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, 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

Source:
https://www.cgtlive.com/view/immix-biopharma-car-t-nxc-201-snags-fda-rmat-designation-light-chain-amyloidosis

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