Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Investor Presentation (January 2020)
24 slides from Cartesian Therapeutics's investor presentation, Jan 13, 2020
Slide 1: Corporate Presentation January 2020
Corporate Presentation January 2020 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 Tolerogenic platform – Selecta’s immune tolerance platform, ImmTOR, could unlock the full potential of biologic therapies by mitigating Neutralizing Antibody (Nab) formation — Pipeline focused on therapeutic biologics/enzymes and AAV gene therapies Lead program –
Slide 4: ImmTOR Immune Tolerance Platform
ImmTOR 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 could 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
Dual energy computed tomography (DECT) scan images show reduction of tissue urate burden 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 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 flares occurred i
Slide 13: SEL-212 has been generally well tolerated
SEL-212 has been generally well tolerated 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 reported not to be or unlikely
Slide 14: Comparing the efficacy of SEL-212 to KRYSTEXXA in refractory gout patients
Comparing the efficacy of SEL-212 to KRYSTEXXA® in gout patients refractory to conventional therapy SEL-212 (N=~75) 6 Infusions Once Monthly 0.15 mg/kg ImmTOR + 0.2 mg/kg of pegadricase Primary Endpoint: Statistical superiority for SUA level < 6mg/dL at 6 months ~150 Refractory Randomized Multiple S
Slide 15: ImmTOR in Gene Therapy
ImmTOR in Gene Therapy 15
Slide 16: 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 Goal of improving enrollment in clinical tri
Slide 17: 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 18: 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 19: 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 20: 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 Selecta e
Slide 21: Financial Overview and Projected Milestones
Financial Overview and Projected Milestones 21
Slide 22: Financial snapshot
Financial snapshot State For the Quarter Ended September 30, 2019 (In thousands) Research & Development Expenses $8,104 General & Administrative Expenses $3,690 Total Operating Expenses $11,794 Net Loss $(11,994) As of September 30, 2019 (In thousands, except shares outstanding) Cash, Cash Equivalen
Slide 23: Projected upcoming milestones
Projected upcoming milestones Guidance from FDA meeting regarding Phase 3 clinical development plan (Q1 2020) Report top-line data from SEL-212 vs. KRYSTEXXA® COMPARE trial in chronic refractory gout (mid 2020) Planning to commence clinical trial of ImmTOR in gene therapy (2020) 23
Slide 24: 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 24
Selecta Biosciences — Investor Presentation (January 2020) on 6ix