# 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 (HBI0101)

Source: https://onco.cc/key-papers/paper-nxc-201-hbi0101-asherie-haematologica-2023/  
OnCo record `paper-nxc-201-hbi0101-asherie-haematologica-2023` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A hospital in Jerusalem built and made its own CAR-T cells against the myeloma protein BCMA; in the first 20 heavily treated patients, 75 percent responded and half went into complete remission, with only mild immune reactions.

## Summary

Report of the Hadassah phase 1 dose-escalation study (NCT04720313) of HBI0101, a second-generation optimised anti-BCMA CAR-T therapy developed in an academic setting and given fresh without cryopreservation. Twenty heavily pretreated patients with relapsed or refractory multiple myeloma were treated in three cohorts: 150 million (n = 6), 450 million (n = 7) and 800 million (n = 7) CAR-T cells.

Grade 1 or 2 cytokine release syndrome occurred in 18 patients (90 percent); no grade 3 or 4 cytokine release syndrome, no neurotoxicity of any grade and no dose-limiting toxicity were observed. The overall response rate was 75 percent, (stringent) complete response 50 percent and very good partial response 25 percent; responses were dose dependent, with 85 percent overall response, 71 percent complete response and 57 percent minimal residual disease negativity in the high-dose cohort. Median overall survival was 308 days (range 25 to more than 466), with estimated survival of 55 percent at the data cutoff; median progression-free survival was 160 days, with six patients progression free at the cutoff. The authors argue that the results support decentralised CAR-T production in academic centres to meet local demand.

## Fields

- Kind: Key paper
- Last checked: 2026-09-24
- Journal: Haematologica
- Year: 2023
- DOI: 10.3324/haematol.2022.281628
- Authors: Asherie N, Kfir-Erenfeld S, Avni B, et al.
- Findings: Overall response rate 75 percent, (stringent) complete response 50 percent and very good partial response 25 percent in 20 patients.; Grade 1 to 2 cytokine release syndrome in 90 percent; no grade 3 to 4 cytokine release syndrome, neurotoxicity or dose-limiting toxicity.; Dose-dependent responses: 85 percent response, 71 percent complete response and 57 percent MRD negativity at 800 million cells; median progression-free survival 160 days.
- What it means: The clinical result is in line with commercial BCMA CAR-T products, but the point of the paper is the manufacturing: an academic hospital produced its own CAR-T cells, infused them fresh and treated patients who would otherwise wait for a commercial slot. It is the evidence base for what Immix Biopharma now develops as NXC-201.
- Caveats: Phase 1 in 20 patients at a single centre; follow-up was short and the median progression-free survival of about five months is modest.; No comparison group; the historical comparators are the pivotal trials of idecabtagene vicleucel and ciltacabtagene autoleucel.; The registry lists a much larger continuing enrolment, so later cohorts are not described here.

## Sources

- Haematologica 2023: https://doi.org/10.3324/haematol.2022.281628
- PubMed: https://pubmed.ncbi.nlm.nih.gov/36200421/
- ClinicalTrials.gov NCT04720313: https://clinicaltrials.gov/study/NCT04720313

## Connected records

- cancers: [Multiple myeloma](https://onco.cc/cancers/multiple-myeloma/)
- technologies: [CAR-T cell therapy](https://onco.cc/technologies/car-t/)
- targets: [BCMA](https://onco.cc/targets/bcma/)
- drugs: [NXC-201 CAR-T](https://onco.cc/drugs/nxc-201-car-t/)
- institutions: [Hadassah Medical Center](https://onco.cc/institutions/hadassah/)
- trials: [HBI0101 (NXC-201) multiple myeloma phase 1](https://onco.cc/trials/nxc-201-mm/)
- journals: [Haematologica](https://onco.cc/journals/haematologica/)

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