ARI0002h, a BCMA-targeted chimeric antigen receptor (CAR) T-cell therapy, can produce durable responses in patients with relapsed or refractory multiple myeloma (MM), according to research published in The Lancet Oncology.

ARI0002h produced responses in all patients studied. The median duration of response was not reached at a median follow-up of 18 months, though 40% of patients did ultimately experience disease progression.

Researchers tested ARI0002h in a multicenter pilot study ( Identifier: NCT04309981). The trial included 30 patients with relapsed/refractory MM who received ARI0002h. The patients’ median age at baseline was 61 years, and 60% were men.

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Patients had received a median of 3.5 prior lines of therapy (range, 2.8-5.0). All patients were triple-exposed, 67% were triple-refractory, 37% were penta-exposed, and 23% were penta-refractory.

On study, patients first received a fractionated infusion of ARI0002h at 3 x 10⁶ CAR-T cells/kg (0.3, 0.9, and 1.8 x 10⁶ CAR-T cells/kg on days 0, 3, and 7). At least 100 days after the first infusion, patients received a non-fractionated booster dose of up to 3 x 10⁶ CAR-T cells/kg.

The study’s primary endpoints were overall response rate (ORR) 100 days after the first infusion and the proportion of patients with cytokine release syndrome (CRS) or neurotoxic events in the first 30 days.

The ORR during the first 100 days was 100%. Fifteen patients had a complete response, 9 had a very good partial response, and 6 had a partial response.

The rate of CRS was 80%. Fifteen patients had grade 1 CRS, and 9 patients had grade 2 CRS. There were no cases of grade 3 or higher CRS. There were no cases of immune effector cell-associated neurotoxicity syndrome or late neurologic events.

In a post-hoc analysis with a median follow-up of 18 months, the median overall survival was not reached, and the median progression-free survival was 14.5 months.

Twelve patients ultimately experienced disease progression. Eight patients died, 6 of whom had disease progression. The 2 remaining deaths were due to cranial injury and severe SARS-CoV-2 pneumonia.

“ARI0002h administered in a fractioned manner with a booster dose after 3 months can provide deep and sustained responses in patients with relapsed or refractory multiple myeloma, with a low toxicity, especially in terms of neurological events, and with the possibility of a point-of-care approach,” the researchers wrote.

Disclosures: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


Oliver-Caldés A, González-Calle V, Cabañas V, et al. Fractionated initial infusion and booster dose of ARI0002h, a humanised, BCMA-directed CAR T-cell therapy, for patients with relapsed or refractory multiple myeloma (CARTBCMAHCB-01): A single-arm, multicentre, academic pilot study. Lancet Oncol. Published online July 3, 2023. doi:10.1016/S1470-2045(23)00222-X

This article originally appeared on Cancer Therapy Advisor