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RNA CAR T-Cell Therapy for Myasthenia Gravis Shows Benefits in Early Results

Late-breaking interim data were presented at the 14th Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders.

Interim data from Cartesian Therapeutics’ phase 1/2a clinical trial evaluating its autologous CAR T-cell therapy Descartes-08 in patients with myasthenia gravis showed good safety and promising efficacy.

The findings were presented as late-breaking data at the 14th Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders, taking place in Miami, Florida May 10-12, 2022.

The 2-part, ongoing phase 1/2a clinical trial (NCT 04146051) includes dose escalation and dose expansion phases assessing safety, feasibility, and preliminary efficacy of Descartes-08, autologous, RNA-modified T-cells that express a chimeric antigen receptor directed to B-cell maturation antigen (BCMA) in up to 18 participants with generalized myasthenia gravis.

In the dose escalation part of the study, 3 ascending doses were administered to 3 participants with severe, treatment-refractory myasthenia gravis. Data showed that the therapy was generally well-tolerated, with no reports of cytokine release syndrome or other treatment-related adverse events. Of note, participants experienced at least 1 full class of improvement in MGFA clinical classification, as well as over a 50% mean improvement on the Myasthenia Gravis Composite (MGC) Scale 3 months post-infusion with Descartes-08.

In the dose expansion phase, Cartesian presented data on 2 patients who have thus far been dosed as part of the weekly dosing cohort and have received all 6 infusions and completed 10-week follow-up. Overall, the cell therapy was well-tolerated, and both patients, who had severe disease at baseline (MGC >22) experienced significant improvements in disease severity, with MGC score improvements at 10 weeks from 27 to 2 in patient 1 and from 23 to 3 in patient 2.

"I am very encouraged by the interim analysis findings for this RNA CAR T-Cell therapy," said principal investigator Volkan Granit, MD, in a statement. "The treatment appears safe and well tolerated, and the extent of clinical improvement seen in our early participants is remarkable. If future data in a controlled study corroborate these findings, Descartes-08 would become a welcome addition to our armamentarium for treating MG."

In addition to myasthenia gravis, Descartes-08 is also being studied in an ongoing phase 2a clinical trial (NCT04816526) as a potential frontline therapeutic for multiple myeloma.

Cartesian Therapeutics to Present Late-Breaking Data from Phase 1/2a Trial of RNA CAR T-cell Therapy in Patients with Generalized Myasthenia Gravis. News release. Cartesian Therapeutics. May 10, 2022. https://www.prnewswire.com/news-releases/cartesian-therapeutics-to-present-late-breaking-data-from-phase-12a-trial-of-rna-car-t-cell-therapy-in-patients-with-generalized-myasthenia-gravis-301543090.html