FDA grants RMAT designation to CAR T-cell therapy for advanced gastric cancers
The FDA granted regenerative medicine advanced therapy designation to CT041, a chimeric antigen receptor T-cell therapy for adults with relapsed or refractory gastric adenocarcinoma or gastroesophageal junction adenocarcinoma.
CT041 (CARsgen Therapeutics) is an autologous, gene-edited CAR T-cell therapy that targets claudin 18.2, a stomach-specific isoform of claudin 18 that is highly expressed in gastric adenocarcinoma.
The FDA previously granted orphan drug designation to the investigational therapy for the same indications.
The agency based the regenerative medicine advanced therapy (RMAT) designation on results from an ongoing phase 1B open-label, multicenter clinical trial that evaluated the safety and efficacy of CT041 among patients with advanced gastric, gastroesophageal or pancreatic adenocarcinoma whose tumors express claudin 18.2.
Study results presented at last year’s European Society for Medical Oncology Congress — based on median follow-up of 7.6 months — showed an overall response rate of 48.6% (95% CI, 31.9-65.6) for all study patients and 57.1% (95% CI, 37.2-75.5) for patients with gastric cancers.
Researchers noted an ORR of 61.1% among 18 patients with gastric cancers who received the lowest dose level of 2.5 × 108 cells, with a median PFS of 5.4 months (95% CI, 2.6-not estimable) and OS of 9.5 months (95% CI, 5.2-not estimable).
No dose-limiting toxicities and no cases of grade 3 or greater cytokine release syndrome or neurotoxicity occurred during the study.
CARsgen plans to open a pivotal phase 2 clinical trial of CT041 in North America this year, according to a company-issued press release.
The FDA’s RMAT designation program, part of the 21st Century Cures Act, is designed to expedite review of regenerative medicine therapies intended to treat, modify, reverse or cure serious or life-threatening diseases or conditions. Preliminary clinical evidence must indicate the therapy has the potential to address unmet medical needs.
Qi C, et al. Abstract 1372O. Presented at: European Society for Medical Oncology Congress (virtual meeting); Sept. 17-21, 2021.