Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Corporate Update (July 2024)
29 slides from Cartesian Therapeutics's corporate update, Jul 2, 2024
Slide 1: Topline Data from Phase 2b Trial of Descartes-08 in Myasthenia Gravis
Topline Data from Phase 2b Trial of Descartes-08 in Patients with Myasthenia Gravis July 2024 CARTESIAN THERAPEUTICS
Slide 2: Disclosures and forward-looking statements
Disclosures and 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 b
Slide 3: Met primary endpoint with 71% response rate vs. 25% for placebo
Met primary endpoint 71% response rate at Month 3 for Descartes-08 patients vs. 25% for placebo (p<0.05) Deep, durable responses observed in patients treated with Descartes-08 Safety profile continues to support outpatient administration Data support advancement to Phase 3 2 Deep, durable responses
Slide 4: Myasthenia gravis is a rare, progressive autoimmune disease with significant unmet need
Current treatments require chronic or frequent administration and have limited durability >120,000 Patients in the U.S. and EU Significant unmet need remains Characterized by debilitating fatigue and muscle weakness Limbs Respiratory Ocular Facial Myasthenia gravis is a rare, progressive autoimmune
Slide 5: Cartesian's mRNA approach designed to expand reach of cell therapy to autoimmunity
mRNA cell therapy does not require lymphodepleting chemotherapy No associated cytopenia, secondary malignancies, or other chemotherapy toxicities Reduced burden on patients, caregivers, and healthcare system Convenient dosing schedule (six weekly infusions) Cartesian’s mRNA approach is designed to e
Slide 6: Descartes-08 believed to be first mRNA CAR-T in clinical development for autoimmune disease
5 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 Granted U.S. FDA orphan and RMAT designations for generalized
Slide 7: Phase 2b trial design: double-blind, placebo-controlled trial in myasthenia gravis
Day-70 Day-60 Day-50 Day-40 Day-30 Day-20 Day-10 Phase 2b trial: double-blind, placebo-controlled clinical trial of Descartes-08 in patients with myasthenia gravis Leukapheresis & Cell Processing Days -59 to -10 Randomization WK1 WK 4 WK 7 10 13 16 19 22 25 28 31 34 37 Dosing Six weekly doses Blinde
Slide 8: Patients randomized and primary efficacy dataset
14 patients received Descartes-08 and 12 patients received placebo in the pre-specified primary efficacy dataset 27 Academic medical centers 9 Community clinics 1 Early drop-out 26 Primary efficacy dataset 14 Randomized to Descartes-08 12 Randomized toplacebo 5 Randomized to Descartes-08 4 Randomize
Slide 9: Baseline characteristics: highly symptomatic patient population with severe disease
Baseline characteristics: highly symptomatic patient population with severe disease 1 for AChR, MuSK, and LRP4 antibodies MGC, MG Composite 8
Slide 10: Prior and ongoing treatments: heavily pre-treated patient population
Prior and ongoing treatments: heavily pre-treated patient population *Significant imbalance (p<0.05) 9
Slide 11: Trial met primary endpoint with statistical significance
Trial met primary endpoint with statistical significance 10 p-value: 0.018 Responders: Descartes-08, n= 10/14; placebo, n=3/12 Odds ratio 7.5 (95% CI, 1.3, 43) *Clinically meaningful response is a three-point reduction from baseline Responders observed to have ~3x greater improvements than clinicall
Slide 12: Mean decrease from baseline in primary efficacy population
11 Mean decrease from Baseline in the prespecified primary efficacy population (n=26) p<0.05 by Mann-Whitney U test at Month 3 in MGC and MG-ADL LRP4+, low-density lipoprotein receptor-related protein 4 Statistically significant improvements observed in Descartes-08 patients at Month 3 assessment No
Slide 13: Deep and durable responses in responders through Month 6
12 Mean decrease from Baseline in MGC Responders (participants who achieved a ≥5-point reduction in MGC at Month 3, n=10. Month 4 n=5, Month 6 n=3. Deep and durable responses observed in Descartes-08 responders through Month 6 Results consistent with Phase 2a open-label trial findings 11 Mean decrea
Slide 14: Safety results support outpatient administration
Observed safety results support outpatient administration and in line with Phase 2a observations 13 Descartes-08 (n=19) Placebo (n=17) Grade 1 Grade 2 Grade 3 Grade 1 Grade 2 Grade 3 Headache 6 (32%) 4 (21%) 2 (12%) 3 (18%) Chills 7 (37%) 4 (21%) 1 (6%) Nausea 2 (11%) 5 (26%) 2 (12%) 2 (12%) Fever 6
Slide 15: Phase 2a update
Phase 2a update
Slide 16: Phase 2a update: retreatment led to sustained clinically meaningful responses
Phase 2a update:Descartes-08 retreatment led to sustained clinically meaningful responses Manuscript submitted for peer review; pre-print available at medRxiv.org Patient 1 Patient 2 15 Retreated patients experienced rapid improvement in clinical scores and maintained minimal symptom expression for
Slide 17: Summary and Next Steps for Descartes-08 in MG
Summary and Next Steps for Descartes-08 in MG
Slide 18: Met primary endpoint with 71% response rate vs. 25% for placebo
Met primary endpoint 71% response rate at Month 3 for Descartes-08 patients vs. 25% for placebo (p<0.05) Deep, durable responses observed in patients treated with Descartes-08 Safety profile continues to support outpatient administration Data support advancement to Phase 3 17 Deep, durable responses
Slide 19: Planned next steps for Descartes-08 in MG
Planned next steps for Descartes-08 in MG 18 End of Phase 2 meeting with FDA expected by year-end Initiate Phase 3 clinical trial RMAT designation to support efficient development plan in collaboration with FDA
Slide 20: Leveraging proof-of-concept in MG to expand autoimmune pipeline
SLE Open-label Phase 2 trial Announced dosing of first patient Potential New Indications RA, AIE, SSc Pediatric autoimmune diseases MG End of Phase 2 FDA meeting expected by end of 2024 Potential initiation of Phase 3 trial following FDA meeting Leveraging clinical proof-of-concept of Descartes-08 i
Slide 21: Appendix
Appendix
Slide 22: Trial met primary endpoint with statistical significance
Trial met primary endpoint with statistical significance Responders in pre-specified analysis observed to have ~3x greater improvements than clinically meaningful* Data support advancement to Phase 3 Pre-Specified Responders: Descartes-08, n= 10/14; placebo, n=3/12 Odds ratio 7.5 (95% CI, 1.3, 43) *
Slide 23: Improvement across measures of disease activity in AChR Ab+ MG subjects
22 Descartes-08 demonstrated improvement across important measures of disease activity in AChR Abᐩ MG subjects Statistically significant improvement in Descartes-08 compared to placebo at Month 3 seen across MGC (p=0.002), MG-ADL (p=0.012) and QMG (p=0.029). Placebo responses in AChR Ab+ subjects we
Slide 24: Placebo score reductions driven by three seronegative subjects
23 Score reductions in measures of disease activity in placebo group was driven by responses in three seronegative subjects Seronegative AChR Abᐩ Mean change from baseline in AChR Abᐩ (n=9) and seronegative (n=3) participants randomized to placebo
Slide 25: Similar score reductions across antibody subgroups receiving Descartes-08
24 Score reductions in measures of disease activity were similar in all antibody subgroups of patients receiving Descartes-08 Seronegative AChR or LRP4 Abᐩ Mean change from baseline in AChR or LRP4 Abᐩ (n=10) and seronegative (n=4) participants randomized to placebo
Slide 26: Deepest responses in participants with no prior complement or FcRn inhibitors
25 Descartes-08 Deepest responses seen in participants with no prior exposure to complement or FcRn inhibitors Mean change from baseline in in patients with no prior biologics (Months 1–3 n=8, Month 4 n=4, Month 6 n=2)
Slide 27: Plans for continued advancement in MG and expansion into additional autoimmune indications
Plans for continued advancement of Descartes-08 in MG and expansion into additional autoimmune indications Leverage strong clinical results from Descartes-08 in MG to initiate additional Phase 2 trials with a particular focus on Rheumatology and Neurology Potential new indications targeting both lar
Slide 28: Wholly-owned, in-house manufacturing: 27,000 sq. ft. 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 Potential cost efficiency Clinical and commercial manufacturing scale capabilities support maturing p
Slide 29: Plans to scale operations to support long-term growth
Plans to scale operations to support long-term growth of organization 28 Manufacturing Organizational Structure Commercial Readiness Investment in manufacturing capacity to support additional clinical programs and future commercial launch of MG indication, if approved Expand organizational structure
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