As per its common habit, bluebird bio (BLUE -4.39%) did not announce the timing of it first-quarter earnings report ahead of schedule, nor did the biotech's management hold a conference call to discuss it after they released it. Nevertheless, the release offers an opportunity for investors to gauge the biotech's potential for the year ahead.

bluebird bio results: The only number that really matters

Metric

Q1 2017

Q4 2016

Quarter-over-quarter Change

Cash, cash equivalents, and marketable securities

$799.9 million

$884.8 million

(9.6%)

Data source: bluebird bio

What happened this quarter?

  • In March, a case study of the first patient with sickle cell disease treated with Bluebird's LentiGlobin gene therapy was published in the New England Journal of Medicine. After 15 months of follow-up, the patient hasn't had any sickle cell disease-related clinical events or hospitalizations, a stark contrast to before the treatment.
  • Bluebird refined its plan for getting European Medicines Agency approval for LentiGlobin in transfusion-dependent beta-thalassemia using the new improved manufacturing process. Assuming positive clinical trial data, the agency agreed that Bluebird could gain approval with complete data from the ongoing Northstar and HGB-205 studies. The agency would also like to see whatever data Bluebird has from the Northstar-2 study, but that doesn't have to be complete for LentiGlobin to gain approval.
  • This week, after the close of the quarter, Bluebird announced that it had licensed its lentiviral vector platform to Novartis and GlaxoSmithKline. The biotech will get an upfront milestone payment (presumably a fairly small one, since the amount wasn't disclosed) and potential future milestone and royalty payments.
  • Though it burned through about 10% of its cash in the recently completed quarter, Bluebird seems to have a decent cash runway.
Normal and sickled red blood cells

Image source: Getty Images.

What management had to say

Nick Leschly, who goes by the title "chief bluebird," said the company is prepping now for filing a Marketing Authorization Application (MAA) in the EU and Biologics License Application (BLA) in the U.S. "In the first quarter of 2017, we have been laser-focused on continuing to develop our commercial and manufacturing infrastructure and ensuring that we are prepared for future MAA and BLA filings," he said.

Turning to the earlier-stage programs, Leschly pointed out the importance of data expected this year. "For our phase 1/2 programs in [sickle cell disease] and multiple myeloma, the 2017 data will dictate the timing and path for our planned phase 3 trials," he said.

Looking forward

Bluebird has plenty of data that will be released this year.

Next month, at the American Society of Clinical Oncology (ASCO) meeting, Bluebird will present updated data for bb2121, its lead CAR-T therapy. That same month, at the European Hematology Association (EHA) meeting, investors will see the first data from a trial using the company's new manufacturing procedure for LentiGlobin to treat beta thalassemia.

In December, at the American Society of Hematology meeting, the use of the new manufacturing procedure in patients with sickle-cell disease will be presented. Bluebird has also said it plans to release data from its often-forgotten Lenti-D program in patients with cerebral adrenoleukodystrophy by the end of this year.