Pharma Focus Asia

Cartesian Therapeutics Receives FDA RMAT Designation for Descartes-08 to Treat Myasthenia Gravis

Thursday, May 23, 2024

Cartesian Therapeutics, Inc. (NASDAQ: RNAC), a biotechnology company in the clinical-stage pioneering mRNA cell therapy for autoimmune diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Descartes-08, a treatment for myasthenia gravis (MG).

Descartes-08 is the company's leading product candidate, an autologous mRNA CAR-T therapy that targets the B cell maturation antigen (BCMA). It is designed to treat MG, a chronic autoimmune condition characterized by severe muscle weakness and fatigue.

“The RMAT designation supports our view that Descartes-08, our innovative mRNA CAR-T cell therapy, could significantly impact the MG treatment landscape,” said Carsten Brunn, Ph.D., President and CEO of Cartesian. “We look forward to working closely with the FDA to advance the development of Descartes-08 efficiently for this underserved patient population. We are on track to announce topline data from our ongoing Phase 2b study in mid-2024.”

The RMAT designation, established under the 21st Century Cures Act, is granted to regenerative medicine therapies intended to treat, modify, reverse, or cure serious or life-threatening conditions based on preliminary clinical evidence indicating the potential to address unmet medical needs. This designation provides benefits such as fast track and breakthrough therapy designations, facilitating early and frequent interactions with the FDA to speed up drug development.

Previously, Descartes-08 received an Orphan Drug Designation from the FDA for treating MG.

In January 2024, Cartesian reported positive twelve-month follow-up data from its Phase 2a study of Descartes-08 in patients with generalized MG. The study showed that Descartes-08, administered in an outpatient setting without integrating vectors or preconditioning chemotherapy, resulted in durable depletion of autoantibodies and significant improvements in MG severity scores over one year. The therapy was well-tolerated with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity observed.

The company remains on schedule to report topline data from its Phase 2b randomized, double-blind, placebo-controlled trial of Descartes-08 in MG patients (NCT04146051) in mid-2024.



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