bluebird bio BLUE is a clinical-stage biotech company focused on developing gene therapies for severe genetic diseases.
The company has developed a robust pipeline using its lentiviral gene addition platform. bluebird’s gene therapy pipeline includes candidates for treating transfusion-dependent β-thalassemia ("TDT"), sickle cell disease ("SCD") and cerebral adrenoleukodystrophy ("CALD").
bluebird bio had earned marketing authorizations in Europe for two of its gene-editing therapies, betibeglogene autotemcel (beti-cel, formerly LentiGlobin for β-thalassemia gene therapy), and elivaldogene autotemcel (eli-cel, formerly Lenti-D gene therapy). Beti-cel is used as a treatment for β-thalassemia and eli-cel is used for treating CALD. Last year, the company decided to exit the European market after it failed to reach favorable third-party payers for these treatments.
The company is now focusing on its business in the U.S. market. Presently, bluebird does not have any products in the U.S. market that are anticipated to change soon.
bluebird completed the submission of the rolling biologics license application (BLA) with the FDA for beti-cel to treat TDT in September 2021. The BLA has been accepted for priority review by the FDA. In June, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted unanimously (13-0) that the benefits of beti-cel gene therapy outweigh the risks for patients with beta-thalassemia who require regular red blood cell transfusions. The FDA decision for the same is expected by Aug 19, 2022.
The BLA for eli-cel as a treatment for patients less than 18 years of age with early CALD was accepted for priority review by the FDA. Moreover, recently CTGTAC voted unanimously (15-0) that the benefits of eli-cel gene therapy outweigh the risks for treating any sub-population of children with early active CALD. The PDUFA goal date for the BLA is September 16, 2022.
A potential approval will be a significant boost for the company.
However, earlier this year, the company announced that it will initiate a comprehensive restructuring to save up to $160 million in costs over the next two years. At the same time, BLUE is set to extend the existing cash runway into first-half 2023.
To achieve the aforementioned target, the company intends to reduce its workforce by approximately 30%. The reduction in workforce is expected to reduce bluebird’s 2022 cash burn to less than $340 million, resulting in 35-40% savings in estimated operating costs. This reduction is anticipated to be reflected in BLUE’s operating budget for 2023.
Moreover, in November last year, bluebird completed a spin-off of its oncology programs and portfolio into an independent, publicly-traded company, 2seventy bio, Inc. TSVT, transferring Bristol Myers BMY Abecma to 2seventy bio in the process. The separation was intended to help bluebird focus extensively on its gene therapy pipeline for severe genetic diseases.
BMY’s Abecma received FDA approval in March 2021 for the treatment of adult patients with relapsed or refractory multiple myeloma.
Bluebird also faces stiff competition from Acceleron Pharma’s FDA-approved Reblozyl (luspatercept), developed with BMY, for treating anemia in adult patients with beta thalassemia who require RBC transfusions. Acceleron Pharma was acquired by Merck MRK in 2021.
Apart from the United States, Reblozyl is also approved in Europe, Canada and Australia to treat anemia in certain rare blood disorders. It is also being evaluated for additional indications for hematology therapies.
Though BMY is the principal on the sales transactions for Reblozyl, Merck co-promotes Reblozyl in North America and is eligible to receive future contingent sales-based milestone payments of up to $80 million from Reblozyl.
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