Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Technical Presentation (June 2017)
26 slides from Cartesian Therapeutics's technical presentation, Jun 15, 2017
Slide 1: June 2017 Phase 2 Trial Presentation
June 15, 2017 June 2017 Phase 2 Trial Presentation
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, the potential of SEL-212 to treat severe gout patie
Slide 3: SEL-212 for Chronic Severe Gout
SEL-212 for Chronic Severe Gout
Slide 4: Clinical Objectives of SEL-212 Program
Clinical Objectives of SEL-212 Program Phase 1b Phase 1a Phase 2 Demonstrate that SEL 212: Mitigates ADAs Enables prolonged control of uric acid for >30 days Define effective monthly dose of pegsiticase Demonstrate rapid formation and kinetics of ADAs Demonstrate SEL-212’s safety, tolerability a
Slide 5: Phase 1b Demonstrates SEL-212's Clinical Activity for ≥30 Days
5 0 2 4 6 8 1 0 0 2 4 6 8 1 0 S e ru m U ric A cid ( m g /d L ) 0 2 4 6 8 1 0 0 2 4 6 8 1 0 0.03 mg/kg SVP-Rapamycin 0.4 mg/kg Pegsiticase 0.10 mg/kg SVP-Rapamycin 0.4 mg/kg Pegsiticase 0.4 mg/ kg Pegsiticase only 0.03, 0.1, 0.3 mg/kg SVP-Rapamycin only 0.30 mg/kg SVP-Rapamycin 0.4 mg/kg Pegsiticase
Slide 6: Phase 1b Trial Shows Correlation Between ADA Titers, Pegsiticase Activity and sUA
Phase 1b Trial Shows Correlation Between ADA Titers, Pegsiticase Activity and sUA 6 0 5 1 0 1 5 2 0 2 5 3 0 0 3 6 9 1 2 0 5 1 0 1 5 2 0 2 5 3 0 0 3 6 9 1 2 0 5 1 0 1 5 2 0 2 5 3 0 0 3 6 9 1 2 0 5 1 0 1 5 2 0 2 5 3 0 0 3 6 9 1 2 S e ru m u ri c a cid ( m g /d L ) 0 5 1 0 1 5 2 0 2 5 3 0 1 0 - 1 1 0 0
Slide 7: Phase 2 Trial Overview
Trial Completion Phase 2 Trial Overview 7 • Patients with symptomatic gout and serum uric acid levels >6 mg/dL • Safety, tolerability and pharmacokinetics of multiple doses of SEL-212 and pegsiticase alone • Reduction of serum uric acid levels • Reduction of ADA levels • Multiple ascending dose coho
Slide 8: Status of Phase 2 Trial Cohorts
8 Status of Phase 2 Trial Cohorts Cohort Treatment Weeks 0, 4, 8 Treatment Weeks 12 + 16 Status Pegsiticase SVP-Rapamycin Pegsiticase 1 0.2 mg/kg None 0.2 mg/kg Enrollment terminated 2 0.4 mg/kg None 0.4 mg/kg Enrollment terminated 3 0.2 mg/kg 0.05 mg/kg 0.2 mg/kg Dosing completed 4 0.4 mg/kg 0.05 m
Slide 9: Minimal Effective Dose of SEL-212 Now Defined
9 Minimal Effective Dose of SEL-212 Now Defined Unaudited data as of June 12, 2017 Clinicaltrials.gov NCT02959918 0 1 2 3 4 5 6 7 8 9 1 0 1 1 1 2 1 3 1 4 1 5 1 6 1 7 1 8 1 9 2 0 0 3 6 9 1 2 SU A (m g/d L) 0 1 2 3 4 5 6 7 8 9 1 0 1 1 1 2 1 3 1 4 1 5 1 6 1 7 1 8 1 9 2 0 0 3 6 9 1 2 SU A (m g/d L) 0 1
Slide 10: Minimal Effective Dose of SEL-212
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 11: Higher Dose Cohort: 0.4 mg/kg Pegsiticase + 0.1 mg/kg SVP-Rapamycin
11 Higher Dose Cohort: 0.4 mg/kg of Pegsiticase + 0.1 mg/kg of SVP-Rapamycin Unaudited data as of June 12, 2017 Clinicaltrials.gov NCT02959918 • sUA remains controlled in a majority of patients following repeat doses • Two patients met stopping rules • One of these patients was inadvertently re- dos
Slide 12: Results to Date Suggest Reduction in Flare Frequency During SEL-212 Therapy
12 • Urate lowering therapies typically increase the incidence of flares at the beginning of therapy • SEL-212 lowers flares compared to pegsiticase alone 0 . 0 5 0 . 0 8 0 . 1 0 2 0 4 0 6 0 % P a t i e n t s w i t h f l a r e i n m o n t h s 1 - 3 % P a t ie n t s w it h f la r e s S V P - R a p a
Slide 13: Phase 2 Safety Overview
Phase 2 Safety Overview 13 • SEL-212 has been generally well tolerated at clinically active doses following repeated administrations • SAEs reported to date in the trial: - Seven infusion reactions, four of which were in cohorts receiving pegsiticase alone or pegsiticase in combination with the lowe
Slide 14: Phase 2 Adverse Events
14 Phase 2 Adverse Events Unaudited data as of June 12, 2017 Clinicaltrials.gov NCT02959918 Cohort Entire Study 1 2 3 4 5 6 7 8 N(%) 60 3 3 9 10 6 7 10 10 ≥ 1TEAE 49(81.7) 2 2 9 8 5 5 3 6 ≥ SAE 8 1 1 2 0 0 1* 1#, 1 1* Death 0 0 0 0 0 0 0 0 0 Discontinuation due to TEAE 8 1 1 2 0 0 1 2 1 Specific TEA
Slide 15: The Unmet Need
The Unmet Need
Slide 16: Substantial Unmet Need for Chronic Severe Gout Patients
Substantial Unmet Need for Chronic Severe Gout Patients 16 Tophi Significantly Increase Mortality Risk3 No/not diagnosed tophi 8.3 3.1 5.2 4.7 0.5 Patients Rx treated Primary care, endo, nephro, other Rheumatologists U.S. Gout Patients (million)1 530,000 370,000 Estimated SEL-212 Target Patient Popu
Slide 17: What is Chronic Severe Gout?
What is Chronic Severe Gout? (1) IMS, Desk Research (2) Selecta rheumatologist interviews, Crystal patient registry (3) Choi HK et al, Tophaceous Gout and the Risk of Mortality: A General Population-Based Study, ACR, Sept. 2016 (4) Zhu Y, et al, Comorbidities of gout and hyperuricemia in the US gene
Slide 18: The Clinical Endpoint for Gout Medication Approvals is Well-Defined
6.8 6.0 Seru m U ri c A c id ( m g /d L ) Time Uric acid solubility limit FDA-approved endpoint Urate crystal deposits begin to form The Clinical Endpoint for Gout Medication Approvals is Well-Defined 18
Slide 19: While Oral Therapies May Control sUA, They do not Effectively Resolve Tophi
While Oral Therapies May Control sUA, They do not Effectively Resolve Tophi 19 6.8 6.0 Seru m U ri c A c id ( m g /d L ) Time Oral therapies For illustrative purposes only
Slide 20: Resolving Tophi and Uric Acid Deposits with Monthly Uricase Treatments
Resolving Tophi and Uric Acid Deposits with Monthly Uricase Treatments 20 6.8 6.0 Seru m U ri c A c id ( m g /d L ) Time Transition from uricase to oral therapies Before1 After1 Uric acid deposits Calcium deposits Very low sUA levels enable rapid tophi resolution 12-24 week treatment cycle (1) Arujo
Slide 21: Phase 2 Trial Accomplishments to Date
Phase 2 Trial Accomplishments to Date 21 Controlled sUA and avoided ADAs after multiple doses Induced immune tolerance Demonstrated low incidence of flares SEL-212 generally well tolerated at active dose levels Identified minimum effective dose Data allows initial preparation of Phase 3
Slide 22: Platform Implications
Platform Implications
Slide 23: Immunogenicity's Impact on Biologic Drugs and Product Candidates is Far-Reaching
Immunogenicity’s Impact on Biologic Drugs and Product Candidates is Far-Reaching COMPROMISED EFFICACY Anti-drug antibodies (ADAs) neutralize therapeutic benefit 23 UNPREDICTABLE RESPONSE Changed PK/PD through drug-ADA interaction SAFETY RISK Hypersensitivity reactions can impact patients “Prophylact
Slide 24: SEL-212 Trial Data Inform Platform Expansion
Proprietary program(s) Potential program(s) 24 SEL-212 for Chronic Severe Gout Commercially attractive Rare disease Rapid ADA onset Clear endpoint Adult patients Single Treatment Chronic Treatment Adult Patients Cancer Patients Pediatric Patients Oncology (LMB-100) Enzyme Replacement Thera
Slide 25: Immune Tolerance Pipeline
Indication Description Preclinical Phase 1 Phase 2 Proprietary ADA Mitigation Programs Refractory Gout SVP-Rapamycin co-administered with pegsiticase (SEL-212) Mesothelioma & Pancreatic Cancer* SVP-Rapamycin co-administered with LMB-100 Methylmalonic Acidemia (MMA) SVP-Rapamycin co-administered with
Slide 26: See: SELECTA
See: SELECTA
Selecta Biosciences — Technical Presentation (June 2017) on 6ix