CARsgen® to Present Zevor-cel, CT071 and CT0590 at ASH 2024 Annual Congress

SHANGHAI, Nov. 6, 2024 /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 Company will present the clinical data of zevorcabtagene autoleucel, CT071, and CT0590 as posters at the 66th Annual Congress of the American Society of Hematology ("ASH") which is due to take place between Dec 7 – 10, 2024. The abstracts are available on ASH official website.

"CARsgen is advancing therapeutic options for hematologic malignancies with robust CAR T-cell pipeline, which includes the autologous CAR T-cell therapy zevor-cel, the single-day-culture CT071, and the allogeneic CAR T-cell candidate CT0590. These initiatives underscore CARsgen's strong commitment to innovation in hematology. Leveraging proprietary technology platforms such as CARcelerate^® and THANK-uCAR^®, CARsgen is focused on developing differentiated CAR T-cell therapies to address the critical challenges faced by the clinical community. We are excited to share new data and are confident in the potential of our CAR T-cell therapies to benefit patients worldwide," said Raffaele Baffa, M.D., Ph.D., Chief Medical Officer of CARsgen Therapeutics.

Subgroup Analyses of Phase 2 Study: Evaluating the Efficacy of Fully Human BCMA-Targeting CAR T Cells (Zevorcabtagene Autoleucel) in Patients with Relapsed/Refractory Multiple Myeloma

Publication Number: 4762
Presentation Time: 6:00 PM - 8:00 PM, Monday, December 9, 2024 (PST)

In 102 patients with RRMM who had received at least 3 prior lines of therapy including an immunomodulatory drug and a proteasome inhibitor, the objective response rate (ORR) was 92.2%, the stringent complete response (sCR) or complete response (CR) was 71.6%. The ORR or the CR/sCR rate was not affected by any of the baseline characteristics tested. With a median follow-up of 20.3 (range: 0.4 to 27) months, the median duration of response (DOR), progression-free survival (PFS), and overall survival (OS) data were not mature and therefore, 18-month (18m) and estimated 30-month (30m) event free rates were used as efficacy outcomes for subgroup analyses. The DOR, PFS and OS were not impacted by age or ISS.

These subgroup analyses indicate that baseline characteristics have minimal impact on the clinical efficacy of zevorcabtagene autoleucel, demonstrating that even RRMM patients with poor prognostic factors can benefit from zevorcabtagene autoleucel.

GPRC5D-Targeted CAR T-Cell Therapy CT071 for the Treatment of Refractory/Relapsed Multiple Myeloma

Publication Number: 3451
Presentation Time: 6:00 PM - 8:00 PM, Sunday, December 8, 2024 (PST)

CT071 is a fully human GPRC5D-targeting autologous CAR T-cell product manufactured using an expedited CARcelerate^® platform which shortens the manufacturing process to around 30 hours resulting in shorter vein-to-vein time. Patients with RRMM who had previously received ≥ 3 prior lines of therapy (LOT) or patients who experienced progression or lack of response having been treated with a proteasome inhibitor and an immunomodulatory agent or those who were double class-refractory, all with ECOG score of 0-2 were enrolled. In 17 patients who had been dosed with CT071, 11 patients (64.7%) experienced cytokine release syndrome (CRS), all at Grade 1 (n=8) or 2 (n=3). No immune effector cell-associated neurotoxicity syndrome (ICANS) was observed. No dose limiting toxicity (DLT) occurred.

The overall response rate (ORR) was 94.1% (16/17), and the stringent complete response (sCR) rate was 52.9% (9/17). Notably, 7 patients achieved complete response or better at week 4. All 4 patients with previous exposure to BCMA or BCMA/CD19 CAR T responded to CT071 (2 with sCR and 2 with partial response).

A First-in-Human Study of CT0590, a Triple Knock-out, Allogeneic CAR T-Cell Therapy Targeting BCMA and NKG2A, in Subjects with Relapsed/Refractory Multiple Myeloma

Publication Number: 4843
Presentation Time: 6:00 PM - 8:00 PM, Monday, December 9, 2024 (PST)

CT0590 is an allogeneic dual CAR T-cell therapy deploying THANK-uCAR^® technology that targets both BCMA and NKG2A (a membrane protein expressed in NK and T cells), featuring a triple knockout for TRAC/B2M/NKG2A which mitigates against graft-versus-host disease (GvHD), host immune rejection and fratricide.

This is a first-in-human (FIH), open-label, single center, phase I study of CT0590 in subjects with RRMM to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of CT0590 (NCT05066022). As of 22-Apr-2024, 5 subjects were enrolled (4 subjects with RRMM and 1 subject with primary plasma cell leukemia [pPCL] under compassionate use).

No ≥ 3 grade cytokine release syndrome (CRS) was observed. No cases of immune effector cell-associated neurotoxicity syndrome (ICANS) or GvHD were observed. No dose limiting toxicities were reported and there were no withdrawal from the study or deaths due to adverse events.

With a median follow-up time of 16.6 months (range: 5.1, 24.2), 3 subjects achieved confirmed responses including 2 subjects with stringent complete response (sCR) and 1 subject with partial response (PR). The 2 subjects with sCR include1 RRMM subject [sCR ongoing] with a DOR longer than 23 months as of data cut-off date and 1 pPCL subject with a duration of response (DOR) of 20 months. In the 2 subjects with sCR, CAR copies peaked at > 280,000 copies/µg genomic DNA.

Preliminary results of this FIH study of the CT0590 allogeneic CAR T-cell therapy demonstrated a manageable safety profile while achieving deep and durable clinical responses.

About Zevorcabtagene Autoleucel

Zevorcabtagene autoleucel is a fully human, autologous BCMA CAR T-cell product for the treatment of Multiple Myeloma (MM). Zevorcabtagene autoleucel was approved by the NMPA on February 23, 2024 for the treatment of adult patients with R/R MM who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). CARsgen is conducting a separate Phase 1b/2 LUMMICAR STUDY 2 clinical trial in North America to evaluate the safety and efficacy of zevorcabtagene autoleucel in R/R MM.

Zevorcabtagene autoleucel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019, as well as Priority Medicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA) in 2019 and 2020, respectively.

About CT071

CT071 is a CAR T-cell therapy candidate developed utilizing the proprietary CARcelerate^® platform of CARsgen targeting GPRC5D for the treatment of relapsed/refractory MM or relapsed/refractory plasma cell leukemia (PCL). An IIT (NCT05838131) is ongoing in China to evaluate the preliminary safety and efficacy of CT071 for the treatment of patients with relapsed/refractory multiple myeloma or plasma cell leukemia. A separate investigator-initiated trial (NCT06407947) is ongoing in China for the treatment of patients with newly diagnosed multiple myeloma (NDMM).

About CT0590

CT0590 is a BCMA-targeting allogeneic CAR T-cell product candidate deploying CARsgen's THANK-uCAR^® technology. An IIT is ongoing in China to evaluate the preliminary safety and efficacy of CT0590 for the treatment of R/R MM.

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.

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