Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Investor Presentation (April 2022)
43 slides from Cartesian Therapeutics's investor presentation, Apr 6, 2022
Slide 1: Selecta Biosciences Corporate Presentation April 2022
° 2+ Selecta. O° Biosciences Exhibit 99.1 pl nasdaq | SELB Selecta Biosciences Corporate Presentation April 2022
Slide 2: Forward-looking statements
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 technology platform of the Company, and the unique proprietary pla
Slide 3: @Jess can you pull in design elements from the website for this page? 3
@Jess can you pull in design elements from the website for this page? Selecta Biosciences Corporate Presentation – April 2022 3
Slide 4: Precision immune tolerance platform highlights
• Precision immune tolerance platform has potential to restore self tolerance in autoimmune disease and overcome ImmTOR™ platform has immunogenicity of gene therapies and biologics potentially broad applicability • Preclinical data indicates potentially profound synergy of ImmTOR and engineered Treg
Slide 5: ImmTOR Platform Precision Immune Tolerance
ImmTOR Platform Precision Immune Tolerance 5
Slide 6: ImmTOR combines nanoparticle technology with an FDA approved immunomodulatory drug
ImmTOR combines nanoparticle technology with an FDA approved anti-inflammatory and immunomodulatory drug, and is designed to generate antigen-specific immune tolerance when combined with an antigen of interest Selecta Biosciences Corporate Presentation – April 2022 6
Slide 7: Autoimmune Disease Gene Therapies/Biologics
Autoimmune Disease Gene Therapies/Biologics Selecta Biosciences Corporate Presentation – April 2022 7
Slide 8: ImmTOR-IL synergistic mechanism with Treg-selective IL-2
Treg-selective IL-2 receptor agonist ImmTOR Synergistic mechanism of ImmTOR and a Treg-selective IL- 2: ▪ Observed to greatly increase the magnitude and durability of antigen-specific Treg expansion when compared to either ImmTOR or IL-2 alone ImmTOR-IL ▪ Proof of concept human data in which we obse
Slide 9: ImmTOR applications across tolerogenic, gene and biologic therapies
Tolerogenic Gene Biologic Therapies Therapies Therapies ImmTOR could provide targeted immune ImmTOR potentially enables redosing of ImmTOR is designed to address the tolerance to auto antigens transformative gene therapies immunogenicity of biologics 80% of rare disease has a known Over 160,000 pati
Slide 10: Pipeline
Recent and Expected Commercial Rights Indication Antigen Preclinical Phase 1 Phase 2 Phase 3 Upcoming Milestones TOLEROGENIC THERAPIES Primary biliary cholangitis (PBC) PDC-E2 Undisclosed GENE THERAPIES AAV (serotype undisclosed) SEL-302 Phase 1 start 2H 2022 Methylmalonic acidemia (MMA) AAV-hOTC Or
Slide 11: Strategic collaborations
Collaboration Year 2019 2020 2020 2021 2021 2021 2021 2022 ImmTOR Gene Therapy Biologic Gene Therapy Autoimmune Gene Therapy Gene Therapy Biologic Gene Therapy Approach Strategic licensing Strategic licensing Strategic licensing Strategic Research Option Collaboration to Strategic licensing License
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Slide 13: Autoimmune disease challenges and solution
The current standard of care for autoimmune diseases is broad immunosuppression, which is associated with side effects and leaves patients vulnerable to serious infection and malignancies THE There is a significant need for antigen-specific therapies that can induce immune tolerance to pathogenic au
Slide 14: Synergistic Treg expansion with ImmTOR and IL-2 mutein
▪ 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 diseases ▪ Potential to create a “best in class” IL-2 therapy by combining it with ImmTOR Splenic regulatory T cells Day 4 Day
Slide 15: Antigen-specific Treg expansion
Antigen-specific Treg Total splenic Tregs 70 With superior expansion and 70 durability of total Tregs, Selecta 60 60 potentially has a best in class IL-2 therapy. 50 50 40 40 However, with an approximately 3- fold increase in antigen-specific 30 30 Tregs, this data shows the 20 20 opportunity to ena
Slide 16: AAV8-SEAP redosing with ImmTOR and IL-2 mutein
Day 0 2.7E12 vg/kg AAV8-SEAP +/- ImmTOR +/- IL-2 mutein Day 56 5.0E12 vg/kg AAV8-SEAP +/- ImmTOR +/- IL-2 mutein No immunotherapy 50 mg ImmTOR 100 mg ImmTOR 200 mg ImmTOR nd nd nd 2 dose 2 dose 2 dose 2.0 2 . 0 2 . 0 2 . 0 1.5 1 . 5 1 . 5 1 . 5 1.0 1 . 0 1 . 0 1 . 0 nd 2 dose 0.5 0 . 5 0 . 5 0 . 5 0
Slide 17: Primary biliary cholangitis and ImmTOR
• Autoimmune disorder where the body mistakenly attacks tissue in the liver, leading to inflammation, damage and scarring of the small bile ducts 1 • More common in women, PBC is one of the most common autoimmune diseases affecting nearly 1:1000 women over the age of 40 • Patients with PBC are despe
Slide 18: Gene Therapy
Gene Therapy
Slide 19: Gene therapy immunogenicity challenges and solutions
The formation of neutralizing antibodies (NAbs) after AAV vector administration prevents redosing due to the potentially dangerous immune response that would follow a second or third gene therapy administration. Adverse THE patient events related to high vector doses is inextricably linked to immuno
Slide 20: Selecta Biosciences Corporate Presentation – April 2022 20
Selecta Biosciences Corporate Presentation – April 2022 20
Slide 21: ImmTOR and Xork IgG protease
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 mitigating the de novo formation of anti-AAV antibodies Pre-existing anti-IgG protease Cleavage of human IgG Antibodies in human
Slide 22: ImmTOR enables repeat dosing and is dose sparing
Day 0 Day 70 5e11 vg/kg AAV-SEAP 5e11 vg/kg AAV-SEAP 5e11 vg/kg AAAV-SEAP + ImmTOR 5e11 vg/kg AAV-SEAP + ImmTOR ImmTOR has been 25e11 vg/kg AAV-SEAP 25e11 vg/kg AAV-SEAP observed to enhance transgene expression after Two doses of 5e11 vg/kg with first and second doses of ImmTOR provides comparable A
Slide 23: Empty AAV capsid healthy volunteer study 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 Drug Infusions Study Day 30 45 60 75 90 0 15 Placebo* + Empty Capsid (2E12 vp**/kg) n=3 Placebo + Empty Capsid (2E12 vp/k
Slide 24: NAb titers 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 Linear Scale Log Scale 10 2000 2000 1000 1500 100 1000 Titer 1:25 10 500 Titer 1:5 1 0 Empty Capsid (n=8) Empty Capsid + 0.15 mg/kg ImmTOR (n=9) Empty
Slide 25: Median NAb titers 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 mg/kg ImmTOR at Day 30 100000 Median 10000 Titer 1:1250 1000 50-Fold 250-Fold Difference 100 Difference 1:25 10 1:5 1 0 10 20 30 Days Empty Capsid (n=8) Empty Capsid +
Slide 26: NAb titers at Day 90
Additional doses of ImmTOR may be required to maintain control beyond Day 30 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 Day 90 Day 30 Titer 1:25 Titer 1:5 Empty Capsid (n=8) Empty Capsid + 0.15
Slide 27: Redosing eligibility and NAb titers
1 Human Nonhuman Primate Target For Redosing Single dose Single Dose Three Doses 1000000 100000 Titers too high for re-dosing 100000 10000 200+ 10000 Failure 1000 Antibody levels may interfere with 1000 50-200 efficient re-dosing** Intermediate 100 100 Patients eligible for re-dosing* 10 1-50 10 (Zo
Slide 28: Empty capsid study summary
▪ 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 ▪ After Day 30, 2 of 6 subjects treated with 0.3 mg/kg ImmTOR maintained NAb titers
Slide 29: Methylmalonic acidemia and SEL-302
branch chain amino acids • Methylmalonic acidemia (MMA) is a rare monogenic metabolic odd chain fatty acids propionate disease with a potential live birth incidence of between 1:25,000 1 and 1:48,000 Mitochondria • Majority of patients have mutations in the mitochondrial propionyl-CoA 2-methylcitrat
Slide 30: Selecta Biosciences Corporate Presentation – April 2022 30
Selecta Biosciences Corporate Presentation – April 2022 30
Slide 31: Selecta Biosciences Corporate Presentation – April 2022 31
Selecta Biosciences Corporate Presentation – April 2022 31
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Slide 33: Biologics immunogenicity challenges and solutions
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 THE Challenges Patients that develop an immune response to the current standard of care may be forced to discontinue trea
Slide 34: ImmTOR ameliorates immune response to pegadricase
ImmTOR was observed to ameliorate the immune response to pegadricase and was generally well-tolerated resulting in sustained control of serum uric Pegadricase is highly immunogenic acid (SUA) when given alone Anti-uricase ADAs SEL-212 (combination of pegadricase + ImmTOR) 5 10 5 monthly doses SEL-21
Slide 35: SEL-212 responder data in patients with tophi
Patients with tophi at baseline: • Represent the most severely affected population of gout patients • Are less likely to achieve target SUA levels on conventional oral lowering therapies and have increased gout-related emergency room visits, hospitalizations, gout-related surgeries, and co-morbiditi
Slide 36: DISSOLVE Phase 3 trial design
• 2 double blinded placebo-controlled trials of SEL-212 (0.1 mg/kg & 0.15 mg/kg ImmTOR) − Both studies have a 6-month primary endpoint of serum uric acid (SUA) < 6 mg/dL at month 6, and DISSOLVE I has a 6-month safety extension; secondary endpoints include tender and swollen joint counts, tophus bur
Slide 37: IgA nephropathy
• 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; approximately 100,000 patients in the U.S. and only one approved therapy • Caused by deposits of the protein immunoglobulin A (IgA) ins
Slide 38: IgA protease for IgA nephropathy
• Selecta intends to co-administer IgA protease ImmTOR with its proprietary IgA Saline 5mg/kg IV protease to address IgA nephropathy Glomerular • Mice expressing human IgA1 and IgA1 deposits HEMATURIA sCD89 develop spontaneous IgA nephropathy Complement • Treatment with IgA protease clears depositio
Slide 39: Corporate
Corporate
Slide 40: Management team
Kristen Baldwin Carsten Brunn, Ph.D. Kevin Tan Lloyd Johnston, Ph.D. Kei Kishimoto, Ph.D. Peter G. Traber, M.D. Matthew Bartholomae Chief People Officer President and CEO Chief Financial Officer Chief Operations Officer Chief Scientific Officer Chief Medical Officer General Counsel Selecta Bioscienc
Slide 41: Cash and funded pipeline programs
Current funding expected to support development across pipeline programs including: ~$129.4 MILLION (1) Cash on hand as of December 31, 2021 • Phase 1 clinical trial initiation and preliminary SEL-302 data in MMA • Enzyme candidate selection and IND enabling studies in IgA Nephropathy • Develop a pr
Slide 42: Precision immune tolerance platform highlights
• Precision immune tolerance platform has potential to restore self tolerance in autoimmune disease and overcome ImmTOR™ platform has immunogenicity of gene therapies and biologics potentially broad applicability • Preclinical data indicates potentially profound synergy of ImmTOR and engineered Treg
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