AbelZeta’s CD19/BCMA-targeted CAR-T advances into European fast-track for refractory lupus

AbelZeta Pharma, a clinical-stage biopharmaceutical company with operations in Rockville, Maryland and Shanghai, China, announced receipt of PRIority MEdicines (PRIME) designation from the European Medicines Agency’s Committee for Medicinal Products for Human Use for C-CAR168, an autologous bi-specific CAR-T cell therapy, for refractory systemic lupus erythematosus with or without lupus nephritis. The designation is among the first instances of a CAR-T cell therapy receiving this level of EMA regulatory recognition in a lupus indication, following an RMAT designation from the US FDA granted for the same population in May 2025.

PRIME provides earlier and more frequent scientific advice from the EMA and may support accelerated assessment of a future marketing application. Unlike the US FDA’s RMAT designation, it does not include rolling review but enables earlier regulatory engagement that can help shape pivotal trial design.

C-CAR168 uses a dual CD20/BCMA-targeting construct designed to deplete both autoreactive B cells and the antibody-producing plasma cells that continue to generate pathogenic autoantibodies after B-cell-only depletion. Targeting BCMA alongside CD20 is intended to reach plasma cells that lack CD20 expression, a population thought to sustain disease activity following single-target B-cell depletion approaches such as rituximab or CD19 CAR-T constructs.

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The clinical basis for both regulatory designations comes from an ongoing Phase I investigator-initiated trial, NCT06249438, enrolling patients with refractory autoimmune diseases including SLE and lupus nephritis, neuromyelitis optica spectrum disorder, progressive multiple sclerosis, and inflammatory myopathy. At a data cutoff of September 10, 2025, presented at ACR Convergence in October 2025, 16 patients had been treated, including 11 with SLE, nine of whom had proliferative lupus nephritis. Median follow-up was 177 days. Among 9 evaluable lupus nephritis patients, the renal response rate was 77.8%, reported in the absence of concurrent background standard-of-care immunosuppression. The molecule reportedly demonstrated a favorable safety profile.

The designation supports AbelZeta’s broader strategy of applying its dual-target CAR-T platform beyond lupus into additional autoimmune diseases, including progressive multiple sclerosis. The next milestones will be longer-term follow-up from the ongoing Phase I study and discussions with the EMA under the PRIME framework to define a registrational strategy. AbelZeta has not yet disclosed timelines for pivotal studies in Europe or the US.


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