CARsgen's First Enrolled Subject in the THANK-u Plus™ Platform-Based Allogeneic CAR-T Trial Achieves sCR at Week 4

SHANGHAI, Feb. 9, 2025 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announces that the first subject treated with an allogeneic BCMA CAR-T therapy developed on the THANK-u Plus™ platform, has achieved stringent complete response (sCR) and minimal residual disease (MRD) negativity at the Day-28 assessment.

The patient, diagnosed with relapsed/refractory multiple myeloma, IgA-λ type (R-ISS Stage II), had previously undergone three lines of combination therapy along with autologous hematopoietic stem cell transplantation. After disease progression following the most recent treatment, the patient was enrolled in the clinical trial. Following infusion of CAR‑T cells at the lowest dose level according to the clinical protocol, the patient experienced Grade 1 cytokine release syndrome (CRS), which was effectively managed with antipyretics and tocilizumab, and the patient did not show immune effector cell-associated neurotoxicity syndrome (ICANS) or other CAR-T-related immune adverse events, demonstrating an overall favorable safety profile.

This CAR-T therapy is developed for the treatment of relapsed/refractory multiple myeloma (R/R MM) and relapsed/refractory plasma cell leukemia (R/R PCL). Additional data updates are anticipated in the second half of this year.

"Developed in-house, the THANK-u Plus™ platform is an innovative allogeneic CAR-T technology designed to overcome the accessibility challenges in CAR-T therapy. The expansion of CAR-T cells and the preliminary positive efficacy observed in the first subject further reinforce our confidence in the future of allogeneic CAR-T products development. We extend our sincere gratitude to the investigators for their invaluable collaboration and to the patients for participating in this trial—efforts that accelerate our mission to address unmet medical needs," said Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics.

About THANK-u Plus™

CARsgen has developed the THANK-u Plus™ platform as an enhanced version of its proprietary THANK-uCAR^® allogeneic CAR-T technology to address the potential impact of NKG2A expression levels on therapeutic efficacy. THANK-u Plus™ demonstrates sustained expansion regardless of varying NKG2A expression levels on NK cells and exhibits significantly improved expansion compared to THANK-uCAR^®. Preclinical studies show that THANK-u Plus™ delivers superior antitumor efficacy in the presence of NK cells compared to THANK-uCAR^®. Allogeneic BCMA or dual-targeting CD19/CD20 CAR-T cells developed using this platform exhibit robust antitumor activity in the presence of NK cells, indicating that THANK-u Plus™ has broad potential for developing diverse allogeneic CAR-T therapies.

About CARsgen Therapeutics Holdings Limited

CARsgen is a biopharmaceutical company with operations in China and the U.S., focusing on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. CARsgen has established a comprehensive CAR T-cell research and development platform that covers target discovery, innovative CAR T-cell development, clinical trials, and commercial-scale production. Internally, CARsgen has developed novel technologies and a product pipeline with global rights to address significant challenges faced by existing CAR T-cell therapies. Efforts include improving safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs. CARsgen's mission is to become a global biopharmaceutical leader that provides innovative and differentiated cell therapies for cancer patients worldwide and makes cancer curable.

Forward-looking Statements

All statements in this press release that are not historical fact or that do not relate to present facts or current conditions are forward-looking statements. Such forward-looking statements express the Group's current views, projections, beliefs and expectations with respect to future events as of the date of this press release. Such forward-looking statements are based on a number of assumptions and factors beyond the Group's control. As a result, they are subject to significant risks and uncertainties, and actual events or results may differ materially from these forward-looking statements and the forward-looking events discussed in this press release might not occur. Such risks and uncertainties include, but are not limited to, those detailed under the heading "Principal Risks and Uncertainties" in our most recent annual report and interim report and other announcements and reports made available on our corporate website, https://www.carsgen.com. No representation or warranty is given as to the achievement or reasonableness of, and no reliance should be placed on, any projections, targets, estimates or forecasts contained in this press release.

SOURCE CARsgen Therapeutics