Business Wire

bluebird bio Presents New Data for LentiGlobin Gene Therapy in Transfusion Dependent β-Thalassemia at 60th Annual Meeting of the American Society of Hematology

Del

bluebird bio, Inc. (Nasdaq: BLUE) announced new data from the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) clinical studies of its investigational LentiGlobin gene therapy in the treatment of patients with transfusion-dependent β-thalassemia (TDT) at the 60th Annual Meeting of the American Society of Hematology (ASH).

“Our new data for LentiGlobin in transfusion-dependent β-thalassemia includes a broader population of patients with both non-β00 and β00 genotypes, as well as our first pediatric patients. In all of these patients we observed improved hemoglobin levels and reduced or eliminated requirements for blood transfusions following treatment with LentiGlobin,” said David Davidson, M.D., chief medical officer, bluebird bio. “As our clinical studies advance, we continue to gain insight into the therapeutic potential of LentiGlobin across the spectrum of patients affected by TDT.”

TDT is an inherited blood disorder caused by a mutation in the β-globin gene, which causes ineffective red blood cell production leading to severe anemia. People with TDT require regular transfusions to maintain hemoglobin (Hb) levels in order to survive, but chronic transfusions carry risks, including iron overload that can result in multi-organ damage and shortened life expectancy.

“In my practice, I see the serious complications of transfusion-dependent β-thalassemia and the everyday toll this disease takes on my patients and their families,” said Professor Franco Locatelli, M.D., Ph.D., Full Professor of Pediatrics at the Sapienza University in Rome, Chair of the Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy and lead investigator for the Northstar-2 study. “Patients with transfusion-dependent β-thalassemia are not able to make enough hemoglobin to survive, which is why they need regular blood transfusions every two to four weeks. After treatment with LentiGlobin, patients in this Phase 3 study began to produce gene-therapy derived hemoglobin and near-normal hemoglobin levels, which in the majority of patients abrogated the need for blood transfusions.”

Northstar-2 (HGB-207) Efficacy
After treatment with LentiGlobin, patients are monitored for production of HbAT87Q, which is gene therapy derived-hemoglobin. The production of HbAT87Q increases the overall hemoglobin level in patients with the goal of reducing or eliminating the need for transfusions.

Sixteen patients with non-β00 genotypes (aged 8 – 34 years); two pediatric and 14 adolescents/adults with TDT have been treated in the Phase 3 Northstar-2 study as of September 14, 2018, the data cut-off date.

Eleven of these patients had at least three months of follow-up available at the data cut-off. Ten of the 11 patients had stopped receiving transfusions and had hemoglobin levels of 11.1 – 13.3 g/dL at the time of the last study visit (3 – 18 months post-treatment). HbAT87Q levels in these 10 patients ranged from 7.7 – 10.6 g/dL and significantly contributed to total hemoglobin (67 – 92 percent).

An exploratory analysis was conducted with bone marrow from six patients with 12 months of follow-up after treatment. The samples were evaluated for cellularity and myeloid to erythroid ratio. A low myeloid to erythroid ratio is a key feature of dyserythropoesis, or abnormal bone marrow red blood cell production, characteristic of patients with TDT. In five patients, all of who had stopped chronic transfusions, an increase in the myeloid to erythroid ratio was observed, suggesting improvement in red blood cell production.

Northstar-3 (HGB-212) Efficacy
As of September 14, 2018, three patients with TDT and a β00 genotype or an IVS-I-110 mutation had been treated with LentiGlobin in the Phase 3 Northstar-3 study.

All three patients, as of November 19, 2018, had total hemoglobin of greater than 10 g/dL at their last assessment, including a pediatric patient. Patient 1 had no transfusions following LentiGlobin treatment and their last assessment at month 12, Patient 2 had their last transfusion 1.9 months post-treatment and last assessment at month six, Patient 3 had their last transfusion at 1.4 months post-treatment and last assessment at month three.

Northstar-2 and Northstar-3 Safety
In the Northstar-2 and Northstar-3 studies the safety profile of LentiGlobin gene therapy remained generally consistent with myeloablative busulfan conditioning, including serious adverse events (SAEs) of vaso-occlusive liver disease. One SAE of grade 3 thrombocytopenia was reported and considered possibly related to LentiGlobin.

As of the data cut-off date, September 14, 2018, a total of 37 pediatric, adolescents and adult patients with TDT and a non-β00 or β00 genotype, including patients with IVS-I-110 mutations, have been treated with LentiGlobin in the Northstar, Northstar-2 and Northstar-3 studies.

For more information about the ongoing clinical studies of LentiGlobin in TDT visit www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207) and NCT03207009 for Northstar-3 (HGB-212).

About Transfusion-Dependent β-Thalassemia
TDT is an inherited blood disorder caused by a mutation in the β-globin gene, which causes ineffective red blood cell production leading to severe anemia. Supportive care for people with TDT consists of a lifelong regimen of chronic blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions.

Despite the availability of supportive care, many people with TDT experience serious complications and organ damage due to underlying disease and iron overload. By eliminating or reducing the need for blood transfusions, the long-term complications associated with TDT may be reduced.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been successfully used to treat TDT and is currently the only available option with the potential to correct the genetic deficiency in TDT. Complications of allo-HSCT include a risk of treatment-related mortality, graft failure, graft-versus-host disease (GvHD) and opportunistic infections, particularly in patients who undergo non-sibling matched allo-HSCT.

About LentiGlobin
LentiGlobin is a one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of TDT, which could eliminate or reduce the need for blood transfusions.

bluebird bio’s clinical development program for LentiGlobin includes ongoing studies around the world with sites in Australia, Germany, Greece, France, Italy, Thailand, the United Kingdom and the United States. For more information visit: www.northstarclinicalstudies.com or clinicaltrials.gov using identifier NCT01745120.

In addition, bluebird 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 sickle cell disease.

In October 2018, the European Medicines Agency (EMA) accepted the company’s marketing authorization application (MAA) for LentiGlobin™ gene therapy for the treatment of adolescents and adults with TDT and a non-β00 genotype.

The EMA previously granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT. LentiGlobin is 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 (FDA) also granted LentiGlobin Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built a pipeline with broad potential application in severe genetic diseases and cancer.

bluebird bio's gene therapy clinical programs include investigational treatments for cerebral adrenoleukodystrophy, transfusion-dependent β-thalassemia and sickle cell disease.

bluebird bio's oncology pipeline is built upon the company's 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. The company’s lead oncology programs are anti-BCMA CAR T programs partnered with Celgene.

bluebird bio’s discovery research programs include 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. For more information, visit bluebirdbio.com.

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

LentiGlobin is a trademark 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 views with respect to the potential for its LentiGlobin product candidate to treat transfusion-dependent ß-thalassemia, and the Company’s expectations regarding the review, potential regulatory approval and potential commercial launch of its LentiGlobin product candidate in the United States and Europe. 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 or planned clinical trials of LentiGlobin, the risks that the changes we have made in the LentiGlobin manufacturing will not result in improved patient outcomes, risks that the current or planned clinical trials of LentiGlobin will be insufficient to support future regulatory submissions or to support marketing approval in the US and EU, and the risk that any one or more of our product candidates, 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.

Contact information

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

Media:
Catherine Falcetti, 617-583-3411
cfalcetti@bluebirdbio.com

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Følg saker fra Business Wire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Business Wire

Ras Al Khaimah Marks New Year’s Eve with Amazing Fireworks and New Guinness World Record Bid17.12.2018 11:14Pressemelding

This New Year’s Eve, Ras Al Khaimah is the place to be as the Emirate readies to host the most spectacular celebration with a 12-minute fireworks display - the bid for a new Guinness World Record display - and an array of family-oriented activities. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181217005309/en/ Marjan, Ras Al Khaimah’s master-developer of freehold projects, will manage the show to mark 2019 with a glittering array of activities that will entertain all at its flagship development, Al Marjan Island. With three additional viewing areas around Al Marjan Island, New Year’s Eve in Ras Al Khaimah has fun and thrills in store for local and international visitors. Abdulla Al Abdouli, Managing Director & CEO of Marjan, said: “This year, we are offering exciting family activities that will delight all in the run-up to the amazing fireworks display that aims to set a new Guinness World Record. The event will position t

Mobidiag Announces Joint Venture with Autobio Diagnostics and €10m Equity Investment17.12.2018 09:52Pressemelding

Mobidiag Ltd., a commercial stage molecular diagnostics company addressing the spread of antimicrobial resistance, today announces that it has entered into a joint venture (‘JV’) agreement with Autobio Diagnostics (‘Autobio’), one of the leading clinical diagnostics companies in China, to commercialise the Novodiag® platform in China. In addition, Mobidiag announces that it has secured a separate €10m equity investment from Autobio. Lynx Financial (HK) Ltd, served as the exclusive financial advisor to Mobidiag in this transaction. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181217005272/en/ Mobidiag and Autobio will jointly invest €12.3m to establish a joint venture company in China. Autobio will invest €8m in cash to hold 65%, and Mobidiag will invest €4.3m to hold 35%. Mobidiag will grant to the JV an exclusive license for human infectious disease assays (except for sepsis) for China. Initial activities of the JV will i

Algorand Appoints Pablo Azar as Chief Economist17.12.2018 09:00Pressemelding

Algorand, a foundational blockchain and cryptocurrency company, announced today that it has named Pablo Azar as Chief Economist. Given Algorand's focus on creating sustainable value in a decentralized economy, Mr. Azar will be responsible for guiding Algorand’s economic policies. On the heels of announcing Steve Kokinos and W. Sean Ford as CEO and COO, respectively, Mr. Azar’s appointment as Chief Economist is another advancement for Algorand as it continues to develop its executive team. With a double Ph.D. from MIT, Mr. Azar will bring his unique expertise regarding the application of computer science and cryptography toward the creation of better market mechanisms. Further, he will be responsible for expanding the visibility of Algorand’s economic policy and enhancing the tools necessary for developers to build on the Algorand platform. “As momentum around tokenization increases, so does the need for sound economic management,” said Steve Kokinos, CEO at Algorand. “With his deep bac

Prima Solutions and Effisoft Announce Their Merger to Bolster Global Leadership in Providing Insurance Solutions17.12.2018 09:00Pressemelding

As part of their continuing growth strategies, Prima Solutions and Effisoft announce their merger to create a global InsureTech company. This merger between Effisoft, the leading vendor of reinsurance and regulatory software solutions for insurance and reinsurance companies across the globe, and Prima Solutions, one of Europe’s leading software provider for Life, Health, and P&C insurance companies, will produce one of the largest and most comprehensive players in Europe, North America, and the Asia-Pacific region with cloud-based solutions covering all areas of the insurance market. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181217005249/en/ This merger, concluded on December 13, will enable both groups to accelerate their development in the P&C, health and personal protection insurance markets. This merger is part of an ambitious and coherent growth strategy: it completes Prima Solutions' and Effisoft's existing produc

Arthur D. Little Report Outlines €200 Billion Digital Transformation Opportunity for Telecoms Sector17.12.2018 08:30Pressemelding

Arthur D. Little (ADL) today published a new research report highlighting the need for greater digitalization in the global telecoms industry. The TIME 2018 Flagship Report outlines the scale of the digital opportunity, with analysis estimating that a ‘digital dividend’ of over €200 billion free cash flow awaits telcos that can successfully transform. Industry executives surveyed for the report confirm the findings. 73% say that digital will add value to their businesses, with key benefits including greater agility, increased subscriber growth and cost savings. CAPEX spending on areas such as 5G infrastructure and the Internet of Things is expected to require a 7% CAGR increase from 2017-2022, outpacing forecast revenue growth. Consequently, failing to digitally transform risks commoditization and disintermediation, leaving telcos unable to invest sufficiently. As one senior executive put it bluntly, “Digitalization – there is no other choice if we want to survive.” The report highligh

Denodo Offers Data Virtualization Test Drive on Microsoft Azure17.12.2018 08:30Pressemelding

Denodo, the leader in data virtualization today announced the availability of a free Test Drive environment on Microsoft Azure. Following on the heels of its recently announced Test Drive on AWS, the newest Denodo Test Drive empowers users to quickly and easily create real-time data delivery without replication and explore the benefits of using the Denodo Platform on Azure. Denodo Test Drives are completely free of charge for demonstration, education and evaluation purposes and are available at: http://bit.ly/2AQddE3 Built on Azure, the Test Drive lets individuals discover, explore, prepare, and access data in a user-friendly interface for true self-service. The Denodo Test Drive solves some of the most pressing challenges faced by data scientists and data analysts by enabling them to integrate large volumes of data to support their agile BI, big data analytics and Web/cloud integration objectives. “Microsoft’s commitment to offer an ecosystem of cloud solutions makes it ideal for Deno