Company Announcements

SHANGHAI , March 18, 2025 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, announced its 2024 Annual Results.

Business Highlights

• Zevor-cel was approved by China'sNational Medical Products Administration (NMPA).
• As of December 31, 2024, certification and filings for zevor-cel completed in 23 provinces or cities, covering over 200 healthcare institutes; CARsgen has received a total of 154 valid orders from its commercialization partner Huadong Medicine.
• Satri-cel ST-01 confirmatory Phase II trial for gastric cancer in China: enrollment has been completed, and the study has met its primary endpoint. Satri-cel received BTD designation from CDE of China NMPA. CARsgen plans to submit a New Drug Application (NDA) to the China NMPA during the first half of 2025.
• Results from the investigator-initiated trial (IIT) of satri-cel were published in Nature Medicine and at the 2024 ASCO Annual Meeting.
• The results of the IIT of an allogeneic BCMA-targeting CAR-T product CT0590, which deploys the THANK-uCAR^® technology platform, were presented at the 2024 ASH Annual Meeting.
• Developed THANK-u Plus™ platform as an enhanced version of THANK-uCAR^® allogeneic CAR-T platform. Multiple allogeneic CAR-T products in development, covering treatment areas such as hematologic malignancies, solid tumors, and autoimmune diseases.
• Introduced Zhuhai SB Xinchuang to accelerate allogeneic CAR-T development in mainland China.
• Cash and bank balances were around RMB1,479 million as of December 31, 2024. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,080 million. Expect to have adequate cash into the 2028.

"In 2024, CARsgen achieved its first drug commercialization and made remarkable progresses in key pipeline products and technological innovations. Looking ahead, we will continue developing innovative CAR T-cell therapies to address the significant unmet medical needs," said Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics.

Zevor-cel: Approved by the NMPA and Launched in China

Zevorcabtagene autoleucel (zevor-cel, R&D code: CT053) is an autologous fully human CAR T-cell product against B-cell maturation antigen (BCMA). On February 23, 2024, zevor-cel was approved by China's NMPA for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). CARsgen entered into a collaboration agreement with Huadong Medicine for the commercialization of zevor-cel in mainland China. As of December 31, 2024, certification and regulatory filings for zevor-cel have been completed in 23 provinces or cities, covering over 200 healthcare institutes and we have received a total of 154 valid orders from Huadong Medicine.

Updated results of the pivotal Phase II registrational trial of zevor-cel in China were reported as an oral presentation at the 29th European Hematology Association (EHA) Annual Congress, and a subgroup analysis was presented a poster at the 66th ASH annual congress. We anticipate that growth of sales revenue of zevor-cel will further accelerate with continuous marketing activities and broader insurance coverage.

Satri-cel: Confirmatory Phase II trial Met Primary Endpoint, Plans to Submit an NDA

Satri-cel is an autologous CAR T-cell product candidate against the protein Claudin18.2 that has the potential to be first-in-class globally. Satri-cel targets Claudin18.2 positive solid tumors with a primary focus on gastric cancer/gastroesophageal junction cancer (GC/GEJ) and pancreatic cancer (PC). Patient enrollment has been completed in confirmatory Phase II trial in China (NCT04581473) in advanced GC/GEJ. The study has met its primary endpoint of a statistically significant improvement in progression-free survival (PFS) as assessed by the Independent Review Committee (IRC). Patients treated with satri-cel infusion achieved statistically significant improvement in PFS compared to those treated with physician's choice (paclitaxel, docetaxel, irinotecan, apatinib, or nivolumab). Based on the confirmatory Phase II clinical data, satri-cel has been granted Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of China NMPA.

CARsgen plans to submit an NDA to the NMPA in China during the first half of 2025. Satri-cel is expected to become the world's first CAR-T therapy for solid tumors to be approved for market.

Updated results from the investigator-initiated trial (CT041-CG4006, NCT03874897) were published in Nature Medicine in June 2024 and presented orally at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting in June 2024. Additionally, more research findings have been published in prestigious academic journals such as the Journal of Clinical Oncology and the Journal for ImmunoTherapy of Cancer.

A Phase I clinical trial for PC adjuvant therapy (CT041-ST-05, NCT05911217), and an investigator-initiated trial for GC/GEJ adjuvant therapy (CT041-CG4010, NCT06857786) are ongoing in China.

Advancing the Development of Allogeneic CAR-T Products

In addition to autologous products, CARsgen has also been advancing differentiated allogeneic CAR T-cell products utilizing the proprietary THANK-uCAR^® platform. CARsgen has recently 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.

The results of the IIT proof-of-concept study of an allogeneic BCMA-targeting CAR T-cell product candidate CT0590, which deploys the THANK-uCAR^® technology platform, were presented as a poster at the 66th ASH Annual Meeting in December 2024. The data indicates that CT0590 has achieved a DoR of over 20 months in patients with sCR, with a peak CAR copy number exceeding 280,000 copies/µg gDNA, which is comparable to autologous BCMA CAR-T therapies, preliminarily demonstrating the durability of its efficacy.

In addition, there are several universal CAR-T products under development, covering areas such as hematologic malignancies, solid tumors, and autoimmune diseases:

• CT059X: Targeting BCMA, for the treatment of R/R MM and R/R PCL. The first patient in the IIT achieved sCR at the Day-28 assessment.
• KJ-C2219: Targeting CD19/CD20, for the treatment of B-cell malignancies, as well as SLE and SSc. The IITs are ongoing.
• KJ-C2320: Targeting CD38, for the treatment of AML. The IIT is ongoing.
• KJ-C2114: For the treatment of solid tumors.
• KJ-C2526: Targeting NKG2DL, for the treatment of AML, other malignancies, and senescence.

On February 25, 2025, CARsgen announced reaching agreements with an investment fund managed by Zhuhai Hengqin SB Xinchuang Equity Investment Management Enterprise (Limited Partnership) ("Zhuhai SB Xinchuang") to jointly invest in UCARsgen Biotech Limited ("UCARsgen"), to accelerate allogeneic CAR-T development in mainland China. Under the agreements, UCARsgen has secured the exclusive rights in mainland China for the research, development, manufacture, and commercialization of the following allogeneic CAR-T products from CARsgen Therapeutics: the BCMA-targeted allogeneic CAR-T cell therapy for the treatment of multiple myeloma and plasma cell leukemia and the CD19/CD20 dual-targeted allogeneic CAR-T cell therapy for the treatment of B-cell malignancies. An investment fund managed by Zhuhai SB Xinchuang (currently undergoing registration and filing procedures) subscribed to the newly increased registered capital of UCARsgen for a consideration of RMB 80,000,000, thus retaining an 8% equity stake in the registered capital of UCARsgen upon completion of the transaction, equity stake of CARsgen Therapeutics in UCARsgen will be diluted from 100% to 92%.

Financial Highlights

The revenue was around RMB39.4 million for the year ended December 31, 2024 mainly from zevor-cel, in which was calculated on the basis of ex-works price, rather than 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 year ended December 31, 2024. Due to the inherent time cycle of CAR-T manufacturing, there is a discrepancy between the number of orders obtained from Huadong Medicine and number of ex-works deliveries. The gross profit was around RMB14.8 million for the year ended December 31, 2024, with the selling and distribution expenses around RMB0.88 million. In the commercialization stage, we are demonstrating a strong cost competitive advantage, which is mainly due to self-manufacture for plasmids and vectors with stable output and high yield per batch.

Cash and bank balances were around RMB1,479 million as of December 31, 2024. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,080 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.

About CARsgen Therapeutics Holdings Limited

CARsgen is a biopharmaceutical company focusing on developing innovative CAR T-cell therapies to address the unmet clinical needs including but not limited to hematologic malignancies, solid tumors and autoimmune diseases. CARsgen has established end-to-end capabilities for CAR T-cell research and development covering target discovery, preclinical research, product clinical development, and commercial-scale production. CARsgen has developed novel in-house technologies and a product pipeline with global rights to address challenges faced by existing CAR T-cell therapies. Efforts include improving safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs, etc. CARsgen's mission is to be a global biopharmaceutical leader that provides innovative and differentiated cell therapies for patients worldwide and makes cancer and other diseases 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.

Contact CARsgen

For more information, please visit https://www.carsgen.com/

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SOURCE CARsgen Therapeutics