Inhibikase Therapeutics, Inc. · NASDAQ:IKT
Inhibikase Therapeutics — Investor Presentation (October 2021)
32 slides from Inhibikase Therapeutics's investor presentation, Oct 19, 2021
Slide 1: Clinical Development of Therapies to Reverse Functional Loss in Parkinson's
Clinical Development of Therapies Intended to Reverse the Functional Loss in Parkinson’s and Related Disorders 4Q 2021 BUSINESS PRESENTATION IKT
Slide 2: Forward-looking statements disclaimer
2 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 s
Slide 3: Company Highlights: Driving Functional Reversal of Parkinson's Disease
COMPANY HIGHLIGHTS Driving Functional Reversal of Parkinson’s Disease ▪ Five clinical programs in neurodegeneration and one clinical program in oncology running in 2022 ▪ Multi - therapeutic pipeline with emphasis on neurodegeneration inside and outside of the brain ▪ Our lead inhibitor of the Abels
Slide 4: Multi-Indication Pipeline in Neurodegeneration, Oncology and Infectious Disease
Multi - Indication Pipeline in Neurodegeneration, Oncology and Infectious Disease 4 COMPANY HIGHLIGHTS: MULTI - THERAPEUTIC PIPELINE
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 AVERAGE AGE OF ONSET 60,000 NEW CASES / YR Other illnesses complicate development ARTHRITIS HEART / CIRCULATORY PSYCHOSIS DEMENTIA 47% 36% 35% 30% 700,000 – 1,000,000 U .S. Patients 38,000 DEATHS / YEAR Chronic Disease for a Long T ime 1/3 of a Pati
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: c-Abl as the Primary Culprit, Not the Misfolded Protein
CAUSE OF NEURODEGENERATION Evaluation of the Misfolded Protein ‘Seed’ in Parkinson’s Reveals c - Abl as the Primary Culprit, NOT the Misfolded Protein ▪ Parkinson’s Disease (PD) is a neurodegenerative disease which limits function of nerve cells throughout the brain and gut following misfolding of n
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 (2021), under review 3 https:// ir.prothena.com /news - releases/news - release - details/updat
Slide 10: Biochemistry of Parkinson's Disease Initiation and Progression
10 Biochemistry of Parkinson’s Disease Initiation and Progression 1 THE PATH TO DISEASE MODIFICATION 1 Werner and Olanow (2021 ), under review, J Clin Invest. 2016; 126: 2970 - 2988 , Brain 2019; 142:2380 - 2401 , Cell 2011; 144: 689 - 702 , Nat Neurosci . 2013; 16: 1392 - 1400 , Adv Neurobiol . 201
Slide 11: How Inhibikase Will Modify Disease
How Inhibikase Will Modify Disease IKT
Slide 12: IkT-148009: 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 600 mg Cardiovascular None Renal None Liver None Bone marrow None Sternum None Blood None PBMCs Slight increase in neutrophils within normal limits Cyt
Slide 13: c-Abl inhibition blocks the four pillars of Parkinson's Disease
MODIFIES DISEASE 13 c - Abl inhibition by IkT - 148009 blocks the four pillars of Parkinson’s Disease in Validated Animal Models a - 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 14: Clinical Development
Inhibikase Therapeutics Nasdaq IKT Clinical Development
Slide 15: Phase 1: Clinical 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 15 Category Demographic Value (% of Total N=56) Gender Female 19 (33.9) Male 37 (66.1) Age Average (SD) 57.2 (6.08) Median 57.0 Range 45, 69 Ethnicity Hispanic or Lati
Slide 16: Phase 1: Dose Proportional Pharmacokinetics and Safety
Phase 1:Dose Proportional Clinical Pharmacokinetics and No Clinically Significant Adverse Events PHASE 1 TRIAL IN SAFETY AND DOSING 16 Human safety to date » No clinically significant adverse events have been observed across 7 dosing cohorts Significance of clinical pharmacokinetics » High exposures
Slide 17: 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 17 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) IkT - 14800
Slide 18: 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 a - synuclein: Dysphagia Unresolvable constipation Gastroesophageal reflux Gastroparesis The Gut - Brain
Slide 19: 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) Phase 1 Chronic Toxicology Studies (4 Months to go) Multiple Ascending Dose (MAD): 7 - Day ▪ 3 dosing cohorts, 3:1 randomized with placebo, doses determined from SAD PK and safety ▪ 8 pa
Slide 20: Selected Financial and Stock Data
COMPANY HIGHLIGHTS Selected Financial and Stock Data 20 20 20 Balance Sheet June 30, 2021 Current Assets: Cash $ 46,836,556 Grants Receivable $ 586,581 Prepaid research and development $ 958,779 Prepaid expenses and other current assets $ 833,963 Total Current Assets $ 49,215,879 Total Current Liabi
Slide 21: Upcoming Milestones
Upcoming Milestones 21 21 COMPANY HIGHLIGHTS » 4Q21 • 148009 » Complete 125 mg SAD in healthy subjects » Completed 25 mg MAD 7 - day dosing in healthy subjects » First dosing of Parkinson’s patients early 4Q21 » Fast - Track Designation request under review; outcome before end of 4Q21 anticipated »
Slide 22: Management Team with Deep Experience in Drug Development
ABOUT US Management Team with Deep Experience in Drug Development and Commercialization 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 Biophysics at T
Slide 23: Board of Directors and Industry-Leading Advisors
Board of Directors Industry - Leading Advisors 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 advisory and venture capit
Slide 24: Proof of the importance of c-Abl in Disease, Target Engagement and Functional Reversal
Proof of the importance of c - Abl in Disease, Target Engagement and Functional Reversal in the Brain and Gut IKT Appendix
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 Patient Brain
STUDY 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 in Brain
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 Inhibition of c - Abl below baseline by IkT - 148009 Baseline
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 Gut
α - Synuclein Aggregates Introduced Into Gut Slows Gut Transit Time METHOD OF MEASUREMENT POST TREATMENT RESULTS 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 A53T Human α - Synuclein Veh
Slide 31: Oral IkT-148009 Clears Toxic α-Synuclein in Brain and Gut
IkT - 148009 drives clearance of pathological α - synuclein (green dots have been cleared) 31 Oral IkT - 148009 Treatment Clears Toxic a - Synuclein in the brain and gut EFFECT ON CAUSE OF DISEASE Clearance of toxicity in the gut Green: Pathological a - synuclein Red: Neural ganglia in gut Clearance
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 a - synuclein ▪ Low oral doses in humans achieve therapeutic exposure levels observed in animal efficacy studies ▪ Early clinical outcomes with IkT - 148009 have accelerated
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