Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Investor Presentation (April 2022)
43 slides from Cartesian Therapeutics's investor presentation, Apr 13, 2022
Slide 1: Selecta Biosciences Corporate Presentation
April 2022 Selecta Biosciences Corporate Presentation SELB
Slide 2: Forward-looking statements
2Selecta Biosciences Investor Presentation – April 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 ability to complete the proposed offering on f
Slide 3: Pioneering Precision Immune Tolerance
3Selecta Biosciences Investor Presentation – April 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 – April 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 • Preclinica
Slide 5: ImmTOR Platform: Precision Immune Tolerance
5 ImmTOR Platform Precision Immune Tolerance
Slide 6: A precision immune tolerance platform with broad applicability
6Selecta Biosciences Investor Presentation – April 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 tolerance w
Slide 7: ImmTOR applications in autoimmune disease and gene therapies/biologics
7Selecta Biosciences Investor Presentation – April 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 – April 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 expansi
Slide 9: Restoring self tolerance and powering biologics
9Selecta Biosciences Investor Presentation – April 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 ge
Slide 10: A diversified and growing wholly-owned pipeline
10Selecta Biosciences Investor Presentation – April 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) Deficie
Slide 11: Strategic collaborations and licensing agreements
11Selecta Biosciences Investor Presentation – April 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 and License Agreement License Agreement
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 – April 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 the
Slide 14: Amplifying the effect of ImmTOR with IL-2
14Selecta Biosciences Investor Presentation – April 2022 Expanding the platform by amplifying the effect of ImmTOR with IL-2 Observed synergistic activity in increasing the percentage and durability of Treg expansion in the spleen Opportunity to restore tolerance in a wide range of autoimmune di
Slide 15: Induction and expansion of antigen-specific Treg
15Selecta Biosciences Investor Presentation – April 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 C D 4+ C D 25 + F o x P 3+ ( % , o f C D 4 + C D 45 .2 + ) 0
Slide 16: Superior anti-AAV antibody inhibition with IL-2 plus ImmTOR
16Selecta Biosciences Investor Presentation – April 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* A n ti- A A V I g G ( O D 4 5 0 -5 7 0 ) 12 19 33 47 61 75 91 104 117 0.0 0.5 1.0 1.5 2.0
Slide 17: ImmTOR-based approach to treating PBC
17Selecta Biosciences Investor Presentation – April 2022 Hepatic regulatory T cells Tolerogenic liver sinusoidal endothelial cells We believe ImmTOR is ideally suited to address PBC PBC is a T-cell mediated disease driven by a well-defined antigen, ImmTOR biodistributes to the liver and induces a to
Slide 18: Gene Therapy
18 Gene Therapy
Slide 19: AAV gene therapies are coming of age but still have challenges
19Selecta Biosciences Investor Presentation – April 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 for
Slide 20: Potential for ImmTOR to enhance AAV gene therapies
20Selecta Biosciences Investor Presentation – April 2022 Potential for ImmTOR to enhance AAV gene therapies Safer, more durable AAV gene therapy treatments are within reach
Slide 21: Addressing two key challenges in AAV gene therapy
21Selecta Biosciences Investor Presentation – April 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 by
Slide 22: ImmTOR enhances transgene expression after first and second AAV doses
22Selecta Biosciences Investor Presentation – April 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 AAV
Slide 23: SEL-399 dose-escalation study: subjects and design
23Selecta Biosciences Investor Presentation – April 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=3 0
Slide 24: Single dose ImmTOR inhibits anti-AAV8 NAb at day 30
24Selecta Biosciences Investor Presentation – April 2022 Single dose ImmTOR observed to inhibit formation of anti-AAV8 NAb 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 10L
Slide 25: Dose-dependent inhibition of median anti-AAV8 NAb at day 30
25Selecta Biosciences Investor Presentation – April 2022 Single dose ImmTOR observed to inhibit formation of median anti-AAV8 NAb in a dose-dependent manner at day 30 1:5 Median NAb titers in subjects dosed with 0.3 mg/kg ImmTOR at Day 30 250-fold lower median NAb titers in subjects dosed with 0.3 m
Slide 26: Delayed NAb formation by day 90
26Selecta Biosciences Investor Presentation – April 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 Titer
Slide 27: Empty capsid data in line with NHP data at day 90
27Selecta Biosciences Investor Presentation – April 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 for r
Slide 28: Summary and conclusions
28Selecta Biosciences Investor Presentation – April 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 A
Slide 29: SEL-302 gene therapy program for MMA
29Selecta Biosciences Investor Presentation – April 2022 SEL-302 - Gene therapy program for the treatment of MMA Phase 1 start expected in 2H 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 • Majority
Slide 30: MMA clinical trial design: schedule of events
30Selecta Biosciences Investor Presentation – April 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 MM
Slide 31: MMA clinical trial design: cohort schedule
31Selecta Biosciences Investor Presentation – April 2022 Cohort 2 Children (n=3) (ages ≥ 2 and <12) 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 Adolescents (n=
Slide 32: Biologics
32 Biologics
Slide 33: Biologic therapies potentially enhanced by ImmTOR
33Selecta Biosciences Investor Presentation – April 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 the
Slide 34: Pegadricase is highly immunogenic when given alone
34Selecta Biosciences Investor Presentation – April 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 10
Slide 35: SEL-212 responder data in patients with tophi
35Selecta Biosciences Investor Presentation – April 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 Differe
Slide 36: SEL-212 phase 3 DISSOLVE program design
36Selecta Biosciences Investor Presentation – April 2022 SEL-212 phase 3 DISSOLVE program design Evaluating SEL-212 in a pivotal phase 3 program vs. placebo, DISSOLVE I topline data expected in Q4 2022 • 2 double blinded placebo-controlled trials of SEL-212 (0.1 mg/kg & 0.15 mg/kg ImmTOR) − Both stu
Slide 37: Unmet medical needs in the treatment of IgAN
37Selecta Biosciences Investor Presentation – April 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; approxima
Slide 38: Combining ImmTOR with IgA protease for IgAN
38Selecta Biosciences Investor Presentation – April 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 Ada
Slide 39: Corporate
39 Corporate
Slide 40: Experienced management team
40Selecta Biosciences Investor Presentation – April 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 Kristen B
Slide 41: Financial information at-a-glance and upcoming milestones
41Selecta Biosciences Investor Presentation – April 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-302
Slide 42: Company Highlights
42Selecta Biosciences Investor Presentation – April 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 • Preclinic
Slide 43: Contact: Selecta Biosciences, Inc.
43 Contact: SELECTA BIOSCIENCES, INC. 65 Grove Street Watertown, MA 02472 617-923-1400 info@selectabio.com
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