bluebird bio, Inc. - BLUE STOCK NEWS

bluebird bio (Nasdaq: BLUE) reported that the recent case of acute myeloid leukemia (AML) in its Phase 1/2 HGB-206 study of LentiGlobin for sickle cell disease is unlikely linked to the BB305 lentiviral vector. Analysis uncovered genetic mutations typical of AML, while vector insertion occurred at a site (VAMP4) not associated with AML risks. Regulatory discussions with the FDA are ongoing to lift clinical holds on relevant studies. The company also reported a separate SUSAR of myelodysplastic syndrome (MDS) under investigation but found no malignancy linked to ZYNTEGLO’s use.

bluebird bio (NASDAQ: BLUE) will participate in the Cowen 41^st Annual Health Care Conference on March 3, at 2:40 p.m. ET. A live webcast of the presentation can be accessed via the Investors & Media section of their website. The replay will be available for 90 days post-event. bluebird bio focuses on developing gene therapies for severe genetic diseases and cancer, aiming to provide treatment options for life-threatening conditions. The company operates from multiple locations, including Cambridge, MA, and Zug, Switzerland.

bluebird bio, Inc. (Nasdaq: BLUE) and Bristol Myers Squibb's investigational CAR T therapy, idecabtagene vicleucel (ide-cel), has shown promising results in a pivotal Phase 2 KarMMa study for relapsed and refractory multiple myeloma. The study, published in The New England Journal of Medicine, met its primary endpoint for overall response rate and secondary endpoint for complete response rate. Of the 128 patients treated, meaningful responses were observed in high-risk subgroups. The therapy's safety profile was consistent with CAR T treatments, with cytopenia and cytokine release syndrome being the most common adverse events.

bluebird bio reported its financial results for Q4 and full year 2020, noting a significant revenue increase from $44.7 million in 2019 to $250.7 million in 2020. Despite a net loss reduction to $618.7 million from $789.6 million, the company faces challenges, including a clinical hold on LentiGlobin studies due to safety events. Upcoming milestones include the PDUFA action date for ide-cel on March 27, 2021, and potential EU approval for eli-cel mid-year. The company is separating its severe genetic disease and oncology ventures by the end of 2021.

bluebird bio (NASDAQ: BLUE) will participate in the 10th Annual SVB Leerink Global Healthcare Conference on February 25 at 12:00 p.m. ET. A live webcast of the event can be accessed on the company’s website, with a replay available for 90 days post-event. bluebird bio specializes in developing gene therapies for severe genetic diseases and cancer, aiming to provide treatments for conditions with limited options. The company operates from multiple locations including Cambridge, Seattle, Durham, and Zug.

bluebird bio (Nasdaq: BLUE) announced a temporary suspension of its Phase 1/2 (HGB-206) and Phase 3 (HGB-210) studies for LentiGlobin gene therapy for sickle cell disease due to a reported serious adverse reaction—a case of acute myeloid leukemia (AML) in a patient treated over five years ago. This investigation is critical in assessing the safety of their therapies, including ZYNTEGLO, which is manufactured utilizing the same BB305 lentiviral vector. The company is actively working with regulatory bodies and has notified their independent safety review board.

bluebird bio (Nasdaq: BLUE) plans to separate its severe genetic disease and oncology businesses into independent companies, maintaining a focus on severe genetic diseases. This tax-free spin-off, called Oncology Newco, is expected to enhance operational efficiency and attract suitable investors. Post-separation, current CEO Nick Leschly will lead Oncology Newco, while Andrew Obenshain will continue as CEO for bluebird bio. The separation aims to optimize innovation and financial performance for both entities, with an anticipated completion by Q4 2021.

bluebird bio (Nasdaq: BLUE) announced promising new data from Group C of its Phase 1/2 HGB-206 study of LentiGlobin gene therapy for sickle cell disease (SCD). Results show a complete elimination of severe vaso-occlusive events (VOEs) between six and 24 months, with sustained levels of the anti-sickling hemoglobin HbA in treated patients. Key findings presented at the American Society of Hematology (ASH) Annual Meeting include improved health-related quality of life and a significant reduction in patient-reported pain, underscoring LentiGlobin's potential as a disease-modifying treatment.

bluebird bio (Nasdaq: BLUE) presented long-term efficacy and safety results for betibeglogene autotemcel gene therapy (beti-cel) for transfusion-dependent β-thalassemia at the 62nd ASH Annual Meeting. Data revealed that 87% of pediatric patients and 64%-90% of adults achieved transfusion independence, with median post-infusion follow-up of 49.1 months. The European Commission granted conditional marketing authorization for beti-cel, while it remains under evaluation in the U.S. No serious adverse events were linked to the therapy beyond two years.