Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Investor Presentation (January 2023)
39 slides from Cartesian Therapeutics's investor presentation, Jan 9, 2023
Slide 1: Selecta Biosciences Corporate Presentation
January 2023 Selecta Biosciences Corporate Presentation SELB
Slide 2: Forward-looking statements
2Selecta Biosciences Investor Presentation – January 2023 Any statements in this presentation about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the “Company”), including without limitation, statements regarding the Company’s cash runway, the unique proprietary technolo
Slide 3: Diversified pipeline of novel therapeutic candidates; proof of concept in biologics and gene therapy
3Selecta Biosciences Investor Presentation – January 2023 Diversified pipeline of novel therapeutic candidates; proof of concept in biologics and gene therapy, expanding into autoimmune disease 1 2 3 4 Strong balance sheet, positioned to reach multiple value inflection points with expected runway in
Slide 4: Proprietary precision immune tolerance platform with potentially broad applicability
4Selecta Biosciences Investor Presentation – January 2023 Proprietary precision immune tolerance platform with potentially broad applicability ImmTOR combines nanoparticle technology with an FDA approved anti-inflammatory and immunomodulatory drug Designed to generate antigen-specific immune toleran
Slide 5: ImmTOR aims to restore self tolerance and overcome immunogenicity
5Selecta Biosciences Investor Presentation – January 2023 ImmTOR aims to restore self tolerance in autoimmune disease and overcome immunogenicity of gene therapies and biologics Autoimmune Disease Gene Therapies/Biologics
Slide 6: A diversified and growing wholly-owned pipeline
6Selecta Biosciences Investor Presentation – January 2023 A diversified and growing wholly-owned pipeline Recent and Expected Upcoming MilestonesIndication Antigen Preclinical Phase 1 Phase 3Phase 2 TOLEROGENIC THERAPIES Primary biliary cholangitis (PBC) PDC-E2 Undisclosed c GENE THERAPIES Phase 1 s
Slide 7: Unlocking the potential of our platform through collaborations
7Selecta Biosciences Investor Presentation – January 2023 c Unlocking the potential of our platform through collaborations Upcoming Milestones Antigen Preclinical Phase 1 Phase 3Phase 2 GENE THERAPIES IgA protease Chronic Refractory Gout DISSOLVE Topline Data Q1 2023Pegadricase Duchenne muscular dys
Slide 8: Restoring Self-Tolerance in Autoimmune Disease
8 Restoring Self-Tolerance in Autoimmune Disease
Slide 9: Striving to restore self-tolerance in autoimmune diseases
9Selecta Biosciences Investor Presentation – January 2023 Striving to restore self-tolerance in autoimmune diseases ImmTOR + IL-2 has the potential to be a best-in-class approach Approach: restore natural self-tolerance by administering ImmTOR with nanoparticle-encapsulated self-antigens and avoid t
Slide 10: ImmTOR-IL: ImmTOR plus IL-2 receptor agonist
10Selecta Biosciences Investor Presentation – January 2023 ImmTOR-IL : ImmTOR plus IL-2 receptor agonist ImmTOR-IL Treg-selective agonist IL-2 receptor • Synergistic mechanism of ImmTOR and a Treg-selective IL-2 • Identified an interleukin-2 (IL-2) cytokine with plans to advance it through the next
Slide 11: Induction and expansion of antigen-specific Treg
11Selecta Biosciences Investor Presentation – January 2023 Induction and expansion of antigen-specific Treg Observed a significant expansion of antigen-specific Treg* with a single dose of ImmTOR in combination with an IL-2 mutein + antigen 0 10 20 30 40 50 60 70 Antigen-specific Treg 0 10 20 30 40
Slide 12: ImmTOR-IL mitigates immunogenicity of high vector dose AAV gene therapy
12Selecta Biosciences Investor Presentation – January 2023 ImmTOR-IL mitigates immunogenicity of high vector dose AAV gene therapy ImmTOR + 4 monthly doses of IL-2 mutein inhibits anti-AAV antibodies at 5E13 vg/kg dose 12 19 33 47 61 75 89 103 117 131 0.0 0.5 1.0 1.5 Days AAV post-Infusion 12 19 33
Slide 13: Initial autoimmune disease focus: Primary Biliary Cholangitis (PBC)
13Selecta Biosciences Investor Presentation – January 2023 Initial autoimmune disease focus: Primary Biliary Cholangitis (PBC) ImmTOR + PDC-E2 antigen has the potential to address underlying autoimmune disease 1. Ursodeoxycholic acid, 2. Obeticholic acid 3. Purohit & Cappell (2015) 4. Lu et al (2018
Slide 14: ImmTOR-based approach to treating primary biliary cholangitis
14Selecta Biosciences Investor Presentation – January 2023 Hepatic regulatory T cells Tolerogenic liver sinusoidal endothelial cells We believe ImmTOR is ideally suited to address the root cause of PBC PBC is a T-cell mediated disease driven by a well-defined antigen, ImmTOR designed to biodistribut
Slide 15: Gene Therapy
15 Gene Therapy
Slide 16: AAV gene therapies are coming of age but still have challenges
16Selecta Biosciences Investor Presentation – January 2023 AAV gene therapies are coming of age but still have challenges Selecta has platform technologies to potentially address many key challenges facing the modality ImmTOR and Xork offer independent value creation opportunities with existing and
Slide 17: Aiming to have the leading toolkit to power AAV gene therapies
17Selecta Biosciences Investor Presentation – January 2023 Aiming to have the leading toolkit to power AAV gene therapies “High doses are needed to ensure therapeutic benefit” “Patient eligibility is limited”“Gene therapy is a one time only treatment” Low transduction efficiency and lack of organ sp
Slide 18: ImmTOR enhances transgene expression after first and second AAV doses
18Selecta Biosciences Investor Presentation – January 2023 Time after initial AAV treatment (d) ImmTOR has been observed to enhance transgene expression after first and second doses of AAV Repeat dosing enabled by ImmTOR is dose sparing 5e11 vg/kg AAV-SEAP 5e11 vg/kg AAAV-SEAP + ImmTOR 25e11 vg/kg A
Slide 19: Addressing two key challenges in AAV gene therapy
19Selecta Biosciences Investor Presentation – January 2023 Aiming to simultaneously address two key challenges in AAV gene therapy + ImmTOR Xork Xork IdeS 0.00 0.25 0.50 0.75 1.00 1.25 1.50 1.75 IdeS Xork Human IgG + + + + + Cleavage of human IgG Pre-existing anti-IgG protease Antibodies in human se
Slide 20: SEL-399 empty-AAV8 capsid dose-escalation study
20Selecta Biosciences Investor Presentation – January 2023 SEL-399 (empty-AAV8 capsid) Phase 1 dose-escalation study Total healthy volunteers enrolled: 23 (14 males and 9 females) All subjects with anti-AAV8 NAb titers <1:5 at baseline Randomized, placebo controlled and double-blind study n=3 0 15 3
Slide 21: Single dose ImmTOR inhibits anti-AAV8 NAb formation at day 30
21Selecta Biosciences Investor Presentation – January 2023 Single dose ImmTOR observed to inhibit anti-AAV8 NAb formation at day 30 2000 1000 100 10 Titer 1:5 Titer 1:25 1 Log10 Scale 2000 1500 1000 500 0 Linear Scale Empty Capsid (n=8) Empty Capsid + 0.15 mg/kg ImmTOR (n=9) Empty Capsid + 0.3 mg/kg
Slide 22: Summary and conclusions
22Selecta Biosciences Investor Presentation – January 2023 Summary and conclusions • We observed AAV8 empty capsids eliciting a strong immune response with peak median anti-AAV8 NAb titers of 1:6875 • We observed ImmTOR inhibiting the formation of anti-AAV8 NAb in a dose-dependent manner at Day 30 •
Slide 23: SEL-302 - Gene therapy program for MMA
23Selecta Biosciences Investor Presentation – January 2023 SEL-302 - Gene therapy program for the treatment of MMA Phase 1 start expected in Q4 2022 • Methylmalonic acidemia (MMA) is a rare monogenic metabolic disease with a potential live birth incidence of between 1:25,000 and 1:48,0001 • Majority
Slide 24: MMA Clinical Trial Design: Schedule of Events
24Selecta Biosciences Investor Presentation – January 2023 ImmTOR ImmTOR ImmTOR MMA Clinical Trial Design: Schedule of Events for Individual Subjects Key Biomarker Evaluations POBT sMMA NAb Pre-dose, Day, 21, 42, 70, and 84 Pre-dose, Day 7, 21, 42, 63, 70, 77, and 84 Pre-dose, Day 14, 28, 42, 56, 70
Slide 25: Biologics
25 Biologics
Slide 26: Biologics challenges and ImmTOR solution
26Selecta Biosciences Investor Presentation – January 2023 cTHE Challenges Many biologics can be highly immunogenic resulting in suboptimal responses to the standard of care due to the development of anti-drug antibodies (ADAs) after multiple treatments Patients that develop an immune response to th
Slide 27: Pegadricase immunogenicity and SEL-212 Phase 2 response
27Selecta Biosciences Investor Presentation – January 2023 Pegadricase is a highly immunogenic enzyme with most patients treated with pegadricase alone developing anti-drug antibodies within 2 weeks after a single treatment 5 monthly doses SEL-212* Pegadricase 0.2 mg/kg ImmTOR 0.1 or 0.15 mg/kg * Da
Slide 28: SEL-212 responders in patients with tophi
28Selecta Biosciences Investor Presentation – January 2023 1. Number of patients with tophi with Responder Assessment 2. Treatment difference = SEL-212 percent responder - pegloticase percent responder. Rounded to nearest integer Evaluation Period (Month) Data Set SEL-212 pegloticase Treatment Diffe
Slide 29: SEL-212 Phase 3 DISSOLVE program design
29Selecta Biosciences Investor Presentation – January 2023 SEL-212 phase 3 DISSOLVE program design Evaluating SEL-212 in a pivotal phase 3 program vs. placebo, joint topline data expected in Q1 2023 • 2 double blinded placebo-controlled trials of SEL-212 (0.1 mg/kg & 0.15 mg/kg ImmTOR) Both studies
Slide 30: Opportunity to address unmet medical needs for the treatment of IgAN
30Selecta Biosciences Investor Presentation – January 2023 Opportunity to address unmet medical needs for the treatment of IgAN • Immunoglobulin A nephropathy (IgAN) is a leading cause of chronic kidney disease (CKD) and renal failure with 30-40% of patients reaching end-stage renal disease; approxi
Slide 31: Combining ImmTOR with IgA protease for IgAN
31Selecta Biosciences Investor Presentation – January 2023 Combining ImmTOR with IgA protease for the treatment of IgAN Building on the clinical data from the SEL-212 program and strong preclinical data in IgA Saline IgA protease 5mg/kg IV Glomerular IgA1 deposits Complement deposition Fibronectin A
Slide 32: Corporate
32 Corporate
Slide 33: Experienced management team positions Selecta for success
33Selecta Biosciences Investor Presentation – January 2023 Peter G. Traber, M.D. Chief Medical Officer Experienced management team positions Selecta for success Lloyd Johnston, Ph.D. Chief Operations Officer Carsten Brunn, Ph.D. President and CEO Kei Kishimoto, Ph.D. Chief Scientific Officer Kristen
Slide 34: Positioned for growth in 2023
34Selecta Biosciences Investor Presentation – January 2023 1. Unaudited 2. Includes cash, cash equivalents, marketable securities and restricted cash. Key 2023 milestones: Licensed next-generation IgG protease candidate, Xork, for development with AT845, Astellas Gene Therapies’ investigational prod
Slide 35: Diversified pipeline of novel therapeutic candidates; proof of concept
35Selecta Biosciences Investor Presentation – January 2023 Diversified pipeline of novel therapeutic candidates; proof of concept in biologics and gene therapy, expanding into autoimmune disease 1 2 3 4 Strong balance sheet, positioned to reach multiple value inflection points with expected runway i
Slide 36: Appendix
36 Appendix
Slide 37: Single dose ImmTOR delayed NAb formation by day 90
37Selecta Biosciences Investor Presentation – January 2023 Subjects treated with a single dose of ImmTOR developed delayed NAb formation by day 90 Titer 1:25 Day 90 Day 30 Empty Capsid (n=8) Empty Capsid + 0.15 mg/kg ImmTOR (n=9) Empty Capsid + 0.3 mg/kg ImmTOR (n=6) Titer 1:5
Slide 38: Empty capsid data in-line with single dose ImmTOR NHP data
38Selecta Biosciences Investor Presentation – January 2023 Empty capsid data in-line with single dose ImmTOR NHP data at day 90 Three monthly doses of ImmTOR provide inhibition of NAbs in NHP Antibody levels may interfere with efficient re-dosing** Titers too high for re-dosing Patients eligible for
Slide 39: MMA Clinical Trial Design: Schedule of Events
39Selecta Biosciences Investor Presentation – January 2023 Cohort 2 Children (n=3) Day 336 448 672560 Assumes 1 month (28 days) between Day 84 cutoff and subsequent participant enrollment to allow for DSMB report generation and review. MMA-101 (1.0E13 vg/kg) Cohort 1 Cohort 1: Adolescents (n=3) Day
Selecta Biosciences — Investor Presentation (January 2023) on 6ix