Cartesian Therapeutics has announced positive topline results from its Phase IIb clinical trial evaluating Descartes-08, an autologous, mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T), in patients with generalised myasthenia gravis (MG).
The double-blind, crossover, placebo-controlled trial involved 36 heavily pre-treated, highly symptomatic MG patients.
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They were randomised into a 1:1 ratio to receive either Descartes-08 or a placebo as six weekly outpatient infusions, without preconditioning chemotherapy.
After the blinded follow-up in the third month, placebo recipients were offered the option to switch over to the Descartes-08 treatment.
The proportion of patients achieving a reduction of five points or more in the MG Composite (MGC) score was the primary efficacy endpoint.
Secondary endpoints included safety, tolerability, and other validated MG severity scales.
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By GlobalDataAccording to the findings, the trial met its primary efficacy endpoint of statistical significance, with 71% of subjects in the Descartes-08 arm reported to have five-point or greater improvements in the MGC score at month three, versus 25% in the placebo arm.
The per-protocol population results were similarly positive, with 69% of Descartes-08 patients improving by five points or more, versus 33% of placebo patients.
Patients responding to Descartes-08 exhibited substantial improvements across all MG severity scales in the third month. These improvements persisted or improved at follow-up visits at months four and six.
Descartes-08 was also shown to have a favourable safety profile and was well tolerated, with most adverse events being transient and mild.
Cartesian Therapeutics president and CEO Carsten Brunn said: “We believe the positive data demonstrate clinical proof-of-concept of our novel mRNA platform and highlight the potential of Descartes-08 to provide deep and durable improvements for patients with MG.
“Our recently granted RMAT designation supports the continued development of Descartes-08 in collaboration with the FDA, with plans to hold an End-of-Phase II meeting by the end of the year. The results also exemplify what we hope to obtain with other assets in our existing and future pipeline.”
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