GenScript Subsidiary Legend Biotech Achieves Breakthrough with Cilta-cel Approval in China, Offering New Hope for Multiple Myeloma Patients

Rhea-AI Summary

Legend Biotech (NASDAQ: LEGN), a GenScript subsidiary, has received approval from China's NMPA for ciltacabtagene autoleucel (cilta-cel), a CAR-T therapy for relapsed or refractory multiple myeloma. This breakthrough treatment is approved for adult patients who have undergone at least three prior therapies. The approval is based on the CARTIFAN-1 clinical trial, which showed impressive results:

• 87.9% overall response rate
• 79.3% complete or stringent complete response
• 32.56 months median duration of response
• 30.13 months median progression-free survival

This milestone reinforces GenScript's commitment to innovation in cell therapy and strengthens its position in the biotech industry.

Positive

• Approval of cilta-cel in China for relapsed or refractory multiple myeloma
• High overall response rate of 87.9% in clinical trials
• Impressive complete or stringent complete response rate of 79.3%
• Long median duration of response at 32.56 months
• Extended median progression-free survival of 30.13 months

Negative

• None.

Oncology Specialist

This approval marks a significant advancement in multiple myeloma treatment. The 87.9% overall response rate and 79.3% complete response rate are exceptionally high for heavily pretreated patients. The 30.13-month median progression-free survival is particularly impressive, suggesting durable responses. These results outperform many existing therapies for relapsed/refractory MM. The BCMA-targeting mechanism of cilta-cel represents a novel approach, potentially offering hope to patients who've exhausted other options. However, long-term safety data and real-world effectiveness will be important to monitor.

Biotech Industry Analyst

Legend Biotech's approval in China is a major milestone, potentially expanding their market reach significantly. With China's large population and increasing cancer rates, this could translate to substantial revenue growth. The approval also validates GenScript's cell therapy platform, possibly attracting more partnerships or investments. However, challenges remain: manufacturing scalability, pricing strategies in the Chinese market and competition from other CAR-T therapies. The company's ability to navigate these hurdles will be important for long-term success and market penetration.

Financial Analyst

This approval could be a game-changer for Legend Biotech's financial outlook. The Chinese multiple myeloma market is substantial and as a novel therapy, cilta-cel could command premium pricing. However, investors should note that CAR-T therapies often face reimbursement challenges due to their high cost. The company's ability to secure favorable reimbursement terms will be crucial. Additionally, the manufacturing process for CAR-T is complex and expensive, which could impact profit margins. While this approval is undoubtedly positive, the true financial impact will depend on successful commercialization and market penetration strategies.

PISCATAWAY, N.J., Aug. 27, 2024 /PRNewswire/ -- Legend Biotech (NASDAQ: LEGN), a subsidiary of GenScript Biotech Corporation (hereinafter referred to as "GenScript"), a global leader in life sciences research and manufacturing services, today announced that they have received approval from the China National Medical Products Administration (NMPA) for its cell therapy product, ciltacabtagene autoleucel (cilta-cel). This groundbreaking treatment is approved for use in adult patients with relapsed or refractory multiple myeloma (MM) who have previously undergone at least three prior lines of therapy, including at least one proteasome inhibitor and one immunomodulatory agent. The approval of cilta-cel provides a novel treatment for patients in China who have not benefit from traditional therapies.

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

Legend Biotech CEO Ying Huang stated: "The approval of cilta-cel in China market marks a key milestone and will bring significant benefits to many patients. Moving forward, we will continue to pursue our goal of curing patients, expand our clinical research, and enhance the accessibility of this innovative product to benefit more patients."

Ongoing Commitment to Cell Therapy Market and Innovation Strategy

The approval of cilta-cel is not only a significant milestone of GenScript and Legend Biotech but also a response of their unwavering commitment to innovation-driven development.  Sherry Shao, GenScript's rotating CEO, said, "Innovation has always been at the heart of GenScript's growth strategy. As the global biopharmaceutical field advances rapidly and patient needs deepen, we recognize that only through relentless innovation and unwavering dedication to cell and gene therapy can we drive the industry forward with outstanding products and services. Congratulations to Legend Biotech on its achievements. We are confident that Legend Biotech will continue to lead in cell therapy, exploring new frontiers and delivering groundbreaking innovations to the industry."

The approval is based on the outstanding results of the CARTIFAN-1 (NCT03758417) multi-center confirmatory Phase II clinical trial. This study evaluated the efficacy and safety of cilta-cel in patients with relapsed or refractory multiple myeloma.

Results from a median follow-up of 37.29 months showed that among 58 patients analyzed for efficacy, the overall response rate (ORR) was 87.9%, with very good partial response (VGPR) or better achieved in 86.2%, and complete response (CR) or stringent complete response (sCR) reached 79.3%. 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) was not reach the point.

Cilta-cel has demonstrated excellent clinical efficacy in trials, offering early and durable responses as well as longer PFS survival benefits in patients who failed multiple prior lines of treatment. This innovative and safe therapy represents a significant benefit for multiple myeloma patients.

This approval of cilta-cel not only highlights the technological strength of Legend Biotech but also enhances the market growth potential of GenScript and reinforces its leading position in the biotech industry. This important progress will lead the way for GenScript to continuously invest and explore in the cell therapy field, accelerate the translation of breakthrough therapies from the lab to clinical practice, and expedite the development and commercialization of innovative drugs and therapies.

With over twenty years of dedicated development and investment in the life science industry and cell and gene therapy, GenScript has built a solid foundation in these areas. GenScript is actively expanding the boundaries of life sciences and strengthening 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 and better treatment options for patients worldwide.

About GenScript Biotech Corporation

GenScript Biotech Corporation (HK.1548) is an important technology and service provider in the world for life science R&D and manufacture. Built upon its solid DNA synthesis technology, GenScript Biotech comprises four major business units: a life-science services and products business unit, a biologics contract development and manufacturing organization (CDMO) business unit, an industrial synthetic products business unit, and an integrated global cell therapy company.

GenScript Biotech was founded in New Jersey, USA in 2002 and listed on the Hong Kong Stock Exchange in 2015. The company's business operations span over 100 countries and regions worldwide with legal entities located in the U.S., China, Japan, Singapore, Netherlands, Ireland, the United Kingdom, Korea, Belgium and Spain. GenScript Biotech provides premium, convenient and reliable services and products for over 200,000 customers.

As of June 30, 2024, GenScript Biotech had more than 7,200 employees globally, and 100,000 peer-reviewed journal articles worldwide had cited GenScript Biotech's services and products. In addition, GenScript Biotech owns a number of intellectual property rights, including over 350 patents, over 1000 patent applications and great numbers of know-how secrets.

Driven by the corporate mission of "Make People and Nature Healthier Through Biotechnology", GenScript Biotech strives to become the most trustworthy biotech company in the world.

For more information, please visit GenScript Biotech's official website
https://www.genscript.com

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, gamma-delta 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.

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SOURCE GenScript Biotech Corporation

FAQ

What is the new treatment approved by China's NMPA for multiple myeloma?

China's NMPA has approved ciltacabtagene autoleucel (cilta-cel), a CAR-T cell therapy developed by Legend Biotech (LEGN), for treating relapsed or refractory multiple myeloma in adult patients who have undergone at least three prior lines of therapy.

What were the key results from the CARTIFAN-1 clinical trial for cilta-cel (LEGN)?

The CARTIFAN-1 trial for cilta-cel showed an overall response rate of 87.9%, with 79.3% achieving complete or stringent complete response. The median duration of response was 32.56 months, and median progression-free survival was 30.13 months.

How does cilta-cel work in treating multiple myeloma?

Cilta-cel is a gene-modified autologous CAR-T therapy targeting B-cell maturation antigen (BCMA). It features two BCMA-targeting, heavy-chain, single-domain antibodies that bind to myeloma cells, activating T cells to eliminate tumors.

What is the significance of Legend Biotech's (LEGN) cilta-cel approval in China?

The approval of cilta-cel in China is a significant milestone for Legend Biotech and GenScript, offering a new treatment option for multiple myeloma patients. It reinforces their position in the cell therapy market and demonstrates their commitment to innovation in biotechnology.