Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — J.P. Morgan Healthcare Conference 2019
26 slides from Cartesian Therapeutics's conference presentation, Jan 7, 2019
Slide 1: Company Presentation January 2019
Company Presentation January 2019 SELB 1
Slide 2: Safe Harbor / Disclaimer
Safe Harbor / Disclaimer Any statements in this presentation about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the company”), including without limitation, statements regarding the progress of the clinical development of SEL-212, the anticipated timing of the head-to-
Slide 3: Investment highlights
Investment highlights • Biologic therapies are a growth market, but therapeutic potential may be limited by formation of neutralizing antibodies (NAbs) • Immune tolerance technology ImmTOR (SVP-Rapamycin) has the potential to unlock the full potential of biologic therapies by mitigating NAb formatio
Slide 4: ImmTOR (SVP-Rapamycin) Immune Tolerance Technology
ImmTOR (SVP-Rapamycin) Immune Tolerance Technology 4
Slide 5: Biologic therapies may trigger neutralizing antibodies that negate their therapeutic benefit
Biologic therapies may trigger neutralizing antibodies (NAbs) that negate their therapeutic benefit • Current options limited • Despite unmet need for a technology that selectively induces immune tolerance, there have been few advances in the last 40 years • Dendritic cells sit at the crossroads of
Slide 6: ImmTOR has the potential to enable sustained therapeutic activity of biologic therapies
ImmTOR has the potential to enable sustained therapeutic activity of biologic therapies • ImmTOR was designed to be co-administered with biologic therapies to mitigate antigen-specific NAb formation • ImmTOR has the potential to induce a tolerogenic message from the dendritic cell to naïve T cells t
Slide 7: SEL-212 (ImmTOR+pegadricase) for Chronic Refractory Gout
SEL-212 (ImmTOR+pegadricase) for Chronic Refractory Gout 7
Slide 8: Chronic refractory gout, a severe form of inflammatory arthritis, significant impact on patients
Chronic refractory gout, a severe form of inflammatory arthritis, significant impact on patients Chronic disease can lead to sequelae How chronic 34% 28% 23% including: refractory gout pts • Bone erosions describe flare pain Severe Breaking Glass • Tophi burn a bone piercing skin • Chronic pain • Jo
Slide 9: Today's unmet needs in chronic refractory gout
9 Today’s unmet needs in chronic refractory gout • Monthly dosing • Ability to complete full therapy cycles (6 months) - Persistent reduction in Serum Uric Acid levels (SUA) - Elimination of tophi • Gout flare reduction • Avoidance of “off-label” and global immunosuppressive therapies We believe SEL
Slide 10: SEL-212 is designed to be co-administered with uricase to enable sustained efficacy
SEL-212 is designed to be co-administered with uricase to enable sustained efficacy in chronic refractory gout • SEL-212 has the potential to induce dendritic cells to send tolerogenic message to naïve T cells • May result in development of regulatory T cells and mitigation of NAb formation, thus un
Slide 11: Sustained reduction of SUA with monthly dosing of SEL-212 observed in Phase 2
Sustained reduction of SUA with monthly dosing of SEL-212 was observed in Phase 2 Phase 2 results after 20 weeks of once-monthly SEL-212 treatment: SEL-212 SEL-212 SEL-212 SEL-212 SEL-212 100 66% (21/32)* 50 0 % Pts. SUA mg/dL <6 SUA % Pts. 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 Weeks
Slide 12: DECT scan images show reduction of tissue urate burden
Dual energy computed tomography (DECT) scan images show reduction of tissue urate burden Baseline Week 11 Week 20 0.15 mg/kg ImmTOR + 0.2 mg/kg pedadricase 0.2 mg/kg pedadricase 12 https://selectabio.com/wp-content/uploads/2018/10/ACR-poster-DECT-2018-FINAL-.pdf
Slide 13: Interim data show reduced frequency and severity of flares during SEL-212 therapy
Interim data show reduced frequency and severity of flares during SEL-212 therapy Percent of SEL-212 patients who had flares Severity of flares % of patients % of patients 64% 29% 24% 32% 12% 9% 3% 4% 1 2 3 4 5 Mild Medium Severe Months after initiation of treatment • Majority of flares have occurre
Slide 14: SEL-212 has been generally well tolerated
SEL-212 has been generally well tolerated SEL-212 was generally well tolerated at clinically active doses following >380 administrations 17 SAEs reported in the ongoing Phase 2 trial • 9 were reported not to be or unlikely to be related to study drug • 8 infusion reactions: — 4 in cohorts receiving
Slide 15: Current efficacy and safety data support further development and registration
Current efficacy and safety data support further development and registration Q1 2019: Planning to initiate head-to-head SEL-212 vs Krystexxa trial 6 Monthly Injections Efficacy goal: Projected readouts: 6-month • Interim 6-month data (Q4 2019) statistical superiority vs • Full statistical superiori
Slide 16: ImmTOR in Gene Therapy
ImmTOR in Gene Therapy 16
Slide 17: The ability to re-dose AAV gene therapy is a key goal to unlocking its therapeutic potential
The ability to re-dose AAV gene therapy is a key goal to unlocking its therapeutic potential Dose titration Dose titration • Potential to increase proportion of patients that Therapeutic target zone achieve therapeutic benefit without risk of overdosing • Goal of improving enrollment in clinical tri
Slide 18: Strategies to reduce immunogenicity are an unmet need for re-dosing of AAV gene therapy
Strategies to reduce immunogenicity are an unmet need for re-dosing of AAV gene therapy AAV gene therapy holds potential to transform the future in a variety of inherited and acquired diseases • Cystic fibrosis, classic hemophilia, cancer, and cardiovascular disease Immunogenicity represents a signi
Slide 19: In preclinical studies, ImmTOR induced antigen-specific immune tolerance
In preclinical studies, ImmTOR induced antigen-specific immune tolerance Treatment Challenge ImmTOR provided AAV-specific AAV8 + ImmTOR AAV8 or AAV5 immune tolerance AAV8 + Empty NP • NAbs did not develop in mice treated with ImmTOR+AAV vector Day 0 Day 21 • Mice treated with empty nanoparticle (NP)
Slide 20: Preclinical data indicates potential of ImmTOR-powered re-dosing in gene therapy
Preclinical data indicates potential of ImmTOR-powered re-dosing in gene therapy ImmTOR-powered AAV8 gene therapy has potential to be re-dosed without NAb formation or loss of therapy expression ImmTOR or Empty NP ImmTOR or Empty NP + AAV8-Luc AAV8-Factor IX Day 0 21 54 Anti-AAV8 Antibody Titer Seru
Slide 21: Opportunities for clinical POC in gene therapy
Opportunities for clinical POC in gene therapy Collaborations Proprietary programs License Agreement • Genethon and the • MMA (MethylMalonic Acidemia); • Spark Therapeutics CureCN consortium IND projected 2H 2019 – Licensed ImmTOR for hemophilia A, – AAV gene therapy–sponsored • OTC (Ornithine as we
Slide 22: Projected 2019 milestones
Projected 2019 milestones • Report interim 6-month data from SEL-212 vs. Krystexxa Head-to-Head trial in chronic refractory gout (H2 19) • Initiate SEL-212 Phase 3 program (H2 19) • Initiate trial with CureCN for first-in-human combination of ImmTOR + AAV gene therapy (H2 19) • Announce new partners
Slide 23: Financial Overview
Financial Overview For the Quarter Ended September 30, 2018 June 30, 2018 (In thousands, except share and per share data) Grant & Collaboration Revenue $ - $- Research & Development Expenses 11,885 14,407 General & Administrative Expenses 4,056 4,362 Net Loss Attributable to Common Stockholders $(16
Slide 24: Thank you
Thank you 24
Slide 25: Appendix
Appendix 25
Slide 26: Mitigation of IL-1β Production by ImmTOR in a Preclinical Model of MSU-Induced Inflammation
Mitigation of IL-1β Production by ImmTOR in a Preclinical Model of MSU-Induced Inflammation IL-1β Neutrophil infiltrates * ) • Monosodium urate crystals 6 * * 0 1 . 0 2 5 0 * * 1 (MSU) are known to cause * * x ( ) s 0 . 8 2 0 0 l L inflammation by activating the l e m c / NLRP3-inflammasome pathway
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