Business Wire

bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD) 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 new data from the ongoing HGB-205 clinical study evaluating its LentiGlobin gene therapy product candidate in patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD).

These data will be presented by Elisa Magrin, Ph.D., Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France in a poster session on Saturday, June 24 at the European Hematology Association (EHA) Annual Meeting in Madrid, Spain. Marina Cavazzana, M.D., Ph.D., Professor of Medicine at Paris Descartes University and Research Director at the Centre for Clinical Research in Biotherapy, Necker Hospital, and at the Institute of Genetic Diseases, Imagine, Paris, France, is the primary investigator of the HGB-205 study.

“HGB-205 was designed as a proof-of-concept study to initially assess the feasibility of treatment with LentiGlobin gene therapy in patients with TDT and severe SCD. Results from this study to date demonstrate the potential for durable treatment effect of LentiGlobin, with stable HbAT87Q production through 3.5 years of follow-up and sustained clinical benefit,” said David Davidson, M.D., chief medical officer, bluebird bio. “The two most recently treated patients with SCD, both of whom show rising HbAT87Q production, illustrate the potential benefit of some of the protocol modifications that we have made in our separate HGB-206 study in SCD. As with Patient 1204, the first patient with SCD treated in HGB-205, these two patients received a more stringent busulfan conditioning regimen and regular blood transfusions prior to stem cell harvest. Longer follow-up will be required to determine their eventual HbAT87Q production and clinical outcome, but it is encouraging that their in vivo VCN shows evidence of early stabilization at a higher level compared to the initial cohort of patients in HGB-206. It is also important to note that in our HGB-206 study, these modifications are further supplemented with manufacturing process improvements and evaluation of plerixafor for stem cell mobilization, which we believe may further optimize patient outcomes.”

“We are beginning to see evidence of the long-term durability of benefit from treatment with LentiGlobin, with some TDT patients even transitioning off of chelation therapy,” said Prof. Cavazzana. “It is exciting to see the outcome in the patient with TDT with the longest follow-up in HGB-205, who has gone from years of regular transfusions to 3.5 years without a single blood transfusion after a one-time treatment with LentiGlobin gene therapy.”

Update on the First Patients with Severe Hemoglobinopathies Treated with LentiGlobin Gene Therapy (HGB-205) (Abstract P631)

Presenter: Elisa Magrin, Ph.D., Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
Poster Session Date & Time: Saturday, June 24, 5:30 – 9:00 p.m. CEST
Location: Poster area (Hall 7)

HGB-205 is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of patients with TDT and severe SCD. Four patients with TDT and three patients with severe SCD have undergone infusion with LentiGlobin drug product in this study as of June 2, 2017.

TDT:

  • All patients with TDT have remained free of transfusions since shortly after receiving LentiGlobin treatment. At last study visit:
    • Patient 1201 (β0E genotype) has been free of transfusions for 41.9 months with total hemoglobin of 11.3 g/dL, of which 8.2 g/dL was HbAT87Q
    • Patient 1202 (β0E genotype) has been free of transfusions for 38.7 months with total hemoglobin of 12.9 g/dL, of which 10.0 g/dL was HbAT87Q
    • Patient 1206 (β0E genotype) has been free of transfusions for 20.3 months with total hemoglobin of 11.4 g/dL, of which 8.4 g/dL was HbAT87Q
    • Patient 1203, who is homozygous for the severe β+ mutation IVS1-110, has been free of transfusions for 20.4 months with total hemoglobin of 8.3 g/dL, of which 6.6 g/dL was HbAT87Q
  • All three patients with TDT and β0E genotype have discontinued iron chelation and transitioned to therapeutic phlebotomy.
  • The safety profile continues to be consistent with autologous transplantation. No drug-product related adverse events (AEs) have been observed, and there is no evidence of clonal dominance.

SCD:

  • Patient 1204 was 13 years old at enrollment. At last follow-up (31.7 months), this patient was producing 50% HbAT87Q – well above the approximately 30% anti-sickling hemoglobin level predicted to have potential clinical impact on the disease.
    • Approximately 30 months post-treatment, Patient 1204 suffered an episode of acute gastroenteritis with vomiting and 2 days of fever up to 40°C (104°F), which was followed by a vaso-occlusive crisis (VOC) and subsequent hospitalization. His HbAT87Q and peripheral blood VCN levels have remained stable (HbAT87Q: 6.1 g/dL, VCN: 2.3 copies/diploid genome at 30 months), suggesting continued durability of the gene therapy.
  • Patient 1207 was 16 years old at enrollment. At last follow-up (6.1 months), this patient was producing 20% HbAT87Q. This patient had a pre-treatment history of frequent episodes of VOC and acute chest syndrome (ACS) despite hydroxyurea prior to beginning regular transfusions and had one episode of ACS and a hospitalization at 6 months post-treatment.
  • Patient 1208 was 21 years old at enrollment. At last follow-up (3.4 months), this patient was producing 15% HbAT87Q.
  • The safety profile continues to be consistent with autologous transplantation. No gene therapy related AEs have been observed, and there is no evidence of clonal dominance.

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 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 SCD
Sickle cell disease (SCD) is an inherited disease caused by a mutation in the beta-globin gene that results in sickle-shaped red blood cells. Common complications include anemia, vaso-occlusive crisis, infections, stroke, overall poor quality of life and sometimes, early death.

Where adequate medical care is available, common treatments for patients with SCD largely revolve around prevention of infection and management and prevention of acute sickling episodes. Chronic management may include hydroxyurea and, in certain cases, chronic transfusions. Given the limitations of these treatments, there is no effective long-term treatment. The only advanced treatment for SCD is allogeneic HSCT. Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, GvHD and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT.

About the HGB-205 Study
HGB-205 is an ongoing, open-label Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of subjects with TDT and SCD. The study enrolled seven subjects who will be followed to evaluate safety and transfusion requirements post-transplant. Among patients with sickle cell disease only, efficacy will also be measured based on the number of vaso-occlusive crises or acute chest syndrome events. For more information on the HGB-205 study, please visit clinicaltrials.gov using identifier NCT02151526.

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 severe sickle cell disease, including statements whether the manufacturing process changes for LentiGlobin will improve outcomes of patients with transfusion-dependent ß-thalassemia and severe sickle cell disease and the potential long-term durable treatment effect of LentiGlobin. 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 or the HGB-206 clinical trial protocol 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

Investors:
bluebird bio, Inc.
Manisha Pai, 617-245-2107
mpai@bluebirdbio.com
or
Media:
bluebird bio, Inc.
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

Edgewater Networks Announces Event Sponsorship of BroadSoft Connections 201721.10.2017 00:00Tiedote

Edgewater Networks, Inc., the industry leader in Network Edge Orchestration, announced today that it is an event sponsor of the BroadSoft Connections 2017 user conference, which takes place October 22-25 at the JW Marriott Phoenix Desert Ridge Resort & Spa in Phoenix, AZ. During the event, Edgewater Networks will feature demonstrations of the latest end-to-end network interoperability capabilities, including Zero Touch Provisioning, UC Analytics from the core to the endpoint, SD-WAN optimized for BroadSoft, and much more. Continuing a long-standing partnership with BroadSoft, Edgewater Networks has completed another level of BroadCloud certification, this time for local survivability which is critical for business continuity for SMB and Enterprise customers. BroadSoft’s PacketSmart is available on all EdgeMarc Intelligent Edges. All elements of the Network Edge Orchestration are

More than 70 Companies to Showcase Top Tech at CES Unveiled Paris20.10.2017 18:08Tiedote

The Consumer Technology Association (CTA) today announced that more than 70 exhibitors will showcase their latest innovations at the now sold-out fifth annual CES Unveiled Paris The event will focus on the Internet of Things (IoT), connectivity and smart cities, and draw more than 600 attendees. CES Unveiled Paris will run from 2-7:30 PM on Tuesday, October 24, 2017 at the Palais Brongniart. Regional technology companies, top-tier media outlets, buyers and key industry influencers will come together at CES Unveiled Paris to get a direct look at the latest industry developments. The day kicks off with a CES news conference and CTA market trends presentation. Conference programming is followed by a tabletop exhibition & networking reception. Curated exhibitors will be there to showcase top tech from robotics and audio to smart home and augmented and virtual reality. Notable

Westinghouse Receives Regulatory Approval for Analysis Methodologies20.10.2017 15:15Tiedote

Westinghouse Electric Company today announced that it has received approval from the U.S. Nuclear Regulatory Commission (NRC) for two new important analysis methodologies that will be used to upgrade Westinghouse- and Combustion Engineering-based nuclear steam supply system (NSSS) safety analyses. The approvals granted are for the new FULL SPECTRUMTM Loss of Coolant Accident (FSLOCATM) methodology and the Performance Analysis and Design Model software code update, PAD5. FSLOCA is capable of improved modeling of the transient response in a pressurized water reactor to the full spectrum of LOCA break sizes that could occur, and PAD5 is the next generation of the Westinghouse fuel rod design performance code. “These codes were developed to work in concert with each other to maximize analytical margin for nuclear utilities,” said Michele DeWitt, senior vice preside

Gilead Announces Multiple Scientific Presentations Demonstrating High Cure Rates in Difficult-to-Cure HCV Patients and Improved Long-Term Bone and Renal Safety of Vemlidy® in HBV Patients Switched from Viread®20.10.2017 15:03Tiedote

Gilead Sciences, Inc. (NASDAQ: GILD) today announced results from Phase 2 and Phase 3 studies of its approved medicines for chronic hepatitis C virus (HCV) and hepatitis B virus (HBV) infection, adding to the body of evidence supporting Gilead’s viral hepatitis therapies in diverse patient populations. These and other data from more than 25 abstracts will be presented this week at The Liver Meeting® 2017, which begins today in Washington, D.C. Positive results from studies of Harvoni® (ledipasvir 90mg/sofosbuvir 400mg) in HCV-infected patients with severe renal impairment, Epclusa® (sofosbuvir 400mg/velpatasvir 100mg) in HCV-infected liver transplant recipients and Vosevi® (sofosbuvir 400mg/velpatasvir 100mg/voxilaprevir 100mg) in NS5A-inhibitor experienced HCV-infected patients will be presented during poster sessions on October 21 and October 22. In addition, updated results f

SP3H Announces That It Has Obtained a €1.2 Million European Union Grant for Its Intelligent and Clean Vehicle (VIP) Project20.10.2017 13:47Tiedote

SP3H is proud to announce joining the very select circle of the 10 French winners of the H2020 SME Instrument Phase 2 program since 2014. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171020005325/en/ Last summer VIP received the very prestigious SME instrument phase 2 H2020 label in the "transport & smart cities mobility" category. With an overall budget of € 1.7 million, VIP is subsidized by the European Union at a rate of 70% or €1.2 million. The framework contract was officially signed early October. VIP is the pre-industrialization program for Fuelbox sensors, the world's first miniaturized scanner capable of analyzing the quality of fuels on board vehicles. The heart of the program remains closely linked to the reduction of CO2 and pollutant emissions from vehicle

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