On May 2, 2018 bluebird bio, Inc. (Nasdaq: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2018 (Press release, bluebird bio, MAY 2, 2018, View Source [SID1234525956]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"Early in 2018, we’ve made important progress against our stated regulatory and commercial goals for the year, including preparing and investing in our team and infrastructure to bring us closer to providing our therapies to patients," said Nick Leschly, chief bluebird. "Our plans remain on-track for our first regulatory filing in Europe for TDT, we are rapidly advancing development of bb2121 for patients, and we are focused on future innovation platforms."
Recent Highlights
COMPLETED NORTHSTAR (HGB-204) STUDY – In February 2018, the final patient to be treated in Northstar, the company’s Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin for the treatment of patients with TDT, reached two years of follow-up. The study, along with data from the HGB-205 study, and available data from the Northstar-2 Study (HGB-207), will form the basis of the European Marketing Authorization Application (MAA) submission, which is planned for the second half of 2018.
ENTERED INTO AGREEMENT WITH CELGENE TO CO-DEVELOP AND CO-PROMOTE bb2121 – In March 2018, bluebird and Celgene Corporation (Celgene) announced that bluebird has exercised its option to co-develop and co-promote bb2121, an investigational anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy for the potential treatment of patients with relapsed/refractory multiple myeloma in the United States. Under the terms of the agreement, bluebird and Celgene have joint responsibility for development, manufacturing and commercialization in the United States. Celgene will assume sole responsibility for drug product manufacturing and commercialization outside the United States.
PUBLISHED LENTIGLOBIN TDT DATA IN NEJM – In April 2018, interim data was published in the New England Journal of Medicine (NEJM) from two separate two-year clinical studies investigating the potential for LentiGlobin gene therapy to eliminate or reduce chronic blood transfusions in patients with TDT. The interim results from the Northstar (HGB-204) and HGB-205 studies showed that a majority of the 22 patients in the two Phase 1/2 studies followed for two years or longer remained free from transfusions.
PRESENTED PRE-CLINICAL GENE EDITING RESEARCH AT AACR (Free AACR Whitepaper) – In April 2018, bluebird researchers presented a poster at the American Academy of Cancer Research entitled "Enhancing CAR T cell activity by simultaneous checkpoint gene knock-out and PDCD1 promoter driven IL-12 expression." The poster outlined the development of an IL-12 delivery system driven by the PDCD1 promoter whose expression depends on tumor specific CAR T activation, and concluded that the approach could improve safety and pharmacokinetics of IL-12, as well as reduce T cell exhaustion to enhance CAR T functions.
Upcoming Anticipated Milestones
TDT
Filing for European approval of LentiGlobin in patients with TDT and non-β0/β0 genotypes in the second half of 2018
Submission of LentiGlobin clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non- β0/β0 genotypes to the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting and at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting
Submission of LentiGlobin clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and the β0/β0 genotype to the ASH (Free ASH Whitepaper) Annual Meeting
SCD
Update on the clinical development plan and registration strategy for LentiGlobin in SCD by year end 2018
Initiation of an investigator-led Phase 1 clinical study of a lentiviral gene therapy targeting BCL11a suppression and fetal hemoglobin upregulation in patients with SCD
Submission of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD to the ASH (Free ASH Whitepaper) Annual Meeting
Multiple Myeloma
Submission of bb2121 clinical data from the CRB-401 study to the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
Submission of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma to the ASH (Free ASH Whitepaper) Annual Meeting
Initiation by Celgene of a Phase 3 clinical study of bb2121 in third line multiple myeloma
CALD
Presentation of Lenti-D clinical data from the ongoing Starbeam clinical study in patients with CALD in the second half of 2018
First Quarter 2018 Financial Results
Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2018 and December 31, 2017 were $1.57 billion and $1.61 billion, respectively.
Revenues: Total revenues were $16.0 million for the first quarter of 2018 compared to $6.8 million for first quarter of 2017. Effective January 1, 2018, bluebird adopted Accounting Standards Codification, Topic 606, Revenue from Contracts with Customers ("Topic 606"), using the modified retrospective transition method. The increase was primarily attributable to the adoption of Topic 606 and, to a lesser extent, increased manufacturing services under the company’s agreement with Celgene. Total revenues under the previous revenue recognition standard would have been $9.8 million for the first quarter of 2018.
R&D Expenses: Research and development expenses were $97.1 million for the first quarter of 2018 compared to $55.0 million for the first quarter of 2017. The increase in research and development expenses was driven by costs incurred to advance and expand the company’s pipeline and is attributable to increased clinical trial-related costs and manufacturing costs for our development programs, as well as increased employee-related costs due to headcount growth supporting overall research and development activities, and increased license milestones and fees under the company’s strategic collaboration and license agreements.
G&A Expenses: General and administrative expenses were $34.9 million for the first quarter of 2018 compared to $20.3 million for the first quarter of 2017. The increase in general and administrative expenses was attributable to increases in employee-related costs due to increased headcount to support overall growth, commercial-readiness activities, and professional and consulting fees.
Net Loss: Net loss was $115.1 million for the first quarter of 2018 compared to $68.7 million for the first quarter of 2017.