Business Wire

bluebird bio Announces Early Data from Phase 3 Northstar-2 (HGB-207) Study of LentiGlobinTM Drug Product at European Hematology Association (EHA) Annual Meeting

Jaa

bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, announced early interim data from the ongoing Northstar-2 (HGB-207) Phase 3 clinical study of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia (TDT) and non-β00 genotypes. These data will be presented by Mark Walters, M.D., UCSF Benioff Children’s Hospital, Oakland, California, in an oral session on Sunday, June 25 at the European Hematology Association (EHA) Annual Meeting in Madrid, Spain.

“Northstar-2 is our first study to utilize our improved LentiGlobin drug product manufacturing process to increase the drug product vector copy number and percent of cells transduced. The first patient treated in this study exemplifies the promise of gene therapy, discontinuing blood transfusions approximately a month after treatment and achieving a normal level of total hemoglobin production at six months post-treatment,” said David Davidson, M.D., chief medical officer, bluebird bio. “These early results suggest that the improved manufacturing process results in consistently higher drug product vector copy numbers (VCN) and lentiviral vector positive (LVV+) cells, which is correlated with higher production of HbAT87Q and ultimately may address known patient-to-patient variability.”

“Although early, these data add to the growing body of clinical evidence that indicate that LentiGlobin may offer a transformative benefit for patients with TDT,” said Alexis Thompson, MD, MPH, Ann & Robert H. Lurie Children’s Hospital of Chicago, Illinois and a primary investigator on the study. “Patients with TDT are dependent on a burdensome cycle of transfusion and chelation, and for these patients, gene therapy with LentiGlobin may offer a long-term solution with a one-time therapy that alleviates many of the complications of the current treatment paradigm.”

A Phase 3 Study to Evaluate Safety and Efficacy of LentiGlobin Gene Therapy for Transfusion-Dependent β-thalassemia in Patients with non-β 0 0 Genotypes: The Northstar-2 (HGB-207) Trial (Abstract S814)

The Northstar-2 Study is an ongoing, open-label, single-dose, international, multicenter Phase 3 study designed to evaluate the safety and efficacy of LentiGlobin drug product for the treatment of patients with TDT and non-β00 genotypes. As of June 2, 2017, drug product had been manufactured for six patients. The median DP VCN for these patients was 3.0 (range: 2.4 – 4.0), compared to a median DP VCN of 0.7 (range: 0.3 – 1.5) in Northstar. Results in treated patients, aged 20-22 years, as of June 2, 2017, include:

                 
        Patient 1       Patient 2       Patient 3
DP VCN in each drug product lot (copies/diploid genome)       2.9       2.4       3.2, 2.4
LVV+ cells       77%       53%       77%, 82%
CD34+ cell dose (x106/kg)       7.0       13.6       8.1
HbAT87Q (g/dl; at last follow-up)       9.5       1.6       4.6
Total hemoglobin       13.3       Not reported       Not reported
Days since last transfusion       140       Not reported       Not reported
Follow-up       6 months       3 months       2 months
 
  • Follow-up on Patients 2 and 3 was not sufficient for total hemoglobin or days since last transfusion to be clinically relevant.
  • The safety profile to date appears consistent with autologous transplantation. No Grade 3 or higher drug-product related adverse events have been observed.

Webcast Information
bluebird bio will host a live webcast at 8:00 a.m. ET on Friday, June 23, 2017. The live webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company's website at www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or (315) 625-3227 from outside the United States. Please refer to conference ID number 39917037.

About Northstar-2 (HGB-207)
Northstar-2 is a Phase 3, global, multi-center study designed to evaluate the safety and efficacy of LentiGlobin drug product in patients with transfusion-dependent beta-thalassemia and non-β00 genotypes. For this study, the manufacturing process by which the patient’s cells are transduced with the LentiGlobin viral vector has been improved, with the intent of increasing vector copy number and the percentage of cells successfully transduced.

The target enrollment of the study is 15 adult and adolescent patients and 8 pediatric patients. The study’s primary endpoint is the proportion of treated subjects who meet the definition of "transfusion independence," defined as total hemoglobin levels of at least 9g/dL without any red blood cell (RBC) transfusions for a continuous period of at least 12 months at any time during the study.

About TDT
Transfusion-dependent β-thalassemia (TDT), also called β-thalassemia major or Cooley’s anemia, is an inherited blood disease that can be fatal within the first few years of life if not treated.

Despite advances in the supportive conventional management of the disease, which consists of frequent and lifelong blood transfusions and iron chelation therapy, there is still a significant unmet medical need, including the risk for significant morbidity and early mortality. Currently, the only advanced treatment option for TDT is allogeneic hematopoietic stem cell transplant (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft vs. host disease and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT.

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 its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-thalassemia, 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 program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial for the treatment of relapsed/refractory multiple myeloma. 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 and Europe.

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 whether the manufacturing process changes for LentiGlobin will improve outcomes of patients with transfusion-dependent ß-thalassemia. 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 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, 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

bluebird bio, Inc.
Investors:
Manisha Pai, 617-245-2107
mpai@bluebirdbio.com
or
Media:
Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com

Tietoja julkaisijasta

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Tilaa tiedotteet sähköpostiisi

Haluatko tietää asioista jo ennen kuin ne uutisoidaan? Kun tilaat tiedotteemme tältä julkaisijalta, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

21st Century Fox to Spin off Businesses and Create New “Fox”; a Growth Company Centered on Live News and Sports Brands and the Iconic Fox Brand14.12.2017 15:12Tiedote

Twenty-First Century Fox, Inc. (“21st Century Fox” or the “Company” – NASDAQ: FOXA, FOX) today announced that it intends to spin off to 21st Century Fox shareholders a portfolio of its highly-rated news, sports and broadcast businesses to create a new “Fox,” which will be a growth company centered on live news and sports brands, anchored by the strength of the Fox Network. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171214005660/en/ Left to right: Robert A. Iger, Chairman and CEO of The Walt Disney Company, and Rupert Murdoch, Executive Chairman, 21st Century Fox (Photo: Business Wire) The new “Fox” will include iconic branded properties Fox News Channel, Fox Business Network, Fox Broadcasting Company, Fox Sports, Fox Television Stations Group, and sports cable networks FS1, FS2, Fox Deportes and B

First Eagle Investment Management to Absorb Costs of External Research14.12.2017 14:00Tiedote

First Eagle Investment Management announced today that effective January 3, 2018, it will absorb the costs of all external research purchased by First Eagle investment teams. Industry practices regarding payments for external research are evolving due to the Markets in Financial Instruments Directive II (MiFID II), an EU directive requiring the unbundling of external research costs from trade execution costs. As a result, asset managers worldwide have been considering whether to pass the costs of external research to their clients or to pay them from their own resources. “While our firm is not subject to MiFID II, we believe that this part of the directive points the way forward for our industry,” said Mehdi Mahmud, President and Chief Executive Officer of First Eagle Investment Management. “To be equitable and consistent across our entire client base, we have decided to use

Cognitec Brings Unique Video Investigation Features to Face Recognition Product for Law Enforcement14.12.2017 12:38Tiedote

Cognitec Systems, the face recognition company, has significantly extended the feature set of its FaceVACS-DBScan product. The latest release combines the company’s renowned image database search technology with powerful video inspection tools for a multitude of investigation use cases. FaceVACS-DBScan LE enables fast import of video footage and detailed investigations of the extracted facial images. Agents can find known or unknown persons in multiple videos to quickly narrow down suspect investigations. The investigation can reveal, for example, if a suspect was seen in various locations within a set time window, always with the same group of persons, or in one location too many times during the day. With one click, the investigator can add the facial image seen in a video frame to a local database. If a person is already known, the technology can quickly compare

The Idle Man Signs Extension with Radial to Support International Business Growth14.12.2017 11:00Tiedote

Radial, the leader in omnichannel commerce technology and operations, today announced London-based menswear fashion retailer, The Idle Man, has signed an extension with Radial to support its international growth and expansion plans. The extension follows Radial’s ability to quickly launch fulfilment, international shipping, and customer service solutions on behalf of The Idle Man from its U.K.-based operations center in Manchester, in just three months. Radial’s services have improved overall customer satisfaction as The Idle Man expands its international footprint. Founded in 2014 by Oliver Tezcan, former menswear buying manager at Asos.com, The Idle Man was established to fill the gap left by mainstream retailers for fashionable men’s clothing at affordable prices. Quickly becoming the third largest menswear destination in the U.K., with over 1.5 million unique visits each mon

SES Drives 5G Satellite Demonstration Testbed Initiative with ESA14.12.2017 10:55Tiedote

SES is expanding its efforts to support 5G developments as part of ESA’s ARTES project, SATis5, where it will be enabling demonstrations of satellite-terrestrial integration for 5G networks. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171213006286/en/ SES Drives 5G Satellite Demonstration Testbed Initiative with ESA (Photo: Business Wire) The testbed infrastructure will comprise SES's fleet of Geostationary Earth Orbit (GEO) and Medium Earth Orbit (MEO) satellites, which will be integrated with terrestrial networks and state-of-the-art technologies. In addition to providing the space segment, SES's headquarters in Luxembourg will also host a SATis5 testbed node with prototypes of networks for satellite integration, along with other nodes located in Berlin and Erlangen, and an additional portable no

Alibaba Cloud Becomes the First Cloud Computing Company to Obtain C5 Attestation with Additional Requirements14.12.2017 10:00Tiedote

Alibaba Cloud, the cloud computing arm of the Alibaba Group, announced today that it had completed its assessment for the Cloud Computing Compliance Controls Catalogue (C5) set out by the Federal Office for Information Security in Germany, also known as Bundesamt für Sicherheit in der Informationstechnik (BSI). Alibaba Cloud is the world’s first cloud provider to achieve this attestation with the additional requirements. The attestation covers Elastic Compute Service (“ECS”), Relational Database Service (“RDS”), Object Storage Service (“OSS”), Content Delivery Network (“CDN”), Server Load Balancer (“SLB”), Virtual Private Cloud (“VPC”) and Alibaba Cloud Security available on Alibaba Cloud’s regions in Singapore and Germany. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171213006271/en/ Alibaba Cloud becomes t

Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.

Tutustu uutishuoneeseemme