On November 7, 2023 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the third quarter ended September 30, 2023, including recent commercial and operational progress, and regulatory updates (Press release, bluebird bio, NOV 7, 2023, View Source [SID1234637124]).
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"We continue to see strong, linear growth for both the ZYNTEGLO and SKYSONA launches, with patients completing the treatment process, and significant demand across our established qualified treatment center network," said Andrew Obenshain, chief executive officer, bluebird bio. "Launch preparations for lovo-cel for sickle cell disease are well underway in anticipation of our December 2023 PDUFA date. In the third quarter, we accelerated the growth of our QTC network, which is one hundred percent synergistic across ZYNTEGLO and lovo-cel, and advanced conversations with payers who recognize the value of lovo-cel and have responded favorably to our innovative contract offerings. We are confident in our commercial gene therapy experience, committed to continued partnership with the sickle cell community, and excited about the opportunity to bring gene therapy to individuals living with SCD."
RECENT HIGHLIGHTS
Commercial launches of ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel)
Continued strong, linear growth for ZYNTEGLO with 16 patient starts (cell collections) since launch. Cell collection has been completed for 6 patients for SKYSONA to date.
In September, bluebird announced an amendment to its agreement with Lonza, which manufactures drug product for ZYNTEGLO and SKYSONA. The amended agreement enables increased manufacturing capacity for both therapies. The agreement also included modified payment terms intended to better align with the Company’s business objectives.
Value of ZYNTEGLO and SKYSONA continues to be recognized among commercial and government payers. Recently, bluebird signed outcomes-based agreements for ZYNTEGLO with Michigan and Massachusetts state Medicaid agencies. To date, the Company has received zero ultimate denials for both therapies and prior authorization approvals for drug product remain consistent at approximately two weeks across all payers.
bluebird’s qualified treatment center (QTC) network has scaled to 29 activated centers (defined as a signed master service agreement or MSA) including both adult and pediatric centers across 16 states. Of these 29 activated QTCs, four have been activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD) in addition to ZYNTEGLO. The Company remains on track to scale to 40-50 activated QTCs by the end of 2023.
lovo-cel FDA Review
On August 16, 2023, bluebird bio announced that the U.S. Food and Drug Administration (FDA) communicated that an advisory committee meeting will not be scheduled for lovotibeglogene autotemcel (lovo-cel).
The lovo-cel Biologics Licensing Application (BLA) review remains on track. The Agency previously accepted the lovo-cel BLA for Priority Review and set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.
Advance Agreement to Sell PRV
On October 30, 2023, bluebird entered into an agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV), if received, in connection with the potential approval of lovo-cel for sickle cell disease. Under the terms of the agreement, rights to the PRV will transfer to the buyer and the Company will receive $103 million upon closing of the sale, which is contingent upon the FDA’s approval of the BLA for lovo-cel and granting of the PRV. bluebird anticipates receipt of a potential PRV should lovo-cel be approved for patients with sickle cell disease ages 12 and older. Proceeds from the potential sale of the PRV are not yet reflected in the Company’s cash runway.
Data Presentations at ASH (Free ASH Whitepaper) 2023
Updated long-term follow-up data from the Company’s gene therapy programs in sickle cell disease and beta-thalassemia will be presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 9-12, 2023 at the San Diego Convention Center and online. bluebird continues to have the most mature gene therapy programs for both SCD and beta-thalassemia, presenting up to five years and up to nine years of long-term data, respectively.
SICKLE CELL DISEASE DATA
Oral Presentation [#1051]: Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received lovotibeglogene autotemcel (lovo-cel) Gene Therapy: Up to 60 Months of Follow-up
Presenting Author: Julie Kanter, M.D., director of the UAB Adult Sickle Cell clinic, associate professor in the Division of Hematology and Oncology, and co-director of the UAB Comprehensive Sickle Cell Disease Center at the University of Alabama in Birmingham
Date/Time: Monday, December 11, 2023, 4:30 p.m. PT
BETA-THALASSEMIA DATA
Poster Presentation [#1102]: Sustained, Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with betibeglogene autotemcel (beti-cel)
Presenting Author: Alexis A. Thompson, M.D., M.P.H., professor of pediatrics (hematology), Perelman School of Medicine, University of Pennsylvania, Philadelphia, and chief, Division of Hematology, Children’s Hospital of Philadelphia
Date/Time: Saturday, December 9, 2023, 5:30 p.m. PT
Poster Presentation [#2480]: Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with betibeglogene autotemcel (beti-cel) Gene Therapy: Up to 9 Years of Follow-up
Presenting Author: Janet L. Kwiatkowski, M.D., MSCE, professor of pediatrics (hematology), Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, and director, Thalassemia Center, Children’s Hospital of Philadelphia
Date/Time: Sunday, December 10, 2023, 6:00 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH (Free ASH Whitepaper) 2023 are available on the ASH (Free ASH Whitepaper) conference website.
UPCOMING ANTICIPATED MILESTONES
For lovo-cel, the FDA has set a PDUFA goal date for December 20, 2023, and if approved, the Company anticipates commercial launch in early 2024. bluebird estimates approximately 20,000 individuals living with SCD (or one-fifth of the U.S. SCD population) may be eligible for gene therapy.
The Company is on track to scale to 40-50 activated QTCs by the end of 2023. bluebird’s QTC network is designed to maximize its commercial opportunity in beta-thalassemia and to prioritize proximity to individuals with living with SCD in anticipation of a 2024 commercial launch for lovo-cel, if approved by the FDA.
For SKYSONA, the Company continues to anticipate 5-10 patient starts this year as previously guided.
THIRD QUARTER 2023 FINANCIAL RESULTS
Cash Position: The Company’s cash, cash equivalents, marketable securities and restricted cash balance was approximately $227 million, as of September 30, 2023. bluebird anticipates full-year 2023 net cash burn to be in the range of $270-$300 million, as previously guided. Based on current operating plans, bluebird expects its cash, cash equivalents and marketable securities including anticipated cash flows from operations, and excluding $53 million of restricted cash, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the second quarter of 2024.
Revenue, net: Total revenue, net was $12.4 million for the three months ended September 30, 2023, compared to $0.1 million for the three months ended September 30, 2022. The increase of $12.3 million was primarily due to SKYSONA and ZYNTEGLO product revenue.
SG&A Expenses: Selling, general and administrative expenses were $40.7 million for the three months ended September 30, 2023, compared to $33.4 million for the three months ended September 30, 2022. SG&A includes lease expense related to 50 Binney Street; however, the associated sublease income is presented in other income (expense), net. Excluding the lease expense for 50 Binney St., SG&A expenses were $31.1 million for the three months ended September 30, 2023, compared to $24.1 million for the three months ended September 30, 2022. This increase is mainly attributable to commercial costs driven by marketing activities for ZYNTEGLO and SKYSONA in the United States and the performance of commercial readiness activities in the United States for lovo-cel, in anticipation of potential approval, as well as increased employee compensation, benefit and other headcount-related expenses, professional fees, and information technology and facility related costs in 2023. These increased costs were partially offset by decreased consulting fees.
R&D Expenses: Research and development expenses were $45.5 million for the three months ended September 30, 2023, compared to $53.1 million for the three months ended September 30, 2022. The decrease of $7.6 million was primarily due to manufacturing costs related to SKYSONA and ZYNTEGLO now being included in inventory and cost of product revenue, as well as decreased employee compensation, benefit and other headcount-related expenses, license and milestone fees, and information technology and facility related costs in 2023. These decreased costs were partially offset by increased clinical costs, lab expenses, and consulting fees.
Net income (loss): Net loss was $71.7 million for the three months ended September 30, 2023, compared to a net loss of $76.5 million for the three months ended September 30, 2022.
CONFERENCE CALL DETAILS
bluebird will hold a conference call to discuss third quarter financial results and commercial launch progress on Tuesday, November 7 at 8:00 am ET.
To access the call via telephone, please register at this link https://register.vevent.com/register/BIee363f414c2f4fd19da314c2b328d635 to 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.