Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Investor Presentation (January 2018)
33 slides from Cartesian Therapeutics's investor presentation, Jan 8, 2018
Slide 1: Investor Presentation January 2018
Investor Presentation Nasdaq: SELB January 2018
Slide 2: Safe Harbor / Disclaimer
Safe Harbor / Disclaimer 2 Any statements in this presentation about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the company”), including without limitation, the progress of the Phase 1/2 clinical program of SEL-212, whether the company participates in an End-of-Phase
Slide 3: Corporate Overview
Corporate Overview 3 • Clinical-stage company applying proprietary Synthetic Vaccine Particle (SVP™) platform to avoid unwanted immunogenicity and unlock the full potential of biologic therapies • Expect to begin Phase 3 in 2018 with SEL-212 (SVP-Rapamycin + pegsiticase) for chronic severe gout • IN
Slide 4: Immunogenicity is Now Recognized as a Serious Challenge for Biologic Therapies
Immunogenicity is Now Recognized as a Serious Challenge for Biologic Therapies 4 COMPROMISED EFFICACY Anti-drug antibodies (ADAs) neutralize therapeutic benefit UNPREDICTABLE RESPONSE Changed PK/PD through drug-ADA interaction SAFETY RISK Hypersensitivity reactions can impact patients I M M U N O G
Slide 5: Preventing Unwanted Immunogenicity via Selecta's SVP-Rapamycin Technology Platform
Preventing Unwanted Immunogenicity via Selecta’s SVP-Rapamycin Technology Platform 5 Spleen Preventing ADAs Inducing a tolerogenic response to a biologic drug (antigen) Implementing the message Sending precise instructions Targeting immune cells • By dosing “free biologic” separate from SVP- Rapamyc
Slide 6: Leveraging Nanoparticles to Target and Deliver Instructions to the Immune System
Leveraging Nanoparticles to Target and Deliver Instructions to the Immune System 6 I.V. Injection (6hr post-injection) Spleen harvested 24 hr after I.V. Injection of fluorescent NPs SVP Macrophages Dendritic cells B cells SVP Antigen Spleen
Slide 7: Selecta's Pipeline Development Strategy
Selecta’s Pipeline Development Strategy 7 Chronic Therapies+ Proprietary program Potential program Single Treatment Chronic Treatment Adult Patients with Chronic Conditions Cancer Patients Pediatric Patients with Life-Threatening Conditions Entering Phase 1: SEL-403 for Mesothelioma Treatment Cycle
Slide 8: SEL-212 for Chronic Severe Gout
SEL-212 for Chronic Severe Gout
Slide 9: SEL-212: Advancing a Potential New Treatment Option for Chronic Severe Gout Patients Toward Phase 3
SEL-212: Advancing a Potential New Treatment Option for Chronic Severe Gout Patients Toward Phase 3 9 Rare and Serious Disease • ~160,000 adults with chronic severe gout treated by U.S. rheumatologists • Debilitating flares and joint-damaging arthritis caused by uric acid deposits; risk of renal and
Slide 10: Today's Unmet Needs in Chronic Severe Gout
Today’s Unmet Needs in Chronic Severe Gout • Monthly dosing (vs. biweekly for today’s approved uricase therapy) • Ability to complete full therapy cycles - Persistent reduction in SUA - Elimination of tophi • Gout flare reduction • Avoidance of “off-label” and global immunosuppressive therapies 10 W
Slide 11: Phase 1b Single Dose Patient Cohorts
Phase 1b Single Dose Patient Cohorts 11 IMVACS 2016 Presentation. Clinicaltrials.gov NCT02648269 S e ru m U ric A cid ( m g /d L ) 0.03 mg/kg 0.4 mg/kg 0.1 mg/kg 0.4 mg/kg 0.4 mg/kg 0.3 mg/kg 0.4 mg/kg 0.15 mg/kg 0.4 mg/kg 0 2 4 6 8 1 0 0 2 4 6 8 1 0 0 2 4 6 8 1 0 0 2 4 6 8 1 0 0 2 4 6 8 1 0 N = 5 N
Slide 12: Phase 2 Trial Overview
Phase 2 Trial Overview 12 Enrollment Criteria Primary/Secondary Endpoints Design Dosing Stopping Rules As of October 23 • Patients with symptomatic gout and SUA levels >6 mg/dL • Safety, tolerability and pharmacokinetics of multiple doses of SEL-212 and pegsiticase alone • Reduction of SUA levels •
Slide 13: 5 1 1 2 1 1 9 1 2 6 1 3 3 1
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 1 1 9 1 2 6 1 3 3 1 4 0 0 2 4 6 8 1 0 1 0 2 1 0 3 1 0 4 1 0 5 0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 1 1 9 1 2 6 1 3 3 1 4 0 0 2 4 6 8 1 0 1 0 2 1 0 3 1 0 4 1 0 5 0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0
Slide 14: 5 1 1 2 1 1 9 1 2 6 1 3 3 1
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 1 1 9 1 2 6 1 3 3 1 4 0 0 2 4 6 8 1 0 1 0 2 1 0 3 1 0 4 1 0 5 0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 1 1 9 1 2 6 1 3 3 1 4 0 0 2 4 6 8 1 0 1 0 2 1 0 3 1 0 4 1 0 5 0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0
Slide 15: Comparison of Clinical Activity and Immune Tolerance Between Mid-Dose and Control Cohorts
0.2 or 0.4 mg/kg Pegsiticase Alone Control Cohorts (Cohorts 1 & 2) % of Patients With SUA <6 mg/dL Comparison of Clinical Activity and Immune Tolerance Between Mid-Dose and Control Cohorts 15 Unaudited data reported as of October 23, 2017 | Clinicaltrials.gov NCT02959918 * Ph1b Day 30 @ 0.1 mg/kg SV
Slide 16: Low Frequency of Gout Flares Observed with SEL-212 Treatment
1 1 2 3 4 5 0 2 0 4 0 6 0 16 • Data indicate SEL-212 lowers flares initially and over time during treatment • Urate lowering therapies typically increase the incidence of flares at the beginning of therapy Unaudited data as of October 23, 2017 | Clinicaltrials.gov NCT02959918 % o f P at ie n ts w /F
Slide 17: SEL-212 Generally Well Tolerated at Clinically Active Doses
SEL-212 Generally Well Tolerated at Clinically Active Doses 17 • SEL-212 has been generally well tolerated at clinically active doses following >200 administrations • SAEs reported in the Phase 2 trial: - Four were reported not to be or unlikely to be related to study drug - Seven infusion reactions
Slide 18: SEL-212 Expected to Enter Phase 3 in 2018
SEL-212 Expected to Enter Phase 3 in 2018 18 • SEL-212 (SVP-Rapamycin + pegsiticase) designed to be the first non- immunogenic uricase treatment for chronic severe gout • Phase 2 trial ongoing: - SEL-212 has been demonstrated to be clinically active and generally well tolerated - Cohorts receiving 0
Slide 19: SEL-403 for Mesothelioma
SEL-403 for Mesothelioma Se) SELECTA
Slide 20: SEL-403: A Highly Potent Recombinant Pseudomonas Immunotoxin Targeting Mesothelin
SEL-403: A Highly Potent Recombinant Pseudomonas Immunotoxin Targeting Mesothelin 20 Rare and Serious Disease • Mesothelin expressed in virtually all mesotheliomas (~3,000 annual U.S. diagnoses1) and pancreatic cancers (~50,000); high percentage of ovarian, lung, breast cancers • Certain solid tumor
Slide 21: Immunotoxin LMB-100
Immunotoxin LMB-100 21 • LMB-100: Pseudomonas exotoxin A linked to antibody Fab targeting mesothelin • Technology was licensed to Roche but later returned to NCI • Efficacy was limited by immunogenicity after one or two cycles in most patients • Currently in Phase 1 clinical trials LMB-100 Mesotheli
Slide 22: Clinical Activity of SS1P (LMB-100 Precursor) in Mesothelioma
Clinical Activity of SS1P (LMB-100 Precursor) in Mesothelioma 22 Day 12 3 months 15 months Before treatment 1.6 months 8 months Patient 1 • Widely metastatic peritoneal mesothelioma • Survived 32 months Patient 2 • Extensive pleural mesothelioma • Survival >6 years (still alive) S u m o f ta rg e t
Slide 23: Preclinical Data Supports the Benefits of SVP-Rapamycin + LMB-100 Combination Therapy
Preclinical Data Supports the Benefits of SVP-Rapamycin + LMB-100 Combination Therapy 23 Prevents formation of anti-drug antibodies Restores LMB-100’s anti-tumor response SVP alone does not accelerate tumor growth T u m o r G r o w t h D a y s s i n c e t u m o r i n o c u l a t i o n T u m o r s iz
Slide 24: SEL-403 Entering the Clinic at NCI
SEL-403 Entering the Clinic at NCI 24 • IND acceptance announced on January 2, 2018 • Expect to commence a dose-escalating Phase 1 trial in Q1 2018 under a CRADA at NCI • Enrolling up to 18 patients with malignant pleural or peritoneal mesothelioma who have undergone at least one regimen of chemothe
Slide 25: Proprietary & Licensed Gene Therapy Programs
Proprietary & Licensed Gene Therapy Programs
Slide 26: Selecta's Proprietary Gene Therapy Programs
Selecta’s Proprietary Gene Therapy Programs 26 Rare and Serious Disease • Two rare inborn error of metabolism: Methylmalonic Acidemia (MMA) and Ornithine Transcarbamylase (OTC) Deficiency • Onset in early infancy; significantly reduces life expectancy Immunogenicity Barrier • Infants require treatme
Slide 27: Benefits of ADA Avoidance in Gene Therapy
Benefits of ADA Avoidance in Gene Therapy 27 Inhibiting Liver Inflammation from First Dose Allowing for Repeat Dosing And Dose Titration CD8 T cell Liver Infiltrates Serum ALT Enzyme Levels + AAV8 SVP or Empty NP 0Day SVP or Empty NP + AAV8-Luc AAV8-Factor IX Day 0 21 54 SVP or Empty NP Anti-AAV8 An
Slide 28: Demonstration of the Role of Regulatory T Cells
Demonstration of the Role of Regulatory T Cells 28 Day 0 19 20 21 32 0 1 28 Effect can be Transferred to a Recipient T Reg Depletion Negates Effect Anti-AAV8 IgG levels in recipient mice (Day +14) DONOR AAV8-Luc +/- SVP 0 14Day Day RECIPIENT AAV8-hFIX AAV8-Luc +/- SVP PRIME Anti-CD25 Treg Depletion
Slide 29: Anc80/synMUT Proof of Concept in Mouse Model of MMA at ASGCT 2017
Anc80/synMUT Proof of Concept in Mouse Model of MMA at ASGCT 2017 29 0 5 0 1 0 0 1 5 0 2 0 0 0 5 0 0 1 0 0 0 1 5 0 0 2 0 0 0 D a y s M M A ( M ) A n c 8 0 -h A A T -M u t4 5 e 1 2 V G A A V 8 -h A A T -M u t4 5 e 1 2 V G 0 5 10 15 20 25 30 35 Weight Gain in 12 days (%) Anc80 or AAV8 Retro Orbital
Slide 30: Spark Therapeutics License Agreement
Spark Therapeutics License Agreement 30 • December 2016 agreement provides Spark Therapeutics with exclusive worldwide rights to Selecta's SVP technology for up to five gene therapy targets • Among the largest gene therapy and SMID-cap to SMID-cap biotech deals announced to date • Initial focus on c
Slide 31: Pipeline
Pipeline 31 * LMB-100 is currently being investigated in two Phase 1 clinical trials at the National Cancer Institute (NCI): one of LMB-100 alone in Mesothelioma and one of LMB-100 in combination with nab-paclitaxel in Pancreatic Cancer. Phase 1 trial of SEL-403 in Mesothelioma is expected to begin
Slide 32: Financial Overview
Financial Overview 32 For the Quarter Ended (In thousands, except share and per share data) September 30, 2017 September 30, 2016 Grant & Collaboration Revenue $27 $1,048 Research & Development Expenses 9,504 6,021 General & Administrative Expenses 4,377 2,495 Net Loss Attributable to Common Stockho
Slide 33: Thank You
Se SELECTA Biosciences Thank You
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