JPM23: Bluebird touts gene therapy launch progress, extends cash runway to 2024

Before bluebird bio nabbed a pair of gene therapy FDA approvals for last summer, the company’s future looked uncertain. Entering 2023, the gene therapy specialist appears to be standing on stronger ground. 

In a corporate update Monday, bluebird said it has treated the first commercial patient with beta thalassemia gene therapy Zynteglo. The company says it’s seeing “significant patient demand” for the newly approved therapy and expects to record the first sales from the product in the first quarter of this year. 

Zynteglo won an FDA approval last August to treat patients with beta thalassemia who require regular red blood cell transfusions. As a one-time gene therapy, the drug carries a multimillion-dollar price tag. In a bid to stand by the med’s real-world results, bluebird is offering outcomes-based pricing to payers. 

On that front, bluebird on Monday said the three largest pharmacy benefit managers in the U.S. have inked outcomes-based agreements for the therapy. The company says 190 million patient lives in the U.S. are covered under “favorable” policies for the medicine. 

Meanwhile, bluebird has activated 10 gene therapy treatment centers and is onboarding another 15, the company said. By the end of the year, the company expects to have 40 to 50 treatment centers in its nationwide network. 

As for Skysona, the company’s cerebral adrenoleukodystrophy (CALD) gene therapy, bluebird said cell collection for the first patient is scheduled for this month. That drug won an FDA approval last September and boasts a $3 million price tag

With the launches underway, bluebird expects to burn between $270 million and $300 million in cash this year. Thanks to the company's recent sales of priority review vouchers that netted nearly $200 million, plus other assets on hand, bluebird figures it can operate at least into the first quarter of 2024. 

That outlook marks an improvement from last year, when the company warned that its cash needs “raise substantial doubt regarding its ability to continue as a going concern." Previously, the company had to pull out of the European market over a pricing dispute with officials there, and its U.S. commercial outlook wasn’t always a sure bet before the FDA nods. 

Aside from its approved drugs, the company also sees a big opportunity in its lovo-cel candidate for sickle cell disease. The drugmaker expects to submit the candidate to the FDA in the first quarter of 2023 after completing vector and drug product analytical comparability studies late last year.   

The update comes as the J.P. Morgan Healthcare Conference kicks off in San Francisco. Bluebird CEO Andrew Obenshain is scheduled to speak at the conference on Thursday, but the company likely didn’t want the first day of the conference to pass without touting its recent progress.