On Aug. 3, 2016 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 for cancer, reported business highlights and financial results for the second quarter ended June 30, 2016 (Press release, bluebird bio, AUG 3, 2016, View Source;p=RssLanding&cat=news&id=2192581 [SID:1234514211]).
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"We continue to advance our LentiGlobinTM, Lenti-D and bb2121 programs through clinical trials, and in recent months, we’ve been pleased to showcase some of the innovative platform work we are doing to continuously improve upon our therapies. At the ASH (Free ASH Whitepaper) Workshop on Genome Editing last month, we provided more detail on our proprietary megaTAL genome editing platform, and at the ASGCT (Free ASGCT Whitepaper) annual meeting, we highlighted our progress in developing next-generation T cell-based immunotherapies and improving manufacturing, transduction efficiency and assay development," said Nick Leschly, chief bluebird. "In the second half of 2016, we look forward to continued progress on our clinical programs, including initiation of the LentiGlobin HGB-207 Phase 3 study in non-β0/β0 transfusion-dependent thalassemia (TDT), the integration of manufacturing process improvements into our LentiGlobin clinical trials, and presenting updated LentiGlobin clinical data at ASH (Free ASH Whitepaper)."
Recent Highlights
MANUFACTURING AGREEMENT WITH LONZA – In June, bluebird and Lonza announced a strategic manufacturing agreement providing for the future commercial production of bluebird bio’s Lenti-D and LentiGlobin drug products. This agreement follows a successful multi-year clinical manufacturing relationship and provides bluebird bio with a path to commercial supply including dedicated production suites within Lonza’s state-of-the-art facility. Under this multi-year agreement, Lonza will complete the suite design, construction and validation along with process validation prior to anticipated commercial launch.
GENOME EDITING DATA PRESENTED AT ASH (Free ASH Whitepaper) WORKSHOP ON GENOME EDITING – In July, pre-clinical data from bluebird’s megaTAL genome editing platform was presented at the ASH (Free ASH Whitepaper) Workshop on Genome Editing in Washington, DC. The data reported at the ASH (Free ASH Whitepaper) workshop highlight recent progress bluebird has made in:
Expanding the number of megaTAL targetable sites in the genome to permit the precise placement of an editing event within a target gene
Refining the specificity of megaTALs to eliminate undesirable off-target activity
Combining megaTALs targeting different target genes to achieve the knockout of multiple genes simultaneously
ORPHAN DRUG DESIGNATION GRANTED FOR BB2121 IN MULTIPLE MYELOMA – In May, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for bb2121 in multiple myeloma. bb2121 is a chimeric antigen receptor T cell (CAR T) therapy targeting B cell maturation antigen (BCMA), and is being developed by bluebird bio in collaboration with Celgene Corporation. In February, the first patient was infused in the CRB-401 study of anti-BCMA CAR T therapy bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgene exercised its option to exclusively license bb2121.
PRESENTED INTERIM DATA FROM STARBEAM STUDY AT AAN ANNUAL MEETING – In April, Dr. Florian Eichler of Massachusetts General Hospital for Children presented interim clinical data from the Starbeam study of Lenti-D in CALD at AAN. Initial Starbeam results suggest Lenti-D gene therapy may have similar efficacy to allogeneic hematopoietic stem cell transplant (HCT), the current standard of care, with a more favorable safety profile. As of March 31, 2016, three of the 17 patients enrolled in the study have reached two years of follow-up and remain free of major functional disabilities (MFDs), the primary endpoint of the study. Sixteen of the 17 patients had stabilization of their neurological function score (NFS), and 14 of 17 had a stable Loes score. The safety profile of Lenti-D treatment appeared consistent with myeloablative conditioning.
TEN ABSTRACTS PRESENTED AT ASGCT (Free ASGCT Whitepaper) 19th ANNUAL MEETING – In April, two oral presentations given by bluebird’s academic collaborators highlighted previously presented data from bluebird bio’s ongoing gene therapy clinical trials, including interim data from the Starbeam Study of Lenti-D in cerebral adrenoleukodystrophy, and interim data from the HGB-205 study of LentiGlobin in severe sickle cell disease and TDT. Eight additional presentations were featured at the meeting, highlighting progress across the company’s preclinical, research and process development activities in both HSC gene therapy and T cell immunotherapy.
Second Half 2016 Anticipated Milestones
Update on LentiGlobin process improvements
Initiation of the HGB-207 study in patients with TDT with the non-β0/β0 genotype
Presentation of updated clinical data for LentiGlobin at the ASH (Free ASH Whitepaper) annual meeting in December 2016
Second Quarter 2016 Financial Results and Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2016 were $779.0 million, compared to $865.8 million as of December 31, 2015, a decrease of $86.8 million.
Revenues: Collaboration revenue was $1.6 million for the second quarter of 2016 compared to $4.9 million for second quarter of 2015. The decrease is a result of an amendment to our collaboration agreement with Celgene in June 2015.
R&D Expenses: Research and development expenses were $41.8 million for the second quarter of 2016 compared to $44.3 million for the second quarter of 2015. The decrease in research and development expenses was primarily attributable to decreased in-licensing milestones and fees and stock-based compensation expense partially offset by increased employee payroll and facilities costs due to increased headcount, and increased manufacturing, clinical, and information technology costs to support the advancement of our clinical and pre-clinical programs.
G&A Expenses: General and administrative expenses were $18.4 million for the second quarter of 2016 compared to $10.7 million for the second quarter of 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting costs to support our overall growth.
Net Loss: Net loss was $58.8 million for the second quarter of 2016 compared to $51.8 million for the second quarter of 2015.
Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $779.0 million as of June 30, 2016 will be sufficient to fund its current operations through 2018.