On May 9, 2023 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the first quarter ended March 31, 2023, including recent commercial and operational progress, and regulatory updates (Press release, bluebird bio, MAY 9, 2023, View Source [SID1234631232]).

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"The first quarter of 2023 saw us continue to establish and scale the commercial model for ex-vivo gene therapy in the US through the launches of ZYNTEGLO and SKYSONA and lay the foundation for revenue generation for years to come for bluebird," said Andrew Obenshain, chief executive officer, bluebird bio. "Additionally, with the submission of the lovo-cel BLA for sickle cell disease in April, bluebird has taken a pivotal step towards realizing its most significant opportunity yet — bringing a transformative gene therapy to individuals living with sickle cell disease."

RECENT HIGHLIGHTS

lovo-cel (lovotibeglogene autotemcel) BLA Submitted

On April 24, 2023, bluebird bio announced it submitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for lovo-cel for patients with sickle cell disease (SCD) who are 12 years and older and have a history of vaso-occlusive events. The Company has requested Priority Review, which, if granted, would shorten the FDA’s review of the application to six months from the time of filing, versus a standard review timeline of 10 months.
ZYNTEGLO (betibeglogene autotemcel) Commercial Launch

In the weeks since the Company’s last commercial update, bluebird continues to build on the launch of ZYNTEGLO for beta-thalassemia. To date, there have been six patient starts (cell collections) for ZYNTEGLO.
The first commercial ZYNTEGLO infusion has been completed and the Company anticipates recognizing revenue in Q2 2023.
Payer uptake continues to be positive and to date, the Company has received zero ultimate denials 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. bluebird does not expect to provide ZYNTEGLO revenue projections for 2023.
SKYSONA (elivaldogene autotemcel) Commercial Launch

First commercial infusion with SKYSONA was completed in March 2023. In total, cell collection has been completed for three patients.
Since approval, bluebird has activated three qualified treatment centers (QTCs) to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD). Two additional QTCs on the West Coast are anticipated in 2023.
UPCOMING ANTICIPATED MILESTONES

LOVO-CEL

The Company anticipates BLA acceptance of lovo-cel for SCD in Q2 2023.
The Company continues to anticipate commercial launch in early 2024, if approved. bluebird estimates approximately 20,000 individuals living with sickle cell disease (or 1/5 of the SCD population in the US) may be eligible for gene therapy.
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.
FIRST QUARTER 2023 FINANCIAL RESULTS

Cash Position: The Company’s cash, cash equivalents, marketable securities and restricted cash balance was approximately $364 million, as of March 31, 2023. As bluebird bio launches two first-in-class gene therapies and readies its third investigational gene therapy for SCD for the commercial setting, full-year 2023 cash burn is expected 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 will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the fourth quarter of 2024. This runway includes approximately $45 million of restricted cash, which is currently unavailable for use. Please see our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023 for further information regarding our cash runway guidance and other financial results.

Revenue, net: Total revenue, net was $2.4 million for the three months ended March 31, 2023, compared to $1.9 million for the three months ended March 31, 2022. The increase of $0.4 million was primarily due to SKYSONA product revenue.

SG&A Expenses: Selling, general and administrative expenses were $37.4 million for the three months ended March 31, 2023, compared to $36.1 million for the three months ended March 31, 2022. SG&A includes lease expense related to the 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 $27.8 million for the three months ended March 31, 2023, compared to $36.1 million for the three months ended March 31, 2022. This decrease is mainly attributable to costs related to employee compensation, benefits, and other head-count related expenses.

R&D Expenses: Research and development expenses were $46.1 million for the three months ended March 31, 2023, compared to $77.9 million for the three months ended March 31, 2022. The decrease of $31.7 million was primarily due to decreased employee compensation, benefit and other headcount-related expenses and a decrease in R&D production costs in 2023.

Gain from sale of priority review voucher: Operating expenses were offset by the one-time gain from sale of the Company’s second priority review voucher, for $92.9 million, net, in the first quarter of 2023.
Net income (loss): Net income was $21.2 million for the three months ended March 31, 2023, compared to a net loss of $122.2 million for the three months ended March 31, 2022.