Miloš Miljković, MD, on mRNA-CAR-T Descartes-08's Potential for Treating Myasthenia Gravis

“mRNA cell therapy seems to be very clinically active in autoimmune diseases. Myasthenia gravis is the lead indication. It's administered without lymphodepletion chemotherapy and in an outpatient setting and has a great safety profile with no cytokine release syndrome, no neurotoxicity or ICANS, and no dose limiting toxicities across all patients who were treated under open label.”

Infusions of Descartes-08 (Cartesian Therapeutics) investigational autologous mRNA-engineered chimeric antigen receptor T-cell therapy (CAR-T) therapy yielded clinical y improvements in myasthenia gravis (MG) Composite (MGC) score for patients treated with the CAR-T compared to patients who received a placebo treatment.¹

New data were announced from the phase 2b portion of a clinical trial (NCT04146051) evaluating Descartes-08 for MG with MGC as a primary end point. At 3 months posttreatment, 10 of 14 patients (71%) in the modified intent-to-treat (mITT) group, all of whom received at least 1 dose of Descartes-08, achieved a 5-point or greater improvement in MGC score. In comparison, only 3 of 12 patients (25%) who received the placebo in the mITT showed a 5-point or greater improvement in MGC score (P = .018).

Cartesian also recently presented follow-up data from the phase 2a portion of the study at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.² CGTLive spoke with Miloš Miljković, MD, chief medical officer, Cartesian Therapeutics, during the meeting, to learn more about Descartes-08 and its potential in treating MG. He discussed follow-up of patients in the phase 2a portion and shared future plans for Descartes-08 in MG and other autoimmune indications.

REFERENCES
1. Cartesian Therapeutics announces positive topline results from phase 2b trial of Descartes-08 in patients with myasthenia gravis. News release. Cartesian Therapeutics, Inc. July 2, 2024. Accessed July 2, 2024. https://ir.cartesiantherapeutics.com/news-releases/news-release-details/cartesian-therapeutics-announces-positive-topline-results-phase
2. Howard JF, Mozaffar T, Vu T, et al. Investigation of mRNACellTherapy as a Treatment for AutoimmuneDisease in Patients withMyastenia Gravis. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #241