The latest data set includes 22 newly evaluable patients with triple-refractory disease.
NXC-201 (Nexcella), a BCMA-targeted chimeric antigen receptor (CAR) T-cell therapy, yielded a 90% overall response rate (ORR) in participants with relapsed/refractory multiple myeloma (R/R MM) treated with the therapeutic dose of the therapy in the phase NEXICART-1 trial (NCT04720313).
"I am pleased to present promising NXC-201 efficacy data from our interim Phase 1 dataset, which brings us one step closer to meeting an urgent need for greater access to CAR-T therapies that can considerably shorten treatment waiting time for patients with relapsed or refractory multiple myeloma,” investigator Polina Stepensky, MD, director, Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, Hadassah Medical Organization, said in a statement. “Also encouraging is that we have not yet reached median progression free survival or overall survival, which means that not only could we improve patient outcomes with efficacy, but we may be able to extend lives as well. I look forward to continuing to enroll patients in our ongoing NXC-201 clinical trial.”
The data presented, by Stepensky at the European Society for Blood and Marrow Transplantation and European Hematology Association 5th European CAR T-cell Meeting, in Rotterdam, Netherlands, during February 9-11, 2023, are from 42 participants with triple-class refractory disease, 23 of which are newly evaluable since last presentation. One of these participants was treated with NXC-201 for light chain amyloidosis.
Participants received 150 million (n=6), 450 million (n=7), or dose-expansion, recommended phase 2 dose (RP2D) of 800 million CAR T cells (n=29). Median follow-up of all participants was 146 days (range, 18-314). Across all doses, 35 participants (83%) responded, 21 (50%) achieved a complete response (CR) or stringent CR, 34 (81%) achieved at least a very good partial response (VGPR), and 1 (2%) achieved a PR. Participants treated with the RP2D had a 90% ORR, a 59% CR rate or stringent CR rate, an 86% VGPR rate, and a 4% PR rate. The therapy has been well-tolerated, with no neurotoxicity and low-grade cytokine release syndrome with a median duration of 2 days (range, 1-7).
Nexcella stated that NXC-201 is structurally designed to cause lower toxicities than traditional CAR T-cell therapies. The ongoing phase 1b of the study is characterizing the safety and confirming the maximum tolerated dose and RP2D. The phase 2 portion of the study will primarily evaluate overall survival, progression-free survival, and response rates.
"We are excited to present 42 patients of NXC-201 clinical trial data in Rotterdam. As of the data cutoff, 29 patients have been treated at our identified recommended phase 2 dose. We plan to continue to enroll additional NXC-201 patients at RP2D in Israel and expand to United States clinical trial sites,” Gabriel Morris, president, Nexcella, added to the statement.