Clinical Development of Disease- Modifying Therapeutics for Parkinson’s and Related Disorders 2Q 2022 BUSINESS PRESENTATION IKT Exhibit 99.1
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 securities laws of any such state or jurisdiction. This presentation contains information that may constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended. Inhibikase Therapeutics, Inc. (the “Company” or “we”) intends for the forward-looking statements to be covered by the safe harbor provisions for forward-looking statements in those sections. Generally, we have identified such forward-looking statements by using the words “believe,” “expect,” “intend,” “estimate,” “anticipate,” “project,” “target,” “forecast,” “aim,” "should," “will,” "may”, “continue” and similar expressions. Such statements are subject to a number of assumptions, risks and uncertainties which may cause actual results, performance or achievements to be materially different from those anticipated in these forward-looking statements. You should read statements that contain these words carefully because they discuss future expectations and plans which contain projections of future clinical studies, regulatory approvals, product candidate development, results of operations or financial condition or state other forward-looking information. However, the absence of these words or similar expressions does not mean that a statement is not forward-looking. Forward-looking statements are not historical facts, but instead represent only the Company’s beliefs regarding future events, many of which, by their nature, are inherently uncertain and outside of the Company’s control. It is possible that the Company’s actual results and financial condition may differ, possibly materially, from the anticipated results and financial condition indicated in these forward-looking statements. Management believes that these forward-looking statements are reasonable as of the time made. However, caution should be taken not to place undue reliance on any such forward-looking statements because such statements speak only as of the date when made. The Company undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. In addition, forward-looking statements are subject to certain risks and uncertainties that could cause actual results to differ materially from the Company's historical experience and our present expectations or projections. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in the Company’s filings with the Securities and Exchange Commission, including its annual report on Form 10-K, including under the caption "Risk Factors”. We do not intend our use or display of other entities’ names, trade names, trademarks or service marks to imply a relationship with, or endorsement or sponsorship of us by, any other entity. Disclaimer
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 function could be essential to identifying truly disease-modifying treatments Utilization of slowly progressive, a-synuclein dependent animal models that reproduce the rate of disease progression relative to lifespan of the human disease have been key to properly identifying potential disease-modifying therapeutics. Our lead asset is an inhibitor of the Abelson Tyrosine Kinase (c-Abl). IkT-148009, halts and reverses functional loss; discovered using slowly progressive animal models. Six clinical programs in 2022 across two assets in Parkinson’s disease, Multiple Systems Atrophy (orphan indication, orphan designation not sought) and Chronic Myelogenous Leukemia (orphan indication, orphan designation granted). Phase 2 programs anticipated to begin early 2Q22 in neurodegeneration, readouts anticipated within 12 months. Multiple patent families for lead compound with expiration of 2036 and beyond $20.8 million in grants and contracts from NIH, DoD, the Michael J. Fox Foundation and the Georgia Research Alliance, all peer-reviewed; $63 million gross proceeds in investor capital in 2021 Highly experienced and respected management team, consultants, Board of Directors and nearly all KOLs in the field on Scientific Advisory Board
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: Treatment Naïve Validated Validating Yes c-Abl IkT-148009 Small molecule Parkinson’s Disease: Early Stage Validated Validating Yes c-Abl IkT-148009 Small molecule Neurogenic Constipation Validated Validating Yes c-Abl IkT-148009 Small molecule Dysphagia Validated Validating Yes c-Abl IkT-148009 Small molecule Multiple System Atrophy Validated Validating Yes Oncology BCR-Abl IkT-001Pro Small molecule Stable-phase CML (orphan indication) Validated Validated Yes Research Phase c-Abl IkT-148x Small molecule Dementia with Lewy Body Validated Validating Unknown c-Abl IkT-148x Small molecule Multiple System Atrophy Validated Validating Unknown c-Abl IkT-1427 Small molecule Progressive multifocal leukoencephalopathy Validated Validating Yes ‘Clinical Development’ progress bars represent the current state of the indicated programs. Blue arrows represent completed or in progress studies; white arrows represent planned approaches for future clinical studies. Four indications will be pursued for IkT-148009 in PD, which will be pursued through two INDs, one focused on treatment in the brain in treatment naïve or early-stage patients and the second focused on GI complications. MSA is a Parkinson’s-like disease to enter clinical development at Phase 2 sharing the Phase 1 data for 148009 with PD. MSA moves forward in clinic ONLY if animal model study ongoing is positive. For biomarker status, ‘Validated’ refers to proof of target engagement in the target tissue which has been performed using rodent tissues and fluids. We are currently developing methods for using clinical samples for validating our ability to confirm target engagement in patients. ‘Validating’ in this context indicates ongoing efforts to prove target engagement using proprietary sources and methods under development from human tissues and fluids. Target engagement measures if and to what extent a compound occupies its target. ‘Can be used for patient selection’ refers to our ability to use one or more markers we are currently ‘Validating’ to screen patients for the presence of that marker as a means of defining the patients most likely to benefit from the proposed treatment. 4 Indications Pursued Through 2 INDs. Shares Same Phase 12 505(b)(2) Path to Market IMPD/CTAs to be filed in EU, IND to be filed FDA,2022. Shares Same Phase 12 Multi-Indication Pipeline in Neurodegeneration, Oncology and Infectious Disease COMPANY HIGHLIGHTS: MULTI-THERAPEUTIC PIPELINE
Parkinson’s Disease Market & Strategy IKT
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 Patient’s Lifespan = 25 years 60 Men twice as likely as women to contract disease Parkinson’s Disease in the U.S.1 THE MARKET 1Parkinson’s Disease Foundation Decisions Resources 2016, ParkinsonismRelatDisord . 2012;18:1073-1078, Neuroepidemiology 2010;34:143–151 , J Neurol Neurosurg Psychiatry. 1997 Jan;62(1):10-5. Double BY 2025, PARKINSON’S DISEASE DRUG SALES ARE EXPECTED TO $6.0 Billion SALES ESTIMATES BY 2025 ARE EXPECTED TO CREST Pharma Insights, 2019 Pharma Insights, 2019 The U.S. THE COUNTRY WITH THE HIGHEST DIAGNOSED PREVALENCE IS DelveInsight, 2019
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)
CAUSE OF NEURODEGENERATION Evaluation of the Misfolded Protein ‘Seed’ in Parkinson’s Reveals c-Abl as the Primary Culprit (video)
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/update-phase-2-pasadena-study-prasinezumab-prx002rg7935 http://media.biogen.com/node/22876/html Immunotherapy acts here and fails2
How Inhibikase Will Modify Disease IKT
Re-engineering Approach with Metabolism Preserved (RAMPTM) IS A SMALL MOLECULE 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 Cytotoxicity None in primary or mature cells Sustained brain concentration > 1 micromolar 113 week and 39 week toxicology data shows IkT-148009 has a more favorable toxicity profile as dosing is extended IS A SMALL MOLECULE c-ABL INHIBITOR Selective Inhibitor of c-Abl and Abl2/Arg bypasses toxicity of cancer drugs1 No organ toxicity High brain penetrance 1See SelleckChem.com, Leuk 23:1689ff (2009) Inhibition of these enzymes leads to toxicity Greater Inhibition
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 restores as much as 90% of lost function Neuroinflammation IkT-148009 suppresses to near baseline in the organs of disease
Clinical Development IKT
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 13 (14.8) Not Hispanic or Latino 75 (85.2) Race Black or African American 54 (61.4) White 33 (37.5) Other 1 (1.1) Adverse events 7 (7.9), all clinically insignificant No GI No Cardiovascular No Hematological
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 imatinib1 1FDA summary data for approval 21-335 Therapeutic dosing range
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 Latino 10 (76.9) Race Black or African American 2 (15.4) White 11 (84.6) Other 0 (0) Adverse events 5 (38.5), (pneumonia, spinal headache, dermatitis, constipation, orthostatic hypotension)
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 27.4 4.67 1770 25400 42.8 1.1 N=6 SD 5.09 1.63 807 9260 15.3 0.384 Day 1 Mean 50 mg 10.1 4.67 1720 19400 37.2 2.51 N=6 SD PD 2.7 1.03 737 9470 22.8 1.2 Day 7 Mean 24.9 3.67 2560 32500 57.1 1.61 N=6 SD 3.86 1.51 564 8500 12.4 0.312 Trends in motor and non-motor scores and assessments and in GI function that may have occurred over 7 day-dosing need more cohorts to be completed to draw any conclusions.
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 total 8 patients/dose 7-day dosing 1x/day Hoehn & Yahr (H&Y) < 3.0 Primary endpoints: safety, tolerability, pharmacokinetics (PK), urine, plasma and spinal fluid concentrations Phase 1b ONGOING Multiple Ascending Dose (MAD): 3 Mos Dosing 3 dosing cohorts, 1 placebo cohort 120 patients total 30 patients/dose 3:1 randomized 13-week dosing 1x/day Treatment naïve/Early state patients (H&Y ≤ 2.0) Primary endpoints: safety, tolerability Phase 2a 2Q22 Phase 2a ( Up to 12 months) Multiple Ascending Dose (MAD): 7-Day Dosing Exploratory endpoints: UPDRS II, III, II+III, NMSS, PDQ-39, CSBM, PAGI-SYM Multiple Ascending Dose (MAD): 3 Mos Dosing Animal GI Recovery < 4 weeks Animal Brain Recovery < 8 weeks Secondary endpoints UPDRS II, III, II+III, PGI-S, CGI-S, Epworth Sleepiness Scale, NMSS, PDQ-39, CSBM, PAGI-SYM, PAC-QOL, PAGI-SYM-QOL Exploratory endpoints: Phospho-a-synuclein GI, Skin and CSF; Whole Gut Transit Time (SmartPillTM) Descriptive statistics Apr/May 2022
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 $ 42,469,999 Total Current Liabilities $ 4,054,450 Working Capital $ 38,415,549 Active grant funding available in accounts held by the U.S. treasury $ 385,888 Total Working Capital $ 38,801,437 $20.8M non-dilutive grant revenue pre-IPO (NIH, DoD, State gov’ts) Capitalization Table March 31, 2022 Common Shares Outstanding 25,227,051 Options (WAEP: $2.47) 4,208,056 Warrants (WAEP: $5.21) 1,561,913 Fully Diluted Shares Outstanding 30,997,020
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 Phase 2 studies in MSA anticipated to commence in 3Q22 Meet with FDA to review Phase 2 and Phase 3 development plans 001Pro File IND and commence bioequivalence clinical studies Design and develop superiority studies for IkT-001Pro relative to standard-of-care Identify and begin developing commercial partnership
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 Rockefeller University in New York City. Throughout his scientific career, Dr. Werner has been an innovator integrating chemistry, physics, and biology into a comprehensive approach to solving problems in medicine. Dr. Werner is the author or co-author of more than 70 research articles, reviews, and book chapters and has given lectures on his research work throughout the world. Joseph Frattaroli, CPA Chief Financial Officer Mr. Frattaroli is a certified public accountant with more than 15 years of experience in public company filings and compliance for Nasdaq and OTC Markets companies. Previously, he provided chief financial officer and consulting services for several emerging biopharmaceutical and medical device companies, with responsibilities that included capital formation, deal structuring, and assisting private companies in their transition to becoming publicly traded SEC registrants. Executive C. Warren Olanow, MD, Interim Chief Medical Officer and Chief Executive Officer of CLINTREX. Dr. Olanow is the former Henry P. and Georgette Goldschmidt Professor and Chairman of the Department of Neurology at the Mount Sinai School of Medicine Prior to joining Mount Sinai, he served on the faculties of McGill University, Duke University, and the University of South Florida. He is the former President of the Movement Disorder Society, past President of the International Society of Motor Disturbances, and former Treasurer of the American Neurological Association. He has served on the executive committee of the Michael J. Fox Foundation Scientific Advisory Board, and he is the former Chairman of the Scientific Advisory Board of the Bachmann-Strauss Parkinson Foundation and of the Dystonia Foundation. Dr. Olanow is the former Co-Editor-in-Chief of the journal Movement Disorders. Dr. Olanow received his medical degree from the University of Toronto, performed his neurology training at the New York Neurological Institute at Columbia Presbyterian Medical Center at Columbia University, and undertook postgraduate studies in neuroanatomy at Columbia University and authored more than 600 articles in the field of neurodegeneration.
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 and was co-founder and Executive Vice President of Corporate Development of Tocagen. Seed investor, co-founder, and/or board member of Intervu, Kintera, Inc., Gensia, Calabrian Dr. Roy Freeman, MD Professor of Neurology at the Harvard Medical School and Director of the Center for Autonomic and Peripheral Nerve Disorders in the Department of Neurology at Beth Israel Deaconess Medical Center Former chairman of the World Federation of Neurology research group on the autonomic nervous system, former President of the American Autonomic Society, and former chairman of the Autonomic Section of the American Academy of Neurology. Editor-in-Chief of Autonomic Neuroscience: Basic and Clinical and on the editorial boards of The Clinical Journal of Pain, Pain: Clinical Updates, and Clinical Autonomic Research. Serial founder of several companies in pain and neurodegenerative disease and is on the scientific advisory boards of many large and small pharmaceutical and biotechnology companies. Dr. Paul Grint, MD 20+ years experience in biologics and small-molecule research and development, including the successful approval and commercialization of products in the infectious diseases, immunology, and oncology therapeutic areas. Director of Amplyx Pharmaceuticals and Synedgen. Served in senior management roles at Cerexa, Forest Laboratories, Kalypsys, Pfizer, IDEC Pharmaceuticals, and Schering-Plough Corporation. Fellow of the Royal College of Pathologists and a medical degree from St. Bartholomew’s Hospital College, University of London. Ms. Elizabeth O’Farrell 25-year career with Eli Lilly and Company, lastly serving as Chief Procurement Officer and Leader, Global Head of Shared Services Served in senior management at Lilly including Senior Vice President, Policy and Finance; Senior Vice President, Finance; Chief Financial Officer, Lilly USA; Chief Financial Officer, Lilly Canada; and General Auditor. Before joining Eli Lilly, Ms. Director of PDL BioPharma, Geron Corporation and Lensar BS in accounting with honors and an MBA in management information systems from Indiana University. Robert Hauser, MD Professor of Neurology, University of South Florida College of Medicine - Director USF Parkinson’s Disease and Movement Disorders Center Jeffrey Kordower, PhD Alla V and Solomon Jesmer Professor of Aging & Neurological Sciences Rush University Medical Center Dr. Ken Marek President and Senior Scientist, Institute of Neurodegenerative Disorders Dr. Ted Dawson, MD, PhD Neurodegeneration and Stem Cell Programs, Institute for Cell Engineering, Departments of Neurology, Physiology, Pharmacology, and Molecular Sciences - The Johns Hopkins University School of Medicine Dr. Valina Dawson, PhD Neurodegeneration and Stem Cell Programs, Institute for Cell Engineering, Departments of Neurology and Physiology The Johns Hopkins University School of Medicine Dr. Warren Olanow, MD, FRCPC Henry P. and Georgette Goldschmidt Professor and Chairman Emeritus, Mount Sinai School of Medicine Clintrex, Inc. Dr. Karl Kieburtz, MD, MPH Robert J. Joynt Professor in Neurology, Senior Associate Dean for Clinical Research, Director of the Clinical &Translational Science Institute, Founder Center for Human Experimental Therapeutics (CHET)- University of Rochester Medical Center Clintrex, Inc. Dr. Jay Pasricha, MBBS, MD Director, Johns Hopkins Center for Neurogastroenterology Professor of Medicine
Proof of the importance of c-Abl in Disease, Target Engagement and Functional Reversal in the Brain and Gut IKT Appendix
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 aggregate Synuclein aggregate with c-Abl deleted Injection (Inj) of an expression vector for the A53T form of synuclein doesn’t degrade neurons until the expression of A53T is turned on Once turned on, A53T induces 50% neurodegeneration in 6 months. NOTE loss is visible in the dark patches of stained neurons on the right half. The left half was an internal control and is unaffected. When the expression of A53T is turned on, but c-Abl is genetically deleted from the mouse brain, you don’t get any neurodegeneration. Thus, even though the clumps of a-syn are present in the right region of the brain, they don’t cause disease until c-Abl acts on them No c-Abl = No neurodegeneration **: p < 0.0001, p = 0.0154, p = 0.0001, p = 0.0225, left-to-right
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)
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 Onset of Conditions Near Normal Behavior Returned Following Treatment Mice completed 30 turns/10 min when treated a dose of 100 mg/kg dose NEARLY COMPLETE RESCUE Defect in one hemisphere makes mice run in circles Oral IkT-148009 in Mice Humanized for Parkinson’s Disease in Brain Reverses Functional Loss ANIMAL STUDY BASIS OF IkT-148009 THERAPY
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 Activated c-Abl in disease Inhibition of c-Abl below baseline by IkT-148009 Baseline
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 for functional recovery
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 (Control) IkT – 148009 A53T Human α-Synuclein NOTES If it weren’t for 6 outliers in 96 measures, average would be 170 min, essentially 100% recovery. We find the neuroanatomy is nearly completely restored by measuring the number and distribution of neural ganglia in the tissue. Oral IkT-148009 in Mice Humanized for Parkinson’s Disease in Gut Reverses Functional Loss EVIDENCE OF FUNCTIONAL RECOVERY IN THE GUT At 3 months defecation slows to 500 min (3x normal); At 6 months defecation slows to 800 min (5x normal); Normal = 165 min
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 in the brain Aggregates Cleared during treatment with IkT-148009
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 neurodegeneration RESEARCH ADVANCES ARE TRANSFORMATIONAL Advances in pre-clinical models and clinical dosing