Milestone Achieved: CFDA Approves China's First CAR-T Clinical Trial Application, with Nanjing Legend Taking the Lead

2018Year3Month13On [date], GenScript Biotech (Hong Kong) Limited (“GenScript”) announced that its subsidiary, Nanjing Legend Biotechnology Co., Ltd. (“Nanjing Legend”), has officially received notification from the National Medical Products Administration (CFDA)Granted RelatedLCAR-B38MCAR-TClinical Trial Approval for Autologous Reinfusion.

This is the first CAR-T therapy clinical trial application approved by the CFDA, and it is currently awaiting certificate issuance.

Nanjing Legend, established in 2014 as a wholly-owned subsidiary of Nanjing GenScript, focuses on the research and development of CAR-T immunotherapy and has achieved multiple breakthroughs in the treatment of multiple myeloma in recent years.

LCAR-B38M CAR-T Cell Preparation is a bispecific chimeric antigen receptor product developed based on Legend Biotech’s proprietary chimeric antigen receptor technology platform, targeting B-cell maturation antigen. The primary indication for LCAR-B38M is multiple myeloma, the second most common malignant tumor among hematologic diseases.

Although more than ten new related drugs have been launched in the past decade, the disease remains incurable. Patients incur annual costs of hundreds of thousands of yuan to sustain life and endure severe physical suffering. Currently, there are approximately 200,000 patients in China, with at least 60,000 new cases each year. Most patients are over 40 years old, with a survival period of only 3–5 years.

According to data disclosed by Legend Biotech at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting, 33 out of 35 patients with relapsed multiple myeloma after prior therapy achieved clinical remission two months after receiving investigational anti-BCMA CAR-T cell therapy (LCAR-B38M). These patients demonstrated significant myeloma response (complete response or very good partial response), yielding an overall response rate of 100%. This breakthrough sent shockwaves through the industry, earning Legend Biotech recognition as a “dark horse” from international media outlets such as the Associated Press, Reuters, and The Washington Post.

It is reported that Nanjing Legend completed the patent application for a key technology in March 2016. Multiple other patents related to chimeric antigen receptor (CAR) technology and products are currently under application, with the aim of securing global patent protection.

In December 2017, the company submitted the first clinical trial application for CAR-T therapy in China, which was formally accepted by the Center for Drug Evaluation (CDE). More recently, Legend Biotech became the first to receive the clinical trial approval from the China Food and Drug Administration (CFDA). Legend Biotech has maintained a leading position in the clinical development of CAR-T therapy.

However, the Company stated that due to the high technological complexity and high-risk nature of the LCAR-B38M CAR-T cell autologous infusion product, the development cycle from research and clinical trials to commercial production is lengthy and involves numerous stages. Furthermore, constrained by technical barriers, regulatory review requirements, and government policies, the development of the LCAR-B38M CAR-T cell autologous infusion product may be subject to various influencing factors.

Therefore, the future competitiveness of LCAR-B38M CAR-T cell autologous infusion product remains uncertain.

CAR-T technology is intrinsically linked to gene synthesis technology, representing an extension into the downstream field of synthetic biology, as well as an expansion of the company’s capabilities in gene synthesis and gene editing. In the future, life science research services will serve as a source of cash flow, the bio-enzyme sector will be a key area for expansion, and R&D investment in the CAR-T therapy field will continue.

On January 29, 2018, the China Food and Drug Administration (CFDA) published the 26th batch of drug registration applications proposed for inclusion in the priority review program, with a total of 39 drugs selected, including three CAR-T therapies.

To date,In addition to Legend Biotech, clinical trial applications from four other companies have been accepted for review: