bluebird bio gets CHMP nod for gene therapy Zynteglo

PharmaPhorum | April 01, 2019

A European Medicines Agency advisory panel has recommended a conditional approval for Zynteglo, bluebird bio’s lead gene therapy for thalassemia, a rare blood disorder. The Committee for Medicinal Products for Human Use (CHMP) confirmed on Friday that it had issued a positive opinion for the therapy – previously known as LentiGlobin – as a treatment for patients aged 12 years and older with transfusion-dependent beta-thalassemia (TDT) who do not have a specific β0/β0 genotype. The announcement came after a couple of medical charities jumped the gun and prematurely issued notices of the panel’s endorsement last week, hailing it as a major advance for patients with TDT who typically need frequent blood transfusions to manage the disease. The positive opinion for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) puts bluebird bio on course for its first gene therapy approval in the coming weeks and could give thalassaemia patients a new one-shot treatment option that tackles the underlying genetic cause of the disease.

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Each year, in recognition of Girls’ Day in Austria, Sandoz invites young women to its Anti-Infectives site in Kundl, Austria, where they have the opportunity to meet scientists and gain hands-on experience in Sandoz laboratories as well as in the technical areas of the pharmaceutical industry.


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BUSINESS INSIGHTS

Clearmind Medicine to Partner with Microdose for Psychedelic Capital

Clearmind Medicine Inc. | March 25, 2022

Clearmind Medicine Inc. a psychedelic medicine biotech company focused on the discovery and development of novel psychedelic-derived therapeutics to solve widespread and undertreated health problems, announced that its CEO, Dr. Adi Zuloff-Shani and Mark Haden, VP Business Development, will be presenting in the Psychedelic Capital: March 2022 virtual event. While the presentation is live streamed, lifetime access to recordings will be available to all ticket holders. About Clearmind Medicine Inc. Clearmind is a psychedelic pharmaceutical biotech company focused on the discovery and development of novel psychedelic-derived therapeutics to solve widespread and underserved health problems, including alcohol use disorder. Its primary objective is to research and develop psychedelic-based compounds and attempt to commercialize them as regulated medicines, foods or supplements. The Company’s intellectual portfolio currently consists of four patent families. The Company intends to seek additional patents for its compounds whenever warranted and will remain opportunistic regarding the acquisition of additional intellectual property to build its portfolio.

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BUSINESS INSIGHTS

CCT Research Announces New Partnership with BEKHealth

CCT Research | February 18, 2022

CCT Research has announced an exciting partnership with BEKHealth. The collaboration streamlines access to electronic medical records (EMR) data, across CCT sites and drives a data-driven approach to trial execution and management. CCT has established itself as an innovator in conducting clinical trials. Using BEKHealth, a provider of software solutions for enhancing clinical research processes, will enable CCT to more accurately identify and fit studies to its sites. The deep commitment by both companies to improving the outcomes of clinical trials made the relationship a natural fit. “One of CCT’s driving forces is to promote an environment of innovation and continuous improvement. Our partnership with BEKHealth strengthens our ability to identify potential research participants, ultimately creating greater accessibility to clinical trials.” Jennifer Kocour, President, CCT Research More than just a “technology” partnership, this collaboration with BEKHealth is also patient-centric– which remains a top priority for CCT. Once studies are awarded, it will power onsite research by identifying patients who fit studies best, saving sites, patients, and staff time and hassle. “At the individual site level, our goal is to optimize research to be as beneficial to the practice and its patients as possible with minimal disruption to the clinics day-to-day operations. We view data and technology as central to that strategy and couldn’t be more excited to work with BEKHealth,” said Kyle McAllister, Vice President, Data & Analytics, CCT Research. “The breadth and depth of CCTs network was an exciting opportunity. The ability to leverage technology to centralize key drivers of success such as feasibility, site selection, patient recruitment and analytics drives a new paradigm in clinical research,” said Jason Baumgartner, CEO and Founder, BEKHealth. “Our platform is secure and activation creates a minimal burden on IT teams. What this means for the provider and patient is our collaboration will have a major impact quickly.” “We provide an integrated clinical operating platform that enables companies to streamline research across multiple key processes,” said Josh Ransom, PhD, Vice President, Customer Experience, BEKHealth. “Ultimately this means our partners can scale their businesses faster by improving operational efficiency while taking out manual and inefficient steps.” About CCT Research CCT Research is a network of clinical research sites embedded in the offices of skilled physicians conducting clinical trials to offer patients with innovative medical treatments and safe, effective care options. CCT Research was founded in 2017 and has more than 20 locations across Arizona, Georgia, Kentucky, Nebraska, Nevada, and Utah. The sites are located within physicians’ offices, within ACO networks, and several dedicated research centers. CCT supports research in Neurology, Family Practice, and Dermatology, with plans to include additional therapy areas soon. The company's unique model simplifies the process for trial participants and provides pharmaceutical sponsors with high-quality data.

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VIEWS AND ANALYSIS

Innovent and IASO Bio Present Updated Data of BCMA CAR-T Cell Therapy (Equecabtagene Autoleucel) at EHA 2022

Innovent Biologics | June 13, 2022

Innovent Biologics, Inc. a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, autoimmune, metabolic, ophthalmology and other major diseases, and IASO Biotherapeutics a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, today jointly announced that the updated data from phase 1/2 study of Equecabtagene Autoleucel (Innovent R&D code: IBI326, IASO Bio R&D code: CT103A), a fully-human anti-B cell maturation antigen chimeric antigen receptor T-cell therapy for the treatment of relapsed and/or refractory multiple myeloma (R/R MM), was presented in the form of an oral presentation at the 27th European Hematology Association Annual Meeting in Vienna on June 9-12, 2022. The updated data from the Phase 1/2 study with a longer duration of follow-up in more patients has showed durable and deepening efficacy, manageable safety and long-term in vivo persistence, indicating that Equecabtagene Autoleucel has the potential to be a breakthrough therapy for patients with R/R MM. The updated data is from the 14 clinical sites involved in the Phase 1/2 clinical study of Equecabtagene Autoleucel (ChiCTR1800018137, NCT05066646) in the treatment of patients with R/R MM. As of the data cutoff date of January 21, 2022, 79 patients received recommended phase 2 dose of 1.0×106 CAR-T cells/kg with the median follow-up of nine months (range 1.2, 19.6) and median prior five lines of therapy (range 3,23). Among the 79 patients, 34.2% (27/79) had high-risk cytogenetic abnormalities, 34.2%(27/79)had extramedullary multiple myeloma (EMM), and 15.2%(12/79)had received prior CAR-T therapy. Equecabtagene Autoleucel demonstrated a favorable and manageable safety profile: Among the 79 patients, 94.9% (75/79) experienced cytokine release syndrome (CRS). The majority experienced 1~2 CRS, and no patient experienced grade 3 CRS. The median time to CRS onset was six days after infusion, and the median duration of CRS was five days. Only two patients experienced immune effector cell-associated neurotoxicity syndrome (ICANS), including one patient who experienced grade 1 ICANS and one who experienced grade 2 ICANS. All patients with CRS or ICANS have recovered. Equecabtagene Autoleucel showed favorable and durable efficacy: Among the 79 patients, the overall response rate (ORR) was 94.9% (75/79), with "89.9% (71/79)" of those patients achieving very good partial response (VGPR) or deeper responses, and the complete response/stringent complete response (CR/sCR) rate was 68.4% (54/79). Equecabtagene Autoleucel also demonstrated favorable efficacy in 10 patients with EMM, achieving an ORR of 100% (10/10) and a CR/sCR rate of 90.0% (9/10). In all 79 patients, 92.4% (73/79) achieved minimal residual disease (MRD) negativity, all CR/sCR subjects achieved MRD negativity, and the median duration of MRD negativity was not reached. Equecabtagene Autoleucel demonstrated favorable efficacy in patients who had received prior CAR-T therapy: Among the 12 patients who previously received CAR-T therapy, the ORR was 75.0% (9/12), with 41.7% (5/12) of those patients achieving CR/sCR. Equecabtagene Autoleucel demonstrated robust expansion and prolonged persistence: The expansion of Equecabtagene Autoleucel in peripheral blood reached the peak at a median of 12 days, with a median Cmax of 92,000 copies/ug DNA. Equecabtagene Autoleucel was still detectable in 62.3% (38/61) and 53.3% (8/15) of the subjects who completed 6-months and 12-month follow-ups after infusion. Soluble BCMA in peripheral blood of patients rapidly declined after Equecabtagene Autoleucel infusion and persistently remained below the detectable limit. Equecabtagene Autoleucel has low immunogenicity: 16.5% (13/79) of the subjects tested anti-drug antibody (ADA)-positive after Equecabtagene Autoleucel infusion. Among them,1.3% (1/79) tested ADA-positive before Equecabtagene Autoleucel infusion, and 2.5% (2/79) tested ADA-positive within three months. Prof. Chunrui Li, MD, PhD, from Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, stated "Chimeric antigen receptor (CAR)-T cell therapy is a revolutionary new pillar in cancer treatment. In our previous studies, Equecabtagene Autoleucel has shown excellent efficacy and manageable safety profiles. Its CAR structure contains fully human single-chain fragment variables (scFvs) to bypass potential anti-CAR immunogenicity of the host while retaining antitumor activity. At the 27th EHA conference, we updated the data on the efficacy and safety of Equecabtagene Autoleucel in R/R MM patients with longer median follow-up extended to 9.0 months, the CR/sCR deepened to 68.4%, compared with the CR/sCR of 58.2% with a median follow-up of 7.0 months, which were released at 63rd ASH conference in 2021. The updated data showed long-lasting safety and deepening efficacy of Equecabtagene Autoleucel. We are glad that Equecabtagene Autoleucel also shows favorable efficacy on patients who have relapsed after receiving prior CAR-T therapy. This has meaningful clinical value and is worthy of further exploration in the clinic to potentially bring forth new hope to patients with R/R MM." About Multiple Myeloma (MM) Multiple Myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia, kidney failure, immune problems, and bone fractures. For multiple myeloma patients, common first-line drug treatments include proteasome inhibitors, immunomodulatory drugs, and alkylating agents. While treatment may result in remission, most patients will inevitably enter the relapsed or refractory stage as there's currently no cure. As a result, there is a significant unmet need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of all cancer cases, and more than 2% of cancer-related deaths. According to Frost & Sullivan, the number of new MM cases in the United States rose from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 by 2025. Additionally, the total number of patients diagnosed with MM increased from 132,200 in 2016 to 144,900 in 2020 and is expected to rise to 162,300 by 2025. In China, the number of new MM cases rose from 18,900 in 2016 to 21,100 in 2020 and is expected to increase to 24,500 by 2025. The total number of patients diagnosed with MM in China increased from 69,800 in 2016 to 113,800 in 2020 and is expected to rise to 182,200 by 2025. About Equecabtagene Autoleucel Equecabtagene Autoleucel is an innovative therapy co-developed by Innovent and IASO Bio, with a fully-human anti- BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully-human scFv, CD8a hinge and transmembrane, and 4-1BB-mediated co-stimulation and CD3ζ activation domains. Based on rigorous screening and comprehensive in vivo and in vitro evaluation, Equecabtagene Autoleucel is proven to have potent and rapid anti-myeloma activity and outstanding safety, efficacy, and persistence results.

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PHARMACY MARKET

BioMatrix Specialty Pharmacy Granted Preferred Status with AscellaHealth for Home Infusion Therapies

BioMatrix Specialty Pharmacy | February 09, 2022

BioMatrix Specialty Pharmacy announced inclusion as a preferred provider in AscellaHealth's home infusion pharmacy network. AscellaHealth, a global specialty pharmacy and healthcare solutions company, has launched a site of care optimization strategy designed to cut costs for third-party payers without compromising quality of care. BioMatrix was included in the network to help the organization achieve its goals of improving drug affordability; increasing price transparency; reducing disparities in quality of care and safety; enabling care delivery in the highest-quality, lowest-cost care setting; and increasing patient convenience. Determining the optimal site of care for patients to receive therapy is an important component of treatment success. BioMatrix Specialty Pharmacy provides robust specialty infusion services, including site of care coordination. Whether administering in-home with assistance from one of our home infusion nurses, in-office, or at an ambulatory infusion center, our clinical team works with patients, payers, and prescribers to make therapy administration as safe and convenient as possible. Using a nationwide network of highly trained specialty infusion nurses, BioMatrix coordinates over 1,200 home nursing visits every month. "We are pleased to be included as a preferred provider in AscellaHealth's home infusion network. Our shared focus on positive outcomes for all patients, reduced cost, and price transparency will help AscellaHealth's members effectively manage their specialty infusion spend without compromising quality care." Ken Trader, BioMatrix VP of Managed Markets "By selecting top pharmacies for preferred status, we are taking an important step toward reducing the cost of specialty biologic medications and encouraging patients to move from higher-cost settings, like big hospitals and institutions, to lower-cost settings, such as physician's in-office infusion suites or stand-alone infusion centers," says Dea Belazi, president and CEO, AscellaHealth. "As part of our mission to help our clients better manage the high costs of specialty medication, this initiative exemplifies our efforts to increase care accessibility by encouraging the use of clinically appropriate, lower-cost care settings." About BioMatrix Specialty Pharmacy BioMatrix Specialty Pharmacy, an Inc. 5000 company, offers comprehensive, nationwide specialty pharmacy services and digital health technology solutions for patients with chronic, difficult to treat conditions. Our commitment to every patient is to provide individualized pharmacy services, timely access to care, and focused education and support. We offer a tailored approach for a wide range of therapeutic categories, improving health and empowering patients to experience a higher quality of life. About AscellaHealth LLC AscellaHealth is a global Specialty Pharmacy and Healthcare services organization serving patients, payers, life sciences and providers, offering a comprehensive portfolio of customized, tech-enabled specialty pharmaceutical and medical management services. An Inc. 5000 2021 winner, AscellaHealth's unique, patient-centric approach is built upon proprietary technology processes for innovative programs and services optimizing health outcomes and quality of life for patients with complex chronic conditions or rare diseases that require specialty medications and/or gene and cell therapies.

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Spotlight

Each year, in recognition of Girls’ Day in Austria, Sandoz invites young women to its Anti-Infectives site in Kundl, Austria, where they have the opportunity to meet scientists and gain hands-on experience in Sandoz laboratories as well as in the technical areas of the pharmaceutical industry.

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