Immix Biopharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted RMAT designation to sterically-optimized CAR-T NXC-201 for the treatment of relapsed/refractory AL amyloidosis. As of June 2024 public information, FDA approved less than half of RMAT applications submitted to the agency during the last eight years. FDA RMAT designation requires that a drug is an advanced regenerative medicine, targets a serious condition, with the potential to treat, modify, reverse, or cure, and preliminary clinical evidence has indicated that the drug has the potential to address these unmet medical needs. The RMAT designation is intended to accelerate the development and review of promising investigational products, including cell therapies. To qualify, a product must be designed to treat, modify, reverse, or cure a serious or life-threatening disease, with preliminary clinical evidence suggesting it can address unmet medical needs. The RMAT designation offers several key advantages, including early and frequent interactions with the FDA to discuss potential surrogate or intermediate endpoints, as well as strategies to meet post-approval requirements, potentially streamlining the path to market approval. NXC-201 is the only CAR-T therapy currently in development in AL Amyloidosis, mentioned in a review article entitled ?Systemic Light Chain Amyloidosis? published in June, 2024New England Journal of Medicine. About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. Initial data from Phase 1b/2 ex-U.S. study NEXICART-1 has demonstrated high complete response rates and no neurotoxicity of any kind in relapsed/refractory AL Amyloidosis. NXC-201 is being studied in a comprehensive U.S. clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, with the potential to expand into select immune-mediated diseases. The NXC-201 NEXICART-2 (NCT06097832) U.S. clinical trial builds on a robust clinical dataset. NXC-201 has been awarded Regenerative Medicine Advanced Therapy (RMAT) by the FDA, and Orphan Drug Designation (ODD) by the US FDA and in the EU by the EMA. About NEXICART-2
NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site U.S. Phase 1b/2 dose expansion clinical trial of CAR-T NXC-201 in relapsed/refractory AL Amyloidosis. NEXICART-2 is expected to enroll 40 patients with preserved heart function (excluding patients with pre-existing heart failure) who have not been exposed to prior BCMA-targeted therapy. The study is designed with a standard 6 patient safety-run in to evaluate two doses (three patients each at 150 million CAR+T cells and 450 million CAR+T cells) (both dose levels were evaluated in the NEXICART-1 study and have produced complete responses in relapsed/refractory AL Amyloidosis patients). The study aims to evaluate the safety and efficacy of NXC-201. Primary endpoints are complete response rate and overall response rate, according to consensus recommendations (Palladini et al. 2012). About AL Amyloidosis AL amyloidosis is caused by abnormal plasma cells in the bone marrow, which produce misfolded amyloid proteins that build-up in the heart, kidney, liver, and other organs. This build-up causes progressive and widespread damage to multiple organs, including heart failure, and leads to high mortality rates. The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at 12% per year according to Staron, et al Blood Cancer Journal, to approximately 33,277 patients in 2024. The Amyloidosis market was $3.6 billion in 2017, and is expected to reach $6 billion in 2025, according to Grand View Research.