CARsgen Presents First-in-human Results of GPRC5D Targeting CAR-T CT071 at EHA 2024 By Investing.com

Shanghai, June 15, 2024 /PRNewswire/ — CARsgen Therapeutics Holdings Limited (ticker symbol: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematological malignancies and solid tumors, announces that preliminary results from the ongoing first-in-human study of CT071 have been presented at the conference. The twenty-ninth annual conference of European Society of Hematology (four).

Preliminary results from phase 1 of CT071 (NCT05838131) were presented as a poster at the 29th Annual Environmental Health Association (EHA) Conference on June 14, 18:00 – 19:00 CESTWhich was titled “First-in-human study of GPRC5D-targeted CAR T cells (CT071) with accelerated manufacturing in patients with relapsed/refractory multiple myeloma (RRMM).”⁽¹⁾

“Multiple myeloma is a common but incurable hematological malignancy with significant unmet need. Despite numerous recent advances, most patients relapse and become resistant to available treatments, and thus new treatments are needed.” GPRC5D, a protein that is expressed in Significant on the surface of malignant plasma Cells with limited expression on normal tissues represent a promising target for the treatment of multiple myeloma CT071 is an autologous second-generation CAR T cell product that targets GPRC5D and was manufactured using accelerator CARcelerate.^™ The platform shortens manufacturing time to approximately 30 hours, supporting shorter vein-to-vein time and younger T cells. “Preliminary results of the ongoing study presented at EHA show that CT071 has the potential to be a best-in-class GPRC5D targeting CAR-T therapy.” Rafael BavaMD, PhD, Chief Medical Officer of Carsgen treatments. “We are excited about the development of CT071 and look forward to sharing future updates with the medical community.”

From February 28, 2024In 10 patients, CT071”7 was dosed at 1.0 x 10⁵ cells/kg and 3 patients at 3.0 x 10⁵ cells/kg. Among them, 80% had high-risk cytogenetics, 30% had one or more extramedullary plasmacytoma (EMD), and 40% had stage III R-ISS. This was a highly pretreated population with a median of 5 prior lines of therapy, including 90% double-grade refractory, 70% triple-refractory, and 40% penta-grade refractory. 50% received autologous stem cell transplantation, and 20% had previously been treated with BCMA/CD19 dual-targeting CAR T cells. None of the patients in the study required bridging therapy due to the rapid manufacturing turnaround.

Median follow-up at the time of data cutoff was 4.07 months (range: 2.8-7.4). There were no grade 3 or higher cytokine release syndrome (CRS) events. No immune cell-associated neurotoxicity syndrome (ICANS) was observed. No adverse events of particular interest or dose-limiting toxicities (DLT) occurred. Four patients experienced treatment-related SAEs, including pneumonia (n = 1), decreased appetite (n = 1) and thrombocytopenia (n = 2), and all of them recovered.

The overall response rate was 90%, including 5 patients (50%) with a strict complete response (sCR), 2 patients (20%) with a very good partial response (VGPR), and 2 patients (20%) with a partial response (PR). . All nine patients who had an evaluable MRD at week 4 achieved MRD negativity (10^-6 threshold) ), including all five patients with sCR/CR. Pharmacokinetic analysis showed robust cell expansion and viability, with a mean T_{the above} 14 days (range: 12-28) and median C_{the above} of 32280.5 copies/g gDNA (range: 8372-106060).

About CT071
CT071 is a CAR T cell therapy candidate developed with CARsgen's proprietary CARcelerate.^™ A platform targeting GPRC5D for the treatment of R/R MM or relapsed/refractory plasma cell leukemia (“R/R PCL”). The investigator-initiated trial (NCT05838131) is ongoing China To evaluate the safety and efficacy of CT071 for the treatment of R/R MM or R/R PCL. Another investigator-initiated trial (NCT06407947) is ongoing China For the treatment of newly diagnosed multiple myeloma (NDMM).

on CARsgen Therapeutics Holdings Limited
CARsgen is a biopharmaceutical company operating in China And the we It focuses on innovative CAR T-cell therapies for the treatment of hematological malignancies and solid tumors. CARsgen has established a comprehensive CAR T cell R&D platform, spanning target discovery, innovative CAR T cell development, clinical trials, and commercial-scale production. CARsgen has developed novel technologies and a product pipeline with global rights to address key challenges of CAR T-cell therapies, such as improving the safety profile, enhancing efficacy in solid tumors and reducing treatment costs. CARsgen's mission is to become a global leader in biopharmaceuticals that deliver innovative, differentiated cell therapies to cancer patients worldwide and make cancer curable.

Forward-looking statements
All statements in this press release that do not represent historical fact or that do not relate to current facts or current circumstances are forward-looking statements. These forward-looking statements express the Group's current views, expectations, beliefs and expectations with respect to future events as of the date of this press release. These 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 may not occur. These risks and uncertainties include, but are not limited to, those detailed under “Principal Risks and Uncertainties” in our most recent Annual Report and Interim Report and other announcements and reports available on our corporate website, https://www. carsgen.com. No representation or warranty is made as to completion or reasonableness, and no reliance should be placed on any expectations, targets, estimates or forecasts contained in this press release.

reference
(1) Jie Du, et al. EHA 2024. June 2024; Poster p. 941