On November 4, 2015 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies, reported business highlights and financial results for the third quarter ended September 30, 2015 (Press release, bluebird bio, NOV 4, 2015, View Source;p=RssLanding&cat=news&id=2106830 [SID:1234507954]).

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"2015 continues to be a year of significant advancement for our programs in beta-thalassemia, sickle cell disease and oncology, which is reflected in our strong presence at ASH (Free ASH Whitepaper) this year. We are excited about the progress we’ve made in the third quarter in enrolling the HGB-206 study in severe sickle cell disease, and we have made the decision to increase the enrollment target in order to gather more data and provide additional regulatory options," said Nick Leschly, chief bluebird. "Our balance sheet remains strong, allowing us to continue to execute on our goals heading into 2016. This includes initiating the Phase 1 study for our first clinical oncology program, bb2121, in early 2016 and our plans to present data from the Starbeam study of LentiD in CCALD in the first half of 2016."

Recent Highlights

DATA FROM HGB-204, HGB-205 AND HGB-206 CLINICAL STUDIES OF LENTIGLOBIN TO BE PRESENTED AT ASH (Free ASH Whitepaper) – Updated data from the HGB-204 study of LentiGlobin in beta-thalassemia major and the HGB-205 study in beta-thalassemia major and severe sickle cell disease (SCD) will be highlighted in separate oral presentations at the ASH (Free ASH Whitepaper) annual meeting in December. Initial early data from the HGB-206 study of LentiGlobin in severe SCD will be presented in a poster.

DATA FROM PRE-CLINICAL STUDIES OF BB2121 TO BE PRESENTED AT ASH (Free ASH Whitepaper) — bluebird bio will also present data from its lead oncology program, bb2121 at ASH (Free ASH Whitepaper) – the first bluebird bio oncology data to be presented at a major medical meeting. Three poster presentations will highlight preclinical data and manufacturing improvements made to the anti-BCMA CAR-T program.

INCREASED ENROLLMENT TARGET FOR HGB-206 STUDY IN SEVERE SCD – Announced plan to increase enrollment in the U.S.-based HGB-206 study in severe SCD from eight patients to 20 patients to gain additional data and experience with LentiGlobin in SCD. bluebird bio plans to provide an update on enrollment in all three LentiGlobin clinical studies at the ASH (Free ASH Whitepaper) meeting in December.

PUBLISHED GENOME EDITING PAPER IN SCIENCE TRANSLATIONAL MEDICINE – Along with collaborators at the Center for Immunity and Immunotherapy at Seattle Children’s Research Institute, published "Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template" in Science Translational Medicine. The findings showed that it is possible to edit the CCR5 gene to enable T cells to both resist and kill HIV. They also showed that the megaTAL nuclease platform allows researchers to place edited genes very precisely in the genome, leading to precise location and control of expression of the edited gene.

Third Quarter 2015 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2015 were $901.7 million, compared to $492.0 million as of December 31, 2014, an increase of $409.7 million, which was primarily driven by the June 2015 equity financing.

Revenues: Collaboration revenue was $1.3 million for the third quarter of 2015 compared to $6.3 million for the third quarter of 2014. The decrease is a result of an amendment to our collaboration agreement with Celgene in the second quarter of 2015.

R&D Expenses: Research and development expenses were $30.4 million for the third quarter of 2015, compared to $16.6 million for the same period in 2014, an increase of $13.8 million. The increase in research and development expenses was primarily attributable to a $6.6 million increase in employee compensation and benefit expense to support our overall growth and a $3.6 million increase in expenses necessary to support the advancement of our clinical and pre-clinical programs.

G&A Expenses: General and administrative expenses were $13.7 million for the third quarter of 2015, compared to $6.6 million for the same period in 2014, an increase of $7.1 million. The increase in general and administrative expenses was primarily attributable to a $5.7 million increase in employee compensation and benefit expense to support our overall growth.

Net Loss: Net loss was $42.9 million for the third quarter of 2015, compared to net loss of $17.0 million for the third quarter of 2014.

Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $901.7 million as of September 30, 2015 will be sufficient to fund its current operations through 2018.