On December 10, 2024 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and select immune-mediated diseases, reported that new NXC-201 NEXICART-1 clinical data in relapsed/refractory AL Amyloidosis has been presented at 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held in San Diego, California (Press release, Immix Biopharma, DEC 10, 2024, View Source [SID1234648983]). The updated results include follow-up and clinical data from 3 new NEXICART-1 patients.

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"We are encouraged by the updated NXC-201 results being presented by our academic collaborators at ASH (Free ASH Whitepaper) 2024. We believe the high percentage of complete responders, combined with the consistent, attractive tolerability profile is critically important in relapsed/refractory AL Amyloidosis. This expanded NXC-201 dataset continues to bolster our leadership in relapsed/refractory AL Amyloidosis, where no drugs are FDA approved today," said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, "We are looking forward to bringing this promising therapy to U.S. relapsed/refractory AL Amyloidosis patients."

At the NXC-201 ASH (Free ASH Whitepaper) 2024 oral presentation, data were presented from 16 relapsed/refractory AL amyloidosis patients (including 3 new patients) in the ongoing Phase 1b/2 NEXICART-1 study, with median 4 lines of therapy prior to NXC-201. 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=13).

Patient characteristics:

81% (13/16) had cardiac involvement
38% (6/16) had New York Heart Association (NYHA) stage 3 or 4 heart failure (3 stage 4, 3 stage 3)
31% (5/16) had Mayo stage 3 (1 stage 3b, 4 stage 3a) AL amyloidosis disease
44% (7/16) had t(11;14) translocation
Relapsed/refractory to a median 4 lines of prior therapy (range: 3-10)
Safety and efficacy data:

Overall response rate of 94% (15/16)
Complete response rate of 75% (12/16) (9 out of 16 were MRD- 10-5)
Organ response rate of 62% (8/13 evaluable)
Best responder had a duration of response of 31.5 months as of December 9, 2024, with complete response ongoing
There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events
Median CRS duration was 2 days (range: 1-5):
No grade 4 cytokine release syndrome (CRS) events
2 experienced no CRS; 3 experienced grade 1 CRS; 8 Experienced grade 2 CRS; 3 experienced grade 3 CRS
The NXC-201 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting oral presentation video can be accessed on the ASH (Free ASH Whitepaper) website: View Source

The NXC-201 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting oral presentation can be accessed on the ImmixBio corporate website at this link: View Source

ASH Presentation Details (CAR-T NXC-201 in relapsed/refractory AL Amyloidosis).

Event 66th ASH (Free ASH Whitepaper) Annual Meeting and Exposition, San Diego, CA
Title "Efficacy and Safety of Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) for the Treatment of Relapsed and Refractory AL Amyloidosis"
Presentation
Date/Time (Pacific Time)
Publication #894
Session Date: Monday, December 9, 2024
Session Name: 652. MGUS, Amyloidosis, and Other Non-Myeloma Plasma Cell Dyscrasias: Clinical and Epidemiological: Ignored no Longer-Progress in AL Amyloidosis
Session Time: 2:45 PM-4:15 PM
Presentation Time: 4:00PM PT
About NEXICART-1
NEXICART-1 (NCT04720313) is an open-label, ex-U.S. Phase 1b/2 clinical trial of NXC-201 (formerly HBI0101) in patients with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis (including AL Amyloidosis patients with impaired cardiac function and including AL Amyloidosis patients exposed to prior BCMA-targeted therapy). The primary objective of the study is to characterize the safety and efficacy of NXC-201. NEXICART-1 clinical results are available at View Source .

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 adequate cardiac function 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 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 AL Amyloidosis.

NXC-201 is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis in the U.S., 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 Orphan Drug Designation (ODD) in AL Amyloidosis by the US FDA and in the EU by the EMA.

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.