bluebird bio to Present New Data from Clinical Studies of LentiGlobin™ Gene Therapy for Transfusion-Dependent β-thalassemia (TDT) and LentiGlobin Gene Therapy for Sickle Cell Disease (SCD) at the 24th EHA Congress

bluebird bio, Inc. (Nasdaq: BLUE) announced today that new data from its investigational gene therapy programs for transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) will be presented during the 24th European Hematology Association (EHA) Congress in Amsterdam, the Netherlands, June 13-16.

bluebird bio will present data from its clinical studies of LentiGlobin™ gene therapy for TDT including updated results up to 54 months from the long-term follow-up period of the completed Phase 1/2 Northstar (HGB-204) study. The company will also present new data from the ongoing Phase 3 Northstar-2 (HGB-207) study in patients who do not have a β⁰/β⁰ genotype and from the ongoing Phase 3 Northstar-3 (HGB-212) study in patients who have β⁰/β⁰ genotype or an IVS-I-110 mutation.

New data from the company’s Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will include additional patients treated in the study and updated data for those previously reported.

Oral Presentations: Transfusion-Dependent β-Thalassemia

Results from the Phase 3 Northstar-3 Study Evaluating LentiGlobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassaemia and a β ⁰ or IVS-I-110 Mutation at Both Alleles of the HBB Gene (HGB-212)
Presenting Author: Andreas Kulozik, M.D., Ph.D., University Hospital Heidelberg, Heidelberg, Germany
Date & Time: Friday, June 14, 2019, 11:30 – 11:45 a.m. CEST (5:30 – 5:45 a.m. EDT)

Clinical Outcomes of LentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassaemia (TDT) Following Completion of the Northstar (HGB-204) Study
Presenting Author: Mark Walters, M.D., Benioff Children’s Hospital, Oakland, Calif., U.S.
Date & Time: Friday, June 14, 2019, 11:45 a.m. – 12:00 p.m. CEST (5:45 – 6:00 a.m. EDT)

Safety and Efficacy of LentiGlobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassaemia and Non-β ⁰ /β ⁰ Genotypes in the Phase 3 Northstar-2 Study (HGB-207)
Presenting Author: Franco Locatelli, M.D., Ph.D. University of Pavia, Pavia, Lombardy, Italy
Date & Time: Sunday, June 16, 2019, 8:00 – 8:15 a.m. CEST (2:00 – 2:15 a.m. EDT)

Oral Presentation: Sickle Cell Disease

Updated Results from the HGB-206 Study in Patients with Severe Sickle Cell Disease Treated Under a Revised Protocol with LentiGlobin Gene Therapy Using Plerixafor-Mobilised Haematopoietic Stem Cells (HGB-206)
Presenting Author: Julie Kanter, M.D., Division of Hematology and Oncology, University of Alabama at Birmingham, Birmingham, Ala., U.S.
Date & Time: Sunday, June 16, 2019, 8:15 – 8:30 a.m. CEST (2:15 – 2:30 a.m. EDT)

Abstracts outlining bluebird bio’s accepted data at EHA have been made available on the EHA conference website.

Investor Event & Webcast Information

bluebird bio will host a conference call and live webcast at 8:00 a.m. EDT Friday, June 14, 2019. To access the webcast, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at http://investor.bluebirdbio.com/. A replay of the webcast will be available on the bluebird bio website for 90 days following the call.

About LentiGlobin for Transfusion-Dependent β-Thalassemia

In March 2019, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization for LentiGlobin gene therapy for TDT, which if approved will be commercialized as ZYNTEGLO™ (autologous CD34+ cells encoding β^A-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β⁰/β⁰ genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. ZYNTEGLO adds functional copies of a modified form of the β-globin gene (β^A-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the β^A-T87Q-globin gene they have the potential to produce HbA^T87Q, which is gene therapy- derived-hemoglobin, at levels that significantly reduce or eliminate the need for transfusions.

Non-serious adverse events (AEs) observed during clinical trials that were attributed to ZYNTEGLO were hot flush, dyspnoea, abdominal pain, pain in extremities and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to ZYNTEGLO.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan including SAEs of veno-occlusive disease.

ZYNTEGLO continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies and the long-term follow-up study LTF-303. If approved, ZYNTEGLO will be the first gene therapy to treat TDT.

About LentiGlobin for Sickle Cell Disease

LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment to address the underlying genetic cause of SCD. bluebird bio’s clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT and LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/.

The European Medicines Association (EMA) granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT. LentiGlobin for TDT was also part of the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines.

The U.S. Food and Drug Administration granted Orphan Drug status and Breakthrough Therapy designation to LentiGlobin for TDT; as well as Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, we’re developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, we’re working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO and LentiGlobin are trademarks of bluebird bio.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the β^A-T87Q-globin gene.

Forward-Looking Statements

This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. 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 risk that our MAA submitted for LentiGlobin for TDT may not be approved by the European Commission when expected, or at all; the risk that the efficacy and safety results from our prior and ongoing clinical trials of LentiGlobin for TDT will not continue or be repeated in our ongoing or planned clinical trials of LentiGlobin for TDT; the risk that the current or planned clinical trials of LentiGlobin for TDT will be insufficient to support regulatory submissions or marketing approval in the US and EU; the risk that the production of Hb ^A-T87Q may not be sustained over extended periods of time; and the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of LentiGlobin for TDT following regulatory approval. 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.

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Contact information

bluebird bio
Investors:
Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com

or

Media:
Catherine Falcetti, 339-499-9436
cfalcetti@bluebirdbio.com