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.
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.
Partek® Incorporated | August 20, 2021
Partek® Incorporated announced today they have entered into an agreement with Agilent to integrate Partek® Flow® bioinformatics software with the Agilent Alissa Clinical Informatics Platform. This integration delivers a complete end-to-end analysis workflow that is robust, flexible, and customizable to meet Agilent customer demands. Partek has also made available the Agilent pipeline for RNA-Seq inside Partek Flow. In addition, the agreement allows Agilent to resell Partek Flow software through the Alissa Portal for a single point solution.
“As next-generation sequencing is more broadly adopted for clinical applications, our customers’ software requirements are also expanding. By integrating Partek Flow with the Agilent Alissa Clinical Informatics Platform we will strengthen our overall offering to address diverse applications and workflows across the translational research to clinical testing continuum,” stated Kevin Meldrum, vice president and general manager for the Agilent Genomics Division.
About Partek Incorporated
Partek Incorporated develops and globally markets quality analysis software and services for life sciences research. Partek software is unique in supporting all major genomic platforms, including bulk and single cell next-generation sequencing. Over eight thousand peer-reviewed scientific papers have used Partek software to streamline the analysis of Gene Expression, miRNA Expression, Copy Number, Allele-Specific Copy Number, LOH, Association, Trio analysis, ChIP-Seq, Single Cell RNA-Seq, RNA-Seq, DNA-Seq, DNA Methylation and qPCR studies. Partek, headquartered in St. Louis, MO, USA, has been turning data into discovery® since 1993.
Ginkgo Bioworks | September 07, 2021
Tantu, a company engineering living biotherapeutic products to treat gastrointestinal diseases, and Ginkgo Bioworks, which is building the leading horizontal platform for cell programming, today announced a partnership to accelerate the research and development of Tantu's therapeutic genes. Ginkgo, which recently announced a business combination with Soaring Eagle Acquisition Corp., serves customers across industries seeking to develop new and better products using biology.
Tantu is working to create an orally administered, living biotherapeutic that will produce and apply anti-inflammatory therapeutic proteins directly into diseased sites in the gut, resulting in improved gut barrier function and faster mucosal healing in patients where systemic anti-inflammatory therapies are not enough. Ginkgo plans to apply its automated foundry to accelerate the traditionally slow steps of candidate strain construction and genomic integration and validation with the aim of accelerating Tantu's first program and potentially helping them reach clinical proof of concept in patients faster.
"Each year, 62 million Americans are diagnosed with a digestive disorder and current treatments don't sufficiently promote gastrointestinal healing, meaning many patients need to undergo invasive surgeries to improve their quality of life," said Neel Joshi, co-founder of Tantu. "A therapeutic to aid intestinal mucosal healing could transform patient care for a significant patient population."
"We are excited to partner with a pre-seed company like Tantu with the goal of reducing their time-to-market because we view it as an opportunity to invest in the biotech industry through the startups that are driving forward so much innovation," said Jason Kelly, CEO and co-founder of Ginkgo Bioworks. "Working with Tantu provides Ginkgo with the opportunity to leverage its expertise in pharmaceuticals to support the development of a potentially transformative therapeutic for the millions struggling with digestive disorders."
Companies across numerous industries use Ginkgo's cell programming platform to find more effective, environmentally friendly ways to create products including food ingredients, fragrances, cosmetics, medicines, and more. By enabling the design of organisms that can produce valuable biological products, Ginkgo helps accelerate the development of innovative, bio-based solutions to the world's most pressing challenges.
About Tantu Therapeutics
Tantu is developing living biotherapeutic products to treat gastrointestinal diseases. Its lead therapeutic candidate is an engineered microbe that produces anti-inflammatory and healing agents from inside the gut lumen – something that no clinically approved therapeutic currently does. Tantu's local delivery strategy is designed to improve patient outcomes by reducing reliance solely on systemic anti-inflammatory drugs that can suppress the immune system. Tantu's living biotherapeutic products are being designed to complement proven approaches to further improve patients' health and quality of life.
About Ginkgo Bioworks
Ginkgo is building a platform to enable customers to program cells as easily as we can program computers. The company's platform is enabling biotechnology applications across diverse markets, from food and agriculture to industrial chemicals to pharmaceuticals. Ginkgo has also actively supported a number of COVID-19 response efforts, including K-12 pooled testing, vaccine manufacturing optimization and therapeutics discovery. In May 2021, Ginkgo announced a business combination with Soaring Eagle Acquisition Corp. (Nasdaq: SRNG), which, if completed, will result in Ginkgo, through a parent entity, Ginkgo Bioworks Holdings, Inc., becoming a public company. The extraordinary general meeting of Soaring Eagle's shareholders in connection with the transaction has been scheduled for September 14, 2021 and the transaction is expected to close shortly thereafter, subject to customary closing conditions.