Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Corporate Update (December 2024)
18 slides from Cartesian Therapeutics's corporate update, Dec 3, 2024
Slide 1: Updated Data from Phase 2b Trial of Descartes-08 in Myasthenia Gravis
Updated Data from Phase 2b Trial of Descartes-08 in Myasthenia Gravis December 3, 2024
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: Call participants
Miloš Miljković, MD CHIEF MEDICAL OFFICER Carsten Brunn, PhD PRESIDENT AND CEO Call participants PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY3 Blaine Davis CHIEF FINANCIAL OFFICER James F. Howard Jr., MD PROFESSOR OF NEUROLOGY, MEDICINE, AND ALLIED HEALTH UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDI
Slide 4: Deep and durable response maintained over months in participants treated with Descartes-08
Deep and durable response maintained over 12 months in participants treated with Descartes-08 PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY4 Deepening responses observed over time Durable responses observed over time Deepest responses observed in participants without exposure to prior biologic thera
Slide 5: AURORA: Randomized double-blind, placebo-controlled Phase 3 trial of Descartes-08
5 AURORA: Randomized double-blind, placebo-controlled Phase 3 trial of Descartes-08 in AChR Ab+ gMG 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 placebo SECONDARY OBJECTIVES •
Slide 6: Myasthenia gravis is a rare, progressive autoimmune disease with significant unmet need
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY6 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 7: Cartesian's mRNA approach is designed to expand the reach of potent cell therapy
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY7 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 8: Descartes-08 is an mRNA CAR T-cell therapy in clinical development for autoimmune disease
RMAT, Regenerative Medicine Advanced Therapy RPDD, Rare Pediatric Disease Designation 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
Slide 9: Deepening responses observed in participants treated with Descartes-08
Deepening responses observed in participants treated with Descartes-08 PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY9 • 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 cli
Slide 10: 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 AUTOIMMUNITY10 • 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 11: Safety profile continues to support outpatient administration
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY11 Safety profile continues to support 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
Slide 12: 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 13: Approximately 15% reduction in AChR antibody titer at Month 3 is in line with Phase 2a data
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY13 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 14: Descartes-08 observed not to deplete broader antibody repertoire or decrease vaccine titers
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY14 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 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: Wholly owned, in-house manufacturing
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY16 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 17: Deep and durable response maintained over months in participants treated with Descartes-08
Deep and durable response maintained over 12 months in participants treated with Descartes-08 PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY17 Deepening responses observed over time Durable responses observed over time Deepest responses observed in participants without exposure to prior biologic ther
Slide 18: Q&A
PIONEERING mRNA CELL THERAPY FOR AUTOIMMUNITY18 Q&A
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