Immix Biopharma’s CAR-T NXC-201 Snags FDA RMAT Designation for Light Chain Amyloidosis
The therapy is currently being evaluated for AL amyloidosis in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832) in the United States.
Immix Biopharma and its subsidiary Nexcella's NXC-201, an investigational autologous BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, has received regenerative medicine advanced therapy (RMAT) designation from the FDA for the treatment of relapsed/refractory (r/r) light chain (AL) amyloidosis.1
The therapy is currently being evaluated for AL amyloidosis in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832) in the United States, which moved onto its expansion cohort in October of last year.1,2 NEXICART-2 is expected to enroll approximately 40 patients in total and is taking place at study sites in California, Michigan, New York, and Ohio.
Notably, NXC-201 is also being assessed for the treatment of both AL amyloidosis and multiple myeloma (MM) in the ongoing phase 1a/1b NEXICART-1 clinical trial (NCT04720313), which began as a single-center study at the Hadassah University Hospital in Jerusalem, Israel. The FDA’s decision regarding the RMAT designation was informed by “proof-of-concept" data from NEXICART-2.1
“We are also pleased to report that the pace of enrollment in NEXICART-2 has accelerated, following successful completion of the safety run-in segment,” Gabriel Morris, the chief financial officer of Immix Biopharma, said in a statement.1 “We look forward to sharing further information on our progress, including an update on NEXICART-2, in the first half of 2025.”
In December 2024, Immix announced data from the first 4 patients to have been dosed in NEXICART-2.3 The company highlighted that all 4 of the patients had responses sustained as of the November 14, 2024, data cut-off. Two of the patients achieved complete responses by this time, whereas the other 2 patients achieved minimal residual disease negativity in the bone marrow (10-6). Furthermore, 1 patient showed an improvement from New York Heart Association (NYHA) class II to class I at 14 days posttreatment.
“Today’s announcement represents a major step forward for Immix,” Ilya Rachman, MD, PhD, the chief executive officer of Immix Biopharma, said in a November 2024 statement.3 “We believe this first data cut of US study NEXICART-2 including rapid and deep responses validates our strategy to focus on relapsed/refractory AL Amyloidosis patients with preserved heart function, who will benefit most from NXC-201 treatment.”
Immix previously presented data from 13 patients with AL amyloidosis treated at various dose levels in NEXICART-1 at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.4 The overall hematological response rate was 92% (12 of 13 patients), with 69% of patients (9 of 13) achieving a complete hematological response.
“Positive data from our ex-US study of NXC-201, the largest CAR-T clinical study in relapsed/refractory AL Amyloidosis to-date, showed a 92% overall response rate and a 28.0 month duration of response (best responder), presented at ASGCT 2024,” Morris said in an October 2024 statement.2 “We credit the resolute efforts of our investigators, sites, and team as we continue on track for interim and final read-outs.”
In addition to the RMAT designation, NXC-201 has previously received orphan drug designation from the FDA for both AL amyloidosis and MM.5 The investigational new drug (IND) application for NEXICART-2 was cleared by the FDA in November 2023.6 The US IND clearance for NXC-201 was preceded by several preliminary steps announced by Nexcella earlier in 2023; these included completion of a preIND meeting with the FDA in June and finishing manufacturing of the first engineering batch for NXC-201 in the US in July.7,8
