Benzinga | Apr 20, 2021 07:49AM EDT

bluebird bio Says Investigator Has Concluded Reported Case Of MDS In LentiGlobin Study Is Not MDS And Has Revised Diagnosis To Transfusion-Dependent Anemia

bluebird bio, Inc. (NASDAQ:BLUE) announced today business and program updates across its severe genetic disease portfolio including a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of LentiGlobin for sickle cell disease (SCD) (bb1111), the company's decision to withdraw ZYNTEGLO(tm) (betibeglogene autotemcel, beti-cel) for transfusion-dependent ?-thalassemia (TDT) from the German market and a targeted reshaping of its workforce intended to enable the company to advance its late-stage gene therapy programs.

The case of MDS reported in February in a patient from Group C of the Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD has been further assessed following the review of results from additional tests. The treating investigator has concluded this is not a case of MDS and has revised the diagnosis to transfusion-dependent anemia. bluebird bio has reported this update to regulatory agencies and study investigators. The company continues to work with the treating investigator to determine the potential cause of this patient's anemia.

Last month, the company reported that it is very unlikely the suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML) reported in the HGB-206 study of LentiGlobin for SCD was related to the BB305 lentiviral vector (LVV). This assessment, along with the re-classification of the originally reported MDS case to transfusion-dependent anemia are important steps in bluebird bio's path to seeking removal of the clinical hold on studies HGB-206 and HGB-210 of LentiGlobin for SCD. bluebird bio continues to work with regulators to resume its clinical studies in sickle cell disease as well as to remove the clinical hold for HGB-207 and HGB-212 clinical studies of beti-cel for ?-thalassemia, with potential lift of all clinical holds in mid-2021.

In Europe, reimbursement negotiations in Germany did not result in a price for ZYNTEGLO that reflects the value of this one-time gene therapy with potential life-long benefit for people living with TDT. The price proposed by the German health authorities fails to recognize the severe burden of living with TDT or the innovation and benefit ZYNTEGLO brings to patients who are impacted every day, throughout their lives by this severe genetic disease.

bluebird bio continues with productive negotiations across countries in Europe and we plan to continue to provide updates on the negotiation processes in the second half of 2021.

In response to these and other events and shifts related to the business over the past year, bluebird bio plans to reduce and reshape its workforce, primarily in Europe. This reduction and reallocation of resources will allow the company to focus on priority European markets and streamline global operations going forward to ensure its ability to deliver gene therapies to patients based on bluebird bio's current business plans.

"We remain committed to our pioneering mission to deliver one-time gene therapies with life-long benefits to our patients. We are grateful for the clinical investigators and healthcare providers helping us better understand the recent safety events in our sickle cell disease studies. We are confident that working with the FDA and EMA, we will be able to determine a positive path forward as we seek to re-open our clinical studies. Further, through our continued engagement across Europe, we are optimistic that countries will reach pricing decisions that recognize the value of one-time gene therapies and provide the necessary access to the people who need them," said Andrew Obenshain, president, severe genetic diseases, bluebird bio. "In terms of operations, we have faced challenges over the last year that have resulted in the difficult decision to reduce our workforce and say goodbye to some valued bluebirds. We want to express our gratitude for their contributions and commitment to patients. As we move into the future, we look forward to bluebird bio advancing as a strong, thriving organization that is dedicated to developing treatments for rare genetic diseases."