bluebird bio Presents New Data for LentiGlobin Gene Therapy in Sickle Cell Disease at 60th Annual Meeting of the American Society of Hematology
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 LentiGlobin™ gene 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 (βA/βA), sickle cell trait donors (βS/βA) and patients with sickle cell disease (βS/βS). Clear and distinct distribution of βS was observed in these control samples, with highest expression in the βS/βS samples, followed by βS/βA and no expression of βS in the healthy donor (βA/βA) 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 βS/βS and the βS/βA 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.
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.
LentiGlobin is a trademark of bluebird bio, Inc.
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.
Om Business Wire
(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
Akaal Pharma Pty Ltd Announces Licensing Agreement for its First-in-Class, Topical Spingosine-1-Phosphate-1 (S1P1) Receptor Modulator for Atopic Dermatitis and Psoriasis18.12.2018 23:56 | Pressemelding
Akaal Pharma Pty Limited (Akaal Pharma) today announced that it has entered into a licensing agreement for Akaal Pharma’s First-in-Class, topical Spingosine-1-Phosphate-1 (S1P1) modulator, AKP-11, with Boston Pharmaceuticals, Inc. (Boston Pharmaceuticals). AKP-11 is a differentiated S1P1 modulator, which potentially has clinical applications in a number of inflammatory, immune and vascular diseases. Akaal Pharma has demonstrated the clinical activity of AKP-11 in multiple Phase-1 clinical studies. Under the terms of the license, Boston Pharmaceuticals is responsible for the further clinical development and commercialisation of AKP-11 for the Americas and Europe. Akaal Pharma has the rights for the rest of the world. Financial terms of the agreement were not disclosed. "We continue to actively prioritize and expand our dermatology and pain programs to serve the wider global community suffering from these diseases and where there is need of safe and effective treatment,” said Chairman of
Takeda Announces Listing of American Depositary Shares on the New York Stock Exchange18.12.2018 22:33 | Pressemelding
NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, IN WHOLE OR IN PART, DIRECTLY OR INDIRECTLY, IN ANY JURISDICTION WHERE TO DO SO WOULD CONSTITUTE A VIOLATION OF THE RELEVANT LAWS OF SUCH JURISDICTION Takeda Pharmaceutical Company Limited (TSE: 4502) (“Takeda”) today announced that the listing and trading of its American Depositary Shares (“ADSs”) on the New York Stock Exchange (“NYSE”) is expected to commence on December 24, 2018. Takeda’s ADSs currently trade over-the-counter. The ADSs will now trade under the ticker symbol “TAK” and The Bank of New York Mellon will continue to act as the depositary bank for the ADS program. Takeda will maintain its headquarters in Japan and its primary listing on the Tokyo Stock Exchange (the “TSE”), as well as its current listings on local Japanese stock exchanges. “Our dual listing on the NYSE and TSE reflects our position as a leading global biopharmaceutical company and will provide wider capital markets access with expanded trading hours for our in
Sestek Speech Analytics Scores the Highest Overall Vendor Satisfaction Mark from Customers in DMG Consulting Survey18.12.2018 19:00 | Pressemelding
Sestek today announced that it was recognized for getting the highest overall vendor satisfaction rating from customers in DMG Consulting’s 2018 – 2019 Speech Analytics Product and Market Report. Compared to the featured competitors, Sestek Speech Analytics achieved the highest customer ratings in 8 major vendor categories out of 10. Here are the 7 categories in which Sestek Speech Analytics received a perfect score of 5.0 by the customer satisfaction ratings: • Training and workshops • Ongoing service and support • Professional services • Innovation • Responsiveness to product enhancement requests • Vendor communication • Overall vendor satisfaction Donna Fluss, the President of DMG Consulting -providing comprehensive coverage and authoritative analysis for the market- stated the importance of speech analytics: “Speech analytics has always been interesting because of its unique contributions to the understanding of voice interactions in the contact center. Companies that invest in the
Brand Marketing and Social Purpose Veteran Larry Koffler Joins BCW18.12.2018 18:31 | Pressemelding
BCW (Burson Cohn & Wolfe), a leading global communications agency, today announced that Larry Koffler has joined as Executive Vice President, Managing Director in the agency’s Brand Solutions Practice in New York. He will join BCW on January 14, 2019. “Larry has had an extraordinary career leading integrated communications programs that bridge brand, corporate and purpose for some of the world’s most iconic brands and organizations,” said Chris Foster, President, North America, BCW. “His award-winning experience across a wide variety of industries will be invaluable to clients – and in particular purpose-driven clients – that need powerful programs that exceed expectations and drive meaningful results. I am excited for what he will add to BCW’s celebrated brand solutions business.” Koffler will report to Thomas Bunn, Executive Vice President, Managing Director, Brand Solutions. Koffler joins BCW from Edelman, where he spent the entirety of his career and most recently served as Executi
Skitude Premium Launches, Offering Personalised 3D Mapping, 3D Tracks, Advanced Statistics, Speed Heat Maps and Apple Watch Compatibility18.12.2018 15:58 | Pressemelding
Skitude, the world’s largest apps community of digitally-connected snow sport lovers, with over a million users, has launched a premium version of its popular profile. Skitude apps are available for Android and Apple devices, and the premium upgrade offers the option to see your day’s skiing/boarding as an interactive 3D map, a speed ‘heat map’ or by statistical analysis of descents, slopes and distances covered. There is also Apple Watch compatibility, so your smartphone battery won’t be dead by lunchtime, as can often be the case with tracking apps. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181218005591/en/ (Photo: Skitude) Premium also offers 3D maps and advanced statistics for over 2,500 resorts worldwide, with real time information on slope conditions and whether runs are open. There are ‘points of interest’, webcams and a wealth of other useful information to help you make the most of your time on the slopes. Skit
PSB Names Mike Chuter Chief Executive Officer18.12.2018 14:15 | Pressemelding
PSB, a global research-based consultancy within BCW, announced today that Mike Chuter has joined the firm as Chief Executive Officer, effective immediately. Chuter is based in New York and reports to Donna Imperato, Global CEO of BCW. He is responsible for the global growth and development of the organization across its Corporate, Political, Media and Entertainment practices. “Mike is exceptionally well-suited to lead PSB given his deep experience interpreting data through both analytical and creative lenses and in driving integrated communications programs rooted in analytics,” said Imperato. “Mike is the right person to help PSB advise on holistic creative communications solutions that leverage the firm’s industry-leading expertise in primary research and behavioral, social and other third-party data analysis.” Chuter is a 25-year agency veteran who joins PSB from global social impact organization Thankful, which he co-founded in 2013, a one-half commercial enterprise, one-half socia