Cartesian therapeutics has recently announced the beginning of the phase 2b clinical trial to assess the safety and efficacy of Descartes-08, intended to treat generalized myasthenia gravis (MG), according to a recently published press release.
Currently, immunosuppressive agents constitute the main treatment for MG. Although these drugs have significantly improved the life expectancy and quality of life of patients with MG, a significant percentage of patients do not experience complete remission, the investigators noted. To this date, Descartes -8 represents the only significant advance in engineered cell therapy for MG, they added.
Read more about MG therapies
Descartes-8 is an RNA-engineered chimeric antigen receptor T-cell therapy (rCAR-T). The modified T cells with the human-made CAR can recognize the BCMA protein on the surface of plasma cells involved in MG pathogenesis, thus reducing the production of auto-reactive antibodies. Unlike DNA-based cell therapies, rCAR-T has no risk of genomic integration and has predictable pharmacokinetics.
Descartes-8 can be administered in the outpatient setting in 6 weekly patient visits, and the treatment does not require preconditioning chemotherapy.
The placebo-controlled phase 2b trial has an estimated enrollment of 30 patients older than 18 years of age, and a confirmed diagnosis of generalized myasthenia gravis, no well-managed chronic illnesses, pregnancy, and lactation were part of the exclusion criteria.
The primary outcome measure compares Descartes-8 against placebo using the Myasthenia Gravis Activities of Daily Living Scale after 12 weeks of treatment. Secondary outcome measures include the reduction of myasthenia-specific autoantibody titers, the effect of therapy after one or several doses according to standardized scales, as well as the comparison of Descartes-8 vs placebo in patients that crossover from the placebo arm to the experimental arm.
A placebo-controlled design will provide a stringent evaluation of Descartes-08’s efficacy while elucidating its mechanisms of action. This will result in important insights on treating MG and other autoantibody-mediated autoimmune diseases with Descartes-08,” said Miloš Miljković, MD, chief medical officer at Cartesian Therapeutics.
Reference
Cartesian therapeutics announces dosing of first participant in a phase 2b randomized clinical trial for generalized myasthenia gravis. News release. Cartesian Therapeutics; January 31, 2023.
