NEW YORK – Cartesian Therapeutics this week said it expects to discuss plans to initiate a Phase III trial of its investigational cell therapy, Descartes-08, for generalized myasthenia gravis (gMG) with the US Food and Drug Administration by year-end.
Descartes-08, the company's lead therapeutic candidate, is an autologous mRNA-engineered CAR T-cell therapy that targets B-cell maturation antigen (BCMA) cell-surface proteins. Unlike many other CAR T-cell therapies, it's designed to be administered without preconditioning chemotherapy.
Gaithersburg, Maryland-based Cartesian will hold an end-of-Phase II meeting with the FDA to discuss positive data from its randomized, controlled Phase IIb trial, in which 36 patients received either Descartes-08 or placebo administered as six weekly outpatient infusions. Patients in the placebo arm were later eligible to cross over to Descartes-08 treatment.
"We look forward to continuing to collaborate with the FDA to determine next steps for the Descartes-08 program for MG," Cartesian President and CEO Carsten Brunn said in a statement.
The Phase IIb trial met its primary endpoint with 10 of 14 patients treated with Descartes-08 improving by at least five points on the MG composite scale three months after treatment, compared to three of 12 patients treated with the placebo. The scale ranges from zero to 50 points, with higher numbers representing more severe disease.
Patients assessed after four and six months had sustained durable improvements in MG severity, Cartesian said.
Cartesian is also testing Descartes-08 as a treatment for systemic lupus erythematosus in a separate Phase II trial and is exploring it as a treatment for pediatric autoimmune diseases in preclinical testing. The company has said it expects to file an investigational new drug application for a pediatric basket trial by the end of 2024.