Immix Biopharma Reports 92% Response Rate in AL Amyloidosis CAR-T Trial

Immix Biopharma announces positive clinical data from its Phase 1b/2a NEXICART-1 study of novel autologous CAR-T cell therapy NXC-201 in patients with relapsed/refractory AL Amyloidosis. The study demonstrates a 92% overall response rate and 100% response rate in patients without prior BCMA-targeted therapy exposure.

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Nitish Verma
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Immix Biopharma Reports 92% Response Rate in AL Amyloidosis CAR-T Trial

Immix Biopharma Reports 92% Response Rate in AL Amyloidosis CAR-T Trial

Immix Biopharma Inc. has announced positive new clinical data from its Phase 1b/2a NEXICART-1 study of novel autologous CAR-T cell therapy NXC-201 in patients with relapsed/refractory AL Amyloidosis (R/R ALA). The study, presented at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) in Baltimore, MD, demonstrated a remarkable 92% overall response rate (ORR) in the 13 patients enrolled.

Why this matters: This breakthrough in CAR-T cell therapy has the potential to significantly improve treatment options for patients with relapsed/refractory AL Amyloidosis, a devastating disease with high mortality rates. As the global Amyloidosis market is expected to reach $6 billion by 2025, this development could have far-reaching implications for the pharmaceutical industry and patient care.

Notably, 12 out of the 12 patients who had not been previously exposed to BCMA-targeted bispecific therapy responded to NXC-201 treatment, achieving a 100% ORR in thissubgroup. Among these responders, 9 patients (75%) achieved a complete response. The study's best responder has maintained an ongoing response for an impressive 28 months as of May 10, 2024.

"Relapsed/refractory AL Amyloidosis remains an unmet medical need with no approved options for treatment," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization's Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children and NEXICART-1 principal study investigator. "We continue to be encouraged by NXC-201's response rates and durable effect in patients without significant pre-existing cardiac damage, exemplified by our 28-month longest responder with response ongoing," Dr. Stepensky added.

The NEXICART-1 study is an ongoing Phase 1b/2a open-label trial evaluating the safety and efficacy of NXC-201 in adults with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis. The primary objective of the Phase 1b portion was to characterize the safety and confirm the recommended Phase 2 dose (RP2D) of NXC-201, which has been successfully established at 800 million CAR T cells.

Building upon the success of NEXICART-1, Immix Biopharma is set to launch NEXICART-2, an open-label, single-arm, multi-site Phase 1b dose expansion clinical trial of NXC-201 in the United States for patients with relapsed/refractory AL Amyloidosis. The study is expected to enroll 40 patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy over approximately 18 months from first patient dosing. The primary endpoints will be complete response rate and overall response rate according to consensus recommendations.

NXC-201 is a sterically optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, with potential expansion into other autoimmune indications. The therapy has been granted Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma, as well as by the European Medicines Agency (EMA).

AL amyloidosis is a systemic disorder caused by an abnormality of plasma cells in the bone marrow, leading to progressive and widespread organ damage and high mortality rates. In the United States, the observed prevalence of relapsed/refractory AL Amyloidosis is growing at a rate of 12% per year and is estimated to reach 33,277 patients in 2024. The global Amyloidosis market was valued at $3.6 billion in 2017 and is expected to reach $6 billion by 2025.

The impressive 92% overall response rate and 100% ORR in patients without prior BCMA-targeted therapy exposure in the NEXICART-1 study highlight the potential of NXC-201 as a groundbreaking treatment for relapsed/refractory AL Amyloidosis. As Immix Biopharma prepares to expand its clinical development program with the NEXICART-2 trial in the United States, hopes are high that this novel CAR-T cell therapy could fill a critical unmet need for patients battling this devastating disease.