Inhibikase Therapeutics, Inc. · NASDAQ:IKT
Inhibikase Therapeutics — Investor Presentation (January 2022)
32 slides from Inhibikase Therapeutics's investor presentation, Jan 20, 2022
Slide 1: Clinical Development of Therapies Intended to Reverse Functional Loss in Parkinson's
Clinical Development of Therapies Intended to Reverse the Functional Loss in Parkinson’s and Related Disorders 1Q 2022 BUSINESS PRESENTATION IKT
Slide 2: Forward-Looking Statements Disclaimer
2 This presentation shall not constitute an offer to sell or a solicitation of an offer to buy any securities, nor shall there be any sale of such securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the sec
Slide 3: Company Highlights: Driving Functional Reversal of Parkinson's Disease
COMPANY HIGHLIGHTS Driving Functional Reversal of Parkinson’s Disease ▪ Multi - therapeutic pipeline with emphasis on neurodegeneration inside and outside of the brain ▪ Our lead inhibitor of the Abelson Tyrosine Kinase (c - Abl), IkT - 148009, halts and reverses functional loss in animal models tha
Slide 4: Multi-Indication Pipeline in Neurodegeneration, Oncology and Infectious Disease
BIOMARKER 3 Multi - Indication Pipeline in Neurodegeneration, Oncology and Infectious Disease CLINICAL DEVELOPMENT 1 D r u g Ta r g e t Drug candidate Modality Disease indication PRECLINICAL D E V E L O P M E N T PHASE 1/1b PHASE 2 PHASE 3 Preclinical target engagement Clinical target engagement Can
Slide 5: Parkinson's Disease Market & Strategy
Parkinson’s Disease Market & Strategy IKT
Slide 6: Large Market, Opportunity For Disease Modification
Large Market, Opportunity For Disease Modification 700,000 – 1,000,000 U.S. Patients 60 AVERAGE AGE OF ONSET 60 , 000 NEW CASES / YR Other illnesses complicate development 47% 36% 35% 30% A R T HR I T I S HEART / CIRCULATORY PSYCHOSIS DEMENTIA 38 , 000 DEATHS / YEAR Chronic Disease for a Long Time 1
Slide 7: Causation in Parkinson's and Alzheimer's is closely related
7 Causation in Parkinson’s and Alzheimer’s is closely related 1 COMMON FEATURES OF MISFOLDED PROTEIN DISEASES What role does the misfolded protein play? 1 Nat. Neurosci. 21: 1332 - 1340 (2018)
Slide 8: Misfolded Protein 'Seed' in Parkinson's Reveals c-Abl as Primary Culprit
CAUSE OF NEURODEGENERATION Evaluation of the Misfolded Protein ‘Seed’ in Parkinson’s Reveals c - Abl as the Primary Culprit (video) 8
Slide 9: The Path to Neurodegeneration
THE PATH TO NEURODEGENERATION Stressors Trigger the Production of Misfolded α - Synuclein Which Activates c - Abl to Drive Neurodegeneration 2 1 Nat Rev Neurosci . 2 , 492 – 501 (2001) 2 Werner and Olanow , Mov Disorders 2021, doi: 10.1002/mds.28858 3 https://ir.prothena.com/news - releases/news - r
Slide 10: How Inhibikase Will Modify Disease
How Inhibikase Will Modify Disease IKT
Slide 11: IkT-148009: Low Toxicity, Selective, Brain Penetrant c-Abl Inhibitor
IkT - 148009: Low Toxicity, Selective, Brain Penetrant c - Abl Inhibitor in Clinical Development Toxicology in Rat/Monkey 1 Human equivalent dose of 1460 mg Cardiovascular None Renal None Liver None Bone marrow None Sternum None Blood None PBMCs Slight increase in neutrophils within normal limits Cy
Slide 12: c-Abl Inhibition Blocks the Four Pillars of Parkinson's Disease
MODIFIES DISEASE 12 c - Abl inhibition by IkT - 148009 blocks the four pillars of Parkinson’s Disease in Validated Animal Models - Synuclein Toxicity IkT - 148009 clears to baseline in the organs of disease Neurodegeneration IkT - 148009 preserves as much as 85% of brain neurons Motor Dysfunction
Slide 13: Clinical Development
Inhibikase Therapeutics Nasdaq| | KT Clinical Development
Slide 14: Phase 1: Dose Proportional Pharmacokinetics and No Significant Adverse Events
Phase 1: Dose Proportional Clinical Pharmacokinetics and No Clinically Significant Adverse Events PHASE 1 TRIAL IN SAFETY AND DOSING 14 Category Demographic Value (% of Total N=72) Gender Female 27 (37.5) Male 45 (62.5) Age Average (SD) 57.9 (5.72) Median 58.0 Range 45, 69 Ethnicity Hispanic or Lati
Slide 15: Phase 1: Dose Proportional Pharmacokinetics and No Significant Adverse Events
Phase 1:Dose Proportional Clinical Pharmacokinetics and No Clinically SigniCcant Adverse Events PHASE 1 TRIAL IN SAFETY AND DOSING 15 Human safety to date » No clinically significant adverse events have been observed across 10 dosing cohorts Significance of clinical pharmacokinetics » High exposures
Slide 16: Phase 1: Low Oral Dose Reaches Therapeutic Exposures of Animal Models
Phase 1: Low Oral Dose in Humans Reaches Therapeutic Exposures of Animal Models PHASE 1 TRIAL IN SAFETY AND DOSING 16 Clinical Pharmacokinetics IkT - 148009 compared to therapeutic dose in animal models of progressive disease 2 t 1/2 t max C max AUC 0 - ∞ mg/day (h) (h) (ng/mL) (h*ng/mL) I k T - 148
Slide 17: Parkinson's May Begin in the Gut
Parkinson’s May Begin in the Gut Easy access Can demonstrate disease benefit with quantitative endpoints Biopsy and Biomarkers Large effect size GI disorders resulting from kinase modification of - synuclein: Dysphagia Unresolvable constipation Gastroesophageal reflux Gastroparesis The Gut - Brain
Slide 18: Updated Phases and Development Intervals for 2021
Updated Phases and Development Intervals for 2021 ONGOING TRIALS IN SAFETY AND DOSING Phase 1b MAD (6 - 7 Months) Multiple Ascending Dose (MAD): 7 - Day ▪ 3 dosing cohorts, 3:1 randomized with placebo, doses determined from SAD PK and safety ▪ 8 patients/dose 7 - day dosing 1x/day ▪ Primary objectiv
Slide 19: Selected Financial and Stock Data
COMPANY HIGHLIGHTS Selected Financial and Stock Data 19 Balance Sheet September 30, 2021 (last reporting period) Current Assets: Cash $ 44 , 845 , 95 0 Grants Receivable $ 217 , 48 2 Prepaid research and development $ 264 , 38 1 Prepaid expenses and other current assets $ 541 , 38 8 Total Current As
Slide 20: Upcoming Milestones
Upcoming Milestones 20 COMPANY HIGHLIGHTS » 1Q22 • 148009 » Complete 250 mg SAD in healthy subjects » Complete two of three planned dosing cohorts in MAD 7 - day dosing in PD patients » Phase 2 study set - up in PD; first patient anticipated to commence in early 2Q22 » Complete first of two animal m
Slide 21: Management Team with Deep Experience in Drug Development
ABOUT US Management Team with Deep Experience in Drug Development and Commercialization Executive Milton Werner, PhD President & CEO Previously, Dr . Werner served as Director of Research at Celtaxsys . From September 1996 until June 2007 , Dr . Werner was a Head of the Laboratory of Molecular Bioph
Slide 22: Board of Directors and Industry-Leading Advisors
Board of Directors I n d u s t r y - L e a d i n g A d v i s o r s Mr . Dennis Berman • Co - founder, board member, and/or seed investor in many private biotechnology and technology companies, five of which have gone public . • Currently serves as the President of Molino Ventures, LLC a board adviso
Slide 23: Appendix: Proof of the Importance of c-Abl in Disease
Proof of the importance of c - Abl in Disease, Target Engagement and Functional Reversal in the Brain and Gut IKT Appendix
Slide 24: Biochemistry of Parkinson's Disease Initiation and Progression
24 Biochemistry of Parkinson’s Disease Initiation and Progression 1 THE PATH TO DISEASE MODIFICATION 1 Werner and Olanow, Mov. Disorders 2021, in press; , J Clin Invest. 2016; 126: 2970 - 2988 , Brain 2019; 142:2380 - 2401 , Cell 2011; 144: 689 - 702 , Nat Neurosci. 2013; 16: 1392 - 1400 , Adv Neuro
Slide 25: Activated c-Abl Is the Gateway in the Disease Path
INHIBIKASE’S PROOF ACTIVATED C - ABL IS THE GATEWAY IN THE DISEASE PATH α - Synuclein Plaques Do Not Cause Disease Without c - Abl Modification in Humanized Preclinical Models 1 α - Synuclein plaque in the ABSENCE OF c - Abl CAUSES NO NEURODEGENERATION AFTER 6 MONTHS 1 Brain 142:2380ff (2019) Contro
Slide 26: Pathologic c-Abl-Modified α-Synuclein Present in Parkinson's Patient Brain
S T U D Y Pathologic, c - Abl - Modified α - Synuclein (pY39) is Present in Parkinson’s Patient Brain 1 Toxic pY39 - a - Synuclein Abundant In Substantia Nigra (SN) of Patients Toxic pY39 - a - Synuclein Prominent in Lewy Bodies 1 J Clin Invest. 126 , 2970 - 88 (2016) 26
Slide 27: Oral IkT-148009 in Mice Reverses Functional Loss
Baseline Neuromuscular Coordination Behavioral Marker Toxic Levels of Synuclein Are Introduced Into One Side of the Mouse Brain Healthy Mice Run In Circles at the Average Rate of 25 Turns : 10 Min Toxicity renders the mice trembling and only able to run in circles Treatment Started 6 Weeks After Ons
Slide 28: Oral IkT-148009 Suppresses c-Abl Activation in the Brain
28 Oral IkT - 148009 Suppresses c - Abl Activation in the Brain that Correlates with Functional Recovery ANIMAL STUDY BASIS OF 148009 THERAPY IkT - 148009 engages the c - Abl target in the brain Activated c - Abl in disease Baseline Inhibition of c - Abl below baseline by IkT - 148009
Slide 29: Oral IkT-148009 Preserves Neural Anatomy in the Brain
29 Placebo IkT - 148009 only, no disease A53T induced disease A53T - induced disease + IkT - 148009 Normal # neurons Rescued # neurons ( ≥ 80%) Disease # neurons (< 50%) Oral IkT - 148009 Preserves Neural Anatomy in the Brain ANIMAL STUDY BASIS OF 148009 THERAPY IkT - 148009 stopped loss of neurons,
Slide 30: Oral IkT-148009 in Mice Reverses Functional Loss in the Gut
METHOD OF MEASUREMENT α - Synuclein Aggregates Introduced Into Gut Slows Gut Transit Time Measure defecation rate Neuron count read out by Whole Gut Transit Time - The time it takes for a mouse to defecate Carmine dye Vehicle (Control) IkT – 148009 Vehicle (Control) IkT – 148009 A53T Human α - Synuc
Slide 31: IkT-148009 Drives Clearance of Pathological α-Synuclein
IkT - 148009 drives clearance of pathological α - synuclein (green dots have been cleared) 31 Oral IkT - 148009 Treatment Clears Toxic - Synuclein in the brain and gut EFFECT ON CAUSE OF DISEASE Clearance of toxicity in the gut Clearance of toxicity in the brain Green: Pathological - synuclein R
Slide 32: Research Advances Are Transformational
▪ IKT - 148009 drives functional recovery inside and outside of the brain ▪ IKT - 148009 drives clearance of the toxic form of - synuclein ▪ Low oral doses in humans achieve therapeutic exposure levels observed in animal efficacy studies Targeting c - Abl we believe is transformational to treatmen
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