bluebird bio, Inc. - BLUE STOCK NEWS

bluebird bio has received FDA approval for ZYNTEGLO® (betibeglogene autotemcel), the first gene therapy for beta-thalassemia patients requiring regular blood transfusions. Priced at $2.8M, ZYNTEGLO aims to eliminate the need for ongoing transfusions, potentially saving patients up to $6.4M in lifetime healthcare costs. An innovative outcomes-based contract allows for a single upfront payment with up to 80% reimbursement if transfusion independence is not achieved. ZYNTEGLO will be available through specialized Qualified Treatment Centers, supported by the 'my bluebird support' program for patients and families.

bluebird bio has received FDA approval for ZYNTEGLO (betibeglogene autotemcel), a one-time gene therapy for treating beta-thalassemia in patients needing regular red blood cell transfusions. This therapy aims to provide transfusion independence and improve hemoglobin levels. Clinical data from Phase 3 studies showed that 89% of evaluable patients achieved transfusion independence, with durable results lasting up to 4 years. ZYNTEGLO is expected to revolutionize treatment for this severe, genetic disease affecting approximately 1,300-1,500 patients in the U.S.

bluebird bio reported significant advancements in gene therapy during Q2 2022, including unanimous FDA advisory committee support for beti-cel and eli-cel. If approved, both therapies could launch in Q4 2022. The Company ended the quarter with approximately $218 million in cash equivalents. Total revenue increased to $1.5 million from $0.1 million YoY, while net loss narrowed to $100.1 million from $155.8 million. The BLA for lovo-cel is anticipated in Q1 2023. bluebird bio aims to resolve clinical holds and is exploring financing options, including equity offerings and monetizing potential FDA vouchers.

bluebird bio announced that the FDA's advisory committee voted 13-0, endorsing betibeglogene autotemcel (beti-cel) as a viable treatment option for beta-thalassemia patients requiring regular red blood cell transfusions. If approved, beti-cel would be the first curative gene therapy for this condition in the U.S. The FDA has set a decision goal date for the Biologics License Application on August 19, 2022. With significant clinical data backing beti-cel, 89% of patients in trials achieved transfusion independence. This is a crucial step toward addressing the unmet medical needs of beta-thalassemia patients.

bluebird bio announced positive recommendations from the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee for their gene therapy eli-cel, aimed at treating early active cerebral adrenoleukodystrophy (CALD) in children. The committee voted 15-0 in favor of the therapy's benefits outweighing the risks. The Biologics License Application (BLA) is under priority review, with a PDUFA goal date of September 16, 2022. If approved, eli-cel will be the first gene therapy for CALD, providing an alternative for over 70% of CALD patients lacking matched sibling donors.

bluebird bio announced a halt in trading of its common stock on June 9 and 10, 2022, as the FDA's Advisory Committee reviews biologics licensing applications (BLAs) for beti-cel and eli-cel. Beti-cel targets β-thalassemia patients needing regular blood transfusions, while eli-cel addresses early active cerebral adrenoleukodystrophy in children without a matched sibling donor. PDUFA goal dates for beti-cel and eli-cel are August 19 and September 16, 2022, respectively. The company aims for FDA approval to further its curative gene therapies.

bluebird bio announced that the FDA has posted briefing documents for its advisory committee meeting regarding two gene therapies: elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) and betibeglogene autotemcel (beti-cel) for β-thalassemia. The meeting is scheduled for June 9-10, 2022. The PDUFA goal dates for decisions on the therapies are August 19, 2022 and September 16, 2022, respectively. Past studies showed high efficacy rates for both therapies, but clinical trials face ongoing FDA scrutiny over potential adverse effects.

bluebird bio reported Q1 2022 financial results, ending with approximately $312 million in cash and equivalents. The company plans to reduce operating costs by 35-40% by year-end 2022 through restructuring, which includes a 30% workforce cut.

Key milestones include an FDA advisory committee meeting for beti-cel and eli-cel on June 9-10, 2022, and anticipated BLA submissions for lovo-cel in Q1 2023. Revenue rose to $1.9 million in Q1 2022 from $0.9 million in Q1 2021, while net loss increased marginally to $122.2 million.

bluebird bio (Nasdaq: BLUE) initiates a comprehensive restructuring aimed at achieving up to $160 million in cost savings over the next two years. The company anticipates a cash burn reduction to less than $340 million in 2022 and plans a 30% workforce reduction. This initiative is expected to extend bluebird's cash runway into the first half of 2023 while maintaining focus on FDA approvals for gene therapies for beta-thalassemia and cerebral adrenoleukodystrophy. Key PDUFA dates are set for August 19 and September 16, 2022.