SOMERVILLE, Mass.–(BUSINESS WIRE)–bluebird bio, Inc. (NASDAQ: BLUE) today reported third quarter results and business highlights for the quarter ended September 30, 2024, including recent commercial and operational progress.
“Patient starts more than doubled from our second to third quarter update, providing clear evidence that our commercial launches continued to accelerate,” said Andrew Obenshain, chief executive officer. “This momentum, coupled with steps we took in the third quarter to increase manufacturing capacity for ZYNTEGLO and optimize our cost structure, is propelling bluebird forward on our path to becoming a sustainable commercial gene therapy company. We remain focused on securing additional cash resources to extend our runway, which we believe would enable us to achieve this vision and reach cash flow break-even in the second half of 2025.”
COMMERCIAL LAUNCH UPDATES
Continued commercial momentum across the portfolio
- 57 patient starts completed to date in 2024 (35 ZYNTEGLO, 17 LYFGENIA, 5 SKYSONA).
- 17 additional starts scheduled through the remainder of 2024.
- Evidence of strong commercial demand, with 30 patient starts already scheduled in 2025, supporting the potential for cash flow breakeven in the second half of 2025.
- More than 70 activated QTCs, with 40% having initiated or completed treatment for at least one patient.
Validated access and reimbursement strategy is driving favorable coverage landscape
- To date, more than half of all states have affirmed coverage for LYFGENIA through a preferred drug list or published coverage criteria.
- Nearly 50% of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has already completed prior authorization approval for the use of LYFGENIA for at least one patient.
- Multiple outcomes-based agreements are published and in place for LYFGENIA with national commercial payer organizations, representing more than 200 million U.S. lives.
DATA PRESENTATIONS AT ASH 2024
Updated data from the Company’s lentiviral vector (LVV) gene addition programs in patients with sickle cell disease who have a history of vaso-occlusive events and patients with beta-thalassemia who require regular blood transfusions will be presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place December 7-10, 2024 at the San Diego Convention Center and online.
SICKLE CELL DISEASE DATA
- Oral Presentation [#511]: An Update on Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-cel
- Presenting Author: Dr. Stacey Rifkin-Zenenberg (Hackensack)
- Date/Time: Sunday, December 8, 2024, 9:30 a.m. – 11:00 a.m. PT
- Poster Presentation [#3576]: Participants with a History of Stroke in Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials
- Presenting Author: Dr. Jen Jaroscak (The Medical University of South Carolina)
- Date/Time: Sunday, December 8, 2024, 6:00 p.m. – 8:00 p.m. PT
BETA-THALASSEMIA DATA
- Poster Presentation [#2194]: Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy results in durable Hemoglobin A (HbA) Production with up to 10 Years of Follow-Up in Participants with Transfusion-Dependent β-Thalassemia
- Presenting Author: Dr. Alexis A Thompson (Children’s Hospital of Philadelphia)
- Date/Time: Saturday, December 7, 2024, 5:30 p.m. – 7:30 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH 2024 are available on the ASH conference website.
THIRD QUARTER FINANCIAL HIGHLIGHTS
- Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $118.7 million, including restricted cash of approximately $48.0 million, as of September 30, 2024.
bluebird and Hercules are engaging collaboratively as bluebird works to secure adequate cash runway to obtain additional financing and reach cash flow break-even. Based on current forecasts, which assume continued cost-saving initiatives, successfully renegotiating key contracts, and continued collaborative engagement from Hercules, we expect our existing cash and cash equivalents will enable us to fund our operations into the first quarter of 2025.
The Company anticipates quarterly cash flow break-even in the second half of 2025, assuming it scales to approximately 40 drug product deliveries per quarter and obtains additional cash resources to extend its runway.
- Revenue, net: Total revenue, net was $10.6 million for the three months ended September 30, 2024, compared to $12.3 million for the three months ended September 30, 2023, driven by quarter-to-quarter variability in drug product infusions.
Revenue for the third quarter includes revenue from LYFGENIA, following the completion of the first infusion for sickle cell disease.
bluebird previously guided to an anticipated reduction of net revenue in the third quarter; the Company now anticipates net revenue of at least $25 million in the fourth quarter 2024, as previously reported patient starts are infused.
- Cost of Product Revenue: Cost of product revenue was $11.8 million for the three months ended September 30, 2024, compared to $9.1 million for the three months ended September 30, 2023.
- SG&A Expenses: Selling, general and administrative expenses were $39.8 million for the three months ended September 30, 2024, compared to $40.8 million for the three months ended September 30, 2023. The decrease of $1.0 million was primarily driven by decrease in employee compensation, benefit, and other headcount related expenses, commercial expenses, and facility fees, partially offset by increased professional services fees.
- R&D Expenses: Research and development expenses were $23.2 million for the three months ended September 30, 2024, compared to $58.5 million for the three months ended September 30, 2023. The decrease of $35.3 million was primarily driven by material production shift to inventory and cost of product revenue as well as decreased employee compensation, benefit, and other headcount related expenses, consulting fees, and facility and information technology fees.
- Net income (loss): Net loss was $60.8 million for the three months ended September 30, 2024, compared to a net loss of $87.2 million for the three months ended September 30, 2023.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.
Founded in 2010, bluebird has been setting the standard for gene therapy for more than a decade—first as a scientific pioneer and now as a commercial leader. bluebird has an unrivaled track record in bringing the promise of gene therapy out of clinical studies and into the real-world setting, having secured FDA approvals for three therapies in under two years. Today, we are proving and scaling the commercial model for gene therapy and delivering innovative solutions for access to patients, providers, and payers.
With a dedicated focus on severe genetic diseases, bluebird has the largest and deepest ex-vivo gene therapy data set in the field, with industry-leading programs for sickle cell disease, β-thalassemia and cerebral adrenoleukodystrophy. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.
bluebird continues to forge new paths as a standalone commercial gene therapy company, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.
