bluebird bio to Present New Data from LentiGlobinTM Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease Clinical Studies at Annual Congress of the European Hematology Association

May 17, 2018 09:00 am
CAMBRIDGE, Mass. -- 

bluebird bio, Inc. (Nasdaq: BLUE) announced that data from ongoing LentiGlobin clinical studies in transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) will be highlighted in oral presentations at the 23rd Congress of the European Hematology Association (EHA). An encore presentation of data from the ongoing Phase 1 study of bb2121 in relapsed/refractory multiple myeloma, to be debuted at the 2018 American Society of Clinical Oncology meeting, will also be presented.

“As we progress towards our European regulatory filing for LentiGlobin in TDT later this year, we look forward to sharing the updated data from our Phase 3 HGB-207 study of LentiGlobin in people with TDT,” said David Davidson, chief medical officer, bluebird bio. “In addition, we are pleased to be able to share new data from our HGB-206 study of LentiGlobin in people with SCD, which will include data from patients treated under our updated study protocol using plerixafor mobilization of hematopoietic stem cells, and with LentiGlobin gene therapy manufactured using the refined process.”

Oral Presentations
LentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β00 Genotypes: Updated Results from Northstar-2 (Abstract S833)

Presenter: Franco Locatelli, M.D., Ospedale Pediatrico Bambino Gesù, Rome, Italy
Date & Time: Saturday, June 16, 11:45 a.m. CEST (5:45 a.m. EST)
Location: Room A8

Recent Progress in Gene Therapy for Severe Sickle Cell Disease: Updated Interim Results from a Phase 1 Clinical Study of LentiGlobin Gene Therapy (Abstract S836)

Presenter: Julie Kanter, M.D., Medical University of South Carolina, Charleston, SC
Date & Time: Saturday, June 16, 2018, 12:30 p.m. CEST (6:30 a.m. EST)
Location: Room A8

BB2121 Anti-BCMA CAR T Cell Therapy in Patients with Relapsed/Refractory Multiple Myeloma: Updated Results from a Multicenter Phase I Study (Abstract S138)

Presenter: Noopur S. Raje, M.D., Massachusetts General Hospital, Boston, Massachusetts
Date & Time: Friday, June 15, 12:30 p.m. CEST (6:30 a.m. EST)
Location: Room A8

Note: This will be an encore of data presented at the American Society of Clinical Oncology (ASCO) 2018 Annual Meeting.

Poster Presentations
Early MRD Negativity Predicts Deepening Myeloma Response in Relapsed/Refractory Multiple Myeloma (RRMM) Patients Treated with BB2121 Anti-BCMA CAR T Cells

Presenter: Nikhil C. Munshi, M.D., Dana-Farber Cancer Institute, Boston, Massachusetts
Date & Time: Friday, June 15, 5:30 – 7:00 p.m. CEST (11:30 a.m. – 1:00 p.m. EST)
Location: Poster Area

High Baseline Ferritin Is Associated with Grade 2 CRS Requiring Tocilizumab or Grade ≥ CRS in Relapsed/Refractory Multiple Myeloma Patients Treated with BB2121 Anti-BCMA CAR T Cells

Presenter: Jesus G. Berdeja, M.D., Sarah Cannon Research Institute, Nashville, Tennessee
Date & Time: Saturday, June 16, 5:30 – 7:00 p.m. CEST (11:30 a.m. – 1:00 p.m. EST)
Location: Poster Area

Conference Call & Webcast Information
bluebird bio will host a conference call and live webcast at 8:00 a.m. EST on Friday, June 15, 2018. To access the live webcast, please visit the "Events & Presentations" page within the Investors and Media section of the bluebird bio website at Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 4678706. A replay of the webcast will be available on the bluebird bio website for 90 days following the call.

About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio's gene therapy clinical programs include Lenti-D for the treatment of cerebral adrenoleukodystrophy, and LentiGlobin for the treatment of transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease. bluebird bio's oncology pipeline is built upon the company's leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio's lead oncology programs, bb2121 and bb21217, are anti-BCMA CAR T programs partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.

bluebird bio has operations in Cambridge, Massachusetts, Seattle, Washington, Durham, North Carolina and Zug, Switzerland.

LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company's research, development, manufacturing and regulatory approval plans for its LentiGlobin product candidate to treat transfusion-dependent ß-thalassemia and severe sickle cell disease and its bb2121 product candidate to treat relapsed/refractory multiple myeloma. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of LentiGlobin will not continue or be repeated in our ongoing, planned or expanded clinical trials of LentiGlobin, the risks that the changes we have made in the LentiGlobin manufacturing process or the HGB-206 clinical trial protocol will not result in improved patient outcomes, risks that the current or planned clinical trials of LentiGlobin will be insufficient to support regulatory submissions or marketing approval in the US and EU, the risk of a delay in the enrollment of patients in our clinical studies, and the risk that any one or more of our product candidates, including our bb2121 product candidate, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

bluebird bio, Inc.
Elizabeth Pingpank, 617-914-8736
Stephanie Fagan, 201-572-9581