bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today reported financial results and operational highlights for the fiscal year ended December 31, 2013. At fiscal year end bluebird bio held $206.3 million in cash and cash equivalents.
Pipeline Update and 2013 Highlights Lenti-D (Childhood Cerebral Adrenoleukodystrophy)
- Starbeam Study: In October 2013, the first patient in the phase 2/3 Starbeam (ALD-102) study was transplanted with bluebird’s Lenti-D product candidate. This study is planned to enroll 15 patients with childhood cerebral adrenoleukodystrophy with at least 12 patients being evaluable. The primary endpoint of the study is the percentage of patients that do not develop a major functional disability at 24 months after their transplant. We anticipate completing enrollment in 2015.
LentiGlobin (beta Thalassemia and Sickle Cell Disease)
- HGB-205 Study: In November 2013, the first patient in the phase 1/2 HGB-205 study was transplanted with bluebird’s LentiGlobin product candidate. The HGB-205 study is planned to enroll 7 patients with beta thalassemia major or sickle cell disease at a single site in Paris, France. We anticipate that the first sickle cell patient will be transplanted in this study in 2014.
- Northstar Study: The Northstar (HGB-204) study is a US phase 1/2 study that is planned to enroll 15 patients with thalassemia major. The first patient in this study is expected to be transplanted in early 2014.
- bluebird bio plans to present preliminary data from the HGB-205 and Northstar studies in late 2014.
- Sickle Cell Disease Study: bluebird bio anticipates filing an IND in mid-2014 to start a study with LentiGlobin in patients with sickle cell disease.
Financial ResultsTotal revenues were $20.2 million during the year ended December 31, 2013 compared to $0.3 million for the year ended December 31, 2012 and include amounts allocated to research and development services from bluebird bio’s collaboration with Celgene Corporation.