Company: RNAC
Filing Date: 2025-03-13
Form Type: 10-K
Source: 0001453687-25-000060
Chunk: 38

Company: Cartesian Therapeutics, Inc.
Filing Date: 2025-03-13
Form: 10-K
Item: Item 1
Chunk 38
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. Descartes-08 has been granted Orphan Drug Designation and Regenerative Medicine Advanced Therapy, or RMAT, Designation by the FDA for the treatment of MG as well as a Rare Pediatric Disease Designation for the treatment of juvenile dermatomyositis.

Descartes-08 for the Treatment of MG

In July 2024, we reported topline results from a Phase 2b randomized, double-blind, placebo-controlled trial in patients with MG. The trial achieved its primary endpoint with statistical significance in the pre-specified modified intent-to-treat, or mITT, efficacy population, with 71% (10/14) of patients treated with Descartes-08 observed to have 5-point or greater improvements in MG Composite, or MGC, score at Month 3 compared to 25% (3/12) of patients treated with placebo (p=0.018). In addition, the trial also achieved its primary endpoint with statistical significance in the per-protocol population, with 69% (11/16) of patients treated with Descartes-08 observed to have 5-point or greater improvements in MGC score at Month 3 compared to 33% (5/15) of patients treated with placebo (p=0.048).

Consistent with previously reported results from the Phase 2a open-label portion of the trial, Descartes-08 responders experienced deep improvements across the MG severity scales at Month 3 (average MG-ADL (Activities of Daily Living) = -5.6; MGC= -8.3; QMG (Quantitative MG) = -5.0; QoL-15r (Quality of Life Revised Scale) = -7.9). A 2-point improvement in MG-ADL and 3-point improvement in MGC and QMG scales are considered “clinically meaningful” by expert consensus published in peer-reviewed journals. The improvements seen at Month 3 persisted or further improved in patients evaluated at their Month 4 (n=5) and Month 6 (n=3) follow-up visits, as of the June 19, 2024 data cutoff date. 

In December 2024, we reported updated results from this trial. Deepening of responses was observed over time with participants included in the primary efficacy dataset who continued follow-up (n=12) experiencing an average MG-ADL reduction of 5.5 (±1.1) at Month 4. At Month