CARsgen Therapeutics Announces FDA Grants RMAT Designation to CT041 for CLDN18.2-Positive Advanced Gastric/GEJ Adenocarcinoma

CARsgen Therapeutics (Stock Code: 2171.HK), an innovative CAR-T cell therapy company primarily focused on the treatment of hematologic malignancies and solid tumors, announced today that its self-developed CAR-T product, CT041, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of Claudin18.2 (CLDN18.2)-positive advanced gastric cancer/gastroesophageal junction adenocarcinoma (GC/GEJ).According to publicly available information, CT041 is the world’s first solid tumor CAR-T product to receive Regenerative Medicine Advanced Therapy (RMAT) designation. With this milestone, Carsgen Therapeutics has become the only Chinese CAR-T company whose two core candidate products have both been granted RMAT designation.

CT041’s recent designation as a Regenerative Medicine Advanced Therapy (RMAT) means that it can simultaneously benefit from multiple FDA support programs, including “Breakthrough Therapy” and “Fast Track” designations. These benefits include more intensive communication with the FDA and eligibility for accelerated approval. The designation was based on CT041’s promising therapeutic efficacy and favorable safety profile observed in ongoing clinical trials.

CT041 is a potentially global first-in-class CAR-T candidate product independently developed by Carsgen Therapeutics, primarily indicated for the treatment of gastric cancer/gastroesophageal junction adenocarcinoma and pancreatic cancer. As of the announcement date, CT041 is the only CLDN18.2-targeting CAR-T cell candidate product globally that has received IND/CTA approval from the U.S. FDA, China’s National Medical Products Administration (NMPA), and Health Canada, and is currently undergoing clinical trials.

Overview of Key Regulatory Milestones for CT041:

In 2020, CT041 was granted Orphan Drug designation by the U.S. FDA for the treatment of gastric cancer/gastroesophageal junction adenocarcinoma;

In 2021, CT041 was granted orphan drug designation by the European Medicines Agency (EMA) for the treatment of advanced gastric cancer;

In 2021, CT041 was granted Priority Medicines (PRIME) status by the EMA for the treatment of advanced gastric cancer. CT041 is the first solid tumor CAR-T product worldwide to be selected for the PRIME scheme.

In October 2019, CT053, another BCMA CAR-T product independently developed by Carsgen Therapeutics, was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. FDA for the treatment of relapsed/refractory multiple myeloma. CT041 is the second product from Carsgen Therapeutics to receive RMAT designation, following CT053. As of the announcement date, Carsgen Therapeutics remains the only company among all CAR-T companies in China with products awarded RMAT designation, fully demonstrating the company’s R&D strength.

CARsgen Therapeutics has 11 candidate products, all independently developed with global rights, and has obtained Investigational New Drug (IND) approvals for 8 CAR-T products, ranking first among all CAR-T companies in China. As of December 31, 2021, the company had filed more than 300 patent applications worldwide, including 65 granted patents.

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics“CT041’s recent designation by the U.S. Food and Drug Administration (FDA) as a Regenerative Medicine Advanced Therapy (RMAT) further underscores the product’s outstanding performance and its significant potential to address unmet medical needs. The RMAT designation is crucial for accelerating both product development and the review of Biologics License Applications (BLAs), thereby enabling patients to access this advanced therapy more rapidly. We believe that the RMAT, PRIME, and Orphan Drug designations granted to CT041 will facilitate closer collaboration with global regulatory authorities, expediting the market launch of CT041 and bringing benefits to patients worldwide.”

The latest data from the investigator-initiated trial of CT041, presented at the 2021 ESMO Congress, demonstrated an objective response rate (ORR) of 61.1% in 18 patients with gastric cancer who had experienced failure of at least two prior lines of therapy and received the recommended Phase 2 dose (RP2D) of 2.5×10⁸ CT041 cells. Historical data indicate that the ORR for chemotherapy in gastric cancer patients who have failed at least two prior lines of therapy is approximately 4%–8%, while the efficacy rate for anti-PD-1 monoclonal antibodies is around 11%. Therefore, compared with other therapeutic agents used in later-line treatment of gastric cancer, CT041 has achieved a significant improvement in objective response rate.

In addition to investigator-initiated trials conducted in China, CARsgen Therapeutics has launched a Phase Ib/II clinical trial in China for advanced gastric cancer/gastroesophageal junction cancer and pancreatic cancer, as well as a Phase 1b clinical trial in North America for advanced gastric cancer or pancreatic cancer. CARsgen Therapeutics also plans to initiate a pivotal Phase 2 clinical trial in North America in 2022.

Regenerative Medicine Advanced Therapy (RMAT) designation is granted to cell therapies, tissue-engineered products, human cell and tissue products, or any combination products utilizing such therapies or products, intended to treat, modify, reverse, or cure serious or life-threatening diseases or conditions. To qualify for RMAT designation, an investigational drug must have preliminary clinical evidence demonstrating its potential to address unmet medical needs associated with the disease or condition.

RMAT designation confers the benefits of “fast track” review and “breakthrough therapy” status, including more frequent meetings with the FDA to discuss development plans for candidate products, as well as eligibility for rolling review and priority review.

Legend Biotech (Stock Code: 2171.HK) is a biopharmaceutical company with operations in China and the United States, primarily focused on innovative CAR-T cell therapies for the treatment of hematologic malignancies and solid tumors. We have established an integrated cell therapy platform with in-house capabilities spanning target discovery, antibody development, clinical trials, and commercial-scale manufacturing. Through proprietary technology development and a product pipeline with global rights, we aim to address major challenges in CAR-T cell therapy, such as enhancing safety, improving efficacy against solid tumors, and reducing treatment costs. Our mission is to become a global biopharmaceutical leader by providing innovative and differentiated cell therapies that make cancer curable for patients worldwide.