Exicure, Inc. · NASDAQ:XCUR
Exicure — Corporate Update (June 2019)
13 slides from Exicure's corporate update, Jun 27, 2019
Slide 1: Progress in CNS Disorders
Exhibit 99.1 Progress in CNS Disorders
Slide 2: Special Note Regarding Forward Looking Statements
SPECIAL NOTE REGARDING FORWARD LOOKING STATEMENTS This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding our future results of operations, business strategy, current and prospectiv
Slide 3: Management On Today's Call
Management On Today’s Call David Giljohann, PhD David Snyder, MBA Matthias Schroff, PhD Chief Executive Officer Chief Financial Officer Chief Operating Officer 3
Slide 4: Introduction to Exicure
Introduction to Exicure • Clinical-stage biotechnology company developing nucleic acid therapeutics based on our proprietary Spherical Nucleic Acid, or SNA, technology • SNA technology is intended to provided differentiated therapeutic benefits: better uptake, greater stability ➢ Demonstrated local
Slide 5: Development Pipeline
Development Pipeline Therapeutic Development Stage Therapeutic Area Candidate/ Indication Status Preclinical Target Phase 1 Phase 2 Development • Phase 1b/2 opened late 2018(1) Immuno- AST-008 Solid Tumors • 5 clinical sites open oncology (TLR9 agonist) • Preliminary results in 2019 XCUR17 • Phase 1
Slide 6: Oligonucleotide Therapeutics Provide New Treatment Options in Neurological Diseases
Oligonucleotide Therapeutics Provide New Treatment Options in Neurological Diseases • Large number of neurological diseases can potentially be addressed by oligonucleotide therapeutics • Approval of nusinersen (Spinraza™) shows potential of oligonucleotide therapeutics in neurological diseases • SNA
Slide 7: SNAs Persist Longer in the Rodent CNS
SNAs Persist Longer in the Rodent CNS • IT-administered Nusinersen-SNA rapidly penetrated the rat brain, showing enhanced CNS persistence and reduced renal clearance versus Nusinersen 7
Slide 8: Non-human Primate (NHP) Biodistribution
Non-human Primate (NHP) Biodistribution
Slide 9: NHP Biodistribution Data Show SNA Penetration to All Brain Regions
NHP Biodistribution Data Show SNA Penetration to All Brain Regions SNA Nusinersen • Rapid penetration of the NHP brain, showing enhanced CNS persistence • Reduced renal clearance versus Nusinersen 9
Slide 10: SNAs Distribute Widely Throughout the NHP Brain
SNAs Distribute Widely Throughout the NHP Brain SNA Nusinersen • Nusinersen-SNA rapidly penetrated the NHP brain and showed enhanced CNS persistence 10
Slide 11: Key Findings from NHP Study
Key Findings from NHP Study • SNAs distribute throughout the entire NHP brain following intrathecal administration ➢ SNA present in 6 parental regions with 46 sub-regions • SNAs observed at high amounts throughout the brain • These results suggest that SNA drugs may be potentially applied in a large
Slide 12: SNAs Penetrate Disease Relevant Brain Regions
SNAs Penetrate Disease Relevant Brain Regions Indication Disease Regions SNAs Present? SCAs, FA Cerebellum / brain stem Batten, ALS Cerebellum / brain stem / cortical lobes Parkinson’s Basal ganglia Huntington’s Cortical lobes / basal ganglia 12
Slide 13: Contact
Contact David Giljohann, CEO davidg@exicuretx.com David Snyder, CFO dsnyder@exicuretx.com Matthias Schroff, COO mschroff@exicuretx.com