Business Wire

bluebird bio Presents New Data for LentiGlobin Gene Therapy in Sickle Cell Disease at 60th Annual Meeting of the American Society of Hematology

Del

bluebird bio, Inc. (Nasdaq: BLUE) announced new data from patients in Group C of its ongoing Phase 1/2 HGB-206 study of the company’s investigational LentiGlobingene therapy in patients with sickle cell disease (SCD) today at the 60th Annual Meeting of the American Society of Hematology (ASH).

SCD is a serious, progressively debilitating and life-threatening genetic disease. SCD results from production of abnormal sickle hemoglobin (HbS), which leads to sickled red blood cells (RBCs) and hemolysis.

“After patients with sickle cell disease were treated with LentiGlobin they began to produce gene-therapy derived HbAT87Q, which was associated with lower levels of sickling hemoglobin, the type of hemoglobin that damages red blood cells,” said David Davidson, M.D., chief medical officer, bluebird bio. “These clinical findings were consistent with results in newly developed exploratory assays used to evaluate patient samples that demonstrated reduction of HbS in most red blood cells, and a reduction in sickling comparable to sickle-trait, suggesting the potential for LentiGlobin to fundamentally improve the underlying red blood cell pathology responsible for the clinical consequences of sickle cell disease.”

Many patients with SCD live with severe anemia and vaso-occlusive events which include severe, recurrent pain crises that lead to organ damage and shortened life span.

“Before receiving treatment with LentiGlobin, patients in this study experienced frequent vaso-occlusive events, which is not uncommon for people living with sickle cell disease,” said John Tisdale, M.D., National Heart, Lung and Blood Institute, Bethesda, Md. “At nine months post-treatment with LentiGlobin, no vaso-occlusive events had been reported. By understanding the potential correlation between levels of HbAT87Q and clinical outcomes that cause the most significant burden for patients, we will be able to better characterize the potential benefits of LentiGlobin for people living with sickle cell disease.”

HGB-206: Phase 1/2 Study of LentiGlobin for Sickle Cell Disease
HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for the treatment of adults with SCD. A total of nine patients were treated with LentiGlobin in Group C in HGB-206. As of the data cut-off of September 14, 2018, data was available for seven patients who were at least three months post treatment.

A refined LentiGlobin manufacturing process intended to increase vector copy number (VCN) as well as changes to improve engraftment of gene-modified stem cells, was used for Group C. Group C patients also received LentiGlobin gene therapy made from hematopoietic stem cells (HSCs) collected from peripheral blood after mobilization with plerixafor rather than via bone marrow harvest.

HGB-206: Group C Efficacy
In patients who were six months post treatment (n=4), the production of gene therapy-derived hemoglobin, HbAT87Q, ranged from 4.8 – 8.8 g/dL and were comparable to or exceeded the levels of sickle hemoglobin, HbS. These patients did not receive a blood transfusion during this time and had total hemoglobin ranging from 9.9 – 13.7 g/dL at their last visit.

No vaso-occlusive events were reported as of the data cut-off (up to nine months post treatment with LentiGlobin). In an exploratory analysis, key markers of hemolysis, including reticulocyte counts, lactate dehydrogenase (LDH) and total bilirubin concentration had decreased compared to baseline.

To help assess the distribution of HbAT87Q in the red blood cells, bluebird bio has developed an antibody that recognizes βS, the protein present in HbS.

Using this antibody, the amount of βS was measured in the red blood cells obtained from healthy donors (βAA), sickle cell trait donors (βSA) and patients with sickle cell disease (βSS). Clear and distinct distribution of βS was observed in these control samples, with highest expression in the βSS samples, followed by βSA and no expression of βS in the healthy donor (βAA) samples.

Initial results from two patients treated with LentiGlobin gene therapy, who were nine months post treatment, showed that nearly all their red blood cells had lower amounts of βS than the βSS and the βSA control samples. Given that these patients were no longer receiving any blood transfusions, this suggests βS expression was reduced in these patients due to the production of HbAT87Q following treatment with LentiGlobin.

HGB-206 Safety
As of the data cut-off date, the safety profile of LentiGlobin remains generally consistent with underlying SCD and myeloablative conditioning. A serious adverse event (SAE) of myelodysplasia syndrome was reported in a patient who received LentiGlobin approximately three years ago in Group A of the Phase 1/2 HGB-206 study. Analysis of the patient’s cells showed no evidence of vector mediated insertional oncogenesis, and the independent data monitoring committees, along with the treating physician, agreed the SAE was unlikely related to LentiGlobin gene therapy.

About LentiGlobin in Sickle Cell Disease
LentiGlobin is a one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of sickle cell disease (SCD), which could increase the production of normal hemoglobin.

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: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/.

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 transfusion-dependent β-thalassemia (TDT) and SCD.

The European Medicines Agency (EMA) previously granted Orphan Medicinal Product designation to LentiGlobin for the treatment of SCD. 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, Fast Track designation and Regenerative Medicine Advanced Therapy Designation for the treatment of patients with SCD.

In 2019, bluebird bio plans to initiate a Phase 3 study of LentiGlobin in SCD. For more information about the ongoing Phase 1/2 HGB-206 clinical study of LentiGlobin in SCD visit clinicaltrials.gov and use identifier NCT02140554.

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 sickle cell disease. 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 the LentiGlobin product candidate 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

SFL: strong financial position and results in 2018, consolidating the Group’s position as a prime player15.2.2019 18:01:00Pressemelding

Regulatory News: The financial statements for the year ended 31 December 2018 were approved by the Board of Directors of Société Foncière Lyonnaise (Paris:FLY) on 15 February 2019 at a meeting chaired by Juan José Brugera. 2018 business indicators were very robust, with further underlying growth in rental income and historically high EPRA earnings. The portfolio's appraisal value and the Company's net asset value also continued to grow, attesting to SFL’s excellent positioning. The auditors have completed their audit of the annual financial information and are in the process of issuing their report. Consolidated data (€ millions) 2018 2017 Change Rental income 193.5 195.8 -1.2% Adjusted operating profit* 162.1 164.1 -1.2% Attributable net profit 351.6 685.3 - EPRA earnings 106.7 102.4 +4.1% * Operating profit before disposal gains and losses and fair value adjustments 31/12/2018 31/12/2017 Change Attributable equity 4,010 3,763 +6.6% Consolidated portfolio value excluding transfer cost

5 Happening Cultural Destinations to Visit in 201915.2.2019 10:11:00Pressemelding

Hong Kong There’s never really a sleepy year to visit Hong Kong, but 2019 is particularly ripe for visitors. Spearheaded by the dynamic entrepreneur Adrian Cheng of K11 and New World Development, the rejuvenation of Tsim Sha Tsui harbourfront is just the starting point. The city’s iconic harbourside walkway, Avenue of Stars, has just received a much-awaited makeover that’s the brainchild of Cheng and visually conceived by James Corner, the landscape architect responsible for the High Line in New York. It sees the addition of design-driven rest areas, kiosks featuring homegrown brands, hand prints from Asian film stars and interactive digital elements. Featuring Hong Kong’s first wave energy demonstrator that produces electricity for the Avenue, the place is turned into one of the most sustainable promenades in Hong Kong. This is just one part of Cheng and his company’s grander urban revitalisation plan, Victoria Dockside, a new art and design district which will fully open in the third

ISAE-SUPAERO Launches a New MOOC about Flight Mechanics15.2.2019 09:36:00Pressemelding

"ABC of Flight Mechanics: How Do Planes Fly?": a MOOC dedicated to flight mechanics. The notion of flight often defies our human terrestrial instincts. How does an airplane fly? How can a pilot maintain control? How high in the sky can an aircraft climb? How does a fighter pilot fly while spinning? This online course (available in English) will cover many topics, such as atmospheric physics and Newton's laws applied to aircraft, as well as propulsion forces and longitudinal stability the question of traction. The MOOC "ABC of Flight Mechanics" is aimed at general public with a specific level of scientific knowledge. Nevertheless, various levels of learning are available, thus it allows all students to take the course without needing to plenty understand the mathematical principles. Undergraduate students in aerospace engineering, trainees as well as novice and experienced pilots, journalists and professionals in the aeronautics domain (business, engineering, marketing, communications,

REPLY: Red Reply Wins the Oracle “Partner of the Year Autonomous” Award15.2.2019 09:30:00Pressemelding

Red Reply, the Reply group company that specialises in the Oracle Cloud IaaS and PaaS platform has been named as Oracle “Partner of the Year: Autonomous.” The award recognises Red Reply for its work as an Oracle Cloud Managed Service Provider Partner in developing highly innovative projects using the Oracle Autonomous Database technology. Red Reply has launched a project aimed at re-engineering the Policy Enterprise DataWarehouse of Verti Assicurazioni, company operating in the online insurance sector, using the Oracle Cloud Autonomous DataWarehouse service. The Autonomous DataWarehouse is a cloud-based database that uses machine learning to eliminate manual labour in operations such as tuning, security, backups, updates and other routine activities. This selected Oracle solution is the only one of its kind capable of addressing aspects such as security, performance and high availability of data, using automatic update and tuning mechanisms, while maximising the protection of data in l

Posiflex showcases new Interactive Self-Service Kiosks and IoT Retail Product Innovations at EuroCIS 201915.2.2019 08:00:00Pressemelding

The Posiflex Group — a synergy of world-leading POS, kiosk, and industrial computing technologies — will introduce its full product portfolio and latest innovations at EuroCIS, the leading trade fair for retail technology, from February 19th – 21st in Düsseldorf. With a product strategy focus on serviced IoT, the Posiflex Group will be displaying smart and connected retail solutions with fully-integrated middleware, RMS (Remote Monitoring System) and hardware. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190215005008/en/ Posiflex at EuroCIS Hall 9 / C42 (Graphic: Business Wire) “I am excited to introduce the Posiflex Group portfolio to the European market,” says Hans Peter Nüdling, the newly appointed Chief Strategy Officer for the Posiflex Group. “The Posiflex Group references the collective brands of the corporation, including Posiflex, Portwell and KIOSK Information Systems (KIOSK) – all under one corporate umbrella. As

OpenGate Capital Signs Agreement to Acquire SMAC Waterproofing Business from Colas Group15.2.2019 07:00:00Pressemelding

OpenGate Capital, a global private equity firm, announced today that Colas Group (EPA: RE) has agreed to sell SMAC to an affiliate of OpenGate Capital. Financial terms of the agreement were not disclosed. The divestiture is expected to be complete in the next 60-90 days. SMAC is headquartered in Issy-les-Moulineaux, France, and is a global manufacturer of waterproofing and building envelope solutions with two divisions: SMAC Travaux and SMAC Industrie. After Colas Group’s acquisitions of Miller McAsphalt and Alpiq's rail subsidiaries in 2018, Colas is refocusing on its strategic activities: transport infrastructure and construction materials. The sale of SMAC will reduce Colas’ revenues by an estimated €600 million but is not expected to have a significant impact on Colas' operating income. Upon OpenGate Capital’s completion of acquiring SMAC, the business will join a global portfolio of businesses which includes other industrial companies that provide building materials and solutions.