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bluebird bio reports second quarter financial results

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Published:10th Aug 2021
bluebird bio, Inc. reported financial results and business highlights for the second quarter ended June 30, 2021 and provided operational updates, including the announcement that the FDA placed a clinical hold on clinical studies of elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy (licensed as Skysona in Europe) for cerebral adrenoleukodystrophy (CALD).

ELI-CEL SAFETY UPDATE : The company received a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS), that is likely mediated by Lenti-D lentiviral vector (LVV) insertion, in a patient who was treated with eli-cel, or Lenti-D drug product for CALD over one year ago in the Phase III ALD-104 study. Evidence currently available suggests that specific design features of Lenti-D LVV likely contributed to this event. The company has shared this information with the independent data monitoring committee of the study and the FDA has placed the eli-cel program on a clinical hold. The company does not anticipate the clinical hold to impact its programs in sickle cell disease (SCD), beta-thalassemia or oncology. Subject to resolution of the clinical hold, the company anticipates completing the submission of the rolling BLA for eli-cel in 2021.

BUSINESS SEPARATION : In January 2021, bluebird announced its intent to separate into two independent, publicly traded companies (bluebird bio and 2seventy bio). The company expects the separation to be completed by the end of 2021 and to be tax-free to bluebird shareholders.

BLUEBIRD BIO BUSINESS UPDATE ; Today, bluebird bio is announcing that the company intends to focus its SGD (severe genetic disease ) business on the U.S. market and on further investments in research and development to optimize its core three programs in SCD, beta-thalassemia and CALD, as well as on the development of a pipeline exploring new disease indications using in vivo LVV technology. The company remains focused and is on track to complete the rolling submissions of the U.S. BLAs in beta-thalassemia in 3Q 2021 and CALD in 2021, pending resolution of the eli-cel clinical hold.

In connection with the planned completion of the business separation in the fourth quarter of 2021 and pivot to U.S.-centric efforts for SGD, bluebird plans an orderly wind down of its operations in Europe and to explore how to give patients in Europe access to its gene therapies, including potentially out-licensing the ex-U.S. rights to its three lead products to a company with European experience and capabilities. bluebird’s decision to focus on the U.S. market is driven by the challenges of achieving appropriate value recognition and market access for Zynteglo in Europe, which makes bringing its transformative gene therapies like Zynteglo and Skysona to patients and physicians in Europe untenable for a small innovative company at this time,” said Andrew Obenshain, president, severe genetic diseases, bluebird bio.

Condition: Cerebral Adrenoleukodystrophy
Type: drug

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