On August 8, 2023 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the second quarter ended June 30, 2023, including recent commercial and operational progress, and regulatory updates (Press release, bluebird bio, AUG 8, 2023, View Source [SID1234633956]).

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"As we approach the anniversaries of the FDA approvals of ZYNTEGLO and SKYSONA, we have continued to advance our commercial strategy and prove the model for the gene therapy field on our path to profitability," said Andrew Obenshain, chief executive officer, bluebird bio. "Additionally, with the ongoing FDA review of lovo-cel and potential approval by the end of this year, bluebird is preparing for our largest opportunity yet to impact the lives of patients and families – a gene therapy for individuals living with sickle cell disease in the US."

RECENT HIGHLIGHTS

ZYNTEGLO (betibeglogene autotemcel) Commercial Launch

bluebird continues to build on the launch of ZYNTEGLO for beta-thalassemia. To date, there have been 11 patient starts for ZYNTEGLO.
To date, the Company has received zero ultimate denials from commercial or government payers for ZYNTEGLO; prior authorization approvals for drug product remain consistent at approximately two weeks.
As previously communicated, patient starts remain the key commercial metric during the first year of the ZYNTEGLO launch.
SKYSONA (elivaldogene autotemcel) Commercial Launch

Cell collection has been completed for 5 patients for SKYSONA to date.
Since approval, bluebird has activated four QTCs to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD).
lovo-cel BLA Acceptance and Priority Review

On June 21, 2023, bluebird bio announced that the FDA accepted for priority review its BLA for lovotibeglogene autotemcel (lovo-cel), the Company’s gene therapy for individuals living with sickle cell disease (SCD). bluebird is pursuing FDA approval for lovo-cel for patients ages 12 and older who have a history of vaso-occlusive events (VOEs). The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.
lovo-cel ICER Review

In its ongoing review of the cost-effectiveness of gene therapies for sickle cell disease, the Institute for Clinical and Economic Review (ICER) determined that lovo-cel will be cost effective at a price up to $2.26 million when considering the societal perspective. bluebird anticipates setting a price for lovo-cel upon potential FDA approval.
UPCOMING ANTICIPATED MILESTONES

ZYNTEGLO

The Company is on track to scale to 40-50 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.
SKYSONA

The Company continues to anticipate 5-10 patient starts this year as previously guided.
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.
SECOND QUARTER 2023 FINANCIAL RESULTS

Cash Position: The Company’s cash, cash equivalents, marketable securities and restricted cash balance was approximately $291 million, as of June 30, 2023. The Company anticipates full-year 2023 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, marketable securities and restricted cash, inclusive of revenue will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the fourth quarter of 2024. Excluding $45 million of restricted cash, which is currently unavailable for use, bluebird estimates cash runway into the second quarter of 2024. Please see our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023 for further information regarding our cash runway guidance and other financial results.
Revenue, net: Total revenue, net was $6.9 million for the three months ended June 30, 2023, compared to $1.5 million for the three months ended June 30, 2022. The increase of $5.4 million was primarily due to SKYSONA and ZYNTEGLO product revenue.
SG&A Expenses: Selling, general and administrative expenses were $40.3 million for the three months ended June 30, 2023, compared to $36.7 million for the three months ended June 30, 2022. SG&A includes lease expense related to 50 Binney Street; however, sublease income is presented in other income (expense), net. Excluding the lease expense for 50 Binney St., SG&A expenses were $30.7 million for the three months ended June 30, 2023, compared to $28.2 million for the three months ended June 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 costs related to employee compensation, benefits, and other head-count related expenses.
R&D Expenses: Research and development expenses were $42.3 million for the three months ended June 30, 2023, compared to $63.8 million for the three months ended June 30, 2022. The decrease of $21.5 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 and a decrease in information technology and facility related costs in 2023.
Net income (loss): Net loss was $72.9 million for the three months ended June 30, 2023, compared to a net loss of $100.1 million for the three months ended June 30, 2022.
CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss second quarter financial results and commercial launch progress on Tuesday, August 8 at 8:00 am ET.

To access the call via telephone, please register at this link https://register.vevent.com/register/BI4fa1d86317c74333813f6827624e43ae 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.