AbbVie's Skyrizi wins its first FDA approval, springing blockbuster ambitions

biopharmadive | April 24, 2019

AbbVie's Skyrizi wins its first FDA approval, springing blockbuster ambitions
The psoriasis approval for Skyrizi (risankizumab) in the U.S. was expected, following a similar go-ahead from regulators in Japan and a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use. In Phase 3 studies of the drug, roughly 80% of patients with moderate to severe plaque psoriasis achieved 90% clear skin and slightly more than half reached complete skin clearance using Skyrizi. However, the anti-IL23 antibody is far from alone in the next generation of immunology drugs. Other interleukin inhibitors already on the market include J&J's anti-IL 12/23 Stelara (ustekinumab) as well as the IL-17 inhibitors Cosentyx (secukinumab) and Taltz (ixekizumab), respectively marketed by Novartis and Eli Lilly.

Spotlight

Your lecturer is Prof. Dr. Vincent Racaniello. He is teaching microbiology and immunology at Columbia University in New York City. He is a leading expert in the research of viruses and human diseases. Therefore Racaniello has served on the editorial boards of scientific journals, such as the Journal of Virology or PLOS Pathogens. Furthermore he was the 2015 president of the American Society for Virology. Beyond that he is editor of an online virology blog and co-producer of the podcasts Netcast This Week in Virology, This Week in Parasitism and This Week in Microbiology.

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Matica Bio Announces Joint Research Agreement with Sartorius for the Development of Advanced Viral Vector Manufacturing Technology

Matica Biotechnology, Inc. | October 19, 2021

Matica Biotechnology, Inc, (Matica Bio) a contract development and manufacturing organization (CDMO) specializing in the clinical and commercial production of cell and gene therapies, today announced a joint research agreement (JRA) with Sartorius, a leading international partner of the biopharmaceutical industry. Under this agreement, Matica Bio and Sartorius will work on a number of studies together to streamline and optimize PAT technologies, automation software, and single-use platforms offered by Sartorius for large scale vector production. Michael Stewart, Chief Technology Officer at Matica Bio explained, "The generation of in-line real-time process data is one of the most significant obstacles to achieving consistent, high-producing viral vector titers during development that can be translatable to large scale production. In many respects, what is going on inside a bioreactor or within downstream operations is still a black box to us." Mr. Stewart continued, "Matica Bio's development, manufacturing and quality teams have decades of viral vector production experience. Applying our expertise together with Sartorius' industry-leading single-use and PAT technologies will allow us to provide more robust, consistent results for our clients, guiding informed decision-making throughout the manufacturing process and accelerating the overall development timeline to the clinic and market." "We are extremely excited to initiate this partnership with Sartorius. Our number one priority is to deliver for our clients. Our expert staff will be working to integrate more fully automated processes leading to an increase in the speed of information flow and a reduction in preventable errors. The end result will be increased product yields, higher quality as well as improved flexibility and responsiveness to our clients' ever-changing needs." Dr. Yun Jeong Song, Chief Executive Officer of Matica Bio The JRA with Sartorius underscores Matica Bio's commitment to applying integrated technology and bioprocess solutions to address production complexities like reduction of labor and risk while improving output efficiencies in the production of advanced therapies, including viral vector products. Together Sartorius and Matica Bio are dedicated to solving the challenges of large-scale cell culture and viral vector production, improving manufacturability and reducing the costs of novel cell and gene therapies, oncolytic vectors and vaccines. About Matica Biotechnology, Inc. Matica Bio is a contract development and manufacturing organization for gene therapies, cell therapies, vaccines, oncolytic vectors and other advanced biotherapeutic products. Our GMP facility in College Station, TX is designed for the rapid development, scale-up and production of clinical and commercial supply. Matica Bio offers process development, GMP production, product release and stability assessment, together with the quality oversight and regulatory guidance necessary to ensure our clients' success.

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INDUSTRIAL IMPACT

Legend Biotech Announces Extension of PDUFA Date for Cilta-Cel

Cilta-cel | November 01, 2021

Legend Biotech Corporation (Legend Biotech), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, today announced that the U.S. Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) target date for ciltacabtagene autoleucel (cilta-cel) to February 28, 2022. Cilta-cel is a BCMA-directed chimeric antigen receptor T cell (CAR-T) therapy being investigated for the treatment of adults with relapsed and/or refractory multiple myeloma. The Biologics License Application (BLA) was submitted by Legend Biotech’s collaboration partner Janssen Biotech, Inc. “We are working closely with Janssen and the FDA to facilitate an efficient and thorough review of the BLA for cilta-cel,Financial Officer at Legend Biotech.We remain confident that cilta-cel has shown great promise in patients with relapsed and refractory multiple myeloma, and we are focused on making this therapy available to them in the US as soon as possible.” Ying Huang, Ph.D., Chief Executive Officer and Chief The FDA notified Janssen on October 28, 2021 of the extension of the PDUFA date to allow sufficient time to review information recently submitted pertaining to an updated analytical method following an FDA information request. Legend and Janssen met with the FDA on November 1. No additional clinical data have been requested. About Cilta-cel Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 in the U.S. and Europe and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel. In December 2020, Legend announced initiation of rolling submission of Biologics License Application to the FDA seeking approval of cilta-cel for the treatment of relapsed and/or refractory multiple Myeloma, which was accepted under Priority Review in May 2021. Cilta-cel was previously granted Breakthrough Therapy Designation (BTD) granted in the U.S. in December 2019, and Orphan Drug Designation in February 2019. About Multiple Myeloma Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.1 Although treatment may result in remission, unfortunately, patients will most likely relapse.2 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.3 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.4,5 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.6 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.7 About Legend Biotech Legend Biotech is a global, clinical-stage biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogenic chimeric antigen receptor T-cell, T-cell receptor (TCR-T), and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of safe, efficacious and cutting-edge therapeutics for patients worldwide. We are currently engaged in a strategic collaboration to develop and commercialize our lead product candidate, ciltacabtagene autoleucel, an investigational BCMA-targeted CAR-T cell therapy for patients living with multiple myeloma. Applications seeking approval of cilta-cel for the treatment of patients with RRMM are currently under regulatory review by several health authorities around the world, including the U.S. Food and Drug Administration and the European Medicines Agency. Cautionary Note Regarding Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the timing and outcome of regulatory reviews relating to cilta-cel, including the BLA being reviewed by the FDA. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Legend Biotech’s expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products; unexpected clinical trial results, including as a result of additional analysis of existing clinical data or unexpected new clinical data; unexpected regulatory actions or delays, including requests for additional safety and/or efficacy data or analysis of data, or government regulation generally; as well as the other factors discussed in the “Risk Factors” section of Legend Biotech’s Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 2, 2021. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in this presentation as anticipated, believed, estimated or expected. Legend Biotech specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

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RESEARCH

Bio-Techne And Carterra Initiate A Clinical Research Collaboration For COVID-19 Variant Analysis

Bio-Techne Corporation | September 08, 2021

Bio-Techne, a leading supplier of high-quality and innovative tools for life science research, therapeutic manufacturing and clinical diagnostics and Carterra Inc., the world leader in high-throughput antibody screening and characterization, today announced a clinical research collaboration studying COVID-19 variants. New variants, such as Delta, can be more easily transmitted and more likely to evade the immune response of vaccinated or previously infected people. To assess immune responses to new variants and to plan for intervention mechanisms that avoid vaccine escape, scientists and epidemiologists need a fast and effective assay. The partnership leverages Carterra's flagship analytical platform - the LSA® - to characterize panels of SARS-CoV-2 spike and receptor-binding domain (RBD) variants in an information-rich assay. The SARS-CoV-2 variants were developed by Bio-Techne and used to fast-track the new, scalable assay for measuring the potential risks that new variants may evade the ability of the immune system to block the virus. Bio-Techne has been leading the way in COVID-19 research reagent and immunoassay development enabling scientists to make discoveries leading to effective diagnoses, therapies and vaccines. The company was first to market with COVID-19 research protein variants and has significant expertise with SARS-CoV-2 antibody quantitation. Bio-Techne and Carterra will focus this collaboration on more than 40 variants, including the Delta variant that is prevalent today, and emerging variants as they are discovered. The Carterra LSA will be used to generate high-definition insights with over 120 data points from patient serum samples, including a variant profile, ACE-2 blocking potential, and isotyping profiles of immunoglobin response. The LSA's hands-free, high-throughput setup requires only 2 µL of patient serum and provides for the analysis of new and emerging variants in the ever-evolving field of COVID-19 research. Carterra's LSA instrument is the fastest, most sensitive and flexible biosensor platform on the market. Combining proprietary flow printing microfluidics with High Throughput Surface Plasmon Resonance (HT-SPR) technology, the LSA enables detailed screening and characterization of vast antibody or variant libraries, including full kinetic profiles. With the ability to generate real-time binding data for 384 proteins on one chip and assay up to 1152 patient samples in a single unattended run, the LSA significantly improves efficiency and speed in both drug discovery and clinical research. About Bio-Techne Bio-Techne Corporation is a global life sciences company providing innovative tools and bioactive reagents for the research and clinical diagnostic communities. Bio-Techne products assist scientific investigations into biological processes and the nature and progress of specific diseases. They aid in drug discovery efforts and provide the means for accurate clinical tests and diagnoses. With thousands of products in its portfolio, Bio-Techne generated approximately $931 million in net sales in fiscal 2021 and has approximately 2,700 employees worldwide. About Carterra, Inc. Carterra® is the leading provider of high throughput technologies designed to accelerate and improve the discovery of novel therapeutic candidates. Carterra's LSA™ instrument, software, and consumables for monoclonal antibody (mAb) characterization deliver up to 100 times the throughput of existing platforms in 10% of the time while using only 1% of the sample required by other systems. The LSA combines patented microfluidics technology with real-time high throughput Surface Plasmon Resonance (HT-SPR) and industry-leading data analysis and visualization software to revolutionize mAb screening. Carterra, Inc. is based in Salt Lake City, Utah, and has Customer Experience Centers in San Francisco, Salt Lake City, Boston, and Munich, Germany.

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Spotlight

Your lecturer is Prof. Dr. Vincent Racaniello. He is teaching microbiology and immunology at Columbia University in New York City. He is a leading expert in the research of viruses and human diseases. Therefore Racaniello has served on the editorial boards of scientific journals, such as the Journal of Virology or PLOS Pathogens. Furthermore he was the 2015 president of the American Society for Virology. Beyond that he is editor of an online virology blog and co-producer of the podcasts Netcast This Week in Virology, This Week in Parasitism and This Week in Microbiology.