bluebird bio Reports Second Quarter 2024 Results and Highlights Operational Progress and 2024 Guidance

On August 14, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported second quarter results and business highlights for the quarter ended June 30, 2024, including recent commercial and operational progress (Press release, bluebird bio, AUG 14, 2024, View Source [SID1234645891]).

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"We are seeing clear evidence that our commercial launch is accelerating, with over 20 cell collections completed in sickle cell disease and beta-thalassemia to date in 2024, and more than 40 additional patients already scheduled to initiate the treatment journey for a bluebird gene therapy by the end of this year," said Andrew Obenshain, chief executive officer, bluebird bio. "We are further encouraged by the commitment to provide patient access across both commercial and government payers, most recently conveyed through multiple positive Medicaid decisions and the growing number of published coverage policies for LYFGENIA, and we expect approximately 85 patient starts across our portfolio this year."

COMMERCIAL LAUNCH UPDATES

LYFGENIA launch building (lovotibeglogene autotemcel); continued commercial momentum for ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel)

27 patient starts completed across portfolio to date in 2024 (19 ZYNTEGLO, 4 LYFGENIA, 4 SKYSONA).
Launch momentum building with more than 40 additional patients scheduled for cell collection across commercial portfolio through year end.
Successfully completed manufacturing and release testing for first commercial LYFGENIA patient and the first infusion is being scheduled.
Rapid acceleration projected for ZYNTEGLO following the recent approval of additional manufacturing capacity.
Validated access and reimbursement strategy is driving favorable coverage landscape

Significant progress with formalized paths to market access. To date, more than half of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has affirmed coverage for LYFGENIA through a preferred drug list or published coverage criteria.
Nearly 20% 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.
Timely access to ZYNTEGLO and SKYSONA has continued, with zero ultimate denials to date for either therapy across both Medicaid and commercial payers.
Substantial QTC footprint established

bluebird has activated more than 70 total QTCs for LYFGENIA and ZYNTEGLO (defined as a signed MSA).
Six centers are activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD).
RECENT COMPANY UPDATES

James Sterling appointed as Chief Financial Officer

Effective June 10, 2024, James Sterling became Chief Financial Officer of bluebird bio. Mr. Sterling most recently served as chief financial officer of Renalytix plc, a diagnostics company focused on clinical management of kidney disease. Mr. Sterling was previously managing partner at Renwick Capital LLC, and managing director at investment banks Brock Capital Group LLC and Aleutian Capital Group. He also serves as a board director for a fund managed by Star Mountain Capital. Mr. Sterling has experience as a management consultant at Booz Allen Hamilton. He received his B.A. from Boston University and an M.B.A. from Columbia Business School.
Michael Cloonan appointed to bluebird bio’s Board of Directors

On June 20, 2024, Michael Cloonan was appointed to bluebird bio’s Board of Directors. Mr. Cloonan is currently President and Chief Executive Officer of Sionna Therapeutics. He was previously Chief Operating Officer at Sage Therapeutics leading all business and G&A functions. During his four years with Sage, he helped lead the growth of the organization through multiple capital raises, the launch of the company’s first product, and execution of a transformational, multi-billion-dollar collaboration. An experienced and respected global biotech leader, Mr. Cloonan has over 20 years of biopharma experience across global organizations in various business and commercial roles.
2024 GUIDANCE

The Company anticipates approximately 85 patient starts (cell collections) combined across its portfolio of three FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024. Consistent with previous quarters, bluebird plans to provide quarterly updates on patient starts by indication for each of its therapies.
The Company continues to anticipate gross-to-net discounts across all three products to be in the range of 20% to 25% of gross revenue in 2024 and expects they will fluctuate based on product and payer mix, as well as utilization of outcomes-based agreements for LYFGENIA and ZYNTEGLO.
Based on projected timelines from cell collection to infusion, the Company anticipates recognizing revenue from its first infusion of LYFGENIA in the third or fourth quarter of 2024.
SECOND QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $193 million, including restricted cash of approximately $49 million, as of June 30, 2024.

Based on current operating assumptions, bluebird expects its cash and cash equivalents as of June 30, 2024 will be sufficient to fund its operations into the second quarter of 2025i, not accounting for the cash minimums required under the Company’s loan agreement with Hercules Capital, Inc. and excluding receipt of any future tranches under the agreement.

The Company has also renegotiated certain terms of its loan agreement with Hercules Capital, Inc. and is eligible to receive two future tranches totaling $50 million, contingent upon achievement of patient start and product delivery milestones and completion of additional financing. These tranches take the place of previously agreed tranches tied to patient starts and gross profit.
Revenue, net: Total revenue, net was $16.1 million for the three months ended June 30, 2024, compared to $6.9 million for the three months ended June 30, 2023. The increase of $9.2 million was due to increased ZYNTEGLO product revenue.
On March 26, 2024, bluebird announced that it will restate its consolidated financial statements as of and for the year ended December 31, 2022, and for each of the first three quarters of 2022 and 2023 in its Annual Report on Form 10-K for the year ended December 31, 2023 (the "2023 Form 10-K"). The restatements relate to the identification of leases and the treatment of non-lease components contained in lease agreements. The restatement is not expected to impact the Company’s cash position or revenue. As a result of the restatement, the Company is delayed in filing its 2023 Form 10-K and its Quarterly Reports on Form 10-Q for the quarters ended March 31, 2024 (the "Q1 2024 Form 10-Q") and June 30, 2024 (the "Q2 2024 Form 10-Q"). The Company is continuing to work expeditiously to complete these filings.

The financial results included in this press release represent the most current information available to the Company’s management. The Company expects that its actual results to be reported in its Q2 2024 Form 10-Q will not differ materially from the results included herein, however, these results are subject to change following the completion of the Company’s financial close procedures and the review of its consolidated financial statements for the quarter ended June 30, 2024.

CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its second quarter 2024 results and business updates today, Wednesday, August 14, 2024, at 8:00 am ET.

To participate in the conference call, please dial +1 (800) 715-9871 (U.S. and Canada) and ask to be joined into the bluebird call or provide the Conference ID 2776050.

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.