number that really matters


Q3 2017

Q4 2016

Year-to-Date Change

Cash, cash equivalents, and marketable securities

$1.1 billion

$884.8 million


Data source: Bluebird Bio.

What happened with Bluebird this quarter?

  • Without a doubt, the ASH data was the most important recent release of data because it shows the new manufacturing procedure for its LentiGlobin gene therapy improves the process as expected. Both of the sickle cell disease patients treated with the new protocol that were reported in the abstract had more copies of the globin gene inserted into their cells, and the patient with three-month data available had the highest level of protein expression seen to date in the study.
  • There was also ASH data from the first 18 patients in a phase 1 trial of bb2121, Bluebird's anti-BCMA CAR T therapy, which produced an overall response rate of 89%, or 100% if you exclude the three patients treated at the lowest dose. Bluebird and partner Celgene (NASDAQ:CELG) have started the expansion part of the phase 1 trial at the expected dosage they'll move into later development.
  • Bluebird started the phase 1 trial for its second-generation anti-BCMA CAR T therapy codenamed bb21217.
  • The company published data from the Starbeam trial of its older Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy (CALD). The trial is enrolling more patients and Lenti-D isn't likely to be a big seller given the small number of patients with CALD, but the data looked good so far.
Normal and sickled red blood cells

Image source: Getty Images.

What management had to say

President and CEO Nick Leschly gave a laundry list of additional data for LentiGlobin that will be included in the ASH presentations that wasn't available at the time the abstracts had to be submitted:

Additional follow-up on 204 as well as 205, which will form the basis -- the primary basis of our EU filing from LentiGlobin in [transfusion-dependent beta-thalassemia]; an update on Northstar-2 or 207 study that will include a few more patients that have been treated with the improved manufacturing process and longer follow-up on the patients we showed at EHA; further updates on the 206 sickle cell disease study will include longer follow-up on the 2 patients we just shared in Group B.

And on the ASH presentation for bb2121, Leschly was equally upbeat:

So it will be a pretty significant update in the context of 6 more months across these patients to be able to understand everything from durability to response to safety. So on that sense, the good comprehensive next look at the BCMA program, and both Celgene and us are very excited about that.

Looking forward

Beyond the ASH presentations for LentiGlobin, investors next year will get efficacy data from patients in group C of the 206 sickle cell trial that are using plerixafor mobilization to increase the number of cells collected.

Next year will also bring clarity on the plan for gaining Food and Drug Administration approval for LentiGlobin in patients with transfusion-dependent beta-thalassemia.

And further back in the pipeline, Bluebird is working on a treatment to upregulate expression of fetal hemoglobin in patients with sickle cell disease, which should be ready for the clinic next year.

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