Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — American Academy of Neurology 2025 Annual Meeting
14 slides from Cartesian Therapeutics's conference presentation, Apr 9, 2025
Slide 1: Efficacy and Safety of Autologous BCMA-directed mRNA CAR T-Cell Therapy in Generalized Myasthenia Gravis
Efficacy and Safety of Autologous BCMA-directed American Academy mRNA CAR T-Cell Therapy in Generalized of Neurology Myasthenia Gravis: Results from a Phase 2b 2025 Annual Randomized Placebo-controlled Trial Meeting April 9, 2025 Tuan Vu, Hacer Durmus Tekce, Michael Rivner, Sheetal Shroff, Thomas Ra
Slide 2: COI Disclosures
COI Disclosures • Consultant and/or on speaker bureaus for Alexion/AstraZeneca Rare Disease, Amgen, argenx, CSL Behring, Dianthus, ImmunAbs, NMD Pharma, and Johnson & Johnson. • Research or grant support related to myasthenia gravis from Alexion/AstraZeneca Rare Disease, Amgen, argenx, Cartesians, C
Slide 3: Myasthenia gravis is a rare, progressive autoimmune disease with significant unmet need
Myasthenia gravis is a rare, progressive autoimmune disease with significant unmet need Characterized by debilitating fatigue and muscle weakness >120,000 Patients in the U.S. and EU Limbs Respiratory Ocular Facial Significant unmet need remains Current treatments require chronic or frequent adminis
Slide 4: Descartes-08 is an autologous BCMA-directed mRNA CAR-T in clinical development for gMG
Descartes-08 is an autologous BCMA-directed mRNA CAR-T in clinical development for gMG • Engineered by transfection of autologous CD8+ T cells with mRNA encoding anti- BCMA CAR • B-cell Maturation Antigen (BCMA), a surface antigen preferentially expressed on plasma cells, plasmablasts and plasmacyto
Slide 5: mRNA CAR-T engineering may expand the reach of potent cell therapy products
mRNA CAR-T engineering may expand the reach of potent cell therapy products to address potential autoimmune indications No Lymphodepletion Administered Outpatient No requirement for cell proliferation → no expected need for Convenient dosing schedule pre-treatment chemotherapy → no Grade 3-4 cytopen
Slide 6: Phase 2 study of Descartes-08 in MG (NCT04146051)
Phase 2 study of Descartes-08 in MG (NCT04146051) Patient eligibility Part 1 Identify safe dose • Non-MuSK gMG Part 2 • MG-ADL > 6 (n = 3) Determine • MGFA Class II-IV Complete optimal dosing • Stable medication dosing > 8 wks prior to schedule Part 3 infusion (n = 11) Phase 2b • 4-week washout for
Slide 7: Phase 2b trial: double-blind, placebo-controlled clinical trial of Descartes-08 in patients with myasthenia gravis
Phase 2b trial: double-blind, placebo-controlled clinical trial of Descartes- 08 in patients with myasthenia gravis Leukapheresis & Randomization Descartes-08 Cell Processing Days -59 to -10 1:1 Open Label Follow-Up Month 4, Month 6, Month 9, Month 12 Blinded Dosing Unblinding Six weekly doses of De
Slide 8: Baseline characteristics
Characteristics Descartes-08 Placebo Total Mean Age, Years (SD) 56.7 (16.7) 60 (13.4) 58.2 (15.0) Sex, n (%) Female 10 (71) 6 (50) 16 (62) Baseline MGFA class at screening, n (%) II III 4 (29) 9 (64) 3 (25) 9 (75) 7 (27) 18 (69) characteristics Median age of disease onset IV Years, (range) 1 (7) 55
Slide 9: Trial met primary endpoint
Proportion of MG Composite Responders (≥5-point reduction) at Month 3 Trial met primary endpoint with statistical significance Minimum Point Improvement in MGC and MG-ADL at Month 3 8 Per Protocol Population
Slide 10: Descartes-08 treated participants sustained deep responses through month 12 after a single course of therapy
Descartes-08 treated participants sustained deep responses through month 12 after a single course of therapy Deepest and most sustained responses seen in patients without prior biologics exposure Primary Efficacy Dataset • Average MG-ADL reduction of 5.5 (±1.1) points at Month 4, sustained through M
Slide 11: Safety profile supports outpatient administration
Safety profile supports outpatient administration Descartes-08 (n=20) Placebo (n=16) Grade 1 Grade 2 Grade 3 Grade 1 Grade 2 Grade 3 Headache 7 (35%) 4 (20%) 2 (13%) 3 (19%) Chills 8 (40%) 4 (20%) • Most AEs were transient or mild Nausea 3 (15%) 6 (30%) 1 (6%) 2 (13%) Fever 7 (35%) 4 (20%) 1 (5%) •
Slide 12: Descartes-08 observed not to deplete broader antibody repertoire or decrease vaccine titers
Descartes-08 observed not to deplete broader antibody repertoire or decrease vaccine titers for common viruses No significance change in Ig at No significant change in common vaccine titers at primary end point (D85) vs. Day 11 primary end point (Day 85) relative to Day 12 1.5 IgG Relative to Baseli
Slide 13: Descartes-08 observed not to impact circulating B Cell levels, but to reduce cytokine associated with MG activity
Descartes-08 observed not to impact circulating B Cell levels, but to reduce cytokine associated with MG clinical activity Descartes-08 does alter circulating B Descartes-08 reduces cytokine cell levels1 relative to placebo associated with MG activity (eg, IL-6) Circulating B cells IL-6 Relative cha
Slide 14: Conclusions
Conclusions • Six once-weekly doses of Descartes-08 were associated with a significantly greater proportion of MGC and MG-ADL responders than placebo at Month 3 • More profound improvement in symptoms seen in patients with MG with no prior biologics use • Responses were greater than clinically meani
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