bluebird bio (BLUE 1.13%) released another sneak attack earnings press release with no prior notice on Thursday. Since the company doesn't typically hold a conference call for the earnings, there's apparently no need to give investors a heads-up that earnings are coming like most companies do.
Read the press release -- or this article -- at your leisure. It's an update of what the company has done this year, but doesn't have any new information beyond an update on its cash situation, thus the lack of a need for a conference call, I guess.
For the record, bluebird bio lost $51.8 million during the quarter. The company ended the quarter with $936 million in the bank, which management thinks is enough to get the company through 2018 based on its current business plan. You read that right: bluebird bio has a two-and-half-year runway, something many biotechs can only dream about.
Like its cash situation, bluebird bio's pipeline is in good shape, too. This year, the company worked out a pathway with the FDA and EU regulators to get its lead gene therapy product, LentiGlobin BB305, approved for the treatment of beta-thalassemia major. EU regulators are only requiring data from two ongoing trials -- Northstar and HGB-205 -- while an approval in the U.S. will require two new trials, dubbed HGB-207 and HGB-208.
Clinical trial data for LentiGlobin BB305, which can treat sickle cell disease as well as beta-thalassemia major, has been fantastic. As of the last update in May, two beta-thalassemia major patients hadn't required transfusions for 14 and 16 months, and the one sickle cell patient who's been treated has anti-sickling hemoglobin above the threshold that's believed to be required for minimal sickle cell disease symptoms.
Finally, on the CAR-T front, bluebird bio has signed up a pair of partners -- Kite Pharma and Five Prime Therapeutics -- to help with developing targets on the tumors that the T-cells will attack. The Kite Pharma partnership is developing therapies against human papillomavirus-associated cancers. The Five Prime target hasn't been disclosed.
Mostly perfect
While bluebird has arguably had a great year; it hasn't been entirely perfect -- although you wouldn't know it from the press release since the company failed to mention the negatives for either event.
A deal with Celgene to potentially license multiple CAR-T stocks was narrowed to just a single target BCMA for treating multiple myeloma. Celgene went on to sign a major contract with rival CAR-T developer, Juno Therapeutics. bluebird bio's gene therapy program is further along than the CAR-T products, so the partial shunning by Celgene isn't a major issue.
The aforementioned HGB-207 and HGB-208 trials were reviewed by the National Institutes of Health Recombinant DNA Advisory Committee, which recommended that the company delay the HGB-208 trial in young children until it has more data on LentiGlobin BB305. Fortunately, the committee signed off on the HGB-207 trial in adults and adolescents, although it isn't clear how this might affect the timing for FDA approval.
