Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Investor Presentation (January 2024)
23 slides from Cartesian Therapeutics's investor presentation, Jan 22, 2024
Slide 1: Pioneering mRNA Cell Therapy for Autoimmunity
CARTESIAN THERAPEUTICS Pioneering mRNA Cell Therapy for Autoimmunity January 2024
Slide 2: Forward-Looking Statements
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 “ ompany”) or any person on their behalf, any quest
Slide 3: Clinical-stage company pioneering mRNA cell therapies; near-term catalysts
MULTIPLE ANTICIPATED Clinical-stage company NEAR-TERM CATALYSTS pioneering mRNA cell DESCARTES-08 therapies designed to • Phase 2b topline data in MG expected mid-2024 expand the reach of cell • Initiation of Phase 2 study in SLE expected in 1H 2024 • Initiation of studies in additional autoimmune i
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 M AN A G E M E N T 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 G
Slide 5: Conventional engineered cell therapy uses DNA, which can lead to toxicity and increased patient burden
Conventional engineered cell therapy uses DNA, which can DNA transduced CAR-T associated with: lead to toxicity and increased patient burden • Cytokine release syndrome (CRS) • Neurotoxicity and parkinsonism • Effector function of cell therapy engineered with • Cytopenia (from pre-treatment chemo) D
Slide 6: Cartesian's mRNA approach is designed to expand the reach of potent cell therapy
Cartesian’s mRNA approach is designed to expand the Expectation for cells to be administered at the reach of potent cell therapy therapeutic but sub-toxic doses products to address Descartes-08 has been administered to 66 potential autoimmune patients with autoimmune diseases and cancer1 indications
Slide 7: Wholly-owned pipeline targets autoimmune disease
Wholly-owned pipeline targets autoimmune disease Asset Indications Discovery/Preclinical Phase 1 Phase 2 Pivotal Myasthenia Gravis Descartes-08 Autologous mRNA CAR-T SLE, other Autoimmune Diseases Descartes-15 Autologous Autoimmune Diseases* mRNA CAR-T Descartes-33 Allogeneic mRNA Autoimmune Disease
Slide 8: 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 9: 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 10: Phase 2 study of Descartes-08 in MG (NCT04146051)
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 schedule Part 3 to infusion (n = 11) Phase 2b • 4-week washout for biologics compa
Slide 11: Phase 1/2a study population comprises patients with significant disease
Mean age, years (SD) 52 (18) Previous myasthenia gravis therapies (standard of care) Female 10 (71%) Phase 1/2a Male 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 12: Descartes-08 was observed to be safe and well-tolerated in MG
Descartes-08 Grade* Part 1 (n=3) Part 2: all groups Part 2: group 1 Part 2: group 2 Part 2: group 3 was observed to Hand numbness 2 1 (33%) (n=11) 0 (n=3) 0 (n=7) 0 (n=1) 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 13: 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 14: 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 titers are Retreated patient experienced rapid improvement in clinical scores consistent with the observed clinical which was ongoing at Month 6 of retreatment follow-up w
Slide 15: 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 16: 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 17: 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 that improvement observed in a patient with autoimmune retinitis (AIR) Descartes-08 could lead to clinical 61-year-old man w
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) Descartes-15 is an anti-BCMA mRNA CAR-T with potential 100 Descartes-15 DC-15 100 Descartes-15
Slide 19: In-house manufacturing enhances control of product quality, production schedules and costs
cGMP Cell Manufacturing In-house 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 20: 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 21: Maturing pipeline offers potential for multiple catalysts
Maturing pipeline offers potential for multiple catalysts D e s c a r tes- 08 i n M G D e s c a r tes - 08 i n SLE Mid 2024 1H 2024 Expect to report Phase 2b data mid-2024 Plan to initiate Phase 2 in 1H 2024 D e s c a r te s- 08 Addi t i ona l I ndi c ations D e s c a r tes - 15 2H 2024 Plan to init
Slide 22: Strong Financial Position Expected to Support Pipeline Through Key Milestones
162M common Strong Financial ~$118M 696M basic** Position Expected to Pro forma cash as of 12/31/23* Support Shares outstanding as of 12/31/23 Pipeline Through Key <50 employees Milestones Anticipated cash runway into 2H 2026 Based in Gaithersburg, MD *Reflects the anticipated receipt of $40M throug
Slide 23: 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
Cartesian Therapeutics — Investor Presentation (January 2024) on 6ix