CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced that new data from Group C of its ongoing Phase 1/2 HGB-206 study of investigational LentiGlobin gene therapy (bb1111) for adult and adolescent patients with sickle cell disease (SCD) show a complete elimination of severe VOEs and VOEs between six and 24 months of follow-up. These data are being presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.
Now with more than two years of data, we continue to observe promising results in our studies of LentiGlobin for SCD that further illustrate its potential to eliminate the symptoms and devastating complications of sickle cell disease. Consistently achieving the complete resolution of severe vaso-occlusive events (VOEs) and VOEs between Month 6 and Month 24 follow-up is unprecedented other than with allogeneic stem cell transplantation. Importantly, our data show the potential for LentiGlobin for SCD to produce fundamentally disease-modifying effects with sustained pancellular distribution of gene therapy-derived anti-sickling HbAT87Q and improvement of key markers of hemolysis that approach normal levels, said David Davidson, M.D., chief medical officer, bluebird bio. In addition to these clinical outcomes, for the first time with a gene therapy we now have patient-reported outcomes through the validated PROMIS-57 tool, showing reduction in pain intensity at 12 months after treatment with LentiGlobin for SCD. These results provide insight into the potential real-life impact LentiGlobin for SCD may offer patients.
SCD is a serious, progressive and debilitating genetic disease. In the U.S., the median age of death for someone with sickle cell disease is 43 46 years. SCD is caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS). HbS causes red blood cells to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and unpredictable, painful VOEs.
In the HGB-206 study of LentiGlobin for SCD, VOEs are defined as episodes of acute pain with no medically determined cause other than a vaso-occlusion, lasting more than two hours and severe enough to require care at a medical facility. This includes acute episodes of pain, acute chest syndrome (ACS), acute hepatic sequestration and acute splenic sequestration. A severe VOE requires a 24-hour hospital stay or emergency room visit or at least two visits to a hospital or emergency room over a 72-hour period, with both visits requiring intravenous treatment.
LentiGlobin for SCD was designed to add functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once patients have the A-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin (HbAT87Q) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.
As a hematologist, I regularly see the debilitating effects of pain events caused by sickle cell disease. Pain has an overwhelmingly negative impact on many facets of my patients lives and can lead to prolonged hospitalizations, said presenting study author Alexis A. Thompson, M.D., professor of pediatrics at Northwestern University Feinberg School of Medicine and head of hematology at Ann and Robert H. Lurie Childrens Hospital of Chicago. The results observed with LentiGlobin gene therapy for SCD include the complete elimination of severe vaso-occlusive pain episodes, which is certainly clinically meaningful, but also for the first time, we have documented patients reporting that they are experiencing improved quality of life. This degree of early clinical benefit is extraordinarily rewarding to observe as a provider."
As of the data cut-off date of August 20, 2020, a total of 44 patients have been treated with LentiGlobin for SCD in the HGB-205 (n=3) and HGB-206 (n=41) clinical studies. The HGB-206 total includes: Groups A (n=7), B (n=2) and C (n=32).
HGB-206: Group C Updated Efficacy Results
The 32 patients treated with LentiGlobin for SCD gene therapy in Group C of HGB-206 had up to 30.9 months of follow-up (median of 13.0; min-max: 1.1 30.9 months).
In patients with six or more months of follow-up whose hemoglobin fractions were available (n=22), median levels of gene therapy-derived anti-sickling hemoglobin, HbAT87Q, were maintained with HbAT87Q contributing at least 40% of total hemoglobin at Month 6. At last visit reported, total hemoglobin ranged from 9.6 15.1 g/dL and HbAT87Q levels ranged from 2.7 8.9 g/dL. At Month 6, the production of HbAT87Q was associated with a reduction in the proportion of HbS in total hemoglobin; median HbS was 50% and remained less than 60% at all follow-up timepoints. All patients in Group C were able to stop regular blood transfusions by three months post-treatment and remain off transfusions as of the data cut-off.
Nineteen patients treated in Group C had a history of severe VOEs, defined as at least four severe VOEs in the 24 months prior to informed consent (annualized rate of severe VOE min-max: 2.0 10.5 events) and at least six months follow-up after treatment with LentiGlobin for SCD. There have been no reports of severe VOEs in these Group C patients following treatment with LentiGlobin for SCD. In addition, all 19 patients had a complete resolution of VOEs after Month 6.
In SCD, red blood cells become sickled and fragile, rupturing more easily than healthy red blood cells. The breakdown of red blood cells, called hemolysis, occurs normally in the body. However, in sickle cell disease, hemolysis happens too quickly due to the fragility of the red blood cells, which results in hemolytic anemia.
Patients treated with LentiGlobin for SCD in Group C demonstrated near-normal levels in key markers of hemolysis, which are indicators of the health of red blood cells. Lab results assessing these indicators were available for the majority of the 25 patients with 6 months of follow-up.
The medians for reticulocyte counts (n=23), lactate dehydrogenase (LDH) levels (n=21) and total bilirubin (n=24) continued to improve compared to screening values and stabilized by Month 6. In patients with Month 24 data (n=7), these values approached the upper limit of normal by Month 24. These results continue to suggest that treatment with LentiGlobin for SCD may improve biological markers to near-normal levels for SCD.
As previously reported, assays were developed by bluebird bio to enable the detection of HbAT87Q and HbS protein in individual red blood cells, as well as to assess if HbAT87Q was pancellular, or present throughout all of a patients red blood cells. In 25 patients with at least six months of follow-up, on average, more than 80% of red blood cells contained HbAT87Q, suggesting near-complete pancellularity of HbAT87Q distribution and with pancellularity further increasing over time.
HGB-206: Improvements in Health-Related Quality of Life
Health-related quality of life (HRQoL) findings in Group C patients treated with LentiGlobin for SCD in the HGB-206 study were generated using the Patient Reported Outcomes Measurement Information System 57 (PROMIS-57), a validated instrument in SCD.
Data assessing pain intensity experienced by nine Group C patients were analyzed according to baseline pain intensity scores relative to the general population normative value: 2.6 on a scale of 0-10, where 10 equals the most intense pain. Data were assessed at baseline, Month 6 and Month 12.
Of the five patients with baseline scores worse than the population normative value average, four demonstrated clinically meaningful reductions in pain intensity at Month 12; the group had a mean score of 6.0 at baseline and a mean score of 2.4 at Month 12. Of the four patients with better than or near population normative values at baseline, two reported improvement and two remained stable with a mean score of 2.3 at baseline and 0.8 at Month 12.
HGB-206: Group C Safety Results
As of August 20, 2020, the safety data from Group C patients in HGB-206 remain generally consistent with the known side effects of hematopoietic stem cell collection and myeloablative single-agent busulfan conditioning, as well as underlying SCD. One non-serious, Grade 2 adverse event (AE) of febrile neutropenia was considered related to LentiGlobin for SCD. There were no serious AEs related to LentiGlobin for SCD.
One patient with significant baseline SCD-related and cardiopulmonary disease died 20 months post-treatment; the treating physician and an independent monitoring committee agreed his death was unlikely related to LentiGlobin for SCD and that SCD-related cardiac and pulmonary disease contributed.
LentiGlobin for SCD Data at ASH
The presentation of HGB-206 Group C results and patient reported outcomes research are now available on demand on the ASH conference website:
HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for sickle cell disease (SCD) that includes three treatment cohorts: Groups A (n=7), B (n=2) and C (n=32). A refined manufacturing process designed to increase vector copy number (VCN) and further protocol refinements made to improve engraftment potential of gene-modified stem cells were used for Group C. Group C patients also received LentiGlobin for SCD made from HSCs collected from peripheral blood after mobilization with plerixafor, rather than via bone marrow harvest, which was used in Groups A and B of HGB-206.
About LentiGlobin for SCD (bb1111)
LentiGlobin gene therapy for sickle cell disease (bb1111) is an investigational treatment being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study, and the ongoing Phase 3 HGB-210 study.
The U.S. Food and Drug Administration granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD.
LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.
bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-307) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT04628585 for LTF-307.
LentiGlobin for SCD is investigational and has not been approved in any geography.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell 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, were 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. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, 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: @bluebirdbio, LinkedIn, Instagram and YouTube.
LentiGlobin and bluebird bio are trademarks of bluebird bio, Inc.
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 managements 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: regarding the potential for LentiGlobin for Sickle Cell Disease to treat SCD; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; 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.
- Researchers investigate whether stem cell therapy is safe and effective for treatment-resistant bipolar disease - Newswise - April 18th, 2021
- Stem Cell and Regenerative Therapy Market: Cell Therapy Segment to Dominate Global Market - BioSpace - April 18th, 2021
- First in the nation, FDA-approved Phase II mesenchymal stem cell therapy for Parkinson's disease begins - Newswise - April 18th, 2021
- Cellino Biotech developing tech to help scale stem cell therapies - MedCity News - April 18th, 2021
- Stem cell treatment needed to fight the good fight - Victoria Lookout - April 18th, 2021
- Being bionic: the future of regenerative medicine - Toronto Star - April 18th, 2021
- Treating chronic myeloid leukemia (CML): By phase and more - Medical News Today - April 18th, 2021
- Chemical conversion of human epidermal stem cells into intestinal goblet cells for modeling mucus-microbe interaction and therapy - Science Advances - April 18th, 2021
- Andres Isaias Combining Innovation and Excellence - Influencive - April 18th, 2021
- A Massive New Gene Editing Project Is Out to Crush Alzheimer's - Singularity Hub - April 18th, 2021
- Antibiotic Use Prior to Allogeneic Stem Cell Transplantation May Be Linked to Graft Vs Host Disease - Hematology Advisor - April 18th, 2021
- Mucopolysaccharidosis (MPS) Treatment Market Is Rising With Post COVID-19 Impact Analysis, Development, CAGR - Global Banking And Finance Review - April 18th, 2021
- The Recovery Room: News beyond the pandemic April 16 - Medical News Today - April 18th, 2021
- The Governments Watchful Eye on Fraud Stemming from Stem Cell Therapy - JD Supra - April 4th, 2021
- Uprooting Cancer: Innovative Hydrogel Rapidly Reverts Cancer Cells Back to Cancer Stem Cells - SciTechDaily - April 4th, 2021
- Multiple sclerosis: Recent research on causes and treatments - Medical News Today - April 4th, 2021
- First CAR T-Cell Therapy for Multiple Myeloma: Abecma - Medscape - April 4th, 2021
- Vitro Biopharma Retains Leading Health Care Executive as Acting Director of Regulatory Affairs & Director - Benzinga - April 4th, 2021
- Allogeneic hematopoietic stem cell transplantation for therapy-related myeloid neoplasms following treatment of a lymphoid malignancy - DocWire News - April 4th, 2021
- Orca-T Offers an Alternative to HSCT With Improved Patient Experience - OncLive - April 4th, 2021
- Russell Health Highlighted in the Silicon Review's '50 Leading Companies of the Year 2021' - PRNewswire - April 4th, 2021
- Gracell Biotechnologies Announces Enrollment of First Patient in Registrational Phase 1/2 Clinical Study for GC007g, an Allogeneic CAR-T Cell Therapy... - April 4th, 2021
- LGL Leukemia: Overview, Symptoms, and Treatment - Healthline - April 4th, 2021
- Funding the Next Generation of Cancer Therapies - Genetic Engineering & Biotechnology News - April 4th, 2021
- RenovaCare Announces Organizational Changes and Appointment of New Officers - GlobeNewswire - April 4th, 2021
- Stem Cell Therapy Market expected to reach USD 16.51 Billion by 2025 KSU | The Sentinel Newspaper - KSU | The Sentinel Newspaper - March 8th, 2021
- Creative Medical Technology Holdings Publishes Efficacy in Pain Reduction and Mobility in Patients with Disc Degenerative Disc Using StemSpine... - March 8th, 2021
- New Controversy for Stem Cell Therapy That Repairs Spinal Cords - The Great Courses Daily News - March 8th, 2021
- Cell Therapy for Cartilage Regeneration Gets a Boost With Hyaluronic Acid Enriched Chondrocytes in a 3D Tissue Engineering Platform - Business Wire - March 8th, 2021
- Scar-Forming Cells Switch to Producing New Neurons that Promote Functional Recovery in Mice after Spinal Cord Injury - Genetic Engineering &... - March 8th, 2021
- Moderna Hires Harvard Stem Cell Researcher Jonathan Hoggatt as Director of Hematology: What You Need to Know - Yahoo Finance - March 8th, 2021
- Stem Cell Banking Market Report 2021 | Growth and Opportunities Analysis - BioSpace - February 19th, 2021
- Global Cell Therapy Biomanufacturing Market (2020 to 2025) - Featuring Lonza Group, Merck & Novartis Among Others - ResearchAndMarkets.com -... - February 19th, 2021
- Off-the-Shelf NK Immunotherapy Is Safe and Promising in B-Cell NHL With Chemotherapy and Transplant - Targeted Oncology - February 17th, 2021
- Braunschweig Makes the Case for Earlier Use of CAR T-Cell Therapy in DLBCL - OncLive - February 17th, 2021
- Creative Medical Technology Holdings Files Patent on Prevention of Organ Transplant Rejection using ImmCelz - PRNewswire - February 17th, 2021
- Lineage to Host Virtual OPC1 Investor & Analyst Day on February 22, 2021 - Business Wire - February 17th, 2021
- Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT - February 17th, 2021
- CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2020 Financial Results - GlobeNewswire - February 17th, 2021
- Equillium Presents Positive Interim Clinical Data of Itolizumab in First-line Treatment of Acute Graft-Versus-Host Disease at the 2021 Transplantation... - February 14th, 2021
- Zebrafish reveal regenerative protein that could inspire new treatments for muscle-wasting diseases and aging - FierceBiotech - February 11th, 2021
- Stem cell study illuminates the cause of an inherited heart disorder | Penn Today - Penn Today - February 11th, 2021
- Neurons from patient blood cells enable researchers to test treatments for genetic brain disease - Brown University - February 11th, 2021
- Therapeutic Solutions International Acquires Stem Cell Therapy That Successfully Completed FDA Double Blind Placebo Controlled Efficacy Study for Lung... - February 11th, 2021
- The Role and Activation Mechanism of TAZ in Hierarchical Microgroove/N | IJN - Dove Medical Press - February 11th, 2021
- Stem Cell Therapy Market: Top 5 trends fueling the industry revenue through 2025 - BioSpace - February 9th, 2021
- Magenta Therapeutics to Present Additional Data from Phase 1 MGTA-145 Stem Cell Mobilization Program and Preclinical Updates on Targeting Conditioning... - February 9th, 2021
- Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of... - February 9th, 2021
- Creative Medical Technology Holdings Recruits Internationally Renowned Kidney Expert to Scientific Advisory Board - PRNewswire - February 9th, 2021
- Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with... - February 9th, 2021
- USC scientist Ya-Wen Chen receives American Lung Association grant to advance stem cell-based lung therapies - USC News - February 9th, 2021
- [Full text] Successful Use of Nivolumab in a Patient with Head and Neck Cancer Aft | OTT - Dove Medical Press - February 9th, 2021
- Responses to Liso-Cel Not Influenced by Prior Treatment With Anti-CD19 Agents in R/R Large B-Cell Lymphoma - Targeted Oncology - February 9th, 2021
- Drugs that trip cellular alarm could help clear out hibernating HIV - New Atlas - February 9th, 2021
- Leukemia in children: Symptoms, causes, treatment, outlook, and more - Medical News Today - February 6th, 2021
- Stem Cell Study Illuminates the Cause of a Devastating Inherited Heart Disorder - Newswise - February 4th, 2021
- iSpecimen expands offerings to support regenerative medicine, adding cryopreserved stem and immune cells to existing biospecimens available through... - February 4th, 2021
- CU Researchers Win Prize from National Eye Institute - CU Anschutz Today - February 4th, 2021
- Leading Urologist Doubles Down on CaverStem Regenerative Stem Cell Procedure for Treatment of Erectile Dysfunction in Men - BioSpace - January 25th, 2021
- Research Assistant - Department of Obstetrics and Gynaecology job with NATIONAL UNIVERSITY OF SINGAPORE | 239606 - Times Higher Education (THE) - December 24th, 2020
- Getting to the root of why hair goes gray - messenger-inquirer - December 24th, 2020
- How the COVID Virus Induces Inflammation, Cytokine Storm and Stress in Infected Lung Cells - SciTechDaily - December 24th, 2020
- Medicine by Design symposium highlights importance of convergence in regenerative medicine and human health - News@UofT - December 22nd, 2020
- 2020 in Neuroscience, Longevity, and AIand What's to Come - Singularity Hub - December 22nd, 2020
- Updated Findings Show Continued Efficacy for CAR T-Cell Therapy in Heavily Pretreated Myeloma - Targeted Oncology - December 14th, 2020
- Cell Isolation/ Separation Market worth $15.0 billion by 2025 - Exclusive Report by MarketsandMarkets - PRNewswire - December 14th, 2020
- Cord blood banks sell parents on promising stem cell research, but with no guarantees - The Arizona Republic - December 8th, 2020
- Procyon Technologies LLC and Novo Nordisk A/S to Collaborate on the Development of a Stem-Cell Based Therapy for Type 1 Diabetes - PRNewswire - December 8th, 2020
- Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease - The Conversation US - December 8th, 2020
- Hadassah Medical Center and Neurogenesis Announce Groundbreaking Results from a Phase 2 Study in Progressive Multiple Sclerosis treated with NG-01... - December 8th, 2020
- Worldwide Cell Culture Industry to 2025 - Featuring Thermo Fisher Scientific, Corning Incorporated and Eppendorf Among Others - GlobeNewswire - December 8th, 2020
- The cell culturemarket is projected to reach USD 33.1 billion by 2025 from USD 19.0 billion in 2020, at a CAGR of 11.8% - GlobeNewswire - December 8th, 2020
- New DARZALEX (daratumumab) Data from GRIFFIN Study Show Deeper and Longer Responses in Patients with Newly Diagnosed Multiple Myeloma - BioSpace - December 8th, 2020
- Anatomy of a vaccine: What it takes to create a safe, effective COVID shot - University of California - December 8th, 2020
- Development of New Stem Cell Type May Lead to Advances In Regenerative Medicine - Newswise - December 3rd, 2020
- UCLA receives $7.3 million grant to build state-of-the-art facility for developing gene, cell therapies - UCLA Newsroom - December 3rd, 2020
- Human Embryonic Stem Cells Market in Global : Current and the Future Trends: Astellas Pharma Inc/ Ocata Therapeutics, Stemcell Technologies Inc - The... - December 3rd, 2020
- Possible Role for Comprehensive Molecular ProfilingBased Treatment Selection in Newly Diagnosed AML, Study Suggests - Cancer Therapy Advisor - December 3rd, 2020
- Nobel Prize history from the year you were born - Herald & Review - December 3rd, 2020
- 4D hires a trio of area heads as it ramps up its gene therapy pipeline - FierceBiotech - December 3rd, 2020