Immix Biopharma Moves CAR-T NXC-201 to Expansion Dose in U.S. AL Amyloidosis Trial NEXICART-2

Immix Biopharma, Inc., a clinical-stage biopharmaceutical company focused on developing cell therapies for AL Amyloidosis and other selective immune-mediated diseases, has reported advancements in its NEXICART-2 clinical study. This study evaluates the efficacy and safety of the CAR-T cell therapy, NXC-201, specifically for relapsed or refractory AL Amyloidosis, a severe plasma cell disorder affecting approximately 33,000 individuals in the United States. NXC-201 remains the only one-time CAR-T treatment option currently being studied in U.S. clinical trials for this condition.

Dr. Ilya Rachman, CEO of Immix Biopharma, expressed satisfaction with the study’s progress, emphasizing the potential to offer a new treatment for patients with relapsed/refractory AL Amyloidosis—a condition for which no FDA-approved drugs presently exist. The enthusiasm of clinical investigators for the CAR-T NXC-201 is reflected in the strong patient enrollment numbers.

Gabriel Morris, the company's Chief Financial Officer, highlighted the positive data from an ex-U.S. study of NXC-201, which is considered the largest CAR-T clinical study in relapsed/refractory AL Amyloidosis to date. This study demonstrated a 92% overall response rate and a 28-month duration of response for the best respondent. Morris credited the success to the dedication of the investigators, clinical sites, and the company’s team, as they move towards interim and final data readouts.

The NEXICART-2 study aims to assess the safety and efficacy of NXC-201 in patients with relapsed/refractory AL Amyloidosis who have not been previously treated with BCMA-targeted therapy and possess adequate cardiac function. The study has advanced to the second dosing cohort, which involves administering 450 million CAR-T cells to each patient every 28 days. This dosage has already shown complete responses in previous clinical studies.

The NEXICART-2 study is designed as an open-label, single-arm, multi-site U.S. Phase 1b/2 dose expansion trial. It will enroll 40 patients, beginning with a six-patient safety run-in to evaluate two dosage levels (150 million and 450 million CAR-T cells), with subsequent dose expansion at the higher dose. The primary endpoints of the study are the complete response rate and overall response rate, following consensus recommendations.

NXC-201, the therapy under investigation, is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. Initial clinical data from the ex-U.S. Phase 1b/2 study, NEXICART-1, indicated a high overall and complete response rate, with no neurotoxicity and a brief duration of cytokine release syndrome. These results were presented at the American Society of Hematology (ASH) and the American Society of Gene and Cell Therapy (ASGCT) annual meetings.

In addition to its potential in treating AL Amyloidosis, NXC-201 is also being considered for extension into other immune-mediated diseases. The comprehensive clinical development program for NXC-201 is supported by robust data and has earned Orphan Drug Designation (ODD) from both the U.S. FDA and the EMA in the EU.

AL Amyloidosis is characterized by abnormal plasma cells in the bone marrow producing misfolded amyloid proteins that accumulate in vital organs, leading to significant damage and high mortality rates. The condition's prevalence in the U.S. is estimated to be increasing at a rate of 12% annually, potentially affecting around 33,277 patients by 2024.

Market analysis shows that the Amyloidosis market, valued at $3.6 billion in 2017, is projected to grow to $6 billion by 2025. Immix Biopharma, with its lead candidate NXC-201, is poised to play a significant role in addressing this growing medical need.