China-based CARsgen Therapeutics Holdings Ltd presented the results of a multi-center, open-label Phase Ib/II study assessing the safety and efficacy of its B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T cell therapy CT053 (zevor-cel) at the 7th CAR-TCR Annual Summit. The study is being conducted across North America, focusing on patients with relapsed/refractory multiple myeloma (R/R MM).
Study Results and Highlights
As of August 31, 2022, 17 patients with R/R MM received zevor-cel in a Phase II trial with a median follow-up of 113 days (range: 9 to 373). Among 11 evaluable patients with at least 8 weeks of follow-up, including 4 patients with extramedullary disease (EMD), the objective response rate was 100% (very good partial response, complete response, or strict complete response), with deeper remission observed at longer follow-up. As all responses are ongoing, median progression-free survival, median survival, and median duration of response have not yet been reached, and CR/strict CR rates are not yet mature. All patients with MRD results at week 4 were MRD negative by next-generation sequencing.
Safety Profile
No deaths among enrolled patients occurred, nor did grade 3 or higher cytokine release syndrome. Cytokine release syndrome was observed in 10/17 (59%) patients, all grade 1 or 2.
Regulatory Milestones
CT053 obtained Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designation (ODD) statuses in the US in 2019, and Priority Medicine (PRIME) and ODD statuses in the European Union in 2019 and 2020 respectively. In addition, the drug, awarded breakthrough therapy designation (BTD) status in China in 2020, will be explored as an early treatment of MM.
Future Outlook
The positive results from the Phase Ib/II study underscore CARsgen Therapeutics’ commitment to advancing innovative treatments for R/R MM. With multiple regulatory designations and ongoing trials, CT053 is poised to address significant unmet needs in the treatment of multiple myeloma.-Fineline Info & Tech
