Immix Biopharma : Presentation (IMMX Corporate presentation May 2025)
IMMX
Published on 05/02/2025 at 16:06
Clinical Stage Cell Therapy for AL Amyloidosis
and Other Serious Diseases
May 2025
Pioneering Cell Therapy in AL Amyloidosis and Other Serious Diseases
Dedicated team for NXC-201 in AL Amyloidosis and other serious diseases
Ex-NCI/NIH scientists designed cell therapy for benign tolerability, being developed by Immix
Scientific advisors from Stanford, Memorial Sloan Kettering, Columbia, Tufts, UCLA
Experienced management; board members with recent pharmaceutical acquisitions experience
Sterically-optimized, proprietary CAR-T construct from N-GENIUS platform
Immix N-GENIUS platform produced NXC-201
NXC-201 is our lead, sterically-optimized CAR-T with "digital filter" that reduces non-specific activation
NXC-201 CAR-T construct provides barrier to entry
Sizable AL Amyloidosis market
Relapsed/refractory AL Amyloidosis: 30,000 U.S. patient prevalence
Adding ~2,700 U.S. patients per year (~Billion-dollar annual market increase)
Established billing code for BCMA CAR-T: $425,000 per dose
Typical NXC-201 patient has failed front-line therapy (age >65)
NXC-201: The only CAR-T in
development for AL amyloidosis
No drugs are FDA approved today in relapsed/refractory AL Amyloidosis
We believe NXC-201 clinical results to-date significantly improve treatment options for relapsed/refractory
AL Amyloidosis patients
Source:. E Lebel et al. Efficacy and Safety of Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) for the Treatment of Relapsed and Refractory AL Amyloidosis. Presentation. ASH 2024. M. Assayag, et al. Asherie N, et al. Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial. Haematologica. 2023 Jul 1;108(7):1827-1839. doi: 10.3324/haematol.2022.281628. PMID: 36200421; PMCID: PMC10316256. Quock TP, et al. Epidemiology of AL amyloidosis: a real-world study using US claims data. Blood Adv. 2018 May 22;2(10):1046-1053. doi: 10.1182/bloodadvances.2018016402. PMID: 29748430. Staron A, et al. Marked progress in AL amyloidosis survival: a 40-year longitudinal natural history study. Blood Cancer J. 2021;11(8):139. PMID: 34349108 PMCID: PMC8338947 DOI: 10.1038/s41408-021-00529-w. 129 patients treated composed: a) ex-US: 96 Multiple Myeloma patients at high dose presented at ASH 2024, 13 Multiple Myeloma patient at low dose, 16 AL Amyloidosis patients presented at ASH 2024. b) US: 4 AL Amyloidosis patients.
Significant Near-Term Milestones
Secured rights to NXC-201, N-GENIUS platform
FDA Orphan Drug Designation (ODD) and Regenerative Medicine Advanced Therapy (RMAT) Designation Granted
Mentioned in New England Journal of Medicine
(NEJM) AL Amyloidosis Review
Reported ex-U.S. NEXICART-1 AL Amyloidosis data at ASGCT 2023, ASH 2023, ASGCT 2024,
ASH 2024, JCO published 2024
Additional Academic Trial Sites Added
NEXICART-2 U.S. AL Amyloidosis clinical trial first 6 patients dosed; first patient at Memorial Sloan Kettering Cancer Center
Reported first 4 patients U.S. NEXICART-2 AL Amyloidosis clinical data Q4 2024
Planned FDA Approval Submission (BLA)
2Q/3Q 2025
NXC-201
U.S. NEXICART-2
Trial
>12 patients interim readout
4Q25/1Q26 NXC-201
Initial Clinical Data in Other Serious Diseases
2Q/3Q 2026
NXC-201
U.S. NEXICART-2
Trial
40 patients final readout
N-GENIUS Platform: Sterically-Optimized CAR-T construct "Digital Filter" reduces non-specific activation, leading to better tolerability
ALL BCMA CAR-TS ARE NOT CREATED EQUAL
N-GENIUS PLATFORM
…Resulting in a NXC-201 75% complete response rate with minimal toxicity in relapsed/refractory AL Amyloidosis
Background response rates of 0-10% in
relapsed/refractory AL Amyloidosis
Delivers "Digital"
Intracellular Signaling
Reduces cytokine release
Sterically-optimized key construct modifications
Enhances Plasma Cell Binding
Ensures High Expression
CD8
Signaling Protein
COBRA Binder
CD8 Hinge
CD8
Transmembrane Protein
4-1BB
CD3ζγ
NXC-201
CAR-T
Source: M. Assayag, et al. Academic BCMA-CART cells (HBI0101), a promising approach for the treatment of LC Amyloidosis. 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT). Late Breaking Oral Presentation. Baltimore, MD. May, 2024. Feucht, M. Sadelain, et al. Calibration of CAR activation potential directs alternative T cell fates and therapeutic potency. Nature Medicine. 2019 Jan;25(1):82-88. 5
doi: 10.1038/s41591-018-0290-5. Epub 2018 Dec 17. PMID: 30559421 PMCID: PMC6532069. O. Harush C. J. Cohen, et al. Preclinical evaluation and structural optimization of anti-BCMA CAR to target multiple myeloma. Haematologica. 2022 Oct 1;107(10):2395-2407. doi: 10.3324/haematol.2021.280169. PMID: 35354252 PMCID: PMC9521250. Adapted from PEGS 2021. Zanwar S, et al. Treatment patterns for AL amyloidosis after frontline daratumumab, bortezomib, cyclophosphamide, and dexamethasone treatment failures. Leukemia. 2024 Jun;38(6):1423-1426. doi: 10.1038/s41375-024-02243-5. Epub 2024 Apr 9. Eyal Lebel et al., Efficacy and Safety of Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T-Cell for the Treatment of Relapsed and Refractory AL Amyloidosis. JCO. JCO-24-02252. DOI:10.1200/JCO-24-02252.
Disclaimer
Immix Biopharma Inc. published this content on May 02, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 02, 2025 at 20:03 UTC.