Legend Biotech's Cilta-cel Approved in China, Bringing New Hope for Multiple Myeloma

Legend Biotech, a subsidiary of GenScript Biotech Corporation, has achieved a significant milestone with the approval of its cell therapy product, ciltacabtagene autoleucel (cilta-cel), by the China National Medical Products Administration (NMPA). This approval marks a pivotal moment in the treatment of relapsed or refractory multiple myeloma (MM) in adult patients who have previously undergone at least three lines of therapy, including a proteasome inhibitor and an immunomodulatory agent.

Cilta-cel is a gene-modified autologous chimeric antigen receptor T cell (CAR-T) therapy targeting B-cell maturation antigen (BCMA). The therapy is administered through an intravenous infusion. It features a distinctive CAR structure with two BCMA-targeting, heavy-chain, single-domain antibodies. This allows cilta-cel to bind effectively to BCMA-expressing myeloma cells, leading to the activation and proliferation of T cells to eliminate the tumor.

Ying Huang, CEO of Legend Biotech, emphasized the importance of this approval in the Chinese market and the potential benefits it offers to many patients. The company aims to continue expanding its clinical research and enhancing the accessibility of innovative treatments like cilta-cel to benefit more patients.

Sherry Shao, rotating CEO of GenScript, highlighted that innovation is central to the company's growth strategy. She noted that only through relentless innovation and dedication to cell and gene therapy can the industry advance with outstanding products and services. Shao congratulated Legend Biotech on its achievements and expressed confidence that the company would continue to lead in cell therapy and explore new frontiers.

The NMPA's approval is based on the impressive results of the CARTIFAN-1 (NCT03758417) multi-center confirmatory Phase II clinical trial. This study assessed the efficacy and safety of cilta-cel in patients with relapsed or refractory multiple myeloma. With a median follow-up of 37.29 months, the overall response rate (ORR) among the 58 patients analyzed for efficacy was 87.9%. Moreover, 86.2% of patients achieved very good partial response (VGPR) or better, while 79.3% reached complete response (CR) or stringent complete response (sCR). The median duration of response (mDOR) was 32.56 months, median progression-free survival (mPFS) was 30.13 months, and the median overall survival (mOS) had not been reached at the time of the study.

Cilta-cel has shown exceptional clinical efficacy in trials, providing early and durable responses as well as longer progression-free survival benefits for patients who have failed multiple prior treatments. This innovative and safe therapy represents a significant advancement for multiple myeloma patients.

The approval of cilta-cel underscores Legend Biotech's technological prowess and enhances GenScript's market growth potential, reinforcing its leading position in the biotech industry. This progress paves the way for GenScript to continue investing in and exploring the field of cell therapy. It accelerates the translation of breakthrough therapies from the lab to clinical practice and expedites the development and commercialization of innovative drugs and therapies.

With over two decades of dedicated development and investment in the life science industry, GenScript has established a solid foundation in cell and gene therapy. The company actively expands the boundaries of life sciences and strengthens collaborations with global research institutions, medical organizations, and industry partners. By integrating resources and leveraging synergies, GenScript aims to accelerate the rapid development of the biotech industry and provide more effective treatment options for patients worldwide.

Legend Biotech is committed to treating and ultimately curing life-threatening diseases through advanced cell therapies. Based in Somerset, New Jersey, the company is developing cutting-edge therapeutics for patients globally, utilizing innovative technology platforms.

GenScript Biotech Corporation is a key player in life science research and manufacturing services, providing premium and reliable services and products for over 200,000 customers worldwide. With a mission to make people and nature healthier through biotechnology, GenScript strives to be the most trustworthy biotech company globally.