Inhibikase Therapeutics, Inc. · NASDAQ:IKT
Inhibikase Therapeutics — Investor Presentation (April 2022)
32 slides from Inhibikase Therapeutics's investor presentation, Apr 21, 2022
Slide 1: Clinical Development of Disease-Modifying Therapeutics for Parkinson's and Related Disorders
Clinical Development of Disease- Modifying Therapeutics for Parkinson’s and Related Disorders 2Q 2022 BUSINESS PRESENTATION IKT Exhibit 99.1
Slide 2: Forward-looking statements disclaimer
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 secur
Slide 3: Treating Inside and Outside of the Brain
TREATING INSIDE AND OUTSIDE OF THE BRAIN Simultaneous Evaluation of Brain and GI Function Expands the Definition of a Disease-Modifying Treatment PD is more than just a disease of the brain. GI manifestation in many patients occurs at an early stage, suggesting that evaluation of GI and brain functi
Slide 4: Clinical Development Pipeline
CLINICAL DEVELOPMENT1 BIOMARKER3 Drug Target Drug candidate Modality Disease indication PRECLINICAL DEVELOPMENT PHASE 1/1b PHASE 2 PHASE 3 Preclinical target engagement Clinical target engagement Can be used for patient selection Neurodegeneration c-Abl IkT-148009 Small molecule Parkinson’s Disease:
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 Time 1/3 of a Patien
Slide 7: Causation in Parkinson's and Alzheimer's is closely related
Causation in Parkinson’s and Alzheimer’s is closely related1 COMMON FEATURES OF MISFOLDED PROTEIN DISEASES What role does the misfolded protein play? 1Nat. Neurosci. 21: 1332-1340 (2018)
Slide 8: Cause of Neurodegeneration: 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)
Slide 9: The Path to Neurodegeneration
THE PATH TO NEURODEGENERATION Stressors Trigger the Production of Misfolded α-Synuclein Which Activates c-Abl to Drive Neurodegeneration2 1Nat Rev Neurosci. 2, 492–501 (2001) 2 Werner and Olanow , Mov Disorders 2021, doi: 10.1002/mds.28858 3https://ir.prothena.com/news-releases/news-release-details/
Slide 10: How Inhibikase Will Modify Disease
How Inhibikase Will Modify Disease IKT
Slide 11: Re-engineering Approach with Metabolism Preserved (RAMP)
Re-engineering Approach with Metabolism Preserved (RAMPTM) IS A SMALL MOLECULE c-ABL INHIBITOR
Slide 12: 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/Monkey1 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 Cytotox
Slide 13: c-Abl inhibition blocks the four pillars of Parkinson's Disease
MODIFIES DISEASE 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 IkT-148009 re
Slide 14: Inhibikase Therapeutics Clinical Development
Inhibikase Therapeutics Nasdaq | IKT Clinical Development
Slide 15: 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 Category Demographic Value (% of Total N=88) Gender Female 34 (38.6) Male 54 (61.4) Age Average (SD) 57.9 (5.72) Median 58.0 Range 45, 69 Ethnicity Hispanic or Latino
Slide 16: Phase 1: Dose Proportional Clinical Pharmacokinetics
Phase 1:Dose Proportional Clinical Pharmacokinetics and No Clinically Significant Adverse Events PHASE 1 TRIAL IN SAFETY AND DOSING Significance of clinical pharmacokinetics High exposures at low oral dose, linearly dose proportional up to 175 mg. Exposures at 75 mg IkT-148009 comparable to 500 mg i
Slide 17: Phase 1b: Mild to Moderate PD and No Significant Adverse Events
Phase 1b: Mild to Moderate PD (H&Y < 3.0) and No Clinically Significant Adverse Events PHASE 1 TRIAL IN SAFETY AND DOSING Category Demographic Value (% of Total N=13) Gender Female 6 (42.8) Male 7 (57.2) Age Average 62.5 Median 62 Range 57, 70 Ethnicity Hispanic or Latino 3 (23.1) Not Hispanic or La
Slide 18: Phase 1b: Pharmacokinetics similar to elderly healthy subjects
Phase 1b:Pharmacokinetics similar to elderly healthy subjects PHASE 1 TRIAL IN SAFETY AND DOSING 1FDA summary data for approval 21-335 T1/2 (h) Tmax (h) Cmax (ng/ml) AUC0-24 (ng-h/mL) Vz/F (l) CL (l/h) Day 1 Mean 25 mg 15.4 5 1040 12700 32.5 1.52 N=6 SD Healthy 11.3 4 419 6010 14.7 0.905 Day 7 Mean
Slide 19: Clinical Phase 1b and Phase 2 Programs
Clinical Phase 1b and Phase 2 Programs ONGOING TRIALS IN SAFETY AND DOSING Phase 1b MAD (6-8 Months) Oct. 2021 u 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 Multiple Ascending Dose (MAD): 7-Day Dosing 3 dosing cohorts, 3:1 randomized with placebo, doses determined from SAD PK and safety 24 patients
Slide 20: Company Highlights: Selected Financial and Stock Data
COMPANY HIGHLIGHTS Selected Financial and Stock Data Balance Sheet December 31, 2021 (last reporting period) Current Assets: Cash $ 40,750,133 Grants Receivable $ 110,141 Prepaid research and development $ 107,000 Prepaid expenses and other current assets $ 1,502,725 Total Current Assets $ 42,469,99
Slide 21: Upcoming Milestones
Upcoming Milestones COMPANY HIGHLIGHTS 2Q22 148009 Complete Phase 1b study in Parkinson’s patients with mild to moderate disease Initiate dosing in Phase 2a study in early-stage Parkinson’s patients Complete first of two animal model validation studies of IkT-148009 in MSA Set-up EU/US sites for Pha
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 The R
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 capital firm an
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 Models1 α-Synuclein plaque in the ABSENCE OF c-Abl CAUSES NO NEURODEGENERATION AFTER 6 MONTHS 1Brain 142:2380ff (2019) Control Synuclein
Slide 26: Pathologic c-Abl-Modified α-Synuclein Present in Parkinson's Patient Brain
STUDY Pathologic, c-Abl-Modified α-Synuclein (pY39) is Present in Parkinson’s Patient Brain1 Toxic pY39-a-Synuclein Abundant In Substantia Nigra (SN) of Patients Toxic pY39-a-Synuclein Prominent in Lewy Bodies 1J Clin Invest. 126, 2970-88 (2016)
Slide 27: Oral IkT-148009 Reverses Functional Loss in Mice
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
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 Normal Mice Normal Mice +148009 Parkinson’s Mice Parkinson’s Mice +148009 Parkinson’s Mice +148009 and elacridar Act
Slide 29: Oral IkT-148009 Preserves Neural Anatomy in the Brain
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, accounting fo
Slide 30: Oral IkT-148009 Reverses Functional Loss in the 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 Vehicle
Slide 31: Oral IkT-148009 Clears Toxic α-Synuclein in the brain and gut
IkT-148009 drives clearance of pathological α-synuclein (green dots have been cleared) 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 of toxicity
Slide 32: IKT-148009 drives functional recovery inside and outside of the brain
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 Targeting c-Abl we believe is transformational to treatment of neurodege
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