SOMERSET - Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, announced today that China's National Medical Products Administration (NMPA) has formally accepted its New Drug Application (NDA) for ciltacabtagene autoleucel (cilta-cel).
This submission is based on data from the confirmatory Phase 2 clinical study CARTIFAN-1 (NCT03758417) conducted in China, which evaluated the efficacy and safety of cilta-cel in adult patients with relapsed or refractory multiple myeloma who have received 3 or more prior lines of therapy, including a proteasome inhibitor and immunomodulatory drug.
Saijuan Chen, M.D., hematologist and molecular geneticist, Academician of the Chinese Academy of Engineering, and principal investigator of the CARTIFAN-1 clinical trial, said: 'Incidence and mortality rates of multiple myeloma have recently increased in China, and the disease remains incurable. As a result, there is a huge unmet medical need for new treatment options. The data from the CARTIFAN-1 study showed that cilta-cel provided deep and durable responses in patients with relapsed or refractory multiple myeloma. We hope this drug becomes available to eligible patients as soon as possible.'
Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech, said: 'Meeting medical needs and serving patients around the world has always been the goal of Legend Biotech's innovative research and development. Cilta-cel has been approved for marketing in the United States and Japan and has received conditional marketing authorization in Europe. We look forward to the possibility of providing a new treatment option for appropriate patients with relapsed and refractory multiple myeloma in China.'
About Ciltacabtagene autoleucel (cilta-cel)
Ciltacabtagene autoleucel is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient's own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells. The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.1
In December 2017, Legend Biotech Corporation entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel.
In February 2022, cilta-cel was approved by the U.S. Food and Drug Administration (FDA) under the brand name CARVYKTI for the treatment of adults with relapsed or refractory multiple myeloma.2 In May 2022, the European Commission (EC) granted conditional marketing authorization of CARVYKTI for the treatment of adults with relapsed and refractory multiple myeloma.3 In September 2022, Japan's Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI.4 Cilta-cel was granted Breakthrough Therapy Designation in the U.S. in December 2019 and in China in August 2020. In addition, cilta-cel received a PRIority MEdicines (PRIME) designation from the European Commission in April 2019. Cilta-cel also received Orphan Drug Designation from the U.S. FDA in February 2019, from the European Commission in February 2020, and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In March 2022, the European Medicines Agency's Committee for Orphan Medicinal Products recommended by consensus that the orphan designation for cilta-cel be maintained on the basis of clinical data demonstrating improved and sustained complete response rates following treatment.5
About CARTIFAN-1
CARTIFAN-1 (NCT03758417)6 is a Phase 2 open-label, confirmatory trial evaluating the efficacy and safety of cilta-cel in Chinese patients with relapsed/refractory multiple myeloma who have received at least three prior lines of treatments including a proteasome inhibitor and immunomodulatory drug. The primary endpoint is overall response rate.
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.7 In 2022, it is estimated that more than 21,000 people will be diagnosed with multiple myeloma, and more than 16,000 people will die from the disease in China.8 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.9 Although treatment may result in remission, unfortunately, patients will most likely relapse.10 Patients who relapse after treatment with standard therapies, including protease inhibitors, immunomodulatory agents, and an anti-CD38 monoclonal antibody, have poor prognoses and few treatment options available.11,12
About Legend Biotech
Legend Biotech is a global 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 allogeneic chimeric antigen receptor T-cell 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 cutting-edge therapeutics for patients worldwide.
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 Legend Biotech's strategies and objectives; statements relating to ciltacabtagene autoleucel (cilta-cel), including Legend Biotech's expectations for cilta-cel; statements about submissions for cilta-cel to, and the progress of such submissions with, the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Chinese Center for Drug Evaluation of National Medical Products Administration (CDE) and other regulatory authorities; potential indications for cilta-cel; the anticipated timing of, and ability to progress, clinical trials and the ability to generate, analyze and present data from clinical trials. 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; unexpected delays as a result of actions undertaken, or failures to act, by our third party partners; uncertainties arising from challenges to Legend Biotech's patent or other proprietary intellectual property protection, including the uncertainties involved in the U.S. litigation process; competition in general; government, industry, and general public pricing and other political pressures; the duration and severity of the COVID-19 pandemic and governmental and regulatory measures implemented in response to the evolving situation; as well as the other factors discussed in the 'Risk Factors' section of the Legend Biotech's Annual Report on Form 20-F filed with the Securities and Exchange Commission on March 31, 2022. 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 press release as anticipated, believed, estimated or expected. Any forward-looking statements contained in this press release speak only as of the date of this press release. Legend Biotech specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Contact:
Tina Carter
Tel: (908) 331-5025
Email: tina.carter@legendbiotech.com
LEGEND BIOTECH CORPORATION 
