Anito-cel CAR T cell therapy achieves 79% complete response in refractory myeloma
- In a phase 1 study of 38 patients with relapsed or refractory multiple myeloma, anitocabtagene autoleucel (anito-cel) produced a complete response in 79% of participants.
- At a median follow-up of 38.1 months, the 24-month progression-free survival rate was 57%.
- Of 40 patients enrolled, 38 received anito-cel.
- All 38 patients had an adverse event during the treatment period, and 37 patients (97%) had an adverse event of grade 3 or higher.
- Among patients who received the recommended phase 2 dose (100×106 CAR+ T cells), 94% had cytokine release syndrome of grade 1 or 2, with no events of grade 3 or higher.
The publication
In a phase 1 study of 38 patients with relapsed or refractory multiple myeloma, anitocabtagene autoleucel (anito-cel) produced a complete response in 79% of participants. At a median follow-up of 38.1 months, the 24-month progression-free survival rate was 57%.
Who the findings apply to
The study enrolled patients with relapsed or refractory multiple myeloma who had received three or more lines of therapy previously or had triple-class refractory disease.
Why this matters
The study demonstrates that anito-cel, a BCMA-directed CAR T-cell therapy using a synthetic d-domain binder, can induce high response rates in heavily pretreated patients. The authors conclude that the therapy led to a high incidence of response, with grade 3 or higher cytokine release syndrome and neurotoxicity being rare.
What the study found
Of 40 patients enrolled, 38 received anito-cel. All 38 patients had an adverse event during the treatment period, and 37 patients (97%) had an adverse event of grade 3 or higher.
Among patients who received the recommended phase 2 dose (100×106 CAR+ T cells), 94% had cytokine release syndrome of grade 1 or 2, with no events of grade 3 or higher.
A total of 16% of the patients had immune effector cell-associated neurotoxicity syndrome (ICANS) of grade 1 or 2, and 1 patient (3%) had a grade 3 event. No non-ICANS or delayed neurotoxic effects occurred.
At a median follow-up of 38.1 months, all 38 patients (100%) had had a response, with 79% having a complete response.
The 24-month progression-free survival was 57%; the median progression-free survival was 30.2 months. The 36-month overall survival was 65%.
What the result does not establish
This was a phase 1 study evaluating safety and efficacy, with primary endpoints of adverse events and establishment of the recommended phase 2 dose.
In complementary in vitro studies, the anito-cel ddBCMA binder had a faster off-rate than the dual-VHH binder corresponding to ciltacabtagene autoleucel, though cytotoxicity was similar.
The study was funded by Arcellx and Kite, a Gilead company.
Authors’ conclusion
“Anito-cel therapy led to a high incidence of response among patients with heavily pretreated relapsed or refractory multiple myeloma. Cytokine release syndrome and ICANS of grade 3 or higher were rare.”
Study and sources
Phase 1 Study of Anito-cel, a d-Domain BCMA CAR T Cell for Refractory or Recurrent Myeloma
Early-phase / non-randomised trial · Phase 1 · NEJM · 2026-09-24
Sources and limitations
- PubMed record PMID 42777241 (PubMed / NLM · 2026-09-24)
- Trial registry NCT04155749 (ClinicalTrials.gov · 2026-09-24)
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