Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Canaccord Genuity Presentation August 2018
33 slides from Cartesian Therapeutics's conference presentation, Aug 9, 2018
Slide 1: Canaccord Genuity Presentation August 2018
Canaccord Genuity Presentation Nasdaq: SELB August 2018
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 Phase 2 clinical trial of SEL-212, the anticipated timing for reporting
Slide 3: Corporate Overview
Corporate Overview • Clinical-stage company applying proprietary Synthetic Vaccine Particle (SVP™) platform to mitigate 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; cu
Slide 4: Immunogenicity is Well Recognized as a Serious Challenge for Biologic Therapies
Immunogenicity is Well Recognized as a Serious Challenge for Biologic Therapies I M M U N O G E N I C I T Y ’ S I M P A C T COMPROMISED EFFICACY SAFETY RISK UNPREDICTABLE RESPONSE Anti-drug antibodies (ADAs) Hypersensitivity reactions can Changed PK/PD through neutralize therapeutic benefit impact p
Slide 5: Mitigating Unwanted Immunogenicity via Selecta's SVP-Rapamycin Technology Platform
Mitigating Unwanted Immunogenicity via Selecta’s SVP-Rapamycin Technology Platform Spleen • By dosing the “free biologic” it distributes broadly to desired SVP-Rapamycin Biologic drug Dendritic cell sites of action • Some of the biologic co- localizes with dendritic cells Targeting that have taken u
Slide 6: SEL-212 for Chronic Severe Gout
SEL-212 for Chronic Severe Gout
Slide 7: SEL-212: Advancing a Potential New Treatment Option for Chronic Severe Gout Patients
SEL-212: Advancing a Potential New Treatment Option for Chronic Severe Gout Patients Ownership • In-licensed pegsiticase in 2014; combined with SVP-Rapamycin to form SEL-212 Rare and Serious Disease • ~160,000 adults with chronic severe gout treated by U.S. rheumatologists • Debilitating flares and
Slide 8: Today's Unmet Needs in Chronic Severe Gout
Today’s Unmet Needs in Chronic Severe Gout • Monthly dosing (vs. biweekly for today’s FDA approved uricase therapy) • 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
Slide 9: SEL-212 Clinical Development Plan
Current Stage of SEL- SEL-212 Clinical Development Plan 212 Development DETERMINATION OF DOSE REGIMEN TO TAKE INTO PHASE 3 PHASE 3 PROGRAM WITH DEFINED DOSE REGIMEN Phase 1 a Dose Finding Phase 2 Dose Ranging Pegsiticase Five monthly injections; 6 Monthly Combination Injections of SEL-212 (N= 22) in
Slide 10: Phase 2 Trial Overview
Phase 2 Trial Overview Enrollment Criteria • Patients with symptomatic gout and serum uric acid levels >6 mg/dL Primary/Secondary • Safety, tolerability and pharmacokinetics of multiple doses of SEL-212 and pegsiticase alone • Reduction of serum uric acid levels Endpoints • Reduction of ADA levels D
Slide 11: Expanded Phase 2 Data at 3 Months Show 81% of Patients With Control of SUA <6 mg/dl
Expanded Phase 2 Data at 3 Months Show 81% of Patients With Control of SUA <6 mg/dl Pegsiticase + SVP-Rapamycin SEL-212 1 0 0 Pegsiticase 0.2 or 0.4 mg/kg 81% (22/27) SVP-Rapamycin 5 0 0.125 - 0.15 mg/kg 0 % Pts. SUA mg/dL SUA <6 Pts. % 0 1 2 3 4 5 6 7 8 9 1 0 1 1 1 2 Week 0.2 or 0.4 mg/kg Pegsitica
Slide 12: Cohort 10: 0.125 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037
Cohort 10: 0.125 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037 Patient 0.125 0.125 0.125 0 0 SEL-110 0.4 0.4 0.4 0.4 0.4 SEL-037 1 0 1 0 5 8 4 6 1 0 4 1 0 3 106-0060 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 102-0019 4 1 0 3 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 107-0012 4 3 1 0 2 0 1 0 2 1 0 1 0 5 7 of 8 evaluable patients
Slide 13: Cohort 11: 0.15 mg/kg of SEL-110 + 0.2 mg/kg of SEL-037
Cohort 11: 0.15 mg/kg of SEL-110 + 0.2 mg/kg of SEL-037 0.15 0.15 0.15 0 0 SEL-110 Patient 0.2 0.2 0.2 0.2 0.2 SEL-037 1 0 1 0 5 8 4 6 1 0 117-0003 4 3 1 0 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 106-0086 3 4 1 0 2 0 1 0 2 1 0 1 0 5 8 of 10 evaluable patients 8 4 111-0018 6 1 0 4 1 0 3 2 (80%) maintained UA c
Slide 14: Cohort 12: 0.15 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037
Cohort 12: 0.15 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037 0.15 0.15 0.15 0 0 SEL-110 Patient 0.4 0.4 0.4 0.4 0.4 SEL-037 1 0 1 0 5 8 4 111-0012 6 1 0 4 1 0 3 2 0 1 0 2 1 0 1 0 5 8 4 111-0013 6 1 0 4 1 0 3 2 0 1 0 2 1 2 1 0 5 1 0 8 1 0 4 111-0015 6 4 1 0 3 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 110-0028 Anti )
Slide 15: Data Continue to Suggest Low Flare Rates During SEL-212 Therapy
Data Continue to Suggest Low Flare Rates During SEL-212 Therapy – All Phase 2 Patients to Date % of Patients Experiencing Flares in Month 6 0 5 0 4 0 2 7 1 9 2 0 1 4 % of Patients w/Flare Patients of % 6 3 0 1 1 2 3 4 5 Pegsiticase Alone SEL-212* • Data indicate SEL-212 lowers flares initially and o
Slide 16: SEL-212 Phase 2 Overview and Summary of New Data Presented at EULAR
SEL-212 Phase 2 Overview and Summary of New Data Presented at EULAR Summary of new data presented at EULAR • 3-month data show SEL-212 product profile may provide: • Mitigation of ADAs enabling repeat dosing and sustained serum uric acid control: ~81% of patients with sUA <6 mg/dl • Low flare rate i
Slide 17: SEL-212 Safety For the Total Phase 2 Patient Population
SEL-212 Safety For the Total Phase 2 Patient Population •SEL-212 has been generally well tolerated at clinically active doses following >380 administrations •Seventeen SAEs reported in the ongoing Phase 2 trial: • Nine were reported not to be or unlikely to be related to study drug • Eight infusion
Slide 18: SEL-212 EULAR-Cohort Data Compared to KRYSTEXXA ACR Data
SEL-212 EULAR-Cohort Data Compared to KRYSTEXXA® ACR Data Category SEL-212 (12 weeks) KRYSTEXXA® (16 weeks)+ sUA control 81%++ 44% Gout flare % 33% 52% Dosing regimen 3 monthly injections 3 weekly followed by 7 bi-weekly injections +Krystexxa results from “Initial Clinical Study to determine whether
Slide 19: Next Step for SEL-212 in 2018
Next Step for SEL-212 in 2018 SEL-212 PHASE 3 PROGRAM • Phase 2 data will be presented from patients receiving 6 Monthly Combination Injections of SEL-212 against five combination placebo doses of SEL-212 at ACR October 19th- 24th • Phase 3 program expected to begin in 2018 Primary Clinical Endpoint
Slide 20: SEL-403 for Mesothelioma
SEL-403 for Mesothelioma SSP SELECTA
Slide 21: SEL-403: A Highly Potent Recombinant Pseudomonas Immunotoxin Targeting Mesothelin
SEL-403: A Highly Potent Recombinant Pseudomonas Immunotoxin Targeting Mesothelin Ownership • In-licensed LMB-100 from NCI in April 2017; up to $9.25 million in milestones; low single-digit royalties • Combination with SVP-Rapamycin now known as SEL-403 Rare and Serious Disease • Mesothelin expresse
Slide 22: Immunotoxin LMB-100
Immunotoxin LMB-100 LMB-100 Mesothelin is overexpressed on many solid tumors • LMB-100: Pseudomonas exotoxin A • Mesothelioma (>90%) linked to antibody Fab targeting mesothelin • Pancreatic cancer (>90%) • Technology was licensed to Roche Ira Pastan, M.D. • Ovarian cancer (70%) Head, Molecular but l
Slide 23: Clinical Activity of SS1P (LMB-100 Precursor) in Mesothelioma
Clinical Activity of SS1P (LMB-100 Precursor) in Mesothelioma Patient 1 4 Treatment Cycles • Widely metastatic peritoneal mesothelioma • Survived 32 months Sum of target of target Sum(cm)lesions Months after treatment Before treatment 1.6 months 8 months 6 Treatment Cycles Patient 2 • Extensive pleu
Slide 24: Preclinical Data Supports the Benefits of SVP-Rapamycin + LMB-100 Combination Therapy
Preclinical Data Supports the Benefits of SVP-Rapamycin + LMB-100 Combination Therapy SEL-403 Prevents formation of Restores LMB-100’s SVP alone does not anti-drug antibodies anti-tumor response accelerate tumor growth A n ti-L M B -1 0 0 A n tib o d y T u m o r G r o w t h T u m o r G r o w t h 2 5
Slide 25: SEL-403 In Clinical Phase 1 at NCI
SEL-403 In Clinical Phase 1 at NCI • Enrolled the first patient of a dose-escalating Phase 1 trial in March 2018 under a CRADA at NCI (NCT03436732) • Enrolling up to 18 patients with malignant pleural or peritoneal mesothelioma who have undergone at least one regimen of chemotherapy • Patients to re
Slide 26: Proprietary & Licensed Gene Therapy Programs
Proprietary & Licensed Gene Therapy Programs
Slide 27: Selecta's Proprietary Gene Therapy Programs
Selecta’s Proprietary Gene Therapy Programs Ownership • Two proprietary gene therapies utilizing Anc80 and AAV + SVP-Rapamycin (SEL-302 & SEL-313) Rare and Serious Disease • Two rare inborn error of metabolism: Methylmalonic Acidemia (MMA) and Ornithine Transcarbamylase (OTC) Deficiency • Onset in e
Slide 28: Benefits of ADA Mitigation in Gene Therapy
Benefits of ADA Mitigation in Gene Therapy Inhibiting Liver Allowing for Repeat Dosing Inflammation from First Dose And Dose Titration SVP or Empty NP SVP or Empty NP SVP or Empty NP + AAV8 + AAV8-Luc AAV8-Factor IX Day 0 Day 0 21 54 CD8 T cell Liver Infiltrates Anti-AAV8 Antibody Titer r ) 5 t e 2
Slide 29: Demonstration of the Role of Regulatory T Cells
Demonstration of the Role of Regulatory T Cells Effect can be Transferred to a Recipient T Reg Depletion Negates Effect AAV8-Luc +/- SVP AAV8-Luc +/- SVP PRIME CHALLENGE DONOR Transfer splenocytes Day 0 14 AAV8-hFIX Day 0 19 20 21 32 RECIPIENT Anti-CD25 Treg Depletion Day 0 1 28 Anti-AAV8 IgG levels
Slide 30: 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 Reducing MMA Levels With Increasing Weight Gain Preventing Anti-Anc80 Anc80 and AAV8 Following Treatment Antibodies via SVP-Rapamycin Anc80 or AAV8 Retro Orbital Anti-Anc80 Antibodies injection Weight Gain in 12 days (%) 2 .0 2 0 0 0
Slide 31: Spark Therapeutics License Agreement
Spark Therapeutics License Agreement • 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 comb
Slide 32: Pipeline
Pipeline Indication Preclinical Phase 1 Phase 2 Phase 3 Proprietary ADA Mitigation Programs Chronic Severe Gout SEL-212 Mesothelioma & Pancreatic Cancer SEL-403 Methylmalonic Acidemia (MMA) SEL-302 Ornithine Transcarbamylase Deficiency (OTC) SEL-313 ADA Mitigation Program License Hemophilia A 32
Slide 33: Financial Overview
Financial Overview For the Quarter Ended June 30, 2018 March 31, 2018 (In thousands, except share and per share data) Grant & Collaboration Revenue $ - $- Research & Development Expenses 14,407 11,139 General & Administrative Expenses 4,362 4,674 Net Loss Attributable to Common Stockholders $(18,874
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