Immix Biopharma Begins Dosing in U.S. AL Amyloidosis Trial with CAR-T NXC-201

Immix Biopharma, Inc. (ImmixBio), a clinical-stage biopharmaceutical company, has announced the dosing of the first patient at Memorial Sloan Kettering Cancer Center (MSKCC) in its U.S. NEXICART-2 trial. This trial focuses on NXC-201, a BCMA-targeted CAR-T cell therapy designed to treat relapsed/refractory AL Amyloidosis patients with sufficient cardiac function who have not previously undergone BCMA-targeted therapies.

The NEXICART-2 study builds upon the promising results from the NEXICART-1 trial, an ex-U.S. study, which demonstrated a 92% overall response rate among relapsed/refractory AL Amyloidosis patients. Notably, the best responder had a 28-month duration of response. The findings from the NEXICART-1 study were presented at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT 2024).

Heather Landau, MD, the Amyloidosis Program Director at MSKCC and the principal investigator for NEXICART-2, highlighted the significance of initiating the CAR-T clinical trial for AL Amyloidosis patients who have not benefited from front-line daratumumab (DARZALEX)-combination therapy. Dr. Landau emphasized the potential of NXC-201 to offer a one-time therapeutic option for these patients, noting the lack of approved drugs for relapsed/refractory AL Amyloidosis.

Ilya Rachman, M.D., Ph.D., CEO of Immix Biopharma, expressed optimism about the initiation of the U.S. NEXICART-2 study, viewing it as a significant step forward for AL Amyloidosis treatment. Gabriel Morris, Chief Financial Officer of Immix Biopharma, praised the team's efforts in achieving the mid-2024 milestone for dosing the first patient.

NXC-201 is currently the only CAR-T therapy in development for AL Amyloidosis, as discussed in the June 2024 issue of the New England Journal of Medicine in an article titled "Systemic Light Chain Amyloidosis". The NEXICART-2 trial is an open-label, single-arm, multi-site Phase 1b/2 dose expansion clinical trial in the U.S. It aims to enroll 40 patients and evaluate the safety and efficacy of NXC-201. The trial includes a safety run-in with two dose levels (150 million and 450 million CAR+T cells) and the possibility of escalating to 800 million CAR+T cells, depending on patient responses. The primary endpoints are the complete response rate and overall response rate based on guidelines by Palladini et al. 2012.

The NEXICART-1 trial, an ex-U.S. Phase 1b/2a study, has already established the recommended Phase 2 dose for NXC-201. This trial, which includes patients with relapsed/refractory multiple myeloma and AL amyloidosis, has shown favorable safety and efficacy profiles, including the absence of severe neurotoxicity and a short duration of cytokine release syndrome (CRS).

AL Amyloidosis is a condition driven by abnormal plasma cells in the bone marrow, leading to the production of misfolded amyloid proteins that accumulate in various organs, causing significant damage and high mortality rates. The prevalence of relapsed/refractory AL Amyloidosis in the U.S. is increasing at an estimated rate of 12% per year, with the patient population projected to reach approximately 33,277 by 2024.

The amyloidosis market, valued at $3.6 billion in 2017, is expected to grow to $6 billion by 2025, as noted by Grand View Research. Immix Biopharma’s lead candidate, NXC-201, has received Orphan Drug Designation in both the U.S. and the EU for the treatment of AL Amyloidosis, reflecting its potential impact on this critical and underserved medical need.