Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Investor Presentation (May 2024)
30 slides from Cartesian Therapeutics's investor presentation, May 8, 2024
Slide 1: Pioneering mRNA Cell Therapy for Autoimmunity
® THERAPEUTICS Pioneering mRNA Cell Therapy for Autoimmunity May 2024 C A R T E S I A N T H E R A P E U T I C S Exhibit 99.2
Slide 2: Forward-Looking Statements Disclosures
Forward-Looking Statements 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 quest
Slide 3: Clinical-stage company pioneering mRNA cell therapies to expand reach of cell therapy to autoimmunity
3 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 • Descartes-08: Potential first-in-class mRNA CAR T-ce
Slide 4: Experienced management team to lead the mRNA cell therapy company of the future
Experienced management team to lead the mRNA cell therapy company of the future Matthew Bartholomae General Counsel Metin Kurtoglu, MD, PhD CTO Milos Miljkovic, MD CMO Blaine Davis CFO Chris Jewell, PhD CSO Carsten Brunn, PhD President and CEO MANAGEMENT Emily English, PhD SVP, Head of Manufacturing
Slide 5: Cartesian's mRNA approach is designed to expand the reach of potent cell therapy products
Cartesian’s mRNA approach is designed to expand the reach of potent cell therapy products to address potential autoimmune indications 5 Requires Lymphodepleting Chemotherapy Associated with high rates of toxicity, including cytokine release syndrome Conventional DNA Cell Therapy Permanent Cell Modif
Slide 6: Wholly-owned pipeline targets autoimmune disease
Wholly-owned pipeline targets autoimmune disease SLE, Systemic Lupus Erythematosus * Phase 1 dose escalation study in myeloma underway mRNA MSC, Mesenchymal Stem Cells transfected with mRNA LN, Lymph node Asset Indications Discovery/Preclinical Phase 1 Phase 2 Pivotal Descartes-08 Autologous mRNA CA
Slide 7: Descartes-08 is believed to be the first mRNA CAR-T in clinical development for autoimmune disease
® THERAPEUTICS 7 Descartes-08 is believed to be the first mRNA CAR-T in clinical development for autoimmune disease Engineered by transfection of autologous CD8+ T cells with mRNA encoding anti-BCMA CAR Typical lot processed for infusion within ~3 weeks Positive Phase 2a data in myasthenia gravis un
Slide 8: 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 8 Several autoimmune disease segments involve pathogenic contributions from both PCs/plasmablasts and pDCs, including rheumatology, nephrology, neurology, and ot
Slide 9: Initial indication for Descartes-08: Myasthenia gravis
® THERAPEUTICS Initial indication for Descartes-08: Myasthenia gravis • Affects over 120,000 patients in US and EU • Characterized by debilitating weakness: limbs, respiratory, ocular, facial muscles • Standard of care includes chronic use of immunosuppressants, which are often toxic: • Progressive
Slide 10: Phase 1/2a study of Descartes-08 in MG (NCT04146051)
Phase 2 study of Descartes-08 in MG (NCT04146051) All doses observed to be safe and well-tolerated 10 Patient eligibility • MG-ADL > 6 • MGFA Class II-IV • Stable medication dosing > 8 wks prior to infusion • 4-week washout for biologics • IVIg and plasma exchange not allowed after starting Descarte
Slide 11: Phase 1/2a study population comprises patients with significant disease
® THERAPEUTICS Phase 1/2a study population comprises patients with significant disease • Baseline MG-ADL mean score of 10 • 79% were MGFA Class III-IV at screening • All patients presented with disease that was not controlled with standard of care therapies Volume 22, Issue 7, July 2023, Pages 578-5
Slide 12: Descartes-08 was observed to be safe and well-tolerated in MG
® THERAPEUTICS Descartes-08 was observed to be safe and well-tolerated in MG KEY OBSERVATIONS: • No dose-limiting toxicities • No cytokine release syndrome • No neurotoxicity • No pre-treatment chemotherapy and related cytopenias • Outpatient treatment Volume 22, Issue 7, July 2023, Pages 578-590 12
Slide 13: Descartes-08 observed to induce deep and durable clinical improvement in MG
® THERAPEUTICS Descartes-08 observed to induce deep and durable clinical improvement in MG • Notable magnitude and duration of response across all 4 standard MG severity scales • Responses appear to deepen after completing treatment at Week 6 • Positive twelve-month follow-up data from Phase 2a stud
Slide 14: Durable depletion of autoantibodies consistent with observed clinical responses and MoA
® THERAPEUTICS 14 Descartes-08: Durable depletion of autoantibodies consistent with observed clinical responses and MoA • All three participants with detectable AChR antibody levels at baseline experienced autoantibody reductions by Month 6 • Reductions deepened further by Month 9, and were maintain
Slide 15: Descartes-08 retreatment led to a rapid decrease in MG-specific clinical scores
® THERAPEUTICS 15 Descartes-08 retreatment led to a rapid decrease in MG-specific clinical scores • Retreated patients experienced rapid improvement in clinical scores and minimal symptom expression Manuscript submitted for peer review; pre-print available at medRxiv.org Pa tie nt 1 Pa tie nt 2
Slide 16: Phase 2b randomized, placebo-controlled study of Descartes-08 in MG
Informed Consent/Screening (Days -61 to -12) Screening (Days -60 to -11) Leukapheresis & Cell Processing (Days -59 to -10) Descartes-08 (Day 1, 8, 15, 22, 29, 36) Placebo (Day 1, 8, 15, 22, 29, 36) Follow up visits Day 57 (± 7 days) Day 85* (± 7 days) Day 113 (± 7 days) Month 6 (± 2 weeks) Month 9 (
Slide 17: Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE)
Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE) 17 • Open-label study in up to 30 adults with moderate to severe multi-refractory SLE and no CNS involvement • Designed to assess safety, tolerability, and manufacturing feasibility of Descartes-08 in patients with SLE • Secon
Slide 18: Exploring additional applications for Descartes-08 in autoantibody-associated autoimmune diseases
Exploring additional applications for Descartes-08 in autoantibody- associated autoimmune diseases (AAAD) 18 61-year-old man with DPPX antibody-positive AIR, colitis, encephalitis refractory to prednisone, rituximab, bortezomib, IVIg; positive for 5/5 disease-associated autoantibodies pre-treatment
Slide 19: RNA Armory example: Descartes-15, a next generation anti-BCMA mRNA CAR-T
RNA Armory® example: Descartes-15, a next generation anti-BCMA mRNA CAR-T with >10x potency observed in preclinical studies Potent killing (single target exposure) Persistent killing (multiple exposures) (1:2) (1:8) (1:32) 0 50 100 % S pe ci fic K ill in g DC-15 DC-08 Vehicle EP escartes-08 escartes
Slide 20: In-house manufacturing enhances control of product quality, production schedules and costs
® THERAPEUTICS 20 In-house manufacturing enhances control of product quality, production schedules and costs cGMP Cell Manufacturing ISO14644 certified aseptic facility with dedicated QMS cGMP mRNA Synthesis Clinical grade mRNA production Quality Control Internal assay validation and lot release MSC
Slide 21: Wholly-owned, in-house manufacturing: 27,000 sq ft state-of-the-art cGMP facility
Wholly-owned, in-house manufacturing: 27,000 sq ft state-of-the-art cGMP facility Ownership of quality control and production timelines Flexibility to quickly adapt to changes in processes or needs Cost efficiency Clinical and commercial manufacturing scale capabilities support maturing pipeline and
Slide 22: Platform offers development opportunities via autologous, allogeneic and in situ modalities
Platform offers potential development opportunities via three modalities: autologous, allogeneic and in situ 22 Autologous mRNA CAR-T • Descartes-08 • Descartes-15: next generation anti- BCMA mRNA CAR-T with >10x potency observed in clinical studies Allogeneic mRNA MSC • Descartes-33 rLN: In situ ly
Slide 23: Maturing pipeline offers potential for multiple catalysts
Maturing pipeline offers potential for multiple catalysts 23 Descartes-08 in MG Descartes-08 in SLE Descartes-08 Additional Indications Descartes-15 Expect to report Phase 2b data mid-2024 Plan to initiate Phase 2 in 1H 2024 Plan to initiate basket studies in additional autoimmune indications in 2H
Slide 24: Strong Financial Position Expected to Support Pipeline Through Key Milestones
® THERAPEUTICS Strong Financial Position Expected to Support Pipeline Through Key Milestones <60 EMPLOYEES 2H 2026 17.8M *Shares include approximately 166.3 thousand shares of Series A Non-Voting Convertible Preferred Stock that remain subject to beneficial ownership limitations that are convertible
Slide 25: Pioneering mRNA Cell Therapies
® THERAPEUTICS P I O N E E R I N G m R N A C E L L T H E R A P I E S Pipeline designed to expand the reach of cell therapy to autoimmunity M A T U R I N G P I P E L I N E W I T H E X P E C T E D N E A R - T E R M C A T A L Y S T S Validated lead program, Descartes-08, with Phase 2b data expected mid
Slide 26: Pioneering mRNA Cell Therapy for Autoimmunity
® THERAPEUTICS Pioneering mRNA Cell Therapy for Autoimmunity C A R T E S I A N T H E R A P E U T I C S
Slide 27: Appendix
® THERAPEUTICS Appendix
Slide 28: Differentially expressed B-cell antigens require distinct CAR-T strategies
Differentially expressed B-cell antigens require distinct CAR-T strategies • CD19+ and CD20+ cells represent the vast majority of B cells and are ubiquitously distributed • CD19+ and CD20+ are often expressed on the same B cells • The exceptions are plasma cells, in which neither antigen is expresse
Slide 29: Clinical analyses show BCMA-targeting with CAR-T may enable precision reset of autoreactivity
® THERAPEUTICS Clinical analyses of antigen-depletion therapies show BCMA-targeting with CAR-T may enable precision reset of autoantibody- producing PCs 29Bodansky et al., doi: 10.1101/2023.12.19.23300188 Rituximab (CD20+ cell depletion) Anti-CD19 CAR-T (CD19+ cell depletion) Anti-BCMA CAR-T (BCMA+
Slide 30: Cartesian differentiation: hurdles created by integrating vectors in CAR T therapies
Cartesian differentiation: All approved CAR T therapies and other trials in the autoimmune space face fundamental hurdles created by integrating vectors 30 Conventional CAR-T (integrating viral vectors) targeting CD19 • Creates significant burden for patients in three areas • hospital admission • ly
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