Immix Biopharma is making strides in the treatment of relapsed/refractory AL Amyloidosis with its CAR-T cell therapy, NXC-201. Recent data presented at the 66th American Society of Hematology (ASH) Annual Meeting and the FDA's RMAT designation highlight the potential of this therapy in an area with limited treatment options. The company's ongoing clinical trials and focus on patient safety and efficacy position NXC-201 as a promising candidate for addressing this challenging disease.
Promising Results from NEXICART-1
Updated results from the Phase 1b/2 NEXICART-1 study were presented at ASH 2024, showcasing a 75% complete response rate (12 out of 16 patients) in relapsed/refractory AL Amyloidosis patients. These patients had a median of four prior lines of therapy, indicating the advanced stage of their disease. The best responder in the study experienced a duration of response of 31.5 months, with the complete response ongoing as of December 9, 2024.
The safety profile of NXC-201 in the NEXICART-1 study was also notable. There were no reported cases of immune effector cell-associated neurotoxicity syndrome (ICANS). The median duration of cytokine release syndrome (CRS) was 2 days (range: 1-5), with no grade 4 CRS events reported. These findings suggest that NXC-201 has a manageable safety profile, which is crucial for patients with relapsed/refractory AL Amyloidosis who may have other health complications.
Advancing Clinical Development with NEXICART-2
Building on the positive results from NEXICART-1, Immix Biopharma is conducting the NEXICART-2 trial in the United States. This Phase 1b/2 trial is designed to evaluate NXC-201 in patients with relapsed/refractory AL Amyloidosis who have preserved heart function. The study has completed the six-patient safety run-in segment, and enrollment is now accelerating across U.S. study sites.
Early data from the first four patients in the NEXICART-2 trial showed that all patients normalized their disease markers within 30 days of treatment. Two patients achieved complete responses, and the other two were bone marrow minimal residual disease (MRD) negative. These early results are encouraging and support the continued development of NXC-201.
FDA Grants RMAT Designation
In a significant milestone, the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to NXC-201 for the treatment of relapsed/refractory AL amyloidosis. This designation is intended to accelerate the development and review of promising investigational products, including cell therapies. 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.
The Unmet Need in AL Amyloidosis
AL amyloidosis is a rare and life-threatening disease caused by abnormal plasma cells in the bone marrow. These plasma cells produce misfolded amyloid proteins that build up in organs such as the heart, kidney, and liver, leading to organ damage and failure. The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at 12% per year, reaching approximately 33,277 patients in 2024. There are currently no FDA-approved therapies for patients with relapsed/refractory AL Amyloidosis, highlighting the urgent need for new treatment options.
NXC-201: A Potential New Treatment Option
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. It is designed to target and destroy the abnormal plasma cells that produce amyloid proteins, thereby reducing the amyloid burden in affected organs. The initial data from the NEXICART-1 and NEXICART-2 trials suggest that NXC-201 has the potential to be a safe and effective treatment for patients with relapsed/refractory AL Amyloidosis.
With the RMAT designation from the FDA and the ongoing clinical trials, Immix Biopharma is committed to bringing this promising therapy to patients in need. The company plans to provide the next program update in the first half of 2025, with interim clinical data expected in Q2/Q3 2025 and final topline clinical data expected in Q2/Q3 2026.
