Immix Biopharma, Inc. announced new clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, in patients with relapsed/refractory AL Amyloidosis (R/R ALA) in a late breaking oral presentation at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) in Baltimore, MD. All patients were relapsed/refractory to standards-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone). At the NXC-201 ASGCT 2024 late-breaking oral presentation, data were presented from 13 relapsed/refractory AL amyloidosis patients (including 3 new patients) in the ongoing Phase 1b/2a NEXICART-1 study.

Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=10). Patient characteristics: 85% (11/13) had cardiac involvement; 38% (5/13) had New York Heart Association (NYHA) stage 3 or 4 heart failure; 38% (5/13) had Mayo stage 3 AL amyloidosis disease; Relapsed/refractory to a median 4 lines of prior therapy (range: 3-10); 1 patient, patient 11, was treated and progressed on a BCMA-targeted bispecific antibody before NXC-201 treatment. Safety and efficacy data: Overall response rate (ORR) of 92% (12/13) for relapsed/refractory AL Amyloidosis patients enrolled in NEXICART-1: 12 out of 12 patients not exposed to prior BCMA-targeted bispecific responded to NXC-201 (100% ORR), of which 9 out of 12 were complete responders (75% CRs); 1 patient with prior exposure to BCMA-targeted bispecific treatment did not respond; Best responder had a duration of response of 28.0 months as of May 10, 2024, with response ongoing; There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events; Median cytokine release syndrome (CRS) duration was 2 days (range: 1-5): No grade 4 CRS events; 2 experienced no CRS; 3 experienced grade 1 CRS; 6 Experienced grade 2 CRS; 2 experienced grade 3 CRS.

NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis. The primary objective of the Phase 1b portion of the study is to characterize the safety and confirm the recommended Phase 2 dose (RP2D) of NXC-201. The Phase 1b portion has been successfully completed, with a recommended Phase 2 dose (RP2D) of 800 million CAR+T cells.

NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site Phase 1b dose expansion clinical trial in relapsed/refractory AL Amyloidosis for CAR-T NXC-201 in the United States. Over a period of approximately 18 months from first patient dosing, NEXICART-2 is expected to enroll 40 patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The objectives are the safety and efficacy of NXC-201.

The expected primary endpoints are complete response rate and overall response rate according to consensus recommendations (Palladini et al. 2012). NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, which the company believe has the potential to be the only ?Single-Day CRS?

CAR-T, targeting AL Amyloidosis and other autoimmune diseases. It is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, and expanding into other autoimmune indications. These trials build on a robust NXC-201 clinical dataset initiated in February 2021.

NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma, and awarded EU ODD by the EMA in multiple myeloma and AL Amyloidosis.