bluebird bio Sells Priority Review Voucher for $102 Million

Rhea-AI Summary

bluebird bio announced a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $102 million. The PRV was awarded due to FDA approvals of its gene therapies, ZYNTEGLO and SKYSONA, in 2022. CEO Andrew Obenshain stated that this non-dilutive capital strengthens the company's financial outlook, aiding the launches of these therapies and upcoming milestones, including a BLA submission for lovo-cel for sickle cell disease. The transaction, subject to closing conditions, marks a significant financial maneuver for the company.

Positive

• Sale of a Rare Pediatric Disease Priority Review Voucher for $102 million strengthens financial position.
• Non-dilutive capital enhances the ongoing launches of ZYNTEGLO and SKYSONA.
• Plans for BLA submission for lovo-cel for sickle cell disease indicate future growth potential.

Negative

• None.

- Non-dilutive capital meaningfully strengthens Company’s financial position -

SOMERVILLE, Mass.--(BUSINESS WIRE)-- bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $102 million.

bluebird was granted two PRVs upon the US Food and Drug Administration (FDA) approvals of ZYNTEGLO^® (betibeglogene autotemcel) for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood cell transfusions and SKYSONA^® (elivaldogene autotemcel) for the treatment of early, active cerebral adrenoleukodystrophy, on August 17, 2022 and September 16, 2022 respectively.

“With the sale of our first priority review voucher, we have significantly strengthened our financial outlook,” said Andrew Obenshain, chief executive officer, bluebird bio. “As momentum builds across our business, this non-dilutive capital further bolsters the ongoing launches of our two recently approved gene therapies and the execution of near-term, value-creating milestones, including the planned submission and subsequent FDA review of our biologics licensing application for lovo-cel for sickle cell disease.”

bluebird will receive a payment of $102 million upon closing of the transaction, which remains subject to customary closing conditions, including anti-trust review. The Company continues to explore additional financing opportunities, including the monetization of its second PRV, and anticipates providing full year 2023 guidance early next year.

About the Rare Pediatric Disease Priority Review Voucher Program

The Rare Pediatric Disease Priority Review Voucher Program is intended to encourage development of new drug and biological products for the prevention and treatment of certain rare pediatric diseases. Under this program, upon approval, the US Food and Drug Administration awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The voucher can be redeemed to receive priority review of a subsequent marketing application for a different product. PRVs may be sold or transferred, and there is no limit on the number of times a PRV can be transferred.

About bluebird bio, Inc.

bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.

With a dedicated focus on severe genetic diseases, bluebird has industry-leading programs for sickle cell disease, β-thalassemia and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases. 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.

Founded in 2010, bluebird has the largest and deepest ex-vivo gene therapy data set in the world—setting the standard for the industry. Today, bluebird continues to forge new paths, 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.

bluebird bio, ZYNTEGLO and SKYSONA are registered trademarks of bluebird bio, Inc. All rights reserved.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements, including our statements regarding the Company’s financial condition, results of operations, and anticipated cash runway, as well as statements regarding the Company’s plans and expectations for additional financing opportunities and operations including expected timing relating to its regulatory approvals, commercial launches, plans for future regulatory submissions, expectations regarding the receipt of any Priority Review Vouchers upon potential approval of lovo-cel, and our expectations regarding the timing for a potential BLA submission for lovo-cel, anticipated PDUFA goal dates and anticipated FDA approval of the BLA for lovo-cel. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. These risks include, but are not limited to: the risk that we may not realize expected cost savings from the restructuring, including the anticipated decrease in operational expenses, at the levels we expect; we may encounter additional delays in the development of our programs, including the imposition of new clinical holds or delays in resolving existing clinical holds, that may impact our ability to meet our expected timelines and increase our costs; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected which may cause us to use cash more quickly than we expect or change or curtail some of our plans or both; our expectations as to expenses, cash usage and cash needs may prove not to be correct for other reasons such as changes in plans or actual events being different than our assumptions; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be seen in additional patients treated with our product candidates; the risk that additional insertional oncogenic or other reportable events associated with lentiviral vector, drug product, or myeloablation will be discovered or reported over time; the risk that our eli-cel, beti-cel and lovo-cel programs may be subject to further delays in their development, including but not limited to the imposition of new clinical holds; the risk that lovo-cel may not be approved within the priority review timeframe or at all; and the risk that any one or more of our products and product candidates, including eli-cel, beti-cel or lovo-cel, will not be successfully developed, approved or commercialized. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20221130005434/en/

Investors & Media

Investors:

Courtney O’Leary, 978-621-7347

coleary@bluebirdbio.com

Media:

Sarah Alspach, 857-299-6198

sarah.alspach@bluebirdbio.com

Source: bluebird bio, Inc.

FAQ

What is the significance of bluebird bio's sale of the Priority Review Voucher?

The sale brings in $102 million, significantly strengthening bluebird bio's financial outlook.

When were ZYNTEGLO and SKYSONA approved by the FDA?

ZYNTEGLO was approved on August 17, 2022, and SKYSONA was approved on September 16, 2022.

What are bluebird bio's future plans regarding lovo-cel?

The company plans to submit a biologics licensing application for lovo-cel for sickle cell disease.

How does the Priority Review Voucher program work?

The program incentivizes drug development for rare pediatric diseases by awarding vouchers that can expedite review for future marketing applications.

What financial implications does the PRV sale have for bluebird bio?

The $102 million payment from the sale will enhance bluebird bio's cash position without diluting existing shareholders.