On March 4, 2022 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the fourth quarter and full year ended December 31, 2021, shared recent operational progress, and provided a corporate update (Press release, bluebird bio, MAR 4, 2022, View Source [SID1234609519]).
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"2022 is set up to be a landmark year for bluebird bio, with LVV gene therapies for β-thalassemia and cerebral adrenoleukodystrophy under review by the U.S. Food and Drug Administration (FDA) and plans to submit a Biologics License Application (BLA) for lovo-cel for sickle cell disease (SCD) early next year," said Andrew Obenshain, CEO, bluebird bio. "Underscoring these significant milestones is a continued focus on commercialization and financial discipline to enable the delivery of these transformative therapies to patients and their families."
RECENT HIGHLIGHTS
LOVO-CEL
UPDATE ON PARTIAL CLINICAL HOLD FOR PATIENTS UNDER THE AGE OF 18 – Today, bluebird bio provided an update on the FDA’s partial clinical hold for the lovotibeglogene autotemcel (lovo-cel) gene therapy clinical program for patients under the age of 18 with SCD. As previously communicated, in January 2022, bluebird bio received questions from the FDA related to the partial clinical hold. Following review of the questions from the FDA and an assessment of the timeline for manufacturing drug product lots and collecting analytical comparability data in the HGB-210 study, bluebird bio is reaffirming plans to submit the BLA for lovo-cel in Q1 2023. The Company continues to work with regulators to resume treating patients under the age of 18. In the meantime, the Company is collecting comparability data from drug product lots manufactured for adult patients in the HGB-210 study. As previously communicated, bluebird bio has treated all patients in HGB-206 Group C who will form the primary basis of efficacy for BLA submission, with the demonstration of analytical comparability and validation of the Company’s commercial manufacturing process as the key remaining actions prior to submission of the planned BLA.
HGB-206 FINAL INFUSION – Today, bluebird bio announced the completion of the final patient infusion in the HGB-206 study, the ongoing Phase 1/2 open-label study designed to evaluate the efficacy and safety of lovo-cel for sickle cell disease. A total of 45 patients have been treated with lovo-cel in the HGB-206 study across three treatment cohorts: Groups A (n=7), B (n=2) and C (n=36).
DATA AT ASH (Free ASH Whitepaper) AND PUBLISHED IN THE NEW ENGLAND JOURNAL OF MEDICINE – On December 12, 2021, at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, bluebird bio presented new data showing a complete elimination of severe vaso-occlusive events (VOEs) through up to 36 months of follow-up in 25 patients who had a history of at least four severe VOEs and at least six months follow-up in Group C of its ongoing Phase 1/2 HGB-206 study of lovo-cel for patients with SCD. The safety data presented remain consistent with the known side effects of autologous hematopoietic stem cell collection, myeloablative single-agent busulfan conditioning and underlying SCD. Select data from the Group C cohort of the HGB-206 study were simultaneously published in The New England Journal of Medicine (NEJM).
BETI-CEL
BETI-CEL BLA ACCEPTANCE AND PRIORITY REVIEW – On November 22, 2021, bluebird bio announced that the FDA accepted for priority review its BLA for betibeglogene autotemcel (beti-cel), the Company’s potentially curative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. On January 18, 2022, the FDA extended the review period for the BLA and revised the Prescription Drug User Fee Act (PDUFA) goal date to August 19, 2022. Under priority review, bluebird would be eligible to receive a priority review voucher upon potential approval of beti-cel in 2022.
DATA AT ASH (Free ASH Whitepaper) AND PUBLISHED IN THE NEW ENGLAND JOURNAL OF MEDICINE – On December 11, 2021, at the 63rd ASH (Free ASH Whitepaper) Annual Meeting, bluebird bio presented new data showing that adult and pediatric patients living with β-thalassemia who require regular RBC transfusions can produce normal or near-normal levels of total hemoglobin and continue to remain transfusion-free, and achieve stable iron markers, through up to seven years of follow-up in the ongoing long-term follow-up study (LTF-303) of beti-cel. In the safety data presented there were zero deaths or vector-derived replication-competent lentivirus, and no events of insertional oncogenesis or malignancy in LTF-303. The majority of AEs and SAEs were unrelated to beti-cel and consistent with the known side effects of HSC collection and busulfan conditioning regimen. Data from the pivotal HGB-207 Northstar-2 study were also simultaneously published in an original article in the NEJM.
ELI-CEL
ELI-CEL CLINICAL HOLD – The Company remains in communication with the FDA regarding the clinical hold. The FDA has notified the Company that the clinical hold on the elivaldogene autotemcel (eli-cel) program will remain in place and requested additional information about safety events and monitoring in the eli-cel clinical program.
ELI-CEL BLA ACCEPTANCE AND PRIORITY REVIEW – On December 17, 2021, bluebird bio announced that the FDA accepted for priority review its BLA for eli-cel, the Company’s gene therapy for cerebral adrenoleukodystrophy in patients less than 18 years of age. On January 18, 2022, the FDA extended the review period for the BLA and revised the PDUFA goal date to September 16, 2022. Under priority review, bluebird would be eligible to receive a priority review voucher upon potential approval of eli-cel in 2022.
COMPANY
NEW BOARD OF DIRECTORS APPOINTMENT – Today, bluebird bio announced the appointment of Charlotte Jones-Burton, M.D., M.S., to its Board of Directors. Dr. Jones-Burton is Senior Vice President, Product Development and Strategy at Chinook Therapeutics, a clinical-stage biotechnology company discovering, developing and commercializing precision medicines for rare, severe chronic kidney diseases. With more than 20 years of experience as a clinical development leader, internal medicine and nephrology physician and academician, Dr. Jones-Burton is dedicated to creating healthier communities through drug development, patient advocacy and people engagement. Dr. Jones-Burton earned a medical degree and Master of Science degree in Epidemiology and Preventive Medicine, with a concentration in Clinical Research, from the University of Maryland School of Medicine. Her postgraduate training included an internal medicine residency and a nephrology fellowship at the University of Maryland Medical Systems.
UPCOMING ANTICIPATED MILESTONES
LOVO-CEL
The Company is in active communication with the FDA to resolve the partial clinical hold and resume treating patients under the age of 18.
The Company plans to complete manufacturing of commercial drug product validation lots by mid-2022.
The Company expects to confirm vector and drug product analytical comparability by Q4 2022.
The Company is on track to submit its BLA for lovo-cel in Q1 2023.
BETI-CEL
The FDA has set a PDUFA goal date of August 19, 2022, for a decision on the approval of beti-cel in patients with β-thalassemia with commercial launch expected to follow in mid-2022 if approved.
bluebird bio anticipates an FDA advisory committee meeting for beti-cel and eli-cel will be held over the course of two days on June 9-10, 2022.
ELI-CEL
The FDA has set a PDUFA goal date of September 16, 2022, for a decision on the approval of eli-cel in patients with cerebral adrenoleukodystrophy with commercial launch expected to follow by the end of 2022 if approved.
bluebird bio anticipates an FDA advisory committee meeting for beti-cel and eli-cel will be held over the course of two days on June 9-10, 2022.
FOURTH QUARTER AND FULL YEAR 2021 FINANCIAL RESULTS
Cash Position: The Company’s restricted cash, cash and cash equivalents and marketable securities balance was approximately $442 million, including restricted cash of approximately $46 million, as of December 31, 2021. The full-year 2022 cash burn is expected to be less than $400 million. The Company’s expectation to generate operating losses and negative operating cash flows in the future and the need for additional funding to support its planned operations raise substantial doubt regarding its ability to continue as a going concern for a period of one year after the date that its consolidated financial statements for the year ended December 31, 2021 are issued.
The Company is exploring multiple financing opportunities, including plans for the sale of priority review vouchers, which the Company would be eligible to receive upon potential approval of beti-cel and eli-cel in 2022, while focusing on further cost efficiencies.
Revenues: Total revenue from continuing operations was $1.6 million and $3.7 million for the three and twelve months ended December 31, 2021, respectively. The Company did not recognize revenue from continuing operations in 2020.
R&D Expenses: Research and development expenses from continuing operations were $79.4 million for the three months ended December 31, 2021, compared to $58.8 million for the three months ended December 31, 2020. Research and development expenses were $319.9 million for the twelve months ended December 31, 2021, compared to $319.3 million for the twelve months ended December 31, 2020.
SG&A Expenses: Selling, general and administrative expenses from continuing operations were $53.2 million for the three months ended December 31, 2021, compared to $80.6 million for the three months ended December 31, 2020. Selling, general and administrative expenses were $210.0 million for the twelve months ended December 31, 2021, compared to $240.0 million for the twelve months ended December 31, 2020.
Net Loss: Net loss from continuing operations was $132.3 million for the three months ended December 31, 2021, compared to $136.3 million for the three months ended December 31, 2020. Net loss from continuing operations was $562.6 million for the twelve months ended December 31, 2021, compared to $561.1 million for the twelve months ended December 31, 2020.