On November 7, 2022 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the third quarter ended September 30, 2022 and shared recent operational progress (Press release, bluebird bio, NOV 7, 2022, View Source [SID1234623227]).
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"The third quarter officially launched bluebird bio as a commercial gene therapy company, following FDA approvals of ZYNTEGLO for beta-thalassemia and SKYSONA for cerebral adrenoleukodystrophy," said Andrew Obenshain, CEO bluebird bio. "Positive momentum continues to build through the ZYNTEGLO launch and we are on track to complete our first cell collection this quarter. Additionally, we are readying SKYSONA for commercial availability and are on track to file our biologics licensing application for lovo-cel for sickle cell disease in Q1 2023. As a result of milestones in the quarter, we are adjusting our fourth quarter 2022 financial guidance to account for post-marketing commitment expenses and increased investment in current and upcoming launches, which will enable the Company to fully benefit from its first-in-class position. Today, we are also very pleased to welcome Chris Krawtschuk as bluebird’s next chief financial officer. Chris brings significant experience leading and advising financial and capital strategies for a multitude of commercial organizations and will play a critical role in our financing strategy and the evolution of bluebird’s balance sheet as a commercial company."
RECENT HIGHLIGHTS
ZYNTEGLO FDA APPROVAL AND COMMERCIAL LAUNCH PROGRESS
On August 17, 2022, the U.S. Food and Drug Administration (FDA) approved ZYNTEGLO (betibeglogene autotemcel), the first one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.
Following approval, the Company has made significant progress on its first US commercial launch and remains on track for first apheresis (cell collection) in the fourth quarter of this year. Two months post approval, 27 patients have initiated benefits verification and approximately one-third of those patients have progressed to prior authorization. While we anticipate that not all patients will proceed with treatment, these early indicators demonstrate significant demand across a rare disease population. Of note, the process to initiate gene therapy treatment once a patient and their provider have decided it is right for them may take several weeks as the patient completes the consent process, benefits verification and prior authorization.
bluebird has completed activation of all wave one qualified treatment centers (QTCs) and the Company’s QTC network is on track to reach low double digits by the end of the year, as previously guided. In addition to the Company’s planned list of QTCs, bluebird has received inbound requests for inclusion in its QTC network from more than 30 adult and pediatric institutions, demonstrating eagerness to treat. bluebird’s hemoglobinopathies QTC footprint is expected to scale to 40-50 centers by the end of 2023 and is designed to maximize the beta-thalassemia launch and prepare for the potential future launch of lovotibeglogene autotemcel (lovo-cel) for sickle cell disease (SCD), pending regulatory approval.
To date, the Company has signed outcomes-based agreements with pharmacy benefit managers (PBMs) representing more than 40 national and regional plans. Additionally, to date 12 payers have issued medical coverage policies that are favorably aligned to ZYNTEGLO’s clinical evidence base.
SKYSONA FDA APPROVAL AND COMMERCIAL LAUNCH PROGRESS
On September 16, 2022, the U.S. FDA granted Accelerated Approval for SKYSONA (elivaldogene autotemcel) to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
Concurrent with the FDA approval, the previous clinical hold on the eli-cel clinical development program was lifted.
Since approval, bluebird has activated three QTCs to treat patients with CALD. The Company anticipates commercial readiness for SKYSONA in the fourth quarter of 2022.
BETA-THALASSEMIA AND SICKLE CELL DISEASE DATA AT ASH (Free ASH Whitepaper) 2022
Updates on the Company’s beta-thalassemia and sickle cell disease clinical development programs will be presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 10-13, 2022 at the Ernest N. Morial Convention Center in New Orleans, Louisiana.
BETA-THALASSEMIA DATA
Poster (#2348): Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed Up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials
Presenting Author: Mark C. Walters, MD, Medical Director, Jordan Family Center for BMT & Cellular Therapies Research, UCSF Benioff Children’s Hospital Oakland, Oakland, CA
Date/Time: Sunday, December 11, 2022, 6:00 PM – 8:00 PM
Poster (#3665): Long-Term Patient-Reported Outcomes Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia
Presenting Author: Professor Franco Locatelli, Director, Department of Pediatric Hematology and Oncology, Ospedale Pediatrico Bambino Gesù, Rome, Italy
Date/Time: Monday, December 12, 2022, 6:00 PM – 8:00 PM
SICKLE CELL DISEASE DATA
Oral Presentation (#11): lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study
Presenting Author: Mark C. Walters, MD, Medical Director, Jordan Family Center for BMT & Cellular Therapies Research, UCSF Benioff Children’s Hospital Oakland, Oakland, CA
Date/Time: Saturday, December 10, 2022, 10:30 AM
Abstracts are now live on the ASH (Free ASH Whitepaper) website.
MANAGEMENT UPDATE
Effective today, following the filing of the Company’s 10-Q with the SEC, Chris Krawtschuk has been appointed Chief Financial Officer (CFO). Chris joins bluebird from Jubilant Pharma, a global integrated pharmaceutical company, where he was CFO. He brings over 25 years of experience in finance, accounting, tax, strategy, business development, and capital allocation for commercial execution. Prior to Jubilant, Chris was the CFO of MorphoSys US and VP Global Lead Business Unit Controller at Pfizer. Earlier in his career Chris spent 15 years at PwC, finishing his tenure as Managing Director in PwC’s Capital Markets and Accounting Advisory practice.
Interim CFO Katherine Breedis will continue to lead strategic finance initiatives for bluebird and partner with Chris through a transition period; thereafter, she will continue to serve as a strategic advisor.
NEW LABORATORY SPACE
bluebird reported that the Company has signed a 3-year sublease for 42,000 square feet of lab space located at 100 Hood Park Drive in Charlestown, MA, in close proximity to the Company’s Somerville corporate headquarters. bluebird expects to transition lab operations from its Cambridge location to Charlestown by mid-2023. The new lab space is expected to result in approximately $3 million in annual cost savings beginning in 2024.
UPCOMING ANTICIPATED MILESTONES
COMMERCIAL
ZYNTEGLO: Consistent with previous guidance, the Company anticipates the first commercial ZYNTEGLO infusion in Q1 2023. The Company will realize revenue upon infusion.
SKYSONA: bluebird anticipates commercial readiness for SKYSONA in the fourth quarter of 2022.
REGULATORY
LOVO-CEL: The Company expects to complete vector and drug product analytical comparability for the lovo-cel BLA in the fourth quarter of 2022 and remains on track to submit its BLA for lovo-cel in Q1 2023.
LOVO-CEL: The Company remains in active communication with the FDA to resolve the lovo-cel partial clinical hold and resume enrollment and treatment of patients under the age of 18.
THIRD QUARTER 2022 FINANCIAL RESULTS
Cash Position: The Company’s cash and cash equivalents and marketable securities balance was approximately $141 million as of September 30, 2022. In addition, the Company ended the quarter with $45 million in restricted cash. As a result of increased investment in current and upcoming launches, as well as post-marketing commitments related to the FDA approvals of ZYNTEGLO and SKYSONA, bluebird is adjusting its fourth quarter 2022 projected cash burn to between $75 million to $80 million. bluebird anticipates providing full year 2023 guidance early next year.
As of September 30, 2022, the Company has raised approximately $54.1 million in net proceeds through its At-the-Market (ATM) equity facility. Of this $54.1 million, $46.1 million in net proceeds were realized in the third quarter. The Company expects its cash, cash equivalents and marketable securities, together with the anticipated release of $40 million of the Company’s restricted cash in the fourth quarter of 2022, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the second quarter of 2023.
This quarter the Company received two Priority Review Vouchers (PRVs) following the FDA approvals of ZYNTEGLO and SKYSONA and is exploring options to monetize both PRVs. Concurrently, the Company is exploring additional financing opportunities, including public or private equity and debt financings.
Revenues: Total revenue was $0.1 million for the three months ended September 30, 2022, compared to $1.0 million for the three months ended September 30, 2021.
R&D Expenses: Research and development expenses from continuing operations were $53.1 million for the three months ended September 30, 2022, compared to $73.7 million for the three months ended September 30, 2021. The decrease of $20.6 million was primarily due to decreased employee compensation, benefits, other head-count related expenses, information technology and facility-related costs, clinical trial costs, and material production costs. These decreased costs were partially offset by increased platform costs.
SG&A Expenses: Selling, general and administrative expenses from continuing operations were $33.4 million for the three months ended September 30, 2022, compared to $42.2 million for the three months ended September 30, 2021. The decrease of $8.8 million was 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. These decreased costs were partially offset by increased information technology and facility-related costs, primarily attributed to rent expense due to the addition of the Company’s office leases in Cambridge and Somerville, Massachusetts. Cambridge rent expense was offset by $7.5 million in sublease rental income generated for the three months ended September 30, 2022, captured in "other income" on the Company’s income statement.
Net Loss: Net loss from continuing operations was $76.5 million for the three months ended September 30, 2022, compared to $216.8 million for the three months ended September 30, 2021.