Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Investor Presentation (January 2025)
30 slides from Cartesian Therapeutics's investor presentation, Jan 13, 2025
Slide 1: Pioneering mRNA Cell Therapy for Autoimmunity
Pioneering mRNA Cell Therapy for Autoimmunity January 2025 Exhibit 99.1
Slide 2: Disclosures
Disclosures For the purposes of this notice, the “presentation” that follows shall mean and include the slides that follow, the oral presentation of the slides by members of management of Cartesian Therapeutics, Inc. (the “Company”) or any person on their behalf, any question-and-answer session that
Slide 3: Pioneering mRNA Cell Therapy for Autoimmunity
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY3 Clinical-stage company pioneering mRNA cell therapies designed to expand the reach of cell therapy to autoimmunity • Pipeline of mRNA cell therapies designed to be dosed more reliably and safely in an outpatient setting without lymphodepletion • Descart
Slide 4: Cartesian's mRNA approach is designed to expand the reach of potent cell therapy
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY4 Cartesian’s mRNA approach is designed to expand the reach of potent cell therapy products to address autoimmunity No Lymphodepletion No associated cytopenia, secondary malignancies, or other chemotherapy toxicities Administered Outpatient Convenient dos
Slide 5: Wholly-owned pipeline targets autoimmune disease
Asset Indications Discovery/Preclinical Phase 1 Phase 2 Phase 3 Descartes-08 Autologous mRNA CAR-T Myasthenia Gravis (MG) Systemic Lupus Erythematosus (SLE) Pediatric Autoimmune Diseases* Descartes-15 Autologous mRNA CAR-T Autoimmune Diseases** Wholly-owned pipeline targets autoimmune disease PIONEE
Slide 6: Descartes-08 is an mRNA CAR T-cell therapy in clinical development for autoimmune disease
Engineered by transfection of autologous CD8+ T cells with mRNA encoding anti-BCMA CAR Granted U.S. FDA orphan and RMAT designations for generalized myasthenia gravis, and RPDD for juvenile dermatomyositis Typical lot processed for infusion within as little as ~3 weeks RMAT, Regenerative Medicine Ad
Slide 7: Descartes-08 in Myasthenia Gravis
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY7 Descartes-08 in Myasthenia Gravis
Slide 8: Myasthenia gravis is a rare, progressive autoimmune disease with significant unmet need
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY8 Myasthenia gravis is a rare, progressive autoimmune disease with significant unmet need Current treatments require chronic or frequent administration and have limited durability Characterized by debilitating fatigue and muscle weakness Limbs Respiratory
Slide 9: AURORA: Randomized double-blind, placebo-controlled Phase 3 trial of Descartes-08
9 AURORA: Randomized double-blind, placebo-controlled Phase 3 trial of Descartes-08 in AChR Ab+ gMG expected to commence in 1H 2025 WK1 Masked Dosing Six weekly doses of Descartes-08 or placebo PRIMARY ENDPOINT • Proportion of participants with MG-ADL improvement of ≥3 points at Month 4, relative to
Slide 10: Deep and durable responses maintained over months in Phase 2b
Deep and durable responses maintained over 12 months in participants treated with Descartes-08 in Phase 2b PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY10 Deepening responses observed over time Durable responses observed over time Deepest responses observed in participants without exposure to prior
Slide 11: Deepening responses observed in participants treated with Descartes-08
Deepening responses observed in participants treated with Descartes-08 PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY11 • Average MG-ADL reduction of 5.5 (±1.1) points at Month 4 • 33% of participants achieved minimum symptom expression at Month 6 • 80% of participants reaching Month 12 maintained cl
Slide 12: Deepest responses observed in participants with no prior exposure to complement or FcRn inhibitors
Deepest responses observed in participants with no prior exposure to complement or FcRn inhibitors PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY12 • Average MG-ADL reduction of 6.6 (±1.5) points at Month 4 • 57% of participants achieved minimum symptom expression at Month 6 • 100% of participants re
Slide 13: Safety profile supports outpatient administration
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY13 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%) Nausea 3 (15%) 6 (30%) 1 (6%) 2 (13%) Fever 7 (35%) 4 (
Slide 14: Phase 2a trial update: Descartes-08 retreatment continues to elicit deep and durable responses
Phase 2a trial update: Descartes-08 retreatment continues to elicit deep and durable responses Manuscript submitted for peer review; pre-print available at medRxiv.org. • Three participants retreated to date, two of whom maintain minimum symptom expression 2 years after initial treatment • Third par
Slide 15: Descartes-08 focuses the T-cell repertoire and selectively alters the autoreactome
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY15 Descartes-08 focuses the T-cell repertoire and selectively alters the autoreactome, showing clear biological activity Descartes-08 focuses the T-cell repertoire in a manner that correlates with clinical effect p=0.048 (Wilcoxon signed rank test) TCR Di
Slide 16: Descartes-08 Additional Indications
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY16 Descartes-08 Additional Indications
Slide 17: Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE)
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY17 Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE) • Open-label trial in up to 30 adults with moderate to severe multi-refractory SLE and no CNS involvement • Designed to assess safety, tolerability, and manufacturing feasibility
Slide 18: Intend to leverage the potential of Descartes-08 across multiple clinical programs
Intend to leverage the potential of Descartes-08 across multiple clinical programs PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY18 • Plan to initiate Phase 3 AURORA clinical trial in 1H 2025 • RMAT designation expected to support efficient development plan in collaboration with FDA MG SLE Potential
Slide 19: Descartes-15
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY19 Descartes-15
Slide 20: Descartes-15, a next generation anti-BCMA mRNA CAR-T with >10x potency observed in preclinical studies
Descartes-15, a next generation anti-BCMA mRNA CAR-T with >10x potency observed in preclinical studies PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY20 Potent killing (single target exposure) Persistent killing (multiple exposures) (1:2) (1:8) (1:32) 0 50 100 % S p e c if ic K il li n g DC-15 DC-08 V
Slide 21: Wholly-owned, in-house manufacturing
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY21 Wholly-owned, in-house manufacturing FUTURE GROWTH Clinical and commercial manufacturing scale capabilities support maturing pipeline and future growth QUICK TO ADAPT Flexibility to quickly adapt to changes in processes or needs WHOLLY-OWNED Ownership
Slide 22: Strong financial position: expected to support pipeline through key milestones
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY22 STRONG FINANCIAL POSITION: Expected to Support Pipeline Through Key Milestones * As of 12/31/24. Further includes Series A Non-Voting Convertible Preferred Stock and Series B Non-Voting Convertible Preferred Stock that remain subject to beneficial owne
Slide 23: Our team | Management
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY23 Our team | Management Carsten Brunn, PhD PRESIDENT AND CEO Blaine Davis CHIEF FINANCIAL OFFICER Metin Kurtoǧlu, MD, PhD CHIEF TECHNOLOGY OFFICER Miloš Miljković, MD CHIEF MEDICAL OFFICER Jessica Keliher CHIEF PEOPLE OFFICER Emily English, PhD CHIEF OPE
Slide 24: Key Takeaways
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY24 Key Takeaways Pioneering mRNA Cell Therapies Pipeline designed to expand the reach of cell therapy to autoimmunity Experienced Leadership Team Focused on disciplined investment and creating value for stockholders and patients Strong Balance Sheet to Su
Slide 25: Cartesian Therapeutics
ro Cartesian. therapeutics
Slide 26: Appendix
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY26 Appendix
Slide 27: Phase 2b trial: double-blind, placebo-controlled clinical trial of Descartes-08 in myasthenia gravis
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY27 Phase 2b trial: double-blind, placebo-controlled clinical trial of Descartes-08 in patients with myasthenia gravis Day -70 Day -60 Day -50 Day -40 Day -30 Day -20 Day -10 Leukapheresis & Cell Processing Days -59 to -10 Randomization WK1 6 8 12 Blinded
Slide 28: Approximately 15% reduction in AChR antibody titer at Month 3 is in line with Phase 2a data
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY28 Approximately 15% reduction in AChR antibody titer at Month 3 is in line with Phase 2a data Average reduction (±SEM) in AChR antibody in participants with baseline levels above LLOQ randomized to Descartes-08 (n=12) vs. Placebo (n=9). Phase 2a data Ind
Slide 29: Descartes-08 observed not to deplete broader antibody repertoire or decrease vaccine titers
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY29 Descartes-08 observed not to deplete broader antibody repertoire or decrease vaccine titers for common viruses No significant change in common vaccine titers at primary end point (Day 85) relative to Day 12 No significance change in Ig at primary end p
Slide 30: Descartes-08 is designed for dual action, targeting two key BCMA+ cell populations
Descartes-08 is designed for dual action, precisely targeting two key BCMA+ cell populations involved in a spectrum of autoimmune diseases PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY30 Several autoimmune disease segments involve pathogenic contributions from both PCs/plasmablasts and pDCs, includi
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