On November 1, 2017 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 third quarter ended September 30, 2017 (Press release, bluebird bio, NOV 1, 2017, View Source [SID1234521381]).

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"2017 has been a year focused on execution. In January, we outlined a set of goals intended to bring us closer to our 2022 vision of becoming the gene therapy products company," said Nick Leschly, chief bluebird. "I’m pleased to say that we have accomplished many of these goals. We presented compelling data at ASCO (Free ASCO Whitepaper), EHA (Free EHA Whitepaper) and in the New England Journal of Medicineacross all of our clinical programs, treated our first patient in the expansion cohort of our bb2121 Phase 1 multiple myeloma study, and moved our second generation anti-BCMA CAR T program, bb21217, into the clinic. Coming into ASH (Free ASH Whitepaper), we have an early, but promising, indication that the changes we made in the HGB-206 study have improved engraftment and that mobilization and collection of stem cells using plerixafor may be a safe and viable option for patients with severe sickle cell disease. At ASH (Free ASH Whitepaper), we look forward to sharing additional data across our clinical programs as well as preclinical data that supports our future pipeline."

Recent Highlights

ASH PRESENTATIONS – Today, bluebird bio announced that the company will present clinical and pre-clinical data in 11 abstracts spanning the company’s research and development portfolio. Included among the presentations will be updated clinical data for the company’s clinical studies of LentiGlobin: Northstar (HGB-204) in patients with transfusion-dependent β-thalassemia (TDT), HGB-205 in patients with TDT or severe sickle cell disease (SCD), Northstar-2 (HGB-207) in patients with TDT and non-β0/β0 genotypes, and HGB-206 in patients with SCD. Updated data from the CRB-401 study of bb2121 anti-BCMA CAR T in patients with relapsed/refractory multiple myeloma will also be presented. The company will also present several preclinical and research posters.

HGB-206 UPDATED CLINICAL DATA – In the ASH (Free ASH Whitepaper) abstracts announced today, early results from 2 patients treated in the HGB-206 study using a modified protocol with LentiGlobin drug product (DP) manufactured under a refined manufacturing process demonstrate both higher DP vector copy number (VCN) and higher peripheral VCN after transplant. The toxicity profile observed was consistent with myeloablative conditioning with single-agent busulfan.

HGB-206 PLERIXAFOR SAFETY DATA – Safety data exploring the use of plerixafor mobilization in 3 patients showed an acceptable safety profile and a larger cell dose yield is highlighted in an ASH (Free ASH Whitepaper) abstract. HGB-206 continues to enroll, and patients will be treated under the amended study protocol with DP made from cells obtained through apheresis following plerixafor mobilization.

NORTHSTAR (HGB-204) UPDATED CLINICAL DATA – ASH (Free ASH Whitepaper) abstracts include updated results from the Phase 1/2 Northstar (HGB-204) study in patients with TDT using the original manufacturing process. The updated efficacy outcomes suggested that treatment with LentiGlobin drug product can potentially have a durable effect on eliminating or substantially reducing blood transfusions. Data also indicated that less favorable outcomes were seen in patients who had a low vector copy number (VCN). The safety profile continues to be consistent with autologous transplantation. No drug-product related adverse events (AEs) have been observed, and there is no evidence of clonal dominance.

RMAT FOR LENTIGLOBIN IN SCD – In October, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy Designation for LentiGlobin for the treatment of patients with severe SCD. Under this designation, the FDA will work closely with bluebird bio to provide guidance on the future development of LentiGlobin, including providing advice on generating the evidence needed to support potential approval of the product candidate.

INTERIM LENTI-D DATA IN NEJM – In October, bluebird bio announced the publication in theNew England Journal of Medicineof interim clinical data on the initial 17 patients treated in the Starbeam study of Lenti-D drug product in cerebral adrenoleukodystrophy (CALD). These data were also presented at the Child Neurology Society (CNS) Annual Meeting. As of August 25, 2017, 15/17 patients (88%) in the initial study cohort remained free of major functional disabilities (MFDs) at 24 months, the primary endpoint of the trial. The safety profile of Lenti-D was consistent with myeloablative conditioning. No patients treated with Lenti-D had graft versus host disease (GvHD), and there was no graft rejection or clonal dominance. An expansion cohort of the Starbeam study is enrolling additional patients to enable the manufacture of Lenti-D in Europe and subsequent treatment of subjects in Europe. Findings from this study will, and to add to the overall clinical data package for potential future regulatory filings in the United States and Europe.

FIRST PATIENT TREATED IN BB21217 STUDY – In September, bluebird bio announced that the first patient was treated in CRB-402, the company’s Phase 1 study of bb21217 in patients with relapsed/refractory multiple myeloma. bb21217 is an anti-BCMA CAR T product candidate manufactured in the presence of a PI3 kinase inhibitor, designed to enrich for a more potent, longer-living T cell subtype that in preclinical in vivo studies showed improved anti-tumor activity. Subsequent to study initiation, in September Celgene exercised its option to exclusively license bb21217, resulting in a $15 million option exercise payment from Celgene. Also in September, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for bb21217.

CRB-401 ADVANCES TO EXPANSION COHORT – In September, bluebird bio announced that the first patient was treated in the expansion cohort of CRB-401, the company’s Phase 1 study of bb2121 anti-BCMA CAR T therapy in patients with relapsed/refractory multiple myeloma. Patients in the expansion cohort will be treated at a dose range of 150 to 450 x 106 CAR+ T cells and will be required to have prior exposure to a proteasome inhibitor, an immunomodulatory agent and daratumumab.

NEW BOARD APPOINTMENT – In September, Mary Lynne Hedley, Ph.D., was appointed to the Board of Directors. bluebird bio also announced that with Dr. Hedley’s appointment, John Maraganore, Ph.D., transitioned off the Board of Directors.
Third Quarter 2017 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2017 were $1.1 billion, compared to $884.8 million as of December 31, 2016, an increase of $257.8 million.
Revenues: Total revenue was $7.7 million for the third quarter of 2017 compared to $1.6 million for third quarter of 2016. The increase is attributable to the commencement of revenue recognition for the bb2121 license and manufacturing services under the company’s agreement with Celgene and revenue recognized under bluebird bio’s out-licensing agreement with Novartis Pharma AG (Novartis). In August, Novartis received FDA approval of KYMRIAH and as a result, the company expects to recognize royalty revenue beginning in the fourth quarter of 2017.
R&D Expenses: Research and development expenses were $61.5 million for the third quarter of 2017, compared to $64.0 million for the third quarter of 2016. The decrease in research and development expenses was attributable to decreased platform related expenses as a result of a one-time $15.0 million upfront license payment expensed in the third quarter of 2016, partially offset by increased employee-related costs due to increased headcount to support overall growth, increased clinical trial costs, and increased facility related costs.
G&A Expenses: General and administrative expenses were $23.0 million for the third quarter of 2017, compared to $14.6 million for the third quarter of 2016. The increase in general and administrative expenses was attributable to increased employee-related costs due to increased headcount to support overall growth, increased commercial-related costs attributable to market research costs, increased facility-related expenses, and increased professional and consulting fees.
Cost of License Revenue: Cost of license revenue was $1.1 million for the third quarter of 2017. Cost of license revenue is composed of amounts payable to third party licensors in connection with amounts received under our out-license arrangement with Novartis.
Net Loss: Net loss was $78.8 million for the third quarter of 2017 compared to $77.0 million for the third quarter of 2016.
Webcast Information

bluebird bio will host a live webcast at 8:30 a.m. ET on Wednesday, November 1, 2017. The live webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company’s website at www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or (315) 625-3227 from outside the United States. Please refer to conference ID number 3795968.