Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Cell & Gene Therapy Innovation Summit 2024
26 slides from Cartesian Therapeutics's conference presentation, Feb 8, 2024
Slide 1: Bringing cell therapy to patients outside oncology
Bringing cell therapy to patients outside oncology Dr. Christopher M. Jewell Chief Scientific Officer Cartesian Therapeutics Cell & Gene Therapy Innovation Summit February 8, 2024 (Barcelona)
Slide 2: Disclosures
Disclosures • CM Jewell is employed by and holds an equity position with Cartesian Therapeutics • CM Jewell is an Associate Editor at Nature Regenerative Medicine and receives compensation • CM Jewell holds an equity position with Barinthus Biotherapeutics • CM Jewell is the Minta Martin Professor a
Slide 3: Clinical-stage company pioneering mRNA cell therapies for autoimmunity
Clinical-stage company Multiple anticipated near-term catalysts pioneering mRNA cell DESCARTES-08 therapies for autoimmunity • • Phase 2b topline data in MG expected mid-2024 Initiation of Phase 2 study in SLE expected in 1H 2024 • Initiation of studies in additional autoimmune indications expected
Slide 4: Experienced management team for bringing cell therapy to autoimmunity
Experienced management team for bringing cell therapy to autoimmunity MANAGEMENT Carsten Brunn, PhD Blaine Davis Metin Kurtoglu, MD, PhD Emily English, PhD Chris Jewell, PhD Milos Miljkovic, MD Matthew Bartholomae President and CEO CFO COO VP, Quality Chief Scientific Officer CMO General Counsel BOA
Slide 5: CAR T cell therapy offers transformative potential beyond oncology
The selectivity of chimeric antigen-specific (CAR) T cell therapy offers transformative potential for diverse indications beyond oncology 5
Slide 6: Conventional DNA-based engineered cell therapy can lead to toxicity and patient burden
Conventional engineered cell therapy uses DNA, which can DNA transduced CAR-T can lead to: lead to toxicity and increased patient burden • Cytokine release syndrome (CRS) • Neurotoxicity and parkinsonism • Effector function of cell therapy engineered with DNA • Cytopenia (from pre-treatment chemo) a
Slide 7: Cartesian's mRNA approach expands the reach of cell therapy to autoimmune indications
Cartesian’s mRNA approach Expectation for cells to be administered at the is designed to expand the therapeutic but sub-toxic doses reach of cell therapy to Descartes-08 has been administered to 66 patients autoimmune indications with autoimmune diseases and cancer1 with no CRS, neurotoxicity, or in
Slide 8: DNA-based or RNA-based CAR-T produce distinct patient experiences
DNA-based or RNA-based CAR-T produce distinct patient experiences 8
Slide 9: Autoimmune and inflammatory diseases impact 200M+ people
Autoimmune and inflammatory diseases impact 200M+ people Diverse pathologies but common features Autoimmunity - characterized by attack against self 100+ autoimmune disorders Many poorly defined Other relevant disease classes (e.g., inflammatory disease, allergies) Persistent treatment c
Slide 10: Wholly-owned pipeline targets autoimmune disease
Wholly-owned pipeline targets autoimmune disease Asset Indications Discovery/Preclinical Phase 1 Phase 2 Pivotal Myasthenia Gravis Data from Phase 2b study expected in mid-2024 Descartes-08 Autologous mRNA CAR-T Expect to initiate Phase 2 studies in SLE and additional SLE, other AAAD autoimmune indi
Slide 11: Initial indication for Descartes-08: Myasthenia gravis
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 that is
Slide 12: Descartes-08 is believed to be the first mRNA CAR-T in clinical development for autoimmune disease
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 underscores potenti
Slide 13: Phase 2 study of Descartes-08 in MG
Phase 2 study of Descartes-08 in MG (NCT04146051) Part 1 Patient eligibility Identify safe dose Part 2 • MG-ADL > 6 (n = 3) Determine • MGFA Class II-IV optimal dosing • Stable medication dosing > 8 wks prior Complete Part 3 schedule to infusion (n = 11) Phase 2b • 4-week washout for biologics compa
Slide 14: Phase 1/2a study population comprises patients with significant disease
Mean age, years (SD) 52 (18) Previous myasthenia gravis therapies (standard of care) Phase 1/2a Female Male 10 (71%) 4 (29%) Pyridostigmine 14 (100%) study population Mean weight, kg (SD) 84 (21) Prednisone Other immunosuppressants 14 (100%) 14 (100%) comprises patients Mean BMI, kg/m2 (SD) Race and
Slide 15: Descartes-08 was observed to be safe and well-tolerated in MG
Descartes-08 Grade* Part 1 Part 2: all groups Part 2: group 1 Part 2: group 2 Part 2: group 3 was observed to (n=3) (n=11) (n=3) (n=7) (n=1) Hand numbness 2 1 (33%) 0 0 0 0 be safe and Headache 1 1 (33%) 5 (45%) 1 (33%) 3 (43%) 1 (100%) well-tolerated in MG Muscle soreness Nausea 1 1 1 (33%) 1 (33%)
Slide 16: Descartes-08 observed to induce deep and durable clinical improvement in MG
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 reinforce pri
Slide 17: Descartes-08 demonstrated durable depletion of autoantibodies and retreatment potential in MG
Descartes-08 demonstrated durable depletion of autoantibodies and retreatment potential in MG Lasting reductions in autoantibody titers1 are Retreated patient experienced rapid improvement in clinical scores after consistent with the observed clinical retreatment, which was ongoing with minimal symp
Slide 18: Phase 2b randomized, placebo-controlled, double-masked study of Descartes-08 in MG
Phase 2b randomized, placebo-controlled, double-masked study of Descartes-08 in MG Informed Consent/Screening (Days -61 to -12) Plan to treat ~30 patients Screening (Days -60 to -11) PRIMARY ENDPOINT • Proportion of MG Composite responders (≥5-point Leukapheresis & Cell Processing (Days -59 to -10)
Slide 19: Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE)
Exploring potential of Descartes-08 in Systemic Lupus Erythematosus (SLE) IND CLEARED Screening (Days -60 to -15) PHASE 2 STUDY ON TRACK FOR 1H 2024 Leukapheresis & Cell Processing (Days -59 to -14) • Open-label study in up to 30 adults with moderate to severe multi-refractory SLE and no CNS involve
Slide 20: Exploring additional applications for Descartes-08 in autoantibody-associated autoimmune diseases
Exploring additional applications for Descartes-08 in autoantibody- associated autoimmune diseases (AAAD) Potential significant Descartes-08-associated • Clinical data suggest Descartes-08 improvement observed in a patient with autoimmune retinitis (AIR) could lead to clinical benefit and 61-year-ol
Slide 21: 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) Descartes-15 is an anti-BCMA mRNA CAR-T with potential 100 Descartes-15 DC-15 100 Descartes-15
Slide 22: In-house manufacturing enhances control of product quality, production schedules and costs
cGMP Cell In-house Manufacturing ISO14644 certified aseptic manufacturing facility with dedicated QMS enhances control of product quality, production schedules Quality Control Internal assay cGMP mRNA Synthesis and costs validation and lot release Clinical grade mRNA production Process MSC Cell Deve
Slide 23: Platform offers potential development opportunities via three modalities
Platform offers potential development opportunities via three modalities: autologous, allogeneic and in situ 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
Slide 24: Maturing pipeline offers potential for multiple catalysts
Maturing pipeline offers potential for multiple catalysts D escartes- 0 8 in MG D escartes- 0 8 in S L E Mid 2024 1H 2024 Expect to report Phase 2b data mid-2024 Plan to initiate Phase 2 in 1H 2024 D escartes- 0 8 A d d itio n al In d ic a tio n s D escartes- 1 5 2H 2024 Plan to initiate basket stud
Slide 25: Strong financial position expected to support pipeline through key milestones
Strong Financial ~$118M 162M common Position Expected to Pro forma cash as of 12/31/23* 696M basic** Support Shares outstanding as of 12/31/23 Pipeline Through Key Milestones <50 employees Anticipated cash runway into 2H 2026 Based in Gaithersburg, MD *Reflects the anticipated receipt of $40M throug
Slide 26: Pipeline designed to expand the reach of cell therapy to autoimmunity
PIONEERING mRNA CELL THERAPIES Pipeline designed to expand the reach of cell therapy to autoimmunity MATURING PIPELINE WITH EXPECTED NEAR-TERM CATALYSTS Validated lead program, Descartes-08, with Phase 2b data expected mid-year CASH RESOURCES EXPECTED TO FUND OPERATIONS INTO 2H 2026 Expected to supp
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