Cartesian Therapeutics, a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, has announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation for its lead product candidate, Descartes-08, for the treatment of myasthenia gravis. This designation underscores the potential of Descartes-08 as a first-in-class mRNA CAR-T cell therapy that could significantly impact the MG treatment landscape.
Descartes-08 is an autologous mRNA CAR-T therapy directed against the B cell maturation antigen (BCMA) and is initially being developed for the treatment of MG, a chronic autoimmune disorder that causes disabling muscle weakness and fatigue. The RMAT designation, established under the 21st Century Cures Act, is granted to regenerative medicine therapies that have the potential to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and for which preliminary clinical evidence indicates the potential to address unmet medical needs.
Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian, expressed the company's enthusiasm for the RMAT designation, stating that it reinforces their belief in the potential of Descartes-08 to serve as a meaningful addition to the MG treatment landscape. The designation allows for early, close, and frequent interactions with the FDA, with the goal of expediting drug development. Cartesian looks forward to working closely with the FDA to efficiently advance the development of Descartes-08 for this underserved patient population.
In January 2024, Cartesian announced positive twelve-month follow-up data from its Phase 2a study of Descartes-08 in patients with generalized MG. The study demonstrated that Descartes-08, administered in an outpatient setting without integrating vectors or preconditioning chemotherapy, resulted in durable depletion of autoantibodies and clinically meaningful improvements in MG severity scores during the one-year follow-up period. The therapy was well-tolerated, with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity observed.
Cartesian remains on track to report topline data from its Phase 2b randomized, double-blind, placebo-controlled trial of Descartes-08 in patients with MG (NCT04146051) in mid-2024. The company's clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T therapy. In addition to the ongoing Phase 2b study in MG, Cartesian plans to initiate Phase 2 studies in systemic lupus erythematosus under an allowed IND, as well as basket trials in additional autoimmune indications.
Cartesian Therapeutics is a clinical-stage company pioneering mRNA cell therapies for the treatment of autoimmune diseases. The company's technology enables precision control and optimization of engineered cells for diverse cell therapies leveraging multiple modalities. With a focus on developing novel treatment paradigms to fulfill unmet medical needs, Cartesian is committed to advancing its pipeline of innovative mRNA cell therapies for patients with autoimmune disorders.
The RMAT designation for Descartes-08 in the treatment of MG represents a significant milestone for Cartesian Therapeutics and highlights the potential of the company's mRNA cell therapy platform to address the challenges associated with autoimmune diseases. As the company continues to progress its clinical programs and expand its pipeline, it remains dedicated to its mission of developing transformative therapies that can improve the lives of patients with limited treatment options.
