Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Jefferies Healthcare Conference 2018
32 slides from Cartesian Therapeutics's conference presentation, Jun 4, 2018
Slide 1: Jefferies Presentation June 2018
Jefferies Presentation Nasdaq: SELB June 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, the progress of the Phase 1/2 clinical program of SEL-212, the ability of SVP-Rapamycin to mitigate unwanted imm
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) Mat
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: Phase 2 Data at 12 Weeks Show 74% of Patients with Control of SUA <6 mg/dl
New Phase 2 Data at 12-weeks show 74% of Patients with Control of SUA <6 mg/dl Pegsiticase + SVP-Rapamycin SEL-212 1 0 0 Pegsiticase 74%; (14/19) 0.2 or 0.4 mg/kg SVP-Rapamycin 5 0 0.125 or 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 Patients eva
Slide 12: Patients Dosed With 0.125 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037
Patients Dosed With 0.125 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037 Pegsiticase + SVP-Rapamycin Pegsiticase Alone Patient 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 4 A 3 1 0 106-0061 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 4 F 3 1 0 111-0009 2 0 1 0 2 1 0 1 0 5 8 Anti 4 6 1 0 4 1 0 3 10
Slide 13: Patients Dosed With 0.15 mg/kg of SEL-110 + 0.2 mg/kg of SEL-037
Patients Dosed With 0.15 mg/kg of SEL-110 + 0.2 mg/kg of SEL-037 Pegsiticase + SVP-Rapamycin Pegsiticase Alone Patient 1 0 1 0 5 8 4 6 1 0 4 E 3 106-0084 1 0 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 4 1 0 3 117-0003 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 4 1 0 3 106-0086 2 0 1 0 2 Anti 1 0 1 0 5 8 4 6 1 0 4 A 3 1 0 103
Slide 14: Patients Dosed With 0.15 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037
Patients Dosed With 0.15 mg/kg of SEL-110 + 0.4 mg/kg of SEL-037 Pegsiticase + SVP-Rapamycin Pegsiticase Alone Patient 1 0 1 0 5 8 4 6 1 0 4 A 3 107-0017 1 0 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 4 A 3 1 0 106-0073 2 0 1 0 2 1 0 1 0 5 8 4 6 1 0 4 1 0 3 111-0012 2 0 1 0 2 Anti 1 0 1 0 5 8 4 6 1 0 4 1 0 3 111
Slide 15: SEL-212 Continues to Show Low Overall Incidence of Gout Flares
SEL-212 Continues to Show Low Overall Incidence of Gout Flares In Total Phase 2 Patient Population Percent patients with gout flare by treatment month 6 0 50% 4 0 26% 19% 2 0 13% % of Patients w/Flare Patientsof % 4% 4% 0 month 1 1 2 3 4 5 Pegsiticase Alone SEL-212 15 Unaudited data reported as of A
Slide 16: SEL-212 Safety For Total Phase 2 Patient Population
SEL-212 Safety For Total Phase 2 Patient Population •SEL-212 has been generally well tolerated at clinically active doses following >300 administrations •Fifteen SAEs reported in the ongoing Phase 2 trial: • Seven were reported not to be or unlikely to be related to study drug • Eight infusion react
Slide 17: SEL-212 PANLAR Data Compared to KRYSTEXXA Data
SEL-212 PANLAR Data Compared to KRYSTEXXA® Data Category SEL-212 (12 weeks) KRYSTEXXA® (16 weeks)+ sUA control 74%++ 44% Gout flare % 42% 52% Dosing regimen 3 monthly injections 3 weekly followed by 7 bi-weekly injections +Krystexxa results from “Initial Clinical Study to determine whether a toleriz
Slide 18: Next Step for SEL-212 in 2018
Next Step for SEL-212 in 2018 • Expansion of SEL-212 PHASE 3 PROGRAM PANLAR data set to be presented at EULAR (June 15th) • Data expected in 6 Monthly Combination Injections of SEL-212 third quarter from patients receiving five combination doses of SEL-212 • Patients now receiving 4th of 5 expected
Slide 19: SEL-403 for Mesothelioma
SEL-403 for Mesothelioma SSP SELECTA
Slide 20: 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 21: 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 22: 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 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 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 24: SEL-403 In Clinical Phase 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 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 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 27: 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 28: 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 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 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 30: 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 31: 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 31
Slide 32: Financial Overview
Financial Overview For the Quarter Ended March 31, 2018 December 31, 2017 (In thousands, except share and per share data) Grant & Collaboration Revenue $ - $17 Research & Development Expenses 11,139 13,623 General & Administrative Expenses 4,674 5,671 Net Loss Attributable to Common Stockholders $(1
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