Immix and Nexcella Make Progress in US Trial for Light Chain Amyloidosis CAR-T NXC-201
NEXICART-2 has moved on to its expansion cohort after completing dosing of patients in its initial cohort.
The phase 1b/2 NEXICART-2 clinical trial (NCT06097832), which is evaluating Immix Biopharma and its subsidiary Nexcella's NXC-201, an investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of relapsed/refractory (r/r) light chain (AL) amyloidosis has moved onto its expansion cohort after completing dosing of patients in its initial cohort.1
The study’s initial cohort treated 3 patients at a dose of 150 million CAR+T cells. The expansion cohort, on the other hand, is utilizing a dose of 450 million CAR+T cells. The expansion cohort will sequentially dose 3 patients 28 days apart from one another, after which dosing of multiple patients per month will become possible. NEXICART-2 is the first study to evaluate NXC-201 in the United States; although, the product is also being assessed 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.2
“We are pleased to report that the NEXICART-2 study is making excellent progress,” Ilya Rachman, MD, PhD, the chief executive officer of Immix Biopharma, said in a statement.1 “We are now one step closer to providing a new treatment option for patients with r/r AL Amyloidosis, where no FDA drugs are approved today. Robust enrollment reflects the enthusiasm of clinical investigators for CAR-T NXC-201.”
Immix originally announced that the first patient in NEXICART-2 had been dosed in July 2024.3 The primary end points for the study are the complete response (CR) rate and the overall response rate. The trial is to be carried out at multiple locations, with Memorial Sloan Kettering Cancer Center being the lead site and the location of the first patient’s dosing.1
Immix previously presented data from NEXICART-1 at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.4 As of the update, 13 patients with AL amyloidosis have been treated in the trial, at doses of 150x106 (n = 1), 450x106 (n = 2), and 800x106 (n = 10). 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,” Gabriel Morris, the chief financial officer of Immix Biopharma, added to the statement.1 “We credit the resolute efforts of our investigators, sites, and team as we continue on track for interim and final read-outs.”
Notably, the NEXICART-1 trial includes patients with multiple myeloma (MM) in addition to the aforementioned patients with AL amyloidosis.5 The US investigational new drug (IND) clearance for NXC-201, which occurred in November 2023, was preceded by several preliminary steps announced by Nexcella earlier that year; 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.6,7 NXC-201 has been granted orphan drug designation by the FDA for both MM and AL amyloidosis.8
NXC-201 targets B-cell maturation antigen.1 CGTLive® previously interviewed Ahmad Masri, MD, MS, a cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University, about the idea behind the cell therapy’s approach.
“The whole concept is that if you're able to identify an antigen on the plasma cells, then you are able to use a cell therapy approach in order to deplete and decrease those plasma cells,” Masri told CGTLive. “So, you essentially are able to attack them and deplete them at an earlier stage.”
