bluebird bio (BLUE) Investor Presentation - Slideshow

Pipeline and Approvals - Bluebird bio anticipates 3 approvals by the end of 2023, all with wholly-owned global rights[5] - ZYNTEGLO® (beti-cel) received FDA approval on August 17, 2022, for beta-thalassemia requiring regular red blood cell transfusions, with commercial launch in Q4 2022[6] - The FDA Advisory Committee unanimously endorsed eli-cel (15-0) for treatment of early active CALD[6, 36] - A BLA submission for lovo-cel for sickle cell disease is planned for Q1 2023[6, 33, 34] Clinical Data and Patient Impact - In Phase 3 studies for ZYNTEGLO®, 89% of patients achieved transfusion independence (TI) and normal or near-normal hemoglobin levels[13] - Eli-cel maintained an estimated event-free survival rate of 86.8% (95% CI: 72.7%, 93.9%) through 7 years of follow-up[39] - Lovo-cel demonstrated complete resolution of severe vaso-occlusive events (VOEs) through up to 36 months of follow-up in evaluable patients[32] Commercial Strategy and Market Opportunity - ZYNTEGLO® is priced at $2.8 million with an outcomes-based agreement offering up to 80% rebate if transfusion independence is not achieved within 2 years[17, 18] - The company estimates that 55-60% of the ~1,500 patients with transfusion-dependent beta-thalassemia in the US may be eligible for ZYNTEGLO®[16] - The company plans to expand to ~50 Qualified Treatment Centers (QTCs) by year-end 2023 in anticipation of SCD launch[15] - Over 20,000 SCD patients in the US may be addressed by lovo-cel gene therapy[26, 27] Financial Status - The company's current cash runway extends into 1H23, with $218 million cash on hand as of June 30, 2022[41] - The company is targeting $60 million per quarter net cash burn by year-end 2022 and carry into 2023[41]