CARsgen Therapeutics Holdings Limited announced that the United States Food and Drug Administration 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. 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. 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.

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.