Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Investor Presentation (January 2024)
23 slides from Cartesian Therapeutics's investor presentation, Jan 8, 2024
Slide 1: Pioneering mRNA Cell Therapy for Autoimmunity
® THERAPEUTICS Pioneering mRNA Cell Therapy for Autoimmunity January 2024 C A R T ES I AN T H E R APEUT I CS
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 for 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-cell (
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 COO Milos Miljkovic, MD CMO Blaine Davis CFO Chris Jewell, PhD Chief Scientific Officer Carsten Brunn, PhD President and CEO MANAGEMENT Emily English, PhD VP, Q
Slide 5: DNA CAR-T cell therapy creates increased patient burden
DNA CAR-T cell therapy creates increased patient burden Patients receiving DNA CAR-T require inpatient administration and pre-treatment chemotherapy (lymphodepletion) Indirect costs high due to monitoring/treatment of toxicities DNA transduced CAR-T associated with: Cytokine release syndrome (CRS) N
Slide 6: mRNA CAR-Ts have potential to overcome challenges of DNA CAR-Ts
6 Time Therapeutic window Subtherapeutic Toxicity CAR+ Cells 6 Doses Expectation for cells to be administered at the therapeutic but sub-toxic doses Descartes-08 has been administered to 66 patients with autoimmune diseases and cancer1 with no CRS, neurotoxicity, or infections observed Ability to tr
Slide 7: Wholly-owned pipeline targets autoimmune disease
Wholly-owned pipeline targets autoimmune disease AAAD, autoantibody-associated autoimmune diseases SLE, Systemic Lupus Erythematosus mRNA MSC, Mesenchymal Stem Cells transfected with mRNA LN, Lymph node BCMA, B-cell maturation antigen Asset Indications Discovery/Preclinical Phase 1 Phase 2 Pivotal D
Slide 8: Descartes-08 is believed to be the first mRNA CAR-T in clinical development for autoimmune disease
® THERAPEUTICS 8 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 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 disease
Slide 10: Phase 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 Descartes-08 Patie
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 578-590 11
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 Grade* Pa
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 study rein
Slide 14: Retreated patient experienced rapid improvement in clinical scores
Retreated patient experienced rapid improvement in clinical scores which was ongoing at Month 6 of retreatment follow-up with minimal symptom expression 14 Descartes-08 demonstrated durable depletion of autoantibodies and retreatment potential in MG Lasting reductions in autoantibody titers are cons
Slide 15: 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 16: Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE)
Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE) 16 IND CLEARED PHASE 2 STUDY ON TRACK FOR 1H 2024 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 o
Slide 17: Exploring additional applications for Descartes-08 in autoantibody-associated autoimmune diseases
Exploring additional applications for Descartes-08 in autoantibody- associated autoimmune diseases (AAAD) 17 Potential significant Descartes-08-associated improvement observed in a patient with autoimmune retinitis (AIR) 61-year-old man with DPPX antibody-positive AIR, colitis, encephalitis refracto
Slide 18: 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 % Specific Killing DC-15 DC-08 Vehicle EP Descartes-15 Descartes-08
Slide 19: In-house manufacturing enhances control of product quality, production schedules and costs
® THERAPEUTICS 19 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 20: Platform offers development opportunities via three modalities
Platform offers potential development opportunities via three modalities: autologous, allogeneic and in situ 20 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 lymph no
Slide 21: Maturing pipeline offers potential for multiple catalysts
Maturing pipeline offers potential for multiple catalysts 21 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
Slide 22: Strong Financial Position Expected to Support Pipeline Through Key Milestones
® THERAPEUTICS Strong Financial Position Expected to Support Pipeline Through Key Milestones <50 employees Based in Gaithersburg, MD Anticipated cash runway into 2H 2026 162M common 696M basic** Shares outstanding as of 12/31/23 ~$118M Pro forma cash as of 12/31/23* *Reflects the anticipated receipt
Slide 23: Pioneering mRNA Cell Therapies
® THERAPEUTICS P I O NE E RI NG m RNA CE L L T HE RA P I E S Pipeline designed to expand the reach of cell therapy to autoimmunity M AT U RI N G PI PEL I NE W I T H EX PE C T E D N EAR - T E R M CAT AL YS T S Validated lead program, Descartes-08, with Phase 2b data expected mid-year CA S H RE S O UR
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