▎WuXiEdited by the Kant Content Team

Cartesian Therapeutics today announced positive topline results from its Phase 2b clinical trial of the investigational mRNA cell therapy Descartes-08 in patients with generalized myasthenia gravis (MG).

In this Phase 2b double-blind, placebo-controlled, crossover trial, a total of 36 severely symptomatic, heavily treated MG patients were randomized (1:1) Patients will receive weekly outpatient infusions of Descartes-08 or placebo for a total of 6 weeks, without pre-treatment chemotherapy.At the end of the blind assessment in the third month of the trial, patients receiving placebo were eligible to switch to Descartes-08 treatment.

The primary efficacy endpoint assessed the proportion of patients with a reduction of 5 points or more in the Myasthenia Gravis Composite (MGC) score. Secondary endpoints evaluated safety, tolerability, and other validated MG severity scales, including Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), and the MG Quality of Life Revised Scale (MG-QoL-15R).

The primary efficacy data (n=26) include the modified intent-to-treat (mITT) population, which comprises all participants enrolled at academic medical centers who received at least one dose of Descartes-08 (n=14) or placebo (n=12) and completed at least one post-baseline MGC score follow-up assessment.Safety data include all subjects who received at least one dose of Descartes-08 (n=19) or placebo (n=17).

The trial achieved the primary endpoint in the pre-specified mITT efficacy population.71% (10/14) of patients treated with Descartes-08 showed an improvement of 5 points or more in MGC score at Month 3, compared to 25% (3/12) in the placebo group (p=0.018).

For secondary endpoints, consistent with the results of the previously reported open-label portion of the Phase 2a trial,Descartes-08 responders showed significant improvement on the MG severity scale at Month 3 (mean MG-ADL = -5.6; MGC = -8.3; QMG = -5.0; QoL-15r = -7.9).As of the data cutoff date on June 19, 2024, these improvements were sustained or further improved in patients evaluated at the 4-month (n=5) and 6-month (n=3) follow-ups.

Descartes-08 is aAn mRNA cell therapy candidate, and also a potential "first-in-class" mRNA-engineered chimeric antigen receptor T-cell (mRNA CAR-T)Therapy。It clears plasma cells that secrete autoantibodies by targeting BCMA.Compared with traditional DNA-based CAR-T cell therapy, the administration design of mRNA CAR-T cell therapy does not require pre-treatment chemotherapy, and it has been observed to have predictable and controllable pharmacokinetics, allowing for outpatient administration while avoiding the risks of gene integration and cancer transformation.

References:

[1] Cartesian Therapeutics Announces Positive Topline Results from Phase 2b Trial of Descartes-08 in Patients with Myasthenia Gravis. Retrieved July 2, 2024 from https://www.globenewswire.com/news-release/2024/07/02/2907273/0/en/Cartesian-Therapeutics-Announces-Positive-Topline-Results-from-Phase-2b-Trial-of-Descartes-08-in-Patients-with-Myasthenia-Gravis.html

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