On March 29, 2023 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, 2022 including recent commercial and operational progress, and regulatory updates (Press release, bluebird bio, MAR 29, 2023, View Source [SID1234629492]).
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"Following two milestone FDA approvals in 2022, bluebird is now emerging as a commercial gene therapy leader, fueled by strong momentum and uptake for both ZYNTEGLO and SKYSONA across patients, payers and providers," said Andrew Obenshain, chief executive officer, bluebird bio. "We also remain laser focused on our lovo-cel BLA for sickle cell disease. Following feedback from the FDA in February, bluebird submitted additional information related to CMC comparability analyses to the FDA in early March; we anticipate a response from the Agency within a matter of weeks. Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with more than 50 patients treated and multiple patients followed for more than six years. We remain extremely confident in the quality of our BLA submission. Most importantly, we know that patients and their families are waiting, and we will move quickly to expedite our BLA submission, pending alignment with FDA on product comparability."
RECENT HIGHLIGHTS
lovo-cel (lovotibeglogene autotemcel) BLA Submission Progressing
•In early March, the Company responded to feedback from the FDA on vector and drug product analytical comparability evaluations completed in December 2022; bluebird expects a response from the FDA within a matter of weeks and will move quickly to expedite its BLA submission, pending alignment with FDA on product comparability.
•The Company plans to request priority review for patients 12 and older with a history of vaso-occlusive events. If approved, bluebird continues to anticipate a commercial launch in early 2024.
Momentum Continues in ZYNTEGLO (betibeglogene autotemcel) Commercial Launch
•bluebird has made significant progress in the launch of ZYNTEGLO, with five patient starts (cell collections) for patients with beta-thalassemia to date.
•As launch continues to progress, bluebird is advancing plans to expand manufacturing capacity to meet growing projected demand.
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•On average, prior authorization for the therapy remains at just two weeks, a strong indicator of payer acceptance for ZYNTEGLO; to date, there have continued to be zero ultimate denials for payer coverage.
•bluebird’s qualified treatment center (QTC) network continues to scale as planned with 12 activated QTCs (defined as a signed master service agreement or MSA) and approximately 30 QTCs in the on-boarding or MSA negotiation phase; the Company remains on track to scale to 40-50 centers by the end of 2023.
•As previously announced, during ZYNTEGLO’s 2023 launch year, the Company expects to report key metrics, including the number of patient starts. bluebird does not expect to provide ZYNTEGLO revenue projections for 2023.
SKYSONA (elivaldogene autotemcel) Commercial Launch Continues on Track
•Cell collection has been completed for two patients to be treated with SKYSONA and the first commercial infusion has been completed.
•Since approval, bluebird has activated three QTCs to treat patients with cerebral adrenoleukodystrophy (CALD).
Richard Paulson, MBA appointed to bluebird bio Board of Directors
•On March 24, 2023, Richard Paulson was appointed to bluebird bio’s Board of Directors, effective April 3, 2023. Mr. Paulson is currently President and Chief Executive Officer of Karyopharm Therapeutics. He was previously Executive Vice President and Chief Executive Officer of Ipsen North America, where he focused on innovative therapies and specialty care for oncology, neuroscience and rare diseases. An experienced global biotech and pharmaceutical leader, Mr. Paulson has served in a number of leadership roles across multiple continents, including general management, marketing, sales and market access.
UPCOMING ANTICIPATED MILESTONES
LOVO-CEL
•BLA submission anticipated following response from FDA on analytical comparability data in the coming weeks.
•The Company continues to anticipate commercial launch in early 2024, if approved.
ZYNTEGLO
•The Company is on track to scale to 40-50 centers by the end of 2023.
SKYSONA
•The Company remains on track for 5-10 patient starts this year as previously guided.
FOURTH QUARTER AND FULL YEAR 2022 FINANCIAL RESULTS
•Cash Position: The Company’s cash and cash equivalents, marketable securities and restricted cash balance was approximately $227 million, as of December 31, 2022. As bluebird bio
launches two first-in-class gene therapies and readies its third investigational gene therapy for SCD for the commercial setting, full-year 2023 cash burn is expected to be in the range of $270-$300 million, as previously guided. Based on current operating plans, bluebird expects its cash, cash equivalents, restricted cash and marketable securities, including the net proceeds from the sale of its second priority review voucher (PRV) of $93 million and net proceeds of $131 million from its public offering in January, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the fourth quarter of 2024. This runway includes approximately $45 million of restricted cash, which is currently unavailable for use. Please see our Annual Report filed on Form 10-K for further information regarding our cash runway guidance and other financial results.
•Revenues: Total revenue was $0.06 million for the three months ended December 31, 2022, compared to $1.6 million for the three months ended December 31, 2021. Total revenue was $3.6 million for the twelve months ended December 31, 2022, compared to $3.7 million for the twelve months ended December 31, 2021. The Company anticipates reporting commercial revenue in its Q1 2023 financial statement, as previously guided.
•R&D Expenses: Research and development expenses from continuing operations were $45.9 million for the three months ended December 31, 2022, compared to $79.4 million for the three months ended December 31, 2021. Research and development expenses from continuing operations were $240.8 million for the twelve months ended December 31, 2022, compared to $320.0 million for the twelve months ended December 31, 2021. The decrease in both periods were primarily due to decreased employee compensation, benefits, other head-count related expenses, information technology and facility-related costs, clinical trial costs, and laboratory costs.
•SG&A Expenses: Selling, general and administrative expenses from continuing operations were $30.7 million for the three months ended December 31, 2022, compared to $53.2 million for the three months ended December 31, 2021. Selling, general and administrative expenses from continuing operations were $136.9 million for the twelve months ended December 31, 2022, compared to $210.0 million for the twelve months ended December 31, 2021. The decrease in both periods were primarily due to decreased employee compensation, benefit, and other head-count related expenses and decreased commercial readiness activities due to the Company’s decision to focus its efforts on the U.S. market.
•Gain from sale of priority review voucher: The increase in gain from sale of priority review voucher, net was related to the sale of a priority review voucher for $102 million in the fourth quarter of 2022.
•Net Income/Loss: Net income from continuing operations was $32.2 million for the three months ended December 31, 2022, compared to a loss of $132.3 million for the three months ended December 31, 2021. Net loss from continuing operations was $266.6 million for the twelve months ended December 31, 2022, compared to $562.6 million for the twelve months ended December 31, 2021.
CONFERENCE CALL DETAILS
bluebird will hold a conference call to discuss fourth quarter and full year 2022 financial results and operational progress on Wednesday, March 29 at 8:00 am ET.
To access the call via telephone please follow this link https://register.vevent.com/register/BI0a0b3cf9c17a46cbbabcc02f50c3f12e to register online and receive a dial in number and unique PIN to access the live conference call.
The live webcast of the call may be accessed by visiting the "Events & Presentations" page within the Investors & Media section of the bluebird website at View Source A replay of the webcast will be available on the bluebird website for 90 days following the event.