On May 10, 2024 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, reported 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) (Free ASGCT Whitepaper) in Baltimore, MD (Press release, Immix Biopharma, MAY 10, 2024, View Source [SID1234643082]). All patients were relapsed/refractory to standards-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone).

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"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."

"We are excited to present clinical data from the NEXICART-1 clinical study at ASGCT (Free ASGCT Whitepaper). This study has informed the design of NEXICART-2 clinical trial, expected to open in the U.S. mid-2024," 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 tolerability profile, including lack of neurotoxicity, makes it uniquely suitable as a potential new treatment option for relapsed/refractory AL Amyloidosis patients."

At the NXC-201 ASGCT (Free ASGCT Whitepaper) 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
The NXC-201 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) late-breaking oral presentation can be accessed on the ImmixBio corporate website at this link: View Source

ASGCT 2024 Presentation Details:

Title
"Academic BCMA-CART cells (HBI0101), a promising approach for the treatment of LC Amyloidosis"
Type
Late Breaking Oral Presentation
Oral Presentation Date/Time
Friday, May 10, 2024, 8:45 am – 9:00 am Eastern Time
Session Title
Late-Breaking Abstracts II
Location
Baltimore Convention Center, Baltimore, MD
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.

About NEXICART-2
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).

About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, which we 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.