bluebird bio Plans to Sing in June

bluebird bio released first-quarter earnings today. Management of the gene therapy company didn't hold a conference call, but it doesn't really need to. Without any drugs on the market to report and any new data to present, there isn't much to discuss at the moment.

Waiting for the "moment"In the press release, bluebird bio made some disclosures about timing for its clinical trials.

We'll get to see the first clinical data from the HGB-205 study in patients with sickle cell disease at the European Hematology Association, or EHA, meeting in the middle of June. Abstracts will be available May 21, so we may get some information then, but keep in mind that this is an ongoing trial, so the abstract -- which bluebird had to submitin April -- isn't likely to be as up to date as the actual presentation.

Source: National Institutes of Health

While sickle cell disease is further behind beta-thalassemia major disease, which the HGB-205 trial also enrolled since it's treated with the same LentiGlobin gene therapy, sickle cell disease is more important to bluebird's valuation because it's a much larger market.

The Starbeam study testing Lenti-D in childhood cerebral adrenoleukodystrophy was supposed to enroll 15 patients, but demand was so great that it completed enrollment early with 18 patients expected to receive treatment.

The Northstar trial testing LentiGlobin in patients with beta-thalassemia major also completed enrollment earlier than expected. The 15 patients in the trial will include up to three adolescent patients. Since the goal is to get the beta-thalassemia major patients free from transfusions, if the gene therapy turns out to be a cure -- we don't have enough data to know for sure -- it would be ideal to treat patients as young as possible to eliminate transfusions for the longest period of time. In addition to helping the patient, the elimination of transfusions is important because it'll help justify the high expected cost of gene therapy.

The only trial missing from bluebird's update list in its press release is the HGB-206 study, testing LentiGlobin designed to enroll eight sickle cell disease patients. bluebird bio only said that it's looking forward to "advancing the HGB-206 study" and the study is still listed as enrolling in If the data from the HGB-205 trial presented at EHA are positive, we could see the trial fill up quickly, just like the Northstar trial completed enrolment quickly following the presentation of patients with beta-thalassemia major at the American Society of Hematology meeting in December.

For the record ...bluebird bio recorded a minuscule $6.3 million in revenue from collaborations, but that's primarily from an amortized upfront payment from its 2013 deal with Celgene . The biotech lost $24.8 million in the first quarter, more than double what it lost in the year-ago quarter.

Of course none of that really matters. The most important number is bluebird's $469 million in the bank, which the company believes is enough to last through 2017. With a valuation of $4.5 billion despite not having any drugs on the market, cash -- and raising more of it -- is the least of bluebird bio's concerns.

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