HBI0101 (NXC-201) multiple myeloma phase 1
Hadassah's home-made BCMA CAR-T, now called NXC-201, produced responses in three quarters of heavily pretreated myeloma patients in its first 20-patient study, with only mild cytokine release and no nerve toxicity, showing that a hospital can manufacture its own CAR-T cells.
Overview
NCT04720313 is a phase 1 dose-escalation and safety study at Hadassah Medical Organization of HBI0101, a second-generation anti-BCMA CAR-T cell therapy developed and manufactured in an academic setting and infused fresh without cryopreservation, in relapsed or refractory BCMA-expressing multiple myeloma. Nexcella (now Immix Biopharma) is the collaborator and has taken the product forward as NXC-201.
The Haematologica 2023 report covers the first 20 heavily pretreated patients in three cohorts: 150 million CAR-T cells (6 patients), 450 million (7) and 800 million (7). Grade 1 or 2 cytokine release syndrome occurred in 18 patients (90 percent); there was no grade 3 or 4 cytokine release syndrome, no neurotoxicity of any grade and no dose-limiting toxicity in any cohort. The overall response rate was 75 percent, with a (stringent) complete response in 50 percent and a very good partial response in 25 percent. Responses were dose dependent: in the 800 million cohort the overall response rate was 85 percent, complete response 71 percent and minimal residual disease negativity 57 percent. Across cohorts the median overall survival was 308 days (range 25 to more than 466) and median progression-free survival 160 days at the June data cutoff, with six patients still progression free.
The registry lists the study as active, not recruiting, with an estimated 160 participants and a condition field reading "Dose Escalation and Safety" rather than a cancer, which is why the automated pass never attached it. The product's larger registered programme, NEXICART-2 (NCT06097832), is in AL amyloidosis and is a separate study.
- 75 out of 100 people had their tumour shrink with HBI0101 (NXC-201), all dose cohorts.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- 50 out of 100 people had their tumour shrink with HBI0101 (NXC-201), all dose cohorts.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- HBI0101 (NXC-201), all dose cohorts: 160 days.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- 90 out of 100 people reached this endpoint with HBI0101 (NXC-201), all dose cohorts.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- These results apply to the people the trial enrolled: Relapsed or refractory BCMA-expressing multiple myeloma: academic autologous anti-BCMA CAR-T HBI0101 (now NXC-201), given fresh without cryopreservation, in a dose escalation of 150, 450 and 800 million CAR-T cells. People in a different situation may not see the same effect.
- The trial selected people by a biomarker (BCMA); the result should not be assumed for people whose cancer does not have it.
- Only 20 people took part, so the numbers are less certain than in a large trial.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
20 treated.
(Stringent) complete response 50%, very good partial response 25%; 85% in the 800 million cell cohort (7 patients)
Source71% in the 800 million cell cohort, with 57% minimal residual disease negativity
SourceMedian overall survival 308 days (range 25 to more than 466); six patients progression free at the data cutoff
Source18 of 20; no grade 3 to 4 cytokine release syndrome, no neurotoxicity of any grade, no dose-limiting toxicity
Source| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Overall response rateprimary | HBI0101 (NXC-201), all dose cohorts | 20 | 75% | - | - | link |
| (Stringent) complete response rate | HBI0101 (NXC-201), all dose cohorts | 20 | 50% | - | - | link |
| Median progression-free survival | HBI0101 (NXC-201), all dose cohorts | 20 | 160 days | - | - | link |
| Grade 1 to 2 cytokine release syndrome | HBI0101 (NXC-201), all dose cohorts | 20 | 90% | - | - | link |
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