CARsgen Announces CT041 CAR T-cell Product Candidate Granted RMAT Designation by the FDA

SHANGHAI,Jan. 10,2022 -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK),a company mainly focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors,today announced that the United States Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CT041 for the treatment of patients who have advanced gastric or gastroesophageal junction adenocarcinoma (GC/GEJ) with Claudin18.2-positive tumor. CT041 is the first CAR T-cell product candidate against solid tumor with RMAT designation*,according to public resources.

With the RMAT designation,CT041 enjoys all the benefits of Fast Track and Breakthrough Therapy designations including early and intensive FDA guidance on efficient product candidate development and eligibility for rolling review and priority review. Products granted RMAT designation may also be eligible for accelerated approval.

CT041 is an autologous CAR T-cell product candidate developed by CARsgen,which has the potential to be the first-in-class globally. As of the date of this announcement,CT041 is the only CLDN18.2-targeted CAR T-cell product candidate globally that is being studied in clinical trials with investigational new drug (IND) or clinical trial application approvals from the FDA,the NMPA,and Health Canada.

In October 2019,CARsgen's CT053 CAR T-cell product candidate was first granted RMAT designation by the FDA for the treatment of relapsed and/or refractory multiple myeloma. CT041 is CARsgen's second product candidate with RMAT designation.

CARsgen currently has 11 in-house developed product candidates and obtained 8 IND approvals for CAR T-cell therapies. In addition to the existing clinical pipeline product candidates,CARsgen continues to develop innovative technologies,such as CycloCAR technology to enhance efficacy of CAR T cells against solid tumors,and allogeneic THANK-uCAR technology to reduce costs and increase affordability. As of December 31,2021,CARsgen owns over 300 patent applications and 65 issued patents in more than 19 countries and regions,including the United States,Europe,China,and Japan.

Dr. Zonghai Li,Founder,Chairman of the Board,Chief Executive Officer,and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited,stated that,"The RMAT designation indicates that CT041 has the potential to solve the unmet medical needs of patients with GC/GEJ. RMAT is not only important for accelerating product development and the review of the biologics license application,but also helps patients obtain this advanced therapy as soon as possible. We hope that the RMAT and PRIME designations obtained by CT041 will enable us to work closely with the FDA and the EMA,to obtain approvals from regulatory agencies globally,and thus benefit patients worldwide as soon as possible."

About CT041

CT041 is an autologous CAR T-cell product candidate developed by CARsgen. CT041 targets the treatment of CLDN18.2-positive solid tumors with a primary focus on GC/GEJ and pancreatic cancer (PC). CT041 has demonstrated promising therapeutic efficacy and favorable safety in ongoing clinical trials. CT041 has the potential to become a backbone therapy for GC/GEJ and PC and benefit a large population of patients worldwide.

According to the results of the investigator-initiated trial of CT041 that was orally presented at the European Society of Medical Oncology Congress 2021 (ESMO 2021),18 patients with GC/GEJ who failed at least two prior lines of therapy (including 8 [44%] patients exposed to an anti-PD-1 or PD-L1 antibody) at the dose of 2.5×108 CAR T cells achieved an objective response rate (ORR) of 61.1%. Historical data shows that for patients with GC/GEJ who fail at least two prior lines of therapy,the efficacy rate of chemotherapy is about 4% to 8%,and the efficacy rate of anti-PD-1 antibody is about 11%. Therefore,compared with other treatments for patients with GC/GEJ who fail at least two prior lines of therapy,CT041 shows a significant improvement in ORR.

In addition to the investigator-initiated trials in China,CARsgen has initiated a Phase Ib/II clinical trial for advanced GC/GEJ and PC in China and a Phase 1b clinical trial for advanced gastric or pancreatic adenocarcinoma in North America. CARsgen also intends to conduct a pivotal Phase 2 clinical trial in North America in 2022.

In 2020 and 2021,CT041 received Orphan Drug designation from the FDA for the treatment of GC/GEJ and Orphan Medicinal Product designation from the European Medicines Agency (EMA) for the treatment of advanced gastric cancer. In November 2021,CT041 was granted Priority Medicines (PRIME) eligibility by the EMA for the treatment of advanced gastric cancer.

About Regenerative Medicine Advanced Therapy Designation

The RMAT designation program is intended to help the FDA facilitate an efficient development program for any drug that (1) qualifies as a RMAT,which is defined as a cell therapy,therapeutic tissue engineering product,human cell and tissue product,or any combination product using such therapies or products; (2) is intended to treat,modify,reverse,or cure a serious or life-threatening disease or condition; and (3) has preliminary clinical evidence to indicate the drug has the potential to address unmet medical needs for such a disease or condition.

About CARsgen Therapeutics Holdings Limited

CARsgen is a biopharmaceutical company with operations in China and the U.S. mainly 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. Our vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

Reference

What Is An RMAT? List of RMAT Designations. Retrieved January 10,2022,from https://bioinformant.com/rmat/#list

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