Exicure, Inc. · NASDAQ:XCUR
Exicure — Investor Presentation (January 2022)
23 slides from Exicure's investor presentation, Jan 5, 2022
Slide 1: Pursuing Next Generation Nucleic Acid Therapies
Pursuing Next Generation Nucleic Acid Therapies NASDAQ Ticker: XCUR January 5, 2022 Exhibit 99.1
Slide 2: Cautionary Note Regarding Forward Looking Statements
© 2022 Exicure, Inc. All Rights Reserved. | Cautionary Note Regarding Forward Looking Statements 2 This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "expect," "plan," "may," "will," “continue,
Slide 3: Exicure Snapshot
© 2022 Exicure, Inc. All Rights Reserved. | Exicure Snapshot 3 • Exicure pursues and develops next-generation nucleic acid therapies targeting RNA • Proven team with deep drug development and cutting-edge oligonucleotide chemistry know-how • Exicure addresses cell and tissue uptake limitation of lin
Slide 4: An Established and Experienced Leadership & Management Team
© 2022 Exicure, Inc. All Rights Reserved. | An Established and Experienced Leadership & Management Team 4 Brian Bock Chief Executive Officer Matthias Schroff Chief Scientific Officer Tim Walbert Chairman Jeffrey L. ClelandJames SulatBali Muralidhar Bosun Hau Elizabeth Garofalo Andrew Sassine MANAGEM
Slide 5: Leadership Supported by Scientists With Nucleic Acid Expertise and R&D Engine
© 2022 Exicure, Inc. All Rights Reserved. | Leadership Supported by Scientists With Nucleic Acid Expertise and R&D Engine to Get From Bench to Bedside Rapidly 5 • Diverse scientific staff with expert knowledge in nucleic acid chemistry, drug development and neuroscience • 13 PhDs with more than 100
Slide 6: Exicure has Identified Several Promising Candidates Targeting Severe Neurological Conditions
© 2022 Exicure, Inc. All Rights Reserved. | Exicure has Identified Several Promising Candidates Targeting Severe Neurological Conditions With Its Proprietary Platform 6 • Extensive proprietary IP protection across the platform and composition of matter • Partnerships with Ipsen and AbbVie targeting
Slide 7: Exicure's Next Generation Pipeline and Investment Focus is Neuroscience
© 2022 Exicure, Inc. All Rights Reserved. | Exicure’s Next Generation Pipeline and Investment Focus is Neuroscience DISCOVERY PRECLINICAL DEV PHASE 1 NEUROSCIENCE SCN9A Neuropathic Pain Undisclosed Huntington’s Disease Undisclosed Angelman Syndrome DERMATOLOGY Undisclosed Hair Loss Disorders 1) On N
Slide 8: SNAs: Next Generation Nucleotide Delivery Platform
SNAs: Next Generation Nucleotide Delivery Platform
Slide 9: SNA: A Unique and Effective 3D Architecture for Oligonucleotide Therapeutics
© 2022 Exicure, Inc. All Rights Reserved. | SNA™: A Unique and Effective 3D Architecture For the Next Generation of Oligonucleotide Therapeutics 9 Synthetic Oligonucleotides Benign Lipid Nanoparticle Scaffold1SNA Spherical Nucleic Acid • Compatible with virtually all oligonucleotide classes/chemistr
Slide 10: SNAs Address Current Challenges with Linear Oligonucleotide Therapeutics
© 2022 Exicure, Inc. All Rights Reserved. | SNAs Address Current Challenges with Linear Oligonucleotide Therapeutics – Delivery, Versatility and Multi-Targeting 10 Multi-Targeting Capability to Address Complex Diseases Delivery of defined ratios of oligonucleotides 2 1 3 Oligo vs. Target A Oligo vs.
Slide 11: Example Application: Restoring Function and Increasing Longevity in Mouse Model
© 2022 Exicure, Inc. All Rights Reserved. | Example Application: Restoring Function and Increasing Longevity in Mouse Model of Progressive Muscle Wasting Disease 11 Day 14 Day 62 • Exicure-treated mouse on left, diseased mice1 in the middle and non-diseased mice on right • Based on one-dose with Exi
Slide 12: Three Drivers of Superior Efficacy in Neuroscience
© 2022 Exicure, Inc. All Rights Reserved. | Three Drivers of Superior Efficacy in Neuroscience 12 1 Higher Cell Uptake, Leading to Higher Potency at Same Dose 2 Broad CNS Penetration and Uptake in all Neuronal Cells SNA Linear Superior cell uptake1 vs. linear oligonucleotide with SNA detection obser
Slide 13: Targeting Pain
Targeting Pain exicure’
Slide 14: Sodium Channel 1.7 is a Key Pain Signaling Gatekeeper
© 2022 Exicure, Inc. All Rights Reserved. | Sodium Channel 1.7 is a Key Pain Signaling Gatekeeper 14 Figure adapted using Biorender from: Waxman and Zamponi (2014), Ofra Gohar (2020), Julius & Blasboum (2001), and Alsaloum et al (2020) Pain Signaling Pathway Brain perceives pain by neuronal cell sig
Slide 15: Nav1.7, a Validated Non-Opioid Pain Target, is Ideally Suited to Exicure's Approach
© 2022 Exicure, Inc. All Rights Reserved. | Small Molecule Inhibitor of Nav1.7 (nM) C ha nn el A ct iv at io n • Due to small selectivity margins, small molecules fail to reach target inhibition levels in DRG • Cellular knockdown via SNAs has proven exquisitely selective, with SNA screening hits not
Slide 16: Several Potent Candidates Achieving Greater Than 95% Target Reduction in Sensory Neurons
© 2022 Exicure, Inc. All Rights Reserved. | Exicure has Identified Several Potent Candidates Achieving Greater Than 95% Target Reduction in Sensory Neurons 16 A reduction in SCN9A expression of only ~50% may have therapeutic benefit based on human genetic studies1 Target mRNA Knockdown in iPSC- Deri
Slide 17: SNA Drug Candidate Abolish Key Electrical Transmissions in Pain-Signaling Neurons
© 2022 Exicure, Inc. All Rights Reserved. | SNA Drug Candidate Abolish Key Electrical Transmissions Critical in Pain-Signaling Neurons with Durable Effect 17 • In vitro cultured neurons establish a network communication via electrical signaling • Multi-electrode array measures key signaling (action
Slide 18: Existing Partnerships
Existing Partnerships
Slide 19: Exicure & AbbVie: Collaboration Targeting Hair Loss Disorders
© 2022 Exicure, Inc. All Rights Reserved. | Exicure & AbbVie: Using Exicure’s Discovery & Development Engine for Collaboration Targeting Hair Loss Disorders 19 • $25M upfront • Up to $50M in option/IND- enabling payments • Up to $195M in development and regulatory milestones • Up to $530M in commerc
Slide 20: Exicure and Ipsen: Neuroscience Collaboration Targeting Huntington's and Angelman's
© 2022 Exicure, Inc. All Rights Reserved. | Exicure and Ipsen: Using Exicure’s Discovery & Development Engine for Neuroscience Collaboration Targeting Huntington’s and Angelman’s 20 • $1B+ total deal value • $20M cash upfront • Up to $10M in preclinical milestones • Up to $50M in option exercise pay
Slide 21: Exicure Corporate Financial Snapshot
© 2022 Exicure, Inc. All Rights Reserved. | Exicure Corporate Financial Snapshot(1) 21 • Nasdaq: XCUR • Est. 2021 Year-end Cash: $48.3 million(2) • Outstanding Principal Debt: $7.5 million • Common Stock Outstanding: 108.8 million • Outstanding Warrants: 31.3 million(3) 1) Figures include unaudited
Slide 22: Exicure has Several Promising Therapeutics, Including Non-Opioid Pain Analgesic Candidates
© 2022 Exicure, Inc. All Rights Reserved. | Exicure has Several Promising Therapeutics, Including Candidates for a Highly Coveted Non-Opioid Pain Analgesic 22 • Promising initial data with several candidates targeting Nav1.7, a non-opioid target in pain, with near-term milestones in 2022 – Conservat
Slide 23: exbcure’
exbcure’ www.exicuretx.com