Cartesian Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for Descartes-08 for the Treatment of Myasthenia Gravis
Cartesian Therapeutics, Inc. recently announced the US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Descartes-08 for the treatment of myasthenia gravis (MG). Descartes-08, the company’s lead product candidate, is an autologous mRNA CAR-T directed against the B cell maturation antigen (BCMA) initially being developed for the treatment of MG, a chronic autoimmune disorder that causes disabling muscle weakness and fatigue.
“Receipt of RMAT designation underscores our belief that Descartes-08, our potential first-in-class mRNA CAR-T cell therapy, could serve as a meaningful addition to the MG treatment landscape,” said Carsten Brunn, PhD, President and Chief Executive Officer of Cartesian. “We look forward to working closely with the FDA to efficiently advance the development of Descartes-08 for this underserved population. Looking ahead, we remain on track to report topline data from our ongoing Phase 2b study in patients with MG in the middle of this year.”
Established under the 21st Century Cures Act, a product candidate is eligible for RMAT designation if it is a regenerative medicine therapy intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Receiving RMAT designation offers sponsor companies the benefits of the fast track and breakthrough therapy designation programs, allowing for early, close, and frequent interactions with the FDA with the goal of expediting drug development. Descartes-08 was previously granted Orphan Drug Designation by the FDA for the treatment of MG.
In January 2024, Cartesian announced positive 12-month follow-up data from its Phase 2a study of Descartes-08 in patients with generalized MG. In this study, Descartes-08 was administered in an outpatient setting without integrating vectors or preconditioning chemotherapy, and durable depletion of autoantibodies and clinically meaningful improvements in MG severity scores during the one-year follow-up period were observed. Descartes-08 was observed to be well-tolerated, with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity. The company 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.
Cartesian Therapeutics is a clinical-stage company pioneering mRNA cell therapies for the treatment of autoimmune diseases. The Company’s lead asset, Descartes-08, is a potential first-in-class mRNA CAR-T in Phase 2b clinical development for patients with generalized myasthenia gravis. Additional Phase 2 studies are planned in systemic lupus erythematosus under an allowed IND, as well as basket trials in additional autoimmune indications. The Company’s clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T.
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