Cartesian Therapeutics Highlights Progress and 2024 Strategic Priorities Across Innovative Pipeline of mRNA Cell Therapies for Autoimmunity - Seite 2

Program Updates and Anticipated 2024 Milestones

Cartesian’s internally manufactured portfolio of mRNA cell therapies are purposefully designed to be administered conveniently in an outpatient setting. The Company’s RNA-engineering approach is designed to expand the reach of cell therapy to autoimmunity with potent therapies that can be dosed more reliably and safely in an outpatient setting without lymphodepletion. Cartesian’s proprietary technology platform, RNA Armory, is designed to enable precision control and optimization of engineered cells for diverse cell therapies leveraging multiple modalities, including autologous, allogeneic, and in situ transfection.

Descartes-08

Descartes-08 is an autologous anti-B cell maturation antigen (BCMA) mRNA CAR-T. Compared to conventional DNA-based CAR T-cell therapies, mRNA CAR-T is designed not to require preconditioning chemotherapy, has been observed to have predictable and controllable pharmacokinetics, and is designed to avoid the risk of genomic integration. Descartes-08 has been granted Orphan Drug Designation by the U.S. FDA for the treatment of MG, a chronic autoimmune disorder that causes disabling muscle weakness and fatigue.

• Enrollment remains ongoing in the Company’s Phase 2b randomized, double-blind, placebo-controlled trial of Descartes-08 in patients with MG (NCT04146051), with topline results expected in mid-2024. In the open label Phase 2a portion of the study, Descartes-08 was administered in an outpatient setting without preconditioning chemotherapy. The drug was observed to be safe, well tolerated, and appeared to lead to deep, durable clinical responses. These results were published earlier this year in The Lancet Neurology.
  
  
• In a separate press release issued today, the Company announced positive twelve-month follow-up data from the Phase 2a portion of the Descartes-08 trial in patients with MG. In the study, five out of seven patients maintained clinically meaningful improvements across all four standard MG severity scores approximately 10 months after the last infusion. In addition, all three participants with detectable anti-acetylcholine receptor antibody levels at baseline experienced durable depletion of autoantibodies through the one-year follow-up period. Descartes-08 was observed to be well-tolerated, with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity.