On December 11, 2023 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, reported new clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, in patients with relapsed/refractory AL Amyloidosis (R/R ALA) at an oral presentation by study investigator Moshe E. Gatt, MD at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting being held in San Diego, CA (Press release, Immix Biopharma, DEC 11, 2023, View Source [SID1234638421]). The updated results include follow-up and clinical data from one new patient. All patients were relapsed/refractory to standard-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone) and had experienced a median of 6 prior lines of therapy that failed to stop worsening of disease prior to receiving NXC-201.

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"There is a rising prevalence of relapsed/refractory AL Amyloidosis patients who have 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 principal study investigator. "We continue to be encouraged by NXC-201’s 100% overall response rate, including in this 10th relapsed/refractory AL amyloidosis patient."

"We believe NXC-201 is the first and only CAR-T in clinical development for AL amyloidosis. With our recent IND clearance, we are thrilled to be now activating sites to bring this first-of-a-kind study to U.S. relapsed/refractory AL Amyloidosis patients," said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, "We believe NXC-201’s favorable tolerability profile, including ‘Single Day CRS’ and the ability to overcome neurotoxicity, enables a new potential option for relapsed/refractory AL Amyloidosis patients and potential expansion into select autoimmune indications."

At the NXC-201 ASH (Free ASH Whitepaper) 2023 oral presentation, data were presented from 10 relapsed/refractory AL amyloidosis patients (including one new patient) in the ongoing Phase 1b/2a NEXICART-1 study, with median 6 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=7).

Patient characteristics:

90% (9/10) had high-risk cytogenetics
80% (8/10) had cardiac involvement
50% (5/10) had New York Heart Association (NYHA) stage 3 or 4 heart failure (3 stage 4, 2 stage 3)
40% (4/10) had Mayo stage 3 (1 stage 3b, 3 stage 3a) AL amyloidosis disease
40% (4/10) had t(11;14) translocation
Relapsed/refractory to a median 6 lines of prior therapy (range: 3-10)
Safety and efficacy data:

Overall response rate of 100% (10/10)
Complete response + very good partial response rate of 90% (9/10)
Complete response rate of 70% (7/10) (6 out of 7 were MRD 10-5)
Organ response rate of 60% (6/10)
Best responder had a duration of response of 23.7 months as of December 10, 2023, with response ongoing
Transformation to complete response at month 2 observed in a patient with 7 lines of prior therapy and cardiac involvement
There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events
"Single Day CRS": Median CRS duration was 1 day (range: 1-4):
No grade 4 cytokine release syndrome (CRS) events
2 experienced no CRS; 2 experienced grade 1 CRS; 4 Experienced grade 2 CRS; 2 experienced grade 3 CRS
For the 8 patients with cardiac involvement:

Overall response rate of 100% (8/8)
Complete response rate of 63% (5/8) (4 out of 5 were MRD 10-5)
Organ response rate of 63% (5/8)
For the 4 patients with t(11;14) disease:

Overall response rate of 100% (4/4)
Complete response rate of 75% (3/4) (MRD 10-5)
Organ response rate of 50% (2/4)
The NXC-201 65th 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 65th 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:

Title "Feasibility of a Novel Academic Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis"
Presentation
Date/Time (Pacific Time)
Publication #538
Session Date: Sunday, December 10, 2023
Session Name: 654. MGUS, Amyloidosis and Other Non-Myeloma Plasma Cell Dyscrasias: Clinical and Epidemiological: From Light Chain to Fibril-Novel Diagnostics to Treatments for Amyloidosis
Session Time: 12:00 PM – 1:30 PM PT
Presentation Time: 12:45 PM PT
Presentation
Replay
Accessible at View Source
About NXC-201
NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, relapsed/refractory multiple myeloma, with potential expansion into autoimmune indications.

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

About NEXICART-1
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.

The expected primary endpoint for NXC-201 in relapsed/refractory AL Amyloidosis is overall response rate. ImmixBio plans to submit data to the FDA in relapsed/refractory AL amyloidosis once 30-40 patients are treated with NXC-201.
The expected primary endpoint for the Phase 2 portion in relapsed/refractory multiple myeloma is overall response rate and duration of response. ImmixBio plans to submit data to the FDA in relapsed/refractory multiple myeloma once 100 patients are treated with NXC-201.
About AL Amyloidosis
U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing 12% per year according to Staron, et al Blood Cancer Journal, estimated to reach 29,712 patients in 2023.

The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research. AL amyloidosis is a systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by these cells cause a buildup of misfolded immunoglobulin proteins in and around tissues, nerves, and organs, gradually affecting their function. This can cause progressive and widespread organ damage and high mortality rates.