FDA Grants RMAT Designation to Cartesian Therapeutics' Descartes-08 for Myasthenia Gravis Treatment

Cartesian Therapeutics, Inc., a clinical-stage biotechnology company listed on NASDAQ under the ticker RNAC, announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Descartes-08, their leading product for treating myasthenia gravis (MG). Descartes-08 is an autologous mRNA CAR-T therapy targeting the B cell maturation antigen (BCMA) and represents a significant innovation in the treatment landscape for MG, a chronic autoimmune disorder characterized by muscle weakness and fatigue.

The RMAT designation is significant because it is reserved for regenerative medicine therapies that show potential to treat, modify, or cure serious or life-threatening conditions based on preliminary clinical evidence. This designation facilitates expedited development and review processes, providing benefits similar to fast track and breakthrough therapy designations. The FDA’s decision underscores the potential of Descartes-08 as a groundbreaking treatment for MG.

Dr. Carsten Brunn, President and CEO of Cartesian Therapeutics, expressed optimism about the RMAT designation, noting that it supports their belief in Descartes-08’s potential as a valuable addition to MG treatments. The company is eager to collaborate closely with the FDA to advance the therapy’s development. Brunn reaffirmed that the company is on schedule to release topline data from their Phase 2b trial by mid-2024.

Descartes-08 had already received Orphan Drug Designation from the FDA for treating MG. Earlier in January 2024, Cartesian reported positive follow-up data from a Phase 2a study, showing that Descartes-08, administered without preconditioning chemotherapy and in an outpatient setting, led to durable reduction of autoantibodies and significant improvements in MG severity scores. The therapy was well-tolerated, with no severe toxicities observed.

The ongoing Phase 2b trial, a randomized, double-blind, placebo-controlled study (NCT04146051), continues to progress, with topline data expected by mid-2024.

In addition to Descartes-08, Cartesian Therapeutics is developing a pipeline of mRNA cell therapies for other autoimmune diseases. This includes planned Phase 2 studies of Descartes-08 for systemic lupus erythematosus and other autoimmune conditions. Another candidate in their pipeline, Descartes-15, is a next-generation mRNA CAR-T therapy targeting BCMA, demonstrating the company’s commitment to pioneering advanced treatments for autoimmune diseases.

Cartesian Therapeutics aims to leverage its innovative mRNA technology to develop transformative therapies for patients with significant unmet medical needs. The successful advancement of Descartes-08 through clinical trials could mark a significant milestone in the treatment of myasthenia gravis, potentially offering new hope to patients suffering from this debilitating condition.