Cartesian Therapeutics is moving forward with a Phase 3 clinical trial for Descartes-08, an mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T), as a treatment for myasthenia gravis (MG). The Phase 3 AURORA trial will assess the efficacy and safety of Descartes-08 in patients with acetylcholine receptor autoantibody positive (AChR Ab+) MG.
Phase 2b Trial Results
Updated results from the Phase 2b trial demonstrated a clinically meaningful improvement in patients treated with Descartes-08. The trial showed a 5.5-point average reduction in the MG Activities of Daily Living (MG-ADL) score at Month 4. Furthermore, the treatment responses deepened over time and remained durable through Month 12, suggesting a sustained therapeutic effect.
Phase 3 AURORA Trial Design
The Phase 3 AURORA trial is designed as a randomized, double-blind, placebo-controlled study. It will involve approximately 100 participants with AChR Ab+ MG. The primary endpoint of the trial will be the proportion of participants achieving a three-point or greater improvement in MG-ADL score at Month 4 compared to placebo. This endpoint is intended to provide a clear indication of the treatment's clinical benefit.
Safety Profile
Safety data from the Phase 2b trial indicated that Descartes-08 was well-tolerated. Notably, there were no reported cases of cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS), which are potential concerns with CAR-T cell therapies.
Additional Data
In addition to the Phase 2b trial, Cartesian provided updates on its Phase 2a open-label trial. This trial showed positive results from participants who received a second treatment cycle of Descartes-08, with participants maintaining symptom expression for up to one year following the retreatment.
Analyst Perspective
Mizuho reaffirmed its Outperform rating on Cartesian, highlighting the competitive edge of Descartes-08 after the release of Phase 3 MINT data for Uplizna in treating generalized myasthenia gravis (gMG).
Descartes-08 targets B-cell maturation antigen (BCMA) and is designed to be administered outpatient without preconditioning chemotherapy.
