Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Investor Presentation (June 2020)
29 slides from Cartesian Therapeutics's investor presentation, Jun 18, 2020
Slide 1: Corporate Presentation June 2020
Corporate Presentation J u n e 2 0 2 0 SELB
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-he
Slide 3: Selecta well-positioned for success
Selecta well-positioned for success Pioneering immune tolerance platform, ImmTOR™ Lead program – SEL-212, addressing a $1B+ chronic refractory gout market with high unmet need — Selecta’s immune tolerance platform, ImmTOR, could unlock the full potential of biologic therapies by mitigating Neutraliz
Slide 4: ImmTOR Pioneering Immune Tolerance Platform
ImmTOR Pioneering Immune Tolerance Platform 4
Slide 5: Biologic therapies may trigger NAbs that negate their therapeutic benefit
Biologic therapies may trigger NAbs that negate their therapeutic benefit There is a significant unmet Dendritic cells play a key need for a technology that role in immune tolerance, selectively induces immune providing a promising target tolerance to mitigate unwanted antigen-specific immune respon
Slide 6: ImmTOR may enable sustained therapeutic activity of biologic therapies
ImmTOR has the potential ability to enable sustained therapeutic activity of biologic therapies and unlock their potential Co-administer with biologic therapies to mitigate antigen- specific NAb formation Induce a tolerogenic message from the dendritic cell to naïve T cells to develop into T regulat
Slide 7: SEL-212 (ImmTOR+pegadricase) for Chronic Refractory Gout
SEL-212 (ImmTOR+pegadricase) for Chronic Refractory Gout 7
Slide 8: Chronic refractory gout is a severe form of inflammatory arthritis
Chronic refractory gout is a severe form of inflammatory arthritis with a significant impact on patients Chronic disease can lead How chronic 34% 28% 23% refractory gout to sequelae including: patients describe Severe Breaking Glass Bone erosions their flare pain burn a bone piercing skin Tophi Chro
Slide 9: Significant need for effective new therapies in chronic refractory gout
Significant need for effective new therapies in chronic refractory gout Improved efficacy, allowing patients to complete full 6-month therapy cycle — Persistent reduction in Serum Uric Acid (SUA) levels — Elimination of tophi Monthly dosing Gout flare reduction Avoidance of “off-label” and global im
Slide 10: Sustained reduction of SUA with monthly dosing of SEL-212 in Phase 2
Sustained reduction of SUA with monthly dosing of SEL-212 was observed in Phase 2 dose ranging study 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 <6 mg/dL % Pts. SUA 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16
Slide 11: DECT scan images show reduction of tissue urate burden in Phase 2
Dual energy computed tomography (DECT) scan images show reduction of tissue urate burden in Phase 2 dose ranging study Baseline Week 11 Week 20 https://selectabio.com/wp-content/uploads/2018/10/ACR-poster-DECT-2018-FINAL-.pdf 11
Slide 12: Reduced frequency and severity of flares during SEL-212 therapy
Phase 2 dose ranging data showed 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 flar
Slide 13: SEL-212 generally well-tolerated in the Phase 2 dose ranging study
SEL-212 generally well-tolerated in the Phase 2 dose ranging study SEL-212 was generally well tolerated at clinically Serious infusion reactions (%) active doses following >470 administrations during the Phase 2 trial 4 0 ) 3 3 .3 % ( n 3 0 23 SAEs reported in the Phase 2 trial o i t c 14 were repor
Slide 14: COMPARE Trial: SEL-212 vs KRYSTEXXA in Gout
COMPARE Trial: A Study to Compare the Efficacy of SEL-212 to KRYSTEXXA® in Gout Patients Refractory to Conventional Therapy SEL-212 (N=83) 6 Infusions Once Monthly • Primary Endpoint: Statistical Superiority for Serum uric acid level below 170 Refractory Chronic 6mg/dL at 3 and 6 months Randomized G
Slide 15: SEL-212 Phase 3 Study Design
SEL-212 Phase 3 Study Design 2 double blinded placebo-controlled trials of SEL-212 (0.1 mg/kg & 0.15 mg/kg SEL-110) Randomized 1:1:1 against Placebo with a total of 210 Treated Subjects Enrollment is expected to commence in 2H 2020 SEL-212 -0.1/0.2 mg/kg Study 1 N = 35 subjects / SEL-212 -0.15/0.2 m
Slide 16: Selecta-Sobi Strategic Licensing Agreement
Selecta-Sobi Strategic Licensing Agreement In June 2020, Selecta and Sobi announced a strategic licensing agreement for SEL-212 — Sobi takes on SEL-212 development, regulatory, and commercial activities in all markets outside of China — Selecta runs the phase 3 on behalf of Sobi, at Sobi’s expense S
Slide 17: ImmTOR in Gene Therapy
ImmTOR in Gene Therapy 17
Slide 18: The ability to re-dose AAV gene therapy is a key goal
The ability to re-dose AAV gene therapy is a key goal to unlocking the full therapeutic potential Dose titration Dose titration Potential to increase proportion of patients who achieve Therapeutic target zone therapeutic benefit without risk of overdosing Enrollment in clinical trials could be achie
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) + A
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: First dose benefit of ImmTOR on liver-directed transgene expression
First dose benefit of ImmTOR on liver-directed transgene expression Day 0 Day 56 AAV8-SEAP AAV8-SEAP AAV8-SEAP + ImmTOR AAV8-SEAP + ImmTOR ) U 15000 L R ( P 10000 A E S 5000 m u r e S 0 11 46 63 77 Days First dose benefit is immediate and independent of effect on adaptive immune response Cumulative
Slide 22: Opportunities for clinical POC in gene therapy
Opportunities for clinical POC in gene therapy Collaboration Proprietary Program License Agreements AskBio OTC (Ornithine Transcarbamylase AskBio – Development pipeline and deficiency) – Licensed ImmTOR for Pompe human trials planned for disease in December 2019 repeat dosing of AAV-based gene thera
Slide 23: Strategic Collaborations
Strategic Collaborations 23
Slide 24: Enhancing Value Through Collaborations
Enhancing Value Through Collaborations Selecta has entered into strategic transactions with leading biopharmaceutical companies to further unlock the value of the ImmTOR platform Year 2016 2019 2019 2020 2020 Enable re-dosing of life- Enable re-dosing of life- Enable re-dosing of life- Improving the
Slide 25: Summary
Summary 25
Slide 26: Projected upcoming milestones
Projected upcoming milestones SEL-212 – Report top-line data in head-to-head (COMPARE) trial of SEL-212 against pegloticase in chronic refractory gout (3Q 2020) – Commence Phase 3 clinical program against placebo (2H 2020) Gene Therapy Program – Commence human POC trial under AskBio collaboration in
Slide 27: Strong cash position
Strong cash position Company well-capitalized with funding, pro forma for the strategic license agreement with Sobi, into the first half of 2023 Potential for significant additional non-dilutive funding through milestone and royalty payments from 2023 onward 27
Slide 28: Selecta well-positioned for success
Selecta well-positioned for success Pioneering immune tolerance platform, ImmTOR™ Lead program – SEL-212, addressing a $1B+ chronic refractory gout market with high unmet need — Selecta’s immune tolerance platform, ImmTOR, could unlock the full potential of biologic therapies by mitigating Neutraliz
Slide 29: Relentlessly committed to overcoming immunogenicity
Relentlessly committed to overcoming OUR PURPOSE IMMUNOGENICITY with our pioneering ImmTOR immune tolerance platform to transform the lives of patients and their families 29
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