FDA clears IND application for ‘controllable’ cell therapy to treat advanced multiple myeloma
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The FDA cleared an investigational new drug application for ACLX-001, an engineered T-cell therapy for adults with relapsed and refractory multiple myeloma, according to the agent’s manufacturer.
ACLX-001 (Arcellx) is an autologous, gene-edited T-cell therapy with a novel binding domain that targets the B-cell maturation antigen (BCMA) on the surface of cancer cells.
The investigational therapy’s binding domain was developed using Arcellx’s proprietary platform, in which antigen receptor complex T cells (ARC-T) are controlled by a tumor-targeting protein called a SparX (soluble protein antigen-receptor X-linker).
“ARC-T cells cannot recognize the tumor without SparX proteins and can only destroy a diseased cell when attached to a SparX-antigen complex,” a company-issue press release stated. “By adjusting the dose and frequency of SparX administration, the activity of the ARC-T cells can be managed and controlled.”
The FDA based the IND clearance on results of the first six patients treated in a phase 1 trial evaluating the safety and effectiveness of Arcellx’s CART-ddBCMA, which uses the same proprietary BCMA-directed binding domain. Data presented at last year’s virtual ASH Annual Meeting showed all six patients responded to therapy, with four achieving complete stringent response.
Clinical evaluation of ACLX-001 is the next step in validating the company's ARC-SparX platform, according to Rami Elghandour, chairman and CEO of Arcellx.
“Demonstrating a safe and effective cell therapy that is controllable and adaptable has the potential to be transformative for the field of cell and gene therapy,” he said in the release. “We’re excited about the broad clinical applications this will enable, including our programs in acute myeloid leukemia and solid tumors.”
The IND clearance will allow Arcellx to conduct a phase 1 clinical trial for ACLX-001, which the company plans to initiate in the second half of 2021, according to the release.
The trial will be added as a second arm to the company’s current master protocol trial (NCT04155749) of cell therapies for patients with relapsed and refractory multiple myeloma. The study will include patients who have received at least three previous lines of systemic therapy — including a proteosome inhibitor, immunomodulatory drugs and anti-CD38 antibody — or those with triple-refractory disease.
Reference:
Frigault MJ, et al. Abstract 3199. Presented at: ASH Annual Meeting and Exposition (virtual meeting); Dec. 5-8, 2020.
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