Business Wire

bluebird bio Presents Long-Term Efficacy and Safety Data from Clinical Studies of LentiGlobin® Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) at 24th European Hematology Association (EHA) Congress

Del

bluebird bio, Inc. (Nasdaq:BLUE) announced updated results from the completed Phase 1/2 Northstar (HGB-204) study, and new data from the Phase 3 Northstar-2 (HGB-207) and Phase 3 Northstar-3 (HGB-212) clinical studies of its LentiGlobin® gene therapy for patients with transfusion-dependent β-thalassemia (TDT), at the 24th European Hematology Association (EHA) Congress in Amsterdam, the Netherlands.

“The maturing data from our clinical studies of LentiGlobin for TDT show that patients across genotypes are able to achieve and maintain transfusion independence with stable production of gene therapy-derived-hemoglobin, HbAT87Q, extending for years,” said David Davidson M.D., chief medical officer, bluebird bio. “In patients who achieve transfusion independence, we have observed decreased liver iron concentration over time and improved markers of erythropoiesis, demonstrating the transformative disease-modifying potential of gene therapy for patients with TDT.”

TDT is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or absent hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin for β-thalassemia addresses the underlying genetic cause of TDT by adding functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). This means there is no need for donor HSCs from another person, as is required for allogeneic HSC transplantation (allo-HSCT). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived-Hb, at levels that eliminate or significantly reduce the need for transfusions.

bluebird bio’s clinical development program for LentiGlobin in TDT includes studies across patient genotypes, including those who do not have a β00 genotype as well as those with a β00 genotype.

“Patients living with β-thalassemia who have a β00 genotype or an IVS-I-110 mutation typically have low levels of endogenous hemoglobin,” said Andreas Kulozik, M.D., Ph.D., Heidelberg University Hospital, Heidelberg, Germany. “Transfusion independence is a goal for the treatment of TDT, regardless of genotype. Early results from the ongoing Phase 3 study in patients with a β00 genotype or an IVS-I-110 mutation show gene therapy-derived-hemoglobin significantly contributes to improved total hemoglobin levels.”

Northstar (HGB-204)

The results reported for the completed Phase 1/2 Northstar (HGB-204) study reflect data as of December 13, 2018; of the 18 patients in the study, 10 patients do not have a β00 genotype and eight have a β00 genotype. All 18 patients have completed the two-year study and enrolled in the long-term follow-up study, LTF-303.

Eight of 10 treated patients who do not have a β00 genotype achieved transfusion independence (TI), meaning they had not received a transfusion for at least 12 months or more and maintained a weighted average Hb ≥9 g/dL.

These eight patients had a median weighted average Hb during TI of 10.3 g/dL (min–max: 9.3–13.2 g/dL) and continued to maintain TI for up to 45 months. The patient follow-up period is calculated from infusion of LentiGlobin to the last study visit.

In patients who have a β00 genotype, three of the eight achieved TI and maintained a median weighted average Hb ranging from 9.5–10.1 g/dL for a median duration of 16.4 months (min–max: 16.1–20.8 months).

An exploratory assessment was conducted to assess liver iron concentration (LIC) in the 11 patients from the Northstar study who achieved TI. Increased iron levels are a consequence of frequent transfusions. High iron levels can cause organ damage, which many patients with TDT are at risk of and must manage through chelation regimens.

LIC was measured at baseline and then every 12 months after treatment with LentiGlobin. Patients reinitiated iron chelation therapy at a median of 13 months after LentiGlobin infusion (min–max: 2–16 months). Over time, LIC began to decrease in all 11 patients with the largest decrease observed in patients who had 48 months of data available (n=4). A median 56 percent reduction (min–max: 38–83 percent) was reported in these four patients.

Northstar-2 (HGB-207) Efficacy

As of December 13, 2018, 20 patients who do not have β00 genotypes have been treated in the Phase 3 Northstar-2 study. Patient age ranged from 8–34 years, with five pediatric (<12 years) and 15 adolescent/adult (≥12 years) patients.

Four of five evaluable patients achieved TI and maintained a median weighted average Hb of 12.4 g/dL (min–max: 11.5–12.6 g/dL). These four patients continued to maintain TI for a median duration of 13.6 months (min–max: 12–18.2 months) at the time of the data cut off.

Thirteen of 14 patients with at least three months of follow-up were free from transfusions for at least three months. Total Hb levels in these patients ranged from 8.8–13.3 g/dL at the time of the last study visit. HbAT87Q levels were stable over time in patients who were free from transfusions; at Month 6 (n=10) median HbAT87Q was 9.5 g/dL and at Month 12 (n=7) median HbAT87Q was 9.3 g/dL.

An exploratory analysis was conducted with bone marrow from seven 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 (RBC) production, characteristic of patients with TDT. In these seven patients, all of whom had stopped chronic transfusions, an increase in the myeloid to erythroid ratio was observed, suggesting improvement in RBC production.

Northstar-3 (HGB-212) Efficacy

As of April 12, 2019, 11 patients with TDT and a β00 genotype or an IVS-I-110 mutation had been treated in the Phase 3 Northstar-3 study.

The one patient evaluable for TI achieved and maintained it and had a total Hb of 13.6 g/dL at the Month 16 follow-up.

Five patients had stopped transfusions for at least three months and had Hb levels of 10.2–13.6 g/dL at the time of the last study visit (5 – 16 months post-treatment). Of these patients, all of those who reached six months of follow-up (n=4) had HbAT87Q levels of at least 8 g/dL.

LentiGlobin for TDT Safety

Non-serious adverse events (AEs) observed during clinical studies that were attributed to LentiGlobin for TDT 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 LentiGlobin for TDT.

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.

As of the data cut off dates stated above, a total of 49 pediatric, adolescent and adult patients with TDT and a non-β00 or β00 genotype, including patients with IVS-I-110 mutations, have been treated with LentiGlobin for TDT in the Northstar, Northstar-2 and Northstar-3 studies.

About LentiGlobin for β-Thalassemia

The European Commission (EC) granted conditional marketing authorization for LentiGlobin for TDT, to be marketed as ZYNTEGLO® (autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a β00 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 (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived-hemoglobin, at levels that eliminate or significantly reduce the need for transfusions. Upon engraftment and achievement of transfusion independence, effects of ZYNTEGLO are expected to be lifelong.

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

LentiGlobin for TDT continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies and the long-term follow-up study LTF-303. For more information about the ongoing clinical studies, visit www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT01745120 for Northstar (HGB-204), NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212) and NCT02633943 for LTF-303.

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: @bluebirdbioLinkedInInstagram 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 βA-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 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 future regulatory submissions in the U.S. and EU or additional marketing authorizations; the risk that the production of HbA 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. 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
Investors:
Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
or
Media:
Catherine Falcetti, 339-499-9436
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

Three Finalists Selected Following GA-ASI’s Blue Magic Belgium Event15.6.2019 11:00:00 CESTPressemelding

Three finalists have been selected from GA-ASI’s Blue Magic Belgium event with the goal of supporting GA-ASI and the development of MQ-9B SkyGuardian Remotely Piloted Aircraft (RPA) for Belgium. The three Belgian companies selected are AIRobot, ALX Systems, and Hexagon. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190615005003/en/ "We were very impressed by the many talented companies and particularly by the innovative concepts presented by these three finalists," said Linden Blue, CEO, GA-ASI. (Photo: Business Wire) AIRobot, a company based at DronePort in Sint Truiden, Belgium, focuses on developing drone performance equipment for easy, precise and safe professional operations, while specializing in Artificial Intelligence (AI) for processing hyper-spectral imagery. ALX Systems is an Unmanned Aircraft System (UAS) solution provider based in Liège, Belgium and specializing in AI for processing Full Motion Video. Hexagon’s

World’s First E-Money License for Blockchains Issued to Monerium15.6.2019 08:35:00 CESTPressemelding

Monerium ehf. has received a license from the Financial Supervisory Authority of Iceland to issue e-money on blockchains through its subsidiary, Monerium EMI ehf. The full license is the world’s first e-money license for blockchains issued under EU e-money regulations. The license is passportable within the largest global economic zone, the European Economic Area, and to external jurisdictions subject to regulatory approval and destination country regulations. Designed as a digital alternative to cash, e-money is a proven framework for digital fiat currency already in use for pre-paid cards and mobile wallets. By issuing e-money on blockchains, Monerium removes the need for intermediaries and provides the ability to automate financial transactions in many sectors, including payments, trade finance, securities settlement, and ecommerce. At the same time, e-money on blockchains extends Satoshi Nakamoto’s vision of online peer-to-peer currency transactions to a regulated form of digital f

Mattel® Unveils Hot Wheels™ id, Groundbreaking New Play System Bringing Together Physical and Digital Play, Available Exclusively at Apple.com, Select Apple Stores and on the App Store14.6.2019 13:00:00 CESTPressemelding

Mattel, Inc. (NASDAQ: MAT) today announced the launch of Hot Wheels id, the evolution of the beloved brand that generations of kids have played with to ignite their challenger spirit. Through innovative vehicle play, Hot Wheels id brings together physical and digital in a Mixed Play experience with uniquely identifiable vehicles, a smart Race Portal, Smart Track, and digital hub, so you can build your personalized fleet, measure your performance, and compete like never before. Hot Wheels id is available starting June 14th exclusively at Apple.com, select Apple stores and on the App Store. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190614005084/en/ Hot Wheels Race Portal™ scans your Hot Wheels id vehicles into the app, tracks speed and counts laps via infrared sensors, and easily connects with your classic Hot Wheels track. (Photo: Business Wire) For the first time in history, kids can scan their Hot Wheels id cars digita

S BLOCK Co-host With World Blockchain Forum Singapore & World Blockchain Award Asia14.6.2019 12:45:00 CESTPressemelding

Asia's highly anticipated blockchain event World Blockchain Forum · Singapore & World Blockchain Award · Asia will be held on June 22-23, 2019 at the Marina Bay Sands in Singapore. The conference will bring together more than 4,000 global elites from government, associations, regulatory agencies, investment institutions, blockchain project parties, financial technology, and media to discuss industry trends and innovations, jointly recognizing industry pioneers and contributions to promote the industry and achieve healthy and rapid development! The World Blockchain Forum Singapore & World Blockchain Award Asia will be co-hosted by S BLOCK. As a fast-growing digital asset management platform, S BLOCK was developed for the blockchain industry. This rapidly growing platform aims to serve the digital financial ecosystem and aims to commercialize by the end of 2019. The slogan of S BLOCK is “unleash your digital asset.” The platform also provides investors access to mainstream cryptocurrenci

Janssen Reports Top-Line Phase 3 Results for TREMFYA® (guselkumab) in Adults with Active Psoriatic Arthritis14.6.2019 11:39:00 CESTPressemelding

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced top-line results from the Phase 3 DISCOVER 1 and 2 studies, which evaluated the efficacy and safety of guselkumab compared to placebo in adult patients with active moderate to severe psoriatic arthritis (PsA). Both studies met their primary endpoints of American College of Rheumatology 20% improvement (ACR20), and the safety profiles observed for guselkumab in the DISCOVER programme were consistent with previous studies of guselkumab and current prescribing information.1 The DISCOVER programme comprises the first-ever Phase 3 studies evaluating an IL-23 p19 inhibitor for the treatment of psoriatic arthritis, and data will be presented at upcoming scientific medical meetings. Data from the two DISCOVER studies will serve as the basis of submissions to the U.S. Food and Drug Administration and European Medicines Agency seeking approval of guselkumab as a treatment for psoriatic arthritis, which are anticipated for

Velodyne Features Advanced Lidar for Port Terminal Automation at TOC Europe14.6.2019 10:00:00 CESTPressemelding

Velodyne Lidar, Inc. (Stand A50) will demonstrate how its smart, powerful lidar solutions can advance port terminal automation at TOC Europe in Rotterdam, 18-20 June. Velodyne will highlight customer applications that show how its lidar sensors can be used in crane steering, container handling, internal terminal vehicles (ITV), automated guided vehicles (AGV), forklifts, and terminal security/yard monitoring systems. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190614005040/en/ DGWorld solutions demonstrate how Velodyne’s intelligent lidar sensors are helping port and terminal operators deliver consistent service quality and achieve increased safety. (Photo: Business Wire) DGWorld (Stand B21A), a Dubai-based Velodyne integrator, will showcase autonomous taxis, ITVs, AGVs, and terminal operation vehicles that use Velodyne lidar sensors to run autonomously. These vehicles use advanced artificial intelligence (AI), automation