Cartesian Therapeutics – FDA Grants RMAT Designation for Descartes-08

Cartesian Therapeutics, Inc. (NASDAQ: RNAC) has recently announced that the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation for Descartes-08 for the treatment of myasthenia gravis (MG). The company's lead product candidate, Descartes-08, is an autologous mRNA CAR-T directed against the B cell maturation antigen (BCMA) and is being developed for the treatment of MG, a chronic autoimmune disorder causing muscle weakness and fatigue.

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 diseases. It indicates that preliminary clinical evidence suggests the potential of the therapy to address unmet medical needs for the specified disease or condition. This designation offers benefits such as fast track and breakthrough therapy designation programs, enabling early, close, and frequent interactions with the FDA to expedite drug development.

Additionally, Descartes-08 was previously granted orphan drug designation by the FDA for the treatment of MG. Cartesian Therapeutics also remains on track to report topline data from its ongoing phase 2b randomized, double-blind, placebo-controlled trial of Descartes-08 in patients with MG in mid-2024.

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 revealed durable depletion of autoantibodies and clinically meaningful improvements in MG severity scores during the one-year follow-up period. Furthermore, Descartes-08 was observed to be well-tolerated with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity.

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. Cartesian Therapeutics' clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T. Additional phase 2 studies are planned in systemic lupus erythematosus under an allowed IND, as well as basket trials in additional autoimmune indications. The market has reacted to these announcements by moving the company's shares 2.1% to a price of $25.12. Check out the company's full 8-K submission here.

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