CARsgen® Announces 2024 Interim Results

SHANGHAI, Aug. 29, 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, has announced its 2024 Interim Results.

Business Highlights

• Zevor-cel was approved by NMPA in China.
• Patient enrollment for the confirmatory Phase II trial of satri-cel (CT041) in China has been completed.
• Multiple adjuvant studies for solid tumor CAR T-cell therapies are underway.
• Clinical data on satri-cel, zevor-cel, and CT071 were presented at academic conferences.
• Rapid development of differentiated allogeneic CAR T-cell product pipelines.

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "In the past six months, we have made significant progresses in technological innovation, product development, and business operations. Zevor-cel was successfully launched in China and has been included in nearly 20 provincial and municipal healthcare plans. The pivotal Phase II trial of satri-cel in China has completed patient enrollment. We are also actively exploring the potential of satri-cel in adjuvant therapy, aiming for greater clinical benefit for patients. Additionally, we are rapidly advancing CT071, which is manufactured with our CARcelerate^® platform, while continuing to develop multiple allogeneic CAR T-cell products based on the THANK-uCAR^® platform."

1. Zevor-cel Market Launch and Commercialization Progress in China

Zevor-cel (zevorcabtagene autoleucel, CT053) is a fully human autologous CAR T-cell product targeting B-cell maturation antigen (BCMA). It was approved by the National Medical Products Administration (NMPA) on February 23, 2024 for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least three lines of therapy, (including a proteasome inhibitor and an immunomodulatory agent). CARsgen has been collaborating with Huadong Medicine for the commercialization of zevor-cel in mainland China. As of July 31, 2024, zevor-cel had been included in nearly 20 provincial or municipal government-subsidized insurance programs and private health insurance products; certification and regulatory filings had been completed in over 100 healthcare institutions across 19 provinces or cities nationwide; we had received a total of 52 orders from Huadong Medicine.

The Group's revenue was over RMB6 million for the six months ended June 30, 2024 mainly from zevor-cel in which the primary revenue of zevor-cel was calculated on the basis of ex-works price, rather than on the basis of end-of-market prices. Our revenue is recognized upon completion of ex-works delivery of products. Besides, the Company received a milestone payment of RMB75 million from Huadong Medicine for zevor-cel for the six months ended June 30, 2024. Due to the inherent time cycle of CAR-T manufacturing, this results in a discrepancy between the number of orders obtained from Huadong Medicine and number of ex-works deliveries.

2. Progresses in Development of Differentiated CAR T Pipeline

Satricabtagene autoleucel (CT041) is an autologous humanized CAR T-cell product targeting Claudin18.2. Patient enrollment for the confirmatory Phase II trial (NCT04581473) in China for the treatment of gastric cancer/gastroesophageal junction cancer (GC/GEJ) has been completed. Our collaboration with Moderna, Inc. is ongoing to investigate Moderna's investigational Claudin18.2 mRNA product in combination with satri-cel in preclinical studies.

CARsgen is actively expanding the application of CAR T-cell therapies in post-surgery treatments for solid tumors, including an ongoing Phase I clinical trial for pancreatic cancer (CT041-ST-05, NCT05911217), an investigator-initiated trial that is currently in the start-up phase for GC/GEJ, and a Phase I clinical trial for hepatocellular carcinoma (CT011-HCC-03, NCT06560827).

In the hematological malignancies field, the focus of the pipeline includes CT071, a fully human autologous CAR T-cell product targeting GPRC5D. CT071 was developed using CARsgen's CARcelerate^® platform for the treatment of MM and primary plasma cell leukemia (pPCL). CARcelerate^® is a proprietary platform developed by CARsgen that shortens the manufacturing time to approximately 30 hours, resulting in younger and potentially more potent CAR T cells compared to conventional manufacturing processes. An investigator-initiated trial (NCT05838131) for R/R MM and R/R PCL and another investigator-initiated trial (NCT06407947) for the treatment of newly diagnosed multiple myeloma (NDMM) are currently underway in China. An IND was cleared by the FDA in November 2023 for the treatment of patients with R/R MM and R/R pPCL.

In addition to autologous products, CARsgen is also advancing differentiated allogeneic CAR T-cell products based on the THANK-uCAR^® platform. These include: CT0590, for the treatment of R/R MM and PCL; KJ-C2320, for the treatment of acute myeloid leukemia (AML); KJ-C2219, targeting CD19 and CD20, for the treatment of B-cell-related hematologic malignancies and autoimmune diseases; and KJ-C2114, for the treatment of solid tumors.

3. Clinical Data Disclosure

Updated results of the pivotal Phase II registrational trial of zevor-cel in China were reported as an oral presentation at the European Hematology Association (EHA) 2024 annual meeting. Updated data from the investigator-initiated trial (CT041-CG4006, NCT03874897) of satri-cel were published in Nature Medicine in June and presented orally at the 2024 American Society of Clinical Oncology (ASCO) annual meeting. A summary of safety and efficacy in patients with refractory metastatic pancreatic cancer (PC) (CT041-CG4006 & CT041-ST-01 Ib) was published in Journal of Clinical Oncology. The results from the satri-cel phase 1b clinical trial (CT041-ST-02, NCT04404595) in the U.S. were presented at the 2024 ASCO GI meeting. Results from the investigator-initiated trial (NCT05838131) of CT071 for the treatment of R/R MM were presented as a poster at the EHA 2024 annual meeting. Data updates for CT071 and CT0590 are to be disclosed in the second half of 2024.

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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SOURCE CARsgen Therapeutics