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

Article

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.

REFERENCE
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
Related Videos
Aimee C. Talleur, MD, a physician at St. Jude Children’s Research Hospital
Pat Furlong, BSN, RN
Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center
Heather Lau, MD, MS, the executive director of global clinical development at Ultragenyx Pharmaceutical
Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center
Salvador Rico, MD, PhD
Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center
Michael Kelly, PhD
Pat Furlong, BSN, RN
© 2024 MJH Life Sciences

All rights reserved.