Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Investor Presentation (November 2022)
44 slides from Cartesian Therapeutics's investor presentation, Nov 18, 2022
Slide 1: Selecta Biosciences Corporate Presentation
November 2022 Selecta Biosciences Corporate Presentation SELB
Slide 2: Forward-looking statements
2Selecta Biosciences Investor Presentation – November 2022 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 technol
Slide 3: Pioneering Precision Immune Tolerance
3Selecta Biosciences Investor Presentation – August 2022 PLACEHOLDER FOR SPLASH PAGE @Jess can you pull in design elements from the website for this page? Pioneering Precision Immune Tolerance
Slide 4: Company Highlights
4Selecta Biosciences Investor Presentation – November 2022 Company Highlights ImmTOR™ platform has potentially broad applicability • Precision immune tolerance platform has potential to restore self tolerance in autoimmune disease and overcome immunogenicity of gene therapies and biologics • Preclin
Slide 5: ImmTOR Platform: Precision Immune Tolerance
5 ImmTOR Platform Precision Immune Tolerance
Slide 6: A precision immune tolerance platform with potentially broad applicability
6Selecta Biosciences Investor Presentation – November 2022 A precision immune tolerance platform with potentially broad applicability ImmTOR combines nanoparticle technology with an FDA approved anti-inflammatory and immunomodulatory drug, and is designed to generate antigen-specific immune toleranc
Slide 7: ImmTOR applications in autoimmune disease and gene therapies/biologics
7Selecta Biosciences Investor Presentation – November 2022 ImmTOR could potentially be applied to restore self tolerance in autoimmune disease and overcome immunogenicity of gene therapies and biologics Autoimmune Disease Gene Therapies/Biologics
Slide 8: ImmTOR-IL: ImmTOR plus IL-2 receptor agonist
8Selecta Biosciences Investor Presentation – November 2022 ImmTOR-IL : ImmTOR plus IL-2 receptor agonist ImmTOR-IL Treg-selective IL-2 receptor agonist Synergistic mechanism of ImmTOR and a Treg-selective IL-2: Observed to greatly increase the magnitude and durability of antigen-specific Treg expa
Slide 9: Aiming to restore self tolerance and power biologics
9Selecta Biosciences Investor Presentation – November 2022 Aiming to restore self tolerance to auto antigens and power biologics Biologic Therapies Gene Therapies Tolerogenic Therapies ImmTOR is designed to address the immunogenicity of biologics ImmTOR potentially enables redosing of transformative
Slide 10: A diversified and growing wholly-owned pipeline
10Selecta Biosciences Investor Presentation – November 2022 c A diversified and growing wholly-owned pipeline Recent and Expected Upcoming Milestones TOLEROGENIC THERAPIES Indication Antigen Preclinical Phase 1 Phase 3Phase 2 Commercial Rights BIOLOGIC THERAPIES Ornithine Transcarbamylase (OTC) Defi
Slide 11: Strategic collaborations and partnerships
11Selecta Biosciences Investor Presentation – November 2022 Collaboration Year 2019 2020 2020 2021 2021 2021 2021 2022 ImmTOR Approach Gene Therapy Biologic Gene Therapy Autoimmune Gene Therapy Gene Therapy Biologic Gene Therapy Agreement Strategic Collaboration Agreement License Agreement (Global,
Slide 12: Restoring Self-Tolerance in Autoimmune Disease
12 Restoring Self-Tolerance in Autoimmune Disease
Slide 13: Striving to restore self-tolerance in autoimmune diseases
13Selecta Biosciences Investor Presentation – November 2022 Striving to restore self-tolerance in autoimmune diseases ImmTOR + IL-2 has the potential to be a best-in-class approach There are roughly 80 autoimmune conditions that affect as much as 4.5% of the world’s population*. 24M+ individuals in
Slide 14: Induction and expansion of antigen-specific Treg
14Selecta Biosciences Investor Presentation – November 2022 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 CD 4+ CD 25 + Fo xP 3+ (% , o f C D4 + CD 45 .2 + ) 0 10 2
Slide 15: Superior anti-AAV antibody inhibition with IL-2 combined with ImmTOR
15Selecta Biosciences Investor Presentation – November 2022 Superior anti-AAV antibody inhibition observed when IL-2 is combined with ImmTOR Clear dose sparing effect seen when IL-2 mutein is combined with ImmTOR* An ti- AA V Ig G (O D 45 0- 57 0) 12 19 33 47 61 75 91 104 117 0.0 0.5 1.0 1.5 2.0 No
Slide 16: Immunogenicity of high dose AAV mitigated by ImmTOR-IL
16Selecta Biosciences Investor Presentation – November 2022 Immunogenicity of high vector dose AAV gene therapy mitigated by ImmTOR- IL ImmTOR + 4 monthly doses of IL-2 mutein observed to inhibit anti-AAV antibodies at 5E13 vg/kg dose An ti- AA V8 Ig G (O D 45 0- 57 0) 5E13 vg/kg AAV8-SEAP 5E13 vg/k
Slide 17: Initial autoimmune disease focus: Primary Biliary Cholangitis
17Selecta Biosciences Investor Presentation – November 2022 Initial autoimmune disease focus: Primary Biliary Cholangitis (PBC) We believe ImmTOR-IL + PDC-E2 antigen has the potential to restore immune tolerance in the liver 1. Yeaman SJ, Fussey SP, Danner DJ, et al. Primary biliary cirrhosis: ident
Slide 18: ImmTOR is ideally suited to address PBC
18Selecta Biosciences Investor Presentation – November 2022 Hepatic regulatory T cells Tolerogenic liver sinusoidal endothelial cells We believe ImmTOR is ideally suited to address PBC • Patients with PBC need a highly-targeted, liver-directed approach to treating the root cause of the disorder • Im
Slide 19: Gene Therapy
19 Gene Therapy
Slide 20: AAV gene therapies are coming of age but still have challenges
20Selecta Biosciences Investor Presentation – November 2022 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 – Human proof of concept shows the possibility for ImmTOR to inhibit the
Slide 21: Leading toolkit to power AAV gene therapies
21Selecta Biosciences Investor Presentation – November 2022 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
Slide 22: Potential for ImmTOR to enhance AAV gene therapies
22Selecta Biosciences Investor Presentation – November 2022 Potential for ImmTOR to enhance AAV gene therapies Safer, more durable AAV gene therapy treatments are within reach
Slide 23: Addressing two key challenges in AAV gene therapy
23Selecta Biosciences Investor Presentation – November 2022 Aiming to simultaneously address two key challenges in AAV gene therapy + = ImmTOR Xork • Potential to increase the number of patients eligible for gene therapy by mitigating pre-existing anti-AAV antibodies • Potential to enable re-dosing
Slide 24: ImmTOR enhances transgene expression after first and second AAV doses
24Selecta Biosciences Investor Presentation – November 2022 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
Slide 25: SEL-399 dose-escalation study: subjects and design
25Selecta Biosciences Investor Presentation – November 2022 SEL-399 Phase 1 dose-escalation study: subjects and design 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=
Slide 26: Single dose ImmTOR inhibits anti-AAV8 NAb formation at day 30
26Selecta Biosciences Investor Presentation – November 2022 Single dose ImmTOR observed to inhibit anti-AAV8 NAb formation at day 30 100% of subjects dosed with 0.3 mg/kg ImmTOR had NAb titers ≤1:25 at Day 30 67% of subjects dosed with 0.3 mg/kg ImmTOR had NAb titers ≤1:5 at Day 30 2000 1000 100 10
Slide 27: Delayed NAb formation by day 90
27Selecta Biosciences Investor Presentation – November 2022 Subjects treated with a single dose of ImmTOR developed delayed NAb formation by day 90 2 of 6 subjects dosed with 0.3 mg/kg ImmTOR had NAb titers ≤1:25 at Day 90 1 of 6 subjects dosed with 0.3 mg/kg ImmTOR had NAb titers ≤1:5 at Day 90 Tit
Slide 28: Empty capsid data in line with NHP data at day 90
28Selecta Biosciences Investor Presentation – November 2022 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 fo
Slide 29: Summary and conclusions
29Selecta Biosciences Investor Presentation – November 2022 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 30: SEL-302 - Gene therapy program for MMA
30Selecta Biosciences Investor Presentation – November 2022 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 1:48,000 1 • Majori
Slide 31: MMA Clinical Trial Design: Schedule of Events for Individual Subjects
31Selecta Biosciences Investor Presentation – November 2022 POBT=1-13C -sodium propionate oxidative capacity using breath test; sMMA= serum methylmalonic acid levels; NAb=neutralizing anti-AAV8 antibodies *Interim Endpoint= Data cutoff for Data Safety Monitoring Board evaluation ImmTOR ImmTOR ImmTOR
Slide 32: MMA Clinical Trial Design: Schedule of Events
32Selecta Biosciences Investor Presentation – November 2022 MMA-101 (1.0E13 vg/kg) Cohort 1 Adolescents (n=3) (ages ≥ 12 and < 18) Day 1 DSMB DSMB 84 112 308 336196 224 No ImmTOR; steroid prophylaxis ImmTOR; no steroid prophylaxis MMA-101 (1.0E13 vg/kg) ImmTOR (0.15 mg/kg) MMA-101 (1.0E13 vg/kg) Imm
Slide 33: Biologics
33 Biologics
Slide 34: Biologic therapies potentially enhanced by ImmTOR
34Selecta Biosciences Investor Presentation – November 2022 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 t
Slide 35: Pegadricase is highly immunogenic when given alone
35Selecta Biosciences Investor Presentation – November 2022 Pegadricase is highly immunogenic when given alone Pegadricase is a highly immunogenic enzyme with most patients treated with pegadricase alone developing anti-drug antibodies within 2 weeks after a single treatment Days after treatment 0 5
Slide 36: SEL-212 vs pegloticase responders in patients with tophi
36Selecta Biosciences Investor Presentation – November 2022 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 Diff
Slide 37: SEL-212 phase 3 DISSOLVE program design
37Selecta Biosciences Investor Presentation – November 2022 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 studi
Slide 38: Unmet medical needs in the treatment of IgAN
38Selecta Biosciences Investor Presentation – November 2022 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; approx
Slide 39: Combining ImmTOR with IgA protease for IgAN
39Selecta Biosciences Investor Presentation – November 2022 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
Slide 40: Corporate
40 Corporate
Slide 41: Experienced management team
41Selecta Biosciences Investor Presentation – November 2022 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 Kriste
Slide 42: Financial information at-a-glance
42Selecta Biosciences Investor Presentation – November 2022 1. Unaudited 2. Includes cash, cash equivalents, marketable securities and restricted cash. • Top-line data from Phase 3 DISSOLVE I & II programs of SEL-212 in chronic refractory gout • Phase 1 clinical trial initiation and preliminary SEL-
Slide 43: Company Highlights
43Selecta Biosciences Investor Presentation – November 2022 Company Highlights ImmTOR™ platform has potentially broad applicability • Precision immune tolerance platform has potential to restore self tolerance in autoimmune disease and overcome immunogenicity of gene therapies and biologics • Precli
Slide 44: Contact: Selecta Biosciences, Inc.
44 Contact: SELECTA BIOSCIENCES, INC. 65 Grove Street Watertown, MA 02472 617-923-1400 info@selectabio.com
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