Business Wire

bluebird bio Receives Positive Opinion from CHMP for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia (TDT) Who Do Not Have β0/β0 Genotype

Del

bluebird bio, Inc. (Nasdaq:BLUE) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding β A-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (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. If approved, ZYNTEGLO, formerly referred to as LentiGlobin™ for TDT, will be the first gene therapy to treat TDT.

The CHMP’s positive opinion will now be reviewed by the European Commission (EC), which has the authority to grant marketing authorization for ZYNTEGLO in the European Union (EU). A CHMP positive opinion is one of the final steps before the EC decides on whether to authorize a new medicine. A final decision by the EC for ZYNTEGLO is anticipated in the second quarter of 2019.

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

“The goal of treatment with ZYNTEGLO is to enable patients with transfusion-dependent β-thalassemia to produce hemoglobin at sufficient levels to allow lifelong independence from blood transfusions,” said David Davidson, M.D., chief medical officer, bluebird bio. “The positive CHMP opinion for ZYNTEGLO is a crucial step toward providing what would be the first one-time gene therapy for people living with TDT. We share this achievement with the TDT community, patients and clinical investigators whose dedication made it possible. We look forward to the upcoming decision from the European Commission.”

“For many of my patients, living with TDT means a lifetime of chronic blood transfusions, iron chelation therapy and supportive treatments to manage anemia and other serious complications of this disease,” said Professor Franco Locatelli, M.D., Ph.D., Professor of Pediatrics, Sapienza University of Rome, Italy and Director, Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy. “The burden placed on these patients and their families is significant. It extends beyond immediate health implications to their daily lives, which are affected by the symptoms, hospitalizations and necessary chronic care required for TDT.”

“The present management of TDT, including regular blood transfusions every two to four weeks and daily iron chelation therapy has many psychological and social consequences, including marginalization and isolation. And in many patients TDT-related morbidities can lead to a shortened life span. Therefore, it is with great anticipation and eagerness that the international patient community has closely followed the dynamic rejuvenation of scientific interest and research of gene therapy in TDT over the last few years,” said Dr. Androulla Eleftheriou, Thalassaemia International Federation Executive Director. “Thus, the potential approval of a gene therapy brings hope that we can dramatically change the course of this disease and the health and quality of lives of patients with TDT.”

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). This means there is no need for donor HSCs from another person as is required for allogeneic HSC transplantation (allo-HSCT). A patient’s HSCs are collected and removed from the body through a process called apheresis. These HSCs are taken to a lab where a lentiviral vector is used to insert the β A-T87Q-globin gene into the patient’s HSCs. This step is called transduction. Before their modified HSCs are returned, the patient receives chemotherapy to prepare their bone marrow for the modified HSCs, which are returned through an infusion. Once a patient has the β A-T87Q-globin gene they have the potential to produce HbAT87Q, which is gene therapy- derived-hemoglobin, at levels that significantly reduce or eliminate the need for transfusions.

“The EMA’s collaborative approach and innovative programs have been instrumental in improving timely access of new medicines to patients with significant unmet needs, like those who are living with TDT,” said Anne-Virginie Eggimann, senior vice president of regulatory science at bluebird bio.

ZYNTEGLO was reviewed under an accelerated assessment timeline as part of the EMA’s Priority Medicines (PRIME) and Adaptive Pathways programs, which support medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.

Data Supporting Clinical Profile of ZYNTEGLO

The positive CHMP opinion is supported by efficacy, safety and durability data from the Phase 1/2 HGB-205 study and the completed Phase 1/2 Northstar (HGB-204) study as well as available data from the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies, and the long-term follow-up study LTF-303.

As of September 14, 2018, data from Phase 1/2 Northstar showed that 80 percent (n=8/10) of patients who do not have a β00 genotype achieved transfusion independence, meaning they had not received a transfusion for at least 12 months and maintained hemoglobin ≥9 g/dL. These eight patients had maintained transfusion independence for a median duration of 38 months (21 – 44 months) at the time of data cut off.

In the Phase 3 Northstar-2 and Northstar-3 studies, a refined manufacturing process was used to produce ZYNTEGLO and was intended to further improve the clinical results observed in the Northstar study. As of September 14, 2018, the median (min – max) total hemoglobin for patients six months after ZYNTEGLO infusion in the Northstar-2 study (n=10) was 11.9 (8.4, 13.3) g/dL.

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 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.

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 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, including statements regarding the Company’s plans and expectations for the regulatory approval, and commercialization for ZYNTEGLO™ (autologous CD34+ cells encoding β A-T87Q -globin gene, formerly LentiGlobin™ in TDT) to treat TDT, and the potential implications of clinical data for patients. 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 ZYNTEGLO 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 ZYNTEGLO will not continue or be repeated in our ongoing or planned clinical trials of ZYNTEGLO; the risk that the current or planned clinical trials of ZYNTEGLO will be insufficient to support regulatory submissions or marketing approval in the US and EU; the risk that the production of HbAT87Q 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 ZYNTEGLO 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-K, 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

Secure-D Exposes Suspicious Android App with 500 Million Downloads22.5.2019 13:49:00 CESTPressemelding

Upstream, a leading mobile technology company, has unveiled that the popular Android application VidMate triggers suspicious background activity. Hidden software within the app delivers invisible ads, generates fake clicks and purchases, installs other suspicious apps without consent and collects personal users’ information. Consequently, it depletes users’ data allowance and brings unwanted charges. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190522005501/en/ With over 500 million downloads reported, VidMate is a popular Android application for streaming and downloading videos and songs from services such as Dailymotion, Vimeo, and YouTube. It is not available in the Google Play Store but is distributed through third-party app stores like CNET or Uptodown. According to publicly available information, VidMate was developed by a subsidiary of UC Web, which is owned by Chinese conglomerate Alibaba. Over a recent period Upst

Airship’s Data Study Reveals the State of Global Mobile Permissions One Year into GDPR22.5.2019 13:35:00 CESTPressemelding

With GDPR reaching its one year anniversary May 25, customer engagement company Airship today revealed top-level results of its global benchmark study, examining the state of mobile app user permissions across nearly 700 million people worldwide. While marketers trimmed customer lists to meet new regulatory requirements for "traditional" channels (i.e., email), mobile app audiences continue to grow — up globally by +16.6 percent year over year. Businesses are also sending more notifications — averaging 36 per user per month (up 18.4 percent since last year) — while the average notification opt-in rate remains high at over two-thirds (67 percent) of global app users. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190522005486/en/ With mobile app audiences growing around the world, businesses are more heavily leveraging notifications with negligible impact to opt-in rates. (Graphic: Business Wire) These metrics vary at a regio

Diligent Launches Modern Governance to Address Dangerous Governance Deficits and Announces New Modern Governance Summit22.5.2019 13:27:00 CESTPressemelding

Today, Diligent Corporation launched a new category, called modern governance, designed to provide leaders with the missing key to bring governance into the digital age, along with new technology and a summit to support this well-designed system. Modern governance is the practice of empowering leaders with technology, insights, and processes to fuel good governance that organizations require to thrive and endure. Modern governance helps organizations address governance deficits. A governance deficit means an organization lacks the security, foresight, and accountability to withstand the onslaught of contemporary challenges – such as activist investors, cyber risk, regulation, diversity and global uncertainty. Feedback from Diligent’s 16,000 clients and the 600,000 leaders who use its technology – including more than 50% of the Fortune 1000, 70% of the FTSE 100, and 65% of the ASX – sparked the need for rethinking how companies and their boards should approach governance to navigate tod

Wipro Builds a Blockchain-Based Solution for Travacoin to Enable Digital Currency-Based Payments for the Airline Industry22.5.2019 12:19:00 CESTPressemelding

Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading global information technology, consulting, and business process services company, today announced that it has developed a blockchain-based payment solution for Travacoin, a company specializing in an eponymous digital payment solution. Travacoin is a digital payment system which enables airlines to refund and compensate passengers in a timely manner when a disruption occurs. Passengers will be notified of a delay or cancellation and refunded in this digital currency, which can be used to repurchase airline tickets, book hotels and avail other airport and travel related services. Passengers will benefit by way of a significant reduction in the waiting time for compensation. In addition, passengers can use Travacoins at retail stores in and around the airport and avail attractive discounts offered by retail partners. By compensating passengers with Travacoins, airlines will be able to reduce their administrative overheads and

New Fluid Dispensing Guide for Stock Lubrication and Metal Stamping from Nordson EFD22.5.2019 12:15:00 CESTPressemelding

Nordson EFD , a Nordson company (NASDAQ: NDSN), the world’s leading precision fluid dispensing systems manufacturer, has released a new solutions guide for stock lubrication and metal stamping. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190522005010/en/ The Stock Lubrication Solutions Guide is an overview of how Nordson EFD’s MicroCoat® lubrication system provides more consistent oil coverage and significantly reduces oil waste by up to 90%. By doing so the system quickly pays for itself by extending tool life and reducing oil usage, thus lowering waste disposal costs. In the guide, eight MicroCoat customers share application details that illustrate the benefits of the system’s Low Volume Low Pressure (LVLP) technology. Each customer has a different application with different problems to solve. “Our MicroCoat system is truly the first stock lubrication system to provide consistent oil coverage without overspray,” said Cl

Altasciences Leads Research on Chemical Nerve Agent Therapeutic from Preclinical to Clinical Phase I22.5.2019 12:04:00 CESTPressemelding

Altasciences announced today that they have been selected by Leidos Health to guide and support the development of RAP-103 peptide as a chemical nerve agent therapeutic on behalf of the Medical CBRN Defense Consortium and funded by the Defense Threat Reduction Agency (DTRA). Altasciences will conduct the required Safety Testing, all IND-enabling studies (non-GLP and GLP), the necessary Phase I clinical trials, and associated bioanalytical analysis. “Altasciences’ preclinical and clinical teams are collaborating with Leidos Health on this important program. Together, we have developed a plan to move RAP-103 from preclinical, safely through early clinical trials. As a mid-size CRO specializing in a flexible, innovative approach to providing comprehensive early stage drug development, we have the experience and team in place to complete this program successfully, on time and within budget,” explained Steve Mason, Vice President, Site Operations, at Altasciences. For over 25 years, Altasci