Nexcella becomes the latest biotech to generate positive cell therapy data with interim early-stage results in light chain (AL) amyloidosis.

The company reported new Phase Ib/IIa (NCT04720313) data of its chimeric antigen receptor T (CAR-T) therapy NXC-201 at the Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) in Los Angeles. Hadassah Medical Organization, which is based in Jerusalem, Israel, is listed as the study sponsored.

In the trial, eight patients with AL amyloidosis who were relapsed or refractory to Johnson & Johnson’s Darzalex (daratumumab) had an overall response rate of 100% and a complete response rate of 63%. In addition, Nexcella reported no grade 4 cytokine release syndrome, which can be a toxicity concern for CAR-T therapies.

The ongoing trial targets both AL amyloidosis and multiple myeloma. Nexcella said it intends to submit data in multiple myeloma to the US Food and Drug Administration (FDA) once 100 patients have been treated with NXC-201, and data in AL amyloidosis once 30–40 patients have been treated.

Nexcella and CAR-T cell therapy

With early positive data in hand, Los Angeles, California-based Nexcella is looking to make its mark in the crowded field of CAR-T therapies.

The therapies, which entail genetically altering immune cells in a lab, have shown promise in an array of indications ranging from leukaemia to glioblastoma. However, as more CAR-T therapies earn regulatory approval, questions are rising over challenges in affordability and patient access across different corners of the globe.

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By GlobalData

AL amyloidosis is a rare, potentially life-threatening disease characterised by abnormal light chain proteins gathering on organs and tissues. GlobalData estimates the global market for amyloidosis therapies to surpass $9 billion in 2029.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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