Cartesian Therapeutics Advances mRNA CAR-T Therapy Descartes-08 into Phase III Trial for Myasthenia Gravis

Apr 10, 2025 08:15 CST Updated 08:15

Cartesian Therapeutics

Pharmaceutical R&D Developer

Recently, Cartesian Therapeutics announced its mRNA CAR-T cell therapy Descartes-08In chronic autoimmune disease, systemic typeMyasthenia Gravis（MG）12-Month Follow-Up Data from Phase IIb Clinical Trial in Patients. The data shows that patients who received a single 6-week course of treatment continued to demonstrate symptom improvement at the 12-month assessment. The company willLaunched in the second quarter of this yearIIIFirst Patient Dosed in Phase AURORA Trial.

Descartes-08 is a potential "first-in-class" autologous mRNA CAR-T therapy developed by Cartesian Therapeutics. It targets B-cell maturation antigen.（BCMA）, eliminating plasma cells that secrete autoantibodies.

Compared with traditional DNA-based CAR-T cell therapy, the administration design of mRNA CAR-T cell therapy does not require pre-conditioning chemotherapy and has been observed to have predictable and controllable pharmacokinetics, allowing for outpatient administration and avoiding the risks of gene integration and cancer transformation.

In a Phase IIb double-blind, placebo-controlled crossover trial, a total of 36 severely symptomatic, heavily treated MG patients were randomized.（1：1）Patients receive weekly outpatient infusions of Descartes-08 or placebo for a total of 6 weeks, without pre-treatment chemotherapy.

As previously announced, the trial met its primary endpoint and demonstrated the safety profile of Descartes-08 supporting outpatient administration.

▲ Primary Efficacy Results of Descartes-08

The primary efficacy dataset of the follow-up portion of this Phase IIb clinical trial consists of all patients enrolled at academic medical centers who received at least one dose of Descartes-08 and completed at least one Myasthenia Gravis Activities of Daily Living scale assessment after the third month.（MG-ADL）Modified Intention-to-Treat Analysis of Subjects Composing the Score Follow-Up Assessment（mITT）Population Composition.

As of March 31, 2025, in 12 evaluable patients with generalized MG, 33%(4 cases)After 6 months of Descartes-08 treatment, met the Minimum Symptom Expression (MSE) criteria and maintained until 12 months. 83%（10/12）Patients maintained a clinically meaningful response for 12 months, with a sustained reduction of ≥2 points in the MG-ADL score.

More notably, the "most significant and compelling sustained response" was observed in a subgroup of 7 patients who had not received standard biologic therapy: nearly 60% achieved and maintained the MSE state for 12 months, and all patients in this subgroup maintained a clinically meaningful response.

Descartes-08 demonstrated good safety, with no reports of cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome during the 12-month follow-up period.

In view of the above data, Cartesian Therapeuticsn Therapeutics Planning to launch the Phase III AURORA trial in the second quarter, the study adopts a special protocol assessment agreed upon with the FDA.(SPA) Framework. Notably, Descartes-08 has previously been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. FDA.（RMAT）Designation and Orphan Drug Designation for the treatment of MG.

Reference Source:

1.https://ir.cartesiantherapeutics.com

E.N.D

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