bluebird bio Experiences Fourth Quarter and Full 12 months 2021 Monetary Outcomes, Highlights Operational Progress and Offers Company Replace

CAMBRIDGE, Mass.–(BUSINESS WIRE)–bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Firm”) right this moment reported monetary outcomes and enterprise highlights for the fourth quarter and full yr ended December 31, 2021, shared current operational progress, and offered a company replace.

“2022 is about as much as be a landmark yr for bluebird bio, with LVV gene therapies for β-thalassemia and cerebral adrenoleukodystrophy below assessment by the U.S. Meals and Drug Administration (FDA) and plans to submit a Biologics License Utility (BLA) for lovo-cel for sickle cell illness (SCD) early subsequent yr,” mentioned Andrew Obenshain, CEO, bluebird bio. “Underscoring these important milestones is a continued deal with commercialization and monetary self-discipline to allow the supply of those transformative therapies to sufferers and their households.”

RECENT HIGHLIGHTS

LOVO-CEL

• UPDATE ON PARTIAL CLINICAL HOLD FOR PATIENTS UNDER THE AGE OF 18 – As we speak, bluebird bio offered an replace on the FDA’s partial scientific maintain for the lovotibeglogene autotemcel (lovo-cel) gene remedy scientific program for sufferers below the age of 18 with SCD. As beforehand communicated, in January 2022, bluebird bio obtained questions from the FDA associated to the partial scientific maintain. Following assessment of the questions from the FDA and an evaluation of the timeline for manufacturing drug product heaps and amassing analytical comparability knowledge within the HGB-210 research, bluebird bio is reaffirming plans to submit the BLA for lovo-cel in Q1 2023. The Firm continues to work with regulators to renew treating sufferers below the age of 18. Within the meantime, the Firm is amassing comparability knowledge from drug product heaps manufactured for grownup sufferers within the HGB-210 research. As beforehand communicated, bluebird bio has handled all sufferers in HGB-206 Group C who will type the first foundation of efficacy for BLA submission, with the demonstration of analytical comparability and validation of the Firm’s business manufacturing course of as the important thing remaining actions previous to submission of the deliberate BLA.

• HGB-206 FINAL INFUSION – As we speak, bluebird bio introduced the completion of the ultimate affected person infusion within the HGB-206 research, the continuing Part 1/2 open-label research designed to judge the efficacy and security of lovo-cel for sickle cell illness. A complete of 45 sufferers have been handled with lovo-cel within the HGB-206 research throughout three therapy cohorts: Teams A (n=7), B (n=2) and C (n=36).

• DATA AT ASH AND PUBLISHED IN THE NEW ENGLAND JOURNAL OF MEDICINE – On December 12, 2021, on the 63^rd American Society of Hematology (ASH) Annual Assembly, bluebird bio introduced new knowledge exhibiting a whole elimination of extreme vaso-occlusive occasions (VOEs) by means of as much as 36 months of follow-up in 25 sufferers who had a historical past of a minimum of 4 extreme VOEs and a minimum of six months follow-up in Group C of its ongoing Part 1/2 HGB-206 research of lovo-cel for sufferers with SCD. The protection knowledge introduced stay in step with the recognized unwanted side effects of autologous hematopoietic stem cell assortment, myeloablative single-agent busulfan conditioning and underlying SCD. Choose knowledge from the Group C cohort of the HGB-206 research have been concurrently printed in The New England Journal of Medication (NEJM).

BETI-CEL

• BETI-CEL BLA ACCEPTANCE AND PRIORITY REVIEW – On November 22, 2021, bluebird bio introduced that the FDA accepted for precedence assessment its BLA for betibeglogene autotemcel (beti-cel), the Firm’s doubtlessly healing gene remedy for grownup, adolescent and pediatric sufferers with β-thalassemia throughout all genotypes who require common purple blood cell (RBC) transfusions. On January 18, 2022, the FDA prolonged the assessment interval for the BLA and revised the Prescription Drug Consumer Payment Act (PDUFA) aim date to August 19, 2022. Below precedence assessment, bluebird could be eligible to obtain a precedence assessment voucher upon potential approval of beti-cel in 2022.

• DATA AT ASH AND PUBLISHED IN THE NEW ENGLAND JOURNAL OF MEDICINE – On December 11, 2021, on the 63^rd ASH Annual Assembly, bluebird bio introduced new knowledge exhibiting that grownup and pediatric sufferers dwelling with β-thalassemia who require common RBC transfusions can produce regular or near-normal ranges of whole hemoglobin and proceed to stay transfusion-free, and obtain secure iron markers, by means of as much as seven years of follow-up within the ongoing long-term follow-up research (LTF-303) of beti-cel. Within the security knowledge introduced there have been zero deaths or vector-derived replication-competent lentivirus, and no occasions of insertional oncogenesis or malignancy in LTF-303. The vast majority of AEs and SAEs have been unrelated to beti-cel and in step with the recognized unwanted side effects of HSC assortment and busulfan conditioning routine. Information from the pivotal HGB-207 Northstar-2 research have been additionally concurrently printed in an unique article within the NEJM.

ELI-CEL

• ELI-CEL CLINICAL HOLD – The Firm stays in communication with the FDA relating to the scientific maintain. The FDA has notified the Firm that the scientific maintain on the elivaldogene autotemcel (eli-cel) program will stay in place and requested further details about security occasions and monitoring within the eli-cel scientific program.

• ELI-CEL BLA ACCEPTANCE AND PRIORITY REVIEW – On December 17, 2021, bluebird bio introduced that the FDA accepted for precedence assessment its BLA for eli-cel, the Firm’s gene remedy for cerebral adrenoleukodystrophy in sufferers lower than 18 years of age. On January 18, 2022, the FDA prolonged the assessment interval for the BLA and revised the PDUFA aim date to September 16, 2022. Below precedence assessment, bluebird could be eligible to obtain a precedence assessment voucher upon potential approval of eli-cel in 2022.

COMPANY

• NEW BOARD OF DIRECTORS APPOINTMENT – As we speak, bluebird bio introduced the appointment of Charlotte Jones-Burton, M.D., M.S., to its Board of Administrators. Dr. Jones-Burton is Senior Vice President, Product Growth and Technique at Chinook Therapeutics, a clinical-stage biotechnology firm discovering, creating and commercializing precision medicines for uncommon, extreme persistent kidney ailments. With greater than 20 years of expertise as a scientific improvement chief, inside medication and nephrology doctor and academician, Dr. Jones-Burton is devoted to creating more healthy communities by means of drug improvement, affected person advocacy and folks engagement. Dr. Jones-Burton earned a medical diploma and Grasp of Science diploma in Epidemiology and Preventive Medication, with a focus in Medical Analysis, from the College of Maryland College of Medication. Her postgraduate coaching included an inside medication residency and a nephrology fellowship on the College of Maryland Medical Programs.

UPCOMING ANTICIPATED MILESTONES

LOVO-CEL

• The Firm is in energetic communication with the FDA to resolve the partial scientific maintain and resume treating sufferers below the age of 18.
• The Firm plans to finish manufacturing of economic drug product validation heaps by mid-2022.
• The Firm expects to substantiate vector and drug product analytical comparability by This fall 2022.
• The Firm is on monitor to submit its BLA for lovo-cel in Q1 2023.

BETI-CEL

• The FDA has set a PDUFA aim date of August 19, 2022, for a call on the approval of beti-cel in sufferers with β-thalassemia with business launch anticipated to comply with in mid-2022 if accredited.
• bluebird bio anticipates an FDA advisory committee assembly for beti-cel and eli-cel can be held over the course of two days on June 9-10, 2022.

ELI-CEL

• The FDA has set a PDUFA aim date of September 16, 2022, for a call on the approval of eli-cel in sufferers with cerebral adrenoleukodystrophy with business launch anticipated to comply with by the tip of 2022 if accredited.
• bluebird bio anticipates an FDA advisory committee assembly for beti-cel and eli-cel can be held over the course of two days on June 9-10, 2022.

FOURTH QUARTER AND FULL YEAR 2021 FINANCIAL RESULTS

• Money Place: The Firm’s restricted money, money and money equivalents and marketable securities stability was roughly $442 million, together with restricted money of roughly $46 million, as of December 31, 2021. The total-year 2022 money burn is anticipated to be lower than $400 million. The Firm’s expectation to generate working losses and unfavourable working money flows sooner or later and the necessity for extra funding to help its deliberate operations elevate substantial doubt relating to its means to proceed as a going concern for a interval of 1 yr after the date that its consolidated monetary statements for the yr ended December 31, 2021 are issued.

  The Firm is exploring a number of financing alternatives, together with plans for the sale of precedence assessment vouchers, which the Firm could be eligible to obtain upon potential approval of beti-cel and eli-cel in 2022, whereas specializing in additional value efficiencies.

• Revenues: Complete income from persevering with operations was $1.6 million and $3.7 million for the three and twelve months ended December 31, 2021, respectively. The Firm didn’t acknowledge income from persevering with operations in 2020.
• R&D Bills: Analysis and improvement bills from persevering with operations have been $79.4 million for the three months ended December 31, 2021, in comparison with $58.8 million for the three months ended December 31, 2020. Analysis and improvement bills have been $319.9 million for the twelve months ended December 31, 2021, in comparison with $319.3 million for the twelve months ended December 31, 2020.
• SG&A Bills: Promoting, basic and administrative bills from persevering with operations have been $53.2 million for the three months ended December 31, 2021, in comparison with $80.6 million for the three months ended December 31, 2020. Promoting, basic and administrative bills have been $210.0 million for the twelve months ended December 31, 2021, in comparison with $240.0 million for the twelve months ended December 31, 2020.
• Web Loss: Web loss from persevering with operations was $132.3 million for the three months ended December 31, 2021, in comparison with $136.3 million for the three months ended December 31, 2020. Web loss from persevering with operations was $562.6 million for the twelve months ended December 31, 2021, in comparison with $561.1 million for the twelve months ended December 31, 2020.

About bluebird bio, Inc.

bluebird bio is pursuing healing gene therapies to offer sufferers and their households extra bluebird days.

With a devoted deal with extreme genetic ailments, bluebird has industry-leading scientific packages for sickle cell illness, β-thalassemia and cerebral adrenoleukodystrophy and is advancing analysis to use new applied sciences to those and different ailments. We customized design every of our therapies to handle the underlying reason for illness and have developed in-depth and efficient analytical strategies to know the protection of our lentiviral vector applied sciences and drive the sector of gene remedy ahead.

Based in 2010, bluebird has the biggest and deepest ex-vivo gene remedy knowledge set on the earth—setting the usual for the {industry}. As we speak, bluebird continues to forge new paths, combining our real-world expertise with a deep dedication to affected person communities and a people-centric tradition that pulls and grows a various flock of devoted birds.

bluebird bio is a trademark of bluebird bio, Inc.

Ahead-Trying Statements

This launch incorporates “forward-looking statements” inside the that means of the Personal Securities Litigation Reform Act of 1995. All statements contained on this launch that don’t relate to issues of historic truth ought to be thought-about forward-looking statements, together with with out limitation statements relating to the Firm’s monetary situation, outcomes of operations, in addition to statements relating to the Firm’s plans and expectations for operations together with anticipated timing referring to its manufacturing plans, regulatory approvals and business launches; the Firm’s plans and expectations for the timing of BLA submissions; the Firm’s plans to substantiate vector and drug product analytical comparability; the timing of anticipated FDA advisory committee conferences; and the expectations for being granted any precedence assessment voucher upon approval of any BLA . Any forward-looking statements are based mostly on administration’s present expectations of future occasions and are topic to quite a lot of dangers and uncertainties that would trigger precise outcomes to vary materially and adversely from these set forth in or implied by such forward-looking statements. These dangers and uncertainties embrace, however aren’t restricted to, the chance that the Firm could not be capable to execute an orderly wind down of European operations with the timing or at a value that we anticipated; the chance that further insertional oncogenic or different security occasions related to lentiviral vector, drug product, or myeloablation can be found or reported over time; the chance that the FDA could impose a scientific maintain on further packages using lentiviral vectors; the chance that we could not deal with the FDA’s issues relating to eli-cel shortly or in any respect; the chance that the FDA could require further data, testing, or monitoring that leads to a delay to our regulatory submission plans together with our BLAs for lovo-cel, beti-cel and eli-cel; the dangers that we could not obtain the anticipated advantages of a separation, and a separation might hurt our enterprise, outcomes of operations and monetary situation; devoted monetary and/or strategic funding sources will not be obtainable on favorable phrases; the chance that we’re unable to comprehend the meant advantages of resizing and reshaping our workforce; the COVID-19 pandemic and ensuing financial circumstances can have a larger impression on the Firm’s operations and plans than anticipated; that preliminary optimistic efficacy and security outcomes from our prior and ongoing scientific trials won’t proceed or be repeated in our ongoing or future scientific trials; the chance that the present or deliberate scientific trials of our product candidates can be inadequate to help regulatory submissions or advertising approval in the USA; and the chance that anybody or extra of our product candidates, won’t be efficiently developed, accredited or commercialized. For a dialogue of different dangers and uncertainties, and different essential components, any of which might trigger our precise outcomes to vary from these contained within the forward-looking statements, see the part entitled “Danger Elements” in our most up-to-date Kind 10-Q, in addition to discussions of potential dangers, uncertainties, and different essential components in our subsequent filings with the Securities and Change Fee. All data on this press launch is as of the date of the discharge, and bluebird bio undertakes no obligation to replace this data until required by legislation.