SHANGHAI, July 19, 2022 — 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 results of the investigator-initiated phase I clinical trials of CT053 have been published in Haematologica (https://haematologica.org/article/view/haematol.2022.280629).
The results are from a publication titled “A novel BCMA CAR-T-cell therapy with optimized human scFv for treatment of relapsed/refractory multiple myeloma: results from phase I clinical trials”. To improve safety, overcome limited efficacy, and reduce immunogenicity from non-human derived components, we developed autologous CAR-BCMA T cells (CT053) expressing the fully-human BCMA-specific scFv (25C2). Three investigator-initiated phase I studies investigated CT053’s safety, pharmacokinetics, and preliminary efficacy in patients with R/R MM (clinicaltrials.gov Identifiers: NCT03302403, NCT03380039, NCT03716856). We found that CT053 demonstrated an acceptable safety, pharmacokinetic, and efficacy profile. No ≥ Grade 3 cytokine release syndrome (CRS) or neurotoxicity was observed (4 Grade 1, 11 Grade 2). No immunogenicity was identified. As of the cutoff date of June 30, 2021, median follow-up time was 17.4 months (0.9–38.7 months), and ORR (partial response [PR] or better) was 87.5%, with 79.2% patients experiencing CR (12.5%) or stringent complete response (sCR, 66.7%). The CR/sCR rate was 70% for patients with EMD and 86% in patients without EMD. Median progression-free survival (PFS) was 18.8 months [95% CI, 10.1–not estimable (NE)] in all patients. Median overall survival (OS) was not reached. Median DOR was 21.8 months [95% CI: 9.2–NE] in all patients.
These studies demonstrated the strong efficacy and favorable safety profiles of CT053 in treating patients with R/R MM. It also indicated that selection of scFv for CAR T cells is critical to achieving better clinical outcomes.
About CT053
CT053 is an autologous CAR T-cell product candidate against BCMA being developed for the treatment of relapsed/refractory multiple myeloma (R/R MM). It incorporates a CAR construct engineered by CARsgen that features a fully human BCMA-specific single-chain variable fragment with lower immunogenicity and increased stability, which reduces the self-activation of CAR T-cells in the absence of tumor associated targets.
CARsgen has completed subject enrollment in the pivotal Phase II trial in China (LUMMICAR STUDY 1). In addition, CARsgen has started the pivotal Phase 2 clinical trial in North America (LUMMICAR STUDY 2) and treated the first subject in the pivotal Phase 2 trial in August 2021. As communicated with the U.S. FDA, the company is adding outpatient administration of CT053 into its U.S. clinical investigations.
CARsgen plans to make regulatory submissions for marketing approval to the NMPA in the third quarter of 2022 and plans to submit the BLA to the U.S. FDA in 2023. The company also plans to conduct additional clinical trials to develop CT053 as an earlier line of treatment for multiple myeloma.
Additional data update from the Phase I/II study in China (LUMMICAR STUDY 1) and an integrated analysis in participants with R/R MM by high-risk factors have been available as posters at the 2021 American Society of Hematology (“ASH”) Annual Meeting in December 2021.
CT053 received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019, as well as the PRIority MEdicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA) in 2019 and 2020, respectively. CT053 also received Breakthrough Therapy designation from the NMPA in 2020.
About CARsgen Therapeutics Holdings Limited
CARsgen is a biopharmaceutical company with operations in China and the U.S. and is focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The Company has built an integrated cell therapy platform with in-house capabilities that span target discovery, antibody development, clinical trials, and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors and reducing treatment costs. The Company’s vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.
Contact Us
For more information, please visit https://www.carsgen.com/
Contact CARsgen PR: pr@carsgen.com