Creating Disruptive Therapies

for Neurodegenerative Diseases

About Us

Engineering Therapeutics to Treat Diseases Resulting from Abelson Tyrosine Kinase Dysfunction

ABLi Therapeutics takes aim at neurodegenerative disease by targeting the non-receptor Abelson Tyrosine Kinases (Abl kinases or c-Abl) with small molecule inhibitors to intervene where disease begins.

Our proprietary Re-engineering Approach with Metabolism Preserved (RAMP™) drug innovation engine utilizes clinically validated data of kinase inhibitors to design and develop novel product candidates with enhanced penetration into the brain, with potentially higher potency and improved safety to treat diseases in which kinase activation is implicated.

A sequence of events: A pill. A plus sign. An image of a gear with icons inside of it: a lightning bolt, a wrench, molecules, and bonded molecules. A plus sign with a lightning bolt. Four pills.

Our Focus

Impacting Patient Lives by Treating Disease Where it Begins

We are engineering Abl kinase inhibitors to treat Parkinson’s disease, Parkinson’s-related neurodegenerative diseases and other neurological diseases associated with Abl kinase dysfunction inside and outside of the brain. These protein kinases have been shown to play a critical role in monitoring insults to brain neurons and regulating biological pathways that are associated with neurodegeneration. Recent research has demonstrated that Abl kinases play a central role in Parkinson’s disease initiation and progression.

Watch to learn more about our disease modifying strategy for Parkinson’s disease.

Pipeline

Our RAMP™ drug innovation engine facilitates the development of more selective Abl inhibitors with greater potency and safety than commercially marketed Abl kinase inhibitors used as anti-cancer agents. The c-Abl inhibitors that have emerged from our development can be administered chronically and systemically.

Pipeline
Drug Target Drug Candidate Modality Disease Indication Preclinical Development Phase 1/1b Phase 2 Phase 3
Clinical Programs

c-Abl

Risvodetinib

Small molecule

Parkinson’s Disease Treatment Naive

Preclinical Development Phase complete
Phase 1/1b Phase complete
Phase 2 Phase complete
Phase 3 Phase in progress

c-Abl

Risvodetinib

Small molecule

Multiple System Atrophy (orphan indication)

Preclinical Development Phase complete
Phase 1/1b Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started

c-Abl

Risvodetinib

Small molecule

Dementia with Lewy Body

Preclinical Development Phase complete
Phase 1/1b Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Pre-Clinical

c-Abl

ABLi-0901

Small molecule

PD, DLB, MSA

Preclinical Development Phase in progress
Phase 1/1b Phase not started
Phase 2 Phase not started
Phase 3 Phase not started

c-Abl

ABLi-04BP

Small molecule

PD, DLB, MSA

Preclinical Development Phase in progress
Phase 1/1b Phase not started
Phase 2 Phase not started
Phase 3 Phase not started

c-Abl

ABLi-Ab01

Monoclonal antibody

PD, DLB, MSA

Preclinical Development Phase in progress
Phase 1/1b Phase not started
Phase 2 Phase not started
Phase 3 Phase not started

Clinical Trials

Novel treatments that are inspiring hope

Find out more about our current and planned trials below.

COMPLETED: Risvodetinib (ABLi-148009)-101 ("the 101 Trial")

This Phase 1/1b study investigated the safety and tolerability of the drug Risvodetinib (ABLi-148009) in healthy elderly volunteers (55 to 70 years old) and in Parkinson’s disease patients.

Learn more about the 101 Trial

COMPLETED: Risvodetinib (ABLi-148009)-201 (“the 201 Trial”) in Parkinson’s disease

This Phase 2 study is investigating the safety and tolerability of Risvodetinib (ABLi-148009) at three doses for 12 weeks in 120 untreated Parkinson’s disease patients who do not currently take any medications to control their disease. Thirty-two sites in the U.S. are participating in this study. The study is expected to be extended for an additional 12 months following the 12-week double-blinded period.

Learn more about the 201 Trial

PLANNED: Risvodetinib (ABLi-148009)-202 (“the 202 Trial”) in Multiple Systems Atrophy

This is a Phase 2/3, double-blind, multi-center, randomized, placebo controlled trial of two Risvodetinib doses in participants with MSA designed to assess Risvodetinib safety, tolerability, PK, and efficacy on disease progression. Secondary and exploratory endpoints will evaluate the change in additional clinical features and biomarkers of disease severity and/or progression in the presence or absence of Risvodetinib. It will be conducted in up to 35 sites across the European Union (EU) and the United States (US).

PLANNED: Risvodetinib (ABLi-148009)-OL in Parkinson’s disease

This is an Open Label study that plans to enroll the participants in the 201 Trial into a 12-month open label study with random assignment to one of two doses. All 201 Trial participants that continue to meet trial criteria will be eligible, irrespective of whether they have initiated symptomatic therapy. The OL Trial will evaluate relative changes in individual functional assesssments in motor and non-motor function and perform detailed analysis of pathological synuclein status using mandatory biomarker assessments using skin biopsy and possible CSF collection. It will be conducted in up to 32 sites across the European Union (EU) and the United States (US).

PLANNED: Risvodetinib (ABLi-148009)-301 in untreated Parkinson’s disease

This is two-dose, placebo-controlled double blind study that will evaluate the efficacy of risvodetinib to improve on measures of the Activities of Daily Living, gastrointestinal function and motor function that plans to enroll more than 300 patients with untreated Parkinson’s disease less than 6 years from diagnosis in up to 60 global sites worldwide. In addition to a primary efficacy endpoint focused on the Activities of Daily Living, the 301 trial will evaluate treatment benefit in the central, autonomic and enteric nervous systems and look to establish whether risvodetinib can drive clearance of pathological alpha-synuclein from the body. The potential for recovery of dopamine signaling is also going to be assessed. Finally, a direct measure of altered progression of disease will be conducted using time-to-event analysis based on an agreed upon strategy with regulators.

Team

Rooted in science and expertise, ABLi Therapeutic’s leadership is driving our innovative approaches forward to make a difference in the lives of patients.

Management Team

Formerly, Founder and CEO of Inhibikase Therapeutics, Serial innovator developing c-Abl inhibitors for non-oncology applications in Infections Disease, Neurodegenerative Disease and Cardiopulmonary disease. Raised $345 million in committed capital and more than $24 million in NIH and DoD contract and grant support, building Inhibikase to a $600M+ market value (fully diluted basis) as a public company on Nasdaq. Internationally recognized scientist and thought leader in neurodegenerative disease and mechanisms of human disease. Previously rebuilt Celtaxsys, an immunology company from founding to Series B financing. Prior to entering the industry, Dr. Werner was a Head of Lab at the Rockefeller University where he led a 35 person team with substantial NIH grant support. Dr. Werner completed his PhD in Physical Chemistry and Structural Biology at the University of California, Berkeley and then trained at the National Institutes of Health in Bethesda, MD.

Previously served as CFO and VP of Finance for a Nasdaq-listed public company. Previously Vice President of Finance. For an international public company. Prior to his corporate work, spent 16 years in public practice mostly with Ernst & Young, lastly as a Senior Manager. He is a licensed Certified Public Accountant in the State of Massachusetts and a licensed Chartered Accountant of Australia and New Zealand.

Karl Kieburtz was the founding Director as Director of the Center of Human Experimental Therapeutics (CHET), and served as the Director of the Clinical and Translational Science Institute and Senior Associate Dean for Clinical Research at the University of Rochester. He is director of the clinical coordinating center for the PPMI program sponsored by the Michael J Fox Foundation and CEO of CLINTREX® since its inception in 2008. Dr. Kieburtz served as the Chair of the FDA Peripheral and Central Nervous System Advisory Committee. He has been PI for more than 50 multi-center NIH, industry, and Foundation sponsored clinical trials including the large NIHsponsored multi-center NET-PD study.

Scientific Advisors

Professor of Neurology, University of South Florida College of Medicine - Director USF Parkinson’s Disease and Movement Disorders Center

Dr. Robert Hauser is a Professor of Neurology at the University of South Florida College of Medicine, in Tampa, Florida. He serves as Director of the USF Parkinson’s Disease and Movement Disorders Center, a Parkinson Foundation Center of Excellence. Dr. Hauser earned a medical degree from Temple University School of Medicine in Philadelphia, Pennsylvania, and completed neurology training at the Eastern Virginia Graduate School of Medicine, in Norfolk, Virginia.

Dr. Hauser completed a fellowship in Movement Disorders at the University of South Florida and became Center Director in 1994. Dr. Hauser has authored or co-authored more than 300 peer-reviewed publications and is one of the world’s most cited Parkinson’s Disease investigators. He is Past Chairman of the Interventional Neurology Section of the American Academy of Neurology, has served on the executive committee of the Parkinson Study Group, and was a member of the steering committee for the NIH-sponsored Neuroprotective Exploratory Trials in Parkinson’s Disease program (NET-PD). Dr. Hauser lectures frequently at scientific meetings and served as Chairman of the 2009 World Federation of Neurology International Congress on Parkinson’s Disease and Related Disorders.

He has extensive expertise in clinical trial design and execution. The outcome measures that he developed have become the gold standard for use in clinical trials. He maintains an active patient practice and has been voted a Top Doctor by his peers every year since 1993. His primary research interest is the development of new medical and surgical treatments for Parkinson’s disease and other movement disorders.

Founding Director, ASU-Banner Neurodegenerative Disease Research Center (NDRC) The Charlene and J. Orin Edson Distinguished Director at the Biodesign Institute Professor of Life Sciences Arizona State University

He is an international authority in the area of movement disorders, which special expertise in experimental therapeutics and pathogenesis in movement disorders. Dr. Kordower has been ranked 29th in Parkinson’s disease expertise worldwide. He has performed numerous gene and cell therapy preclinical studies that have been translated into clinical trials. He has published landmark papers in the area of cell replacement strategies, including the first demonstration that fetal dopaminergic grafts can survive, innervate, and form synapses in patients with Parkinson’s disease (NEJM). Furthermore, he demonstrated that long-term grafts in such patients can form Lewy bodies (Nature Medicine). He has co-authored a paper in Nature demonstrating that human dopaminergic stem cells can survive and function in parkinsonian mice, rats, and monkeys. With regard to gene therapy, he published the lead article in Science demonstrating that gene delivery GDNF can prevent the emergence of motor symptoms and prevents nigrostriatal degeneration in nonhuman primate models of Parkinson’s disease. Dr. Kordower was also the first to demonstrate that gene delivery of CNTF can obviate neurodegenerative processes in a nonhuman primate model of Huntington’s disease. Dr. Kordower has published more than 350 manuscripts and chapters, 14 of which are citation classics. He has lectured all over the world and has served on more than 20 journal editorial boards (Sections Head and Associate Editor on two, including Movement Disorders). He has also served on the program committee for the World Parkinson’s Congress, is a Past-President of ASNTR, and is both a founding SAB member and two-time Executive Committee member of The Michael J. Fox Foundation. Dr. Kordower received his Bachelor of Arts, Master of Arts, and Doctor of Philosophy from the City University of New York (CUNY). He was awarded an Honorary Doctor of Science degree from CUNY in 2004.

President and Senior Scientist, Institute of Neurodegenerative Disorders

Kenneth Marek is President and senior scientist at the Institute for Neurodegenerative Disorders. Dr. Marek’s major research interests include the identification of biomarkers for early detection, assessment of disease progression, and development of new treatments for Parkinson’s disease and Alzheimer’s disease and related neurodegenerative disorders. His specific interest has been in in vivo neuroreceptor imaging biomarkers. He has authored numerous neurology and neuroscience publications on these topics. Dr. Marek is the principal investigator of several ongoing multi-center international studies, including the Parkinson’s Progression Marker Initiative (PPMI), the Parkinson Associated Risk Syndrome (PARS) study, and Pathways to Prevention (P2P). Dr. Marek serves on the scientific advisory board of The Michael J. Fox Foundation and is a special advisor to the foundation. He also was a co-founder of Molecular NeuroImaging, LLC, a company providing discovery and clinical neuroimaging research services. He received a Bachelor of Arts in Biochemistry from Princeton University and a Doctor of Medicine from Yale University.

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 CEO, Clintrex Research Corporation

Karl Kieburtz MD MPH is a neurologist and clinical researcher. After an undergraduate degree in Neuroscience at Amherst College, he completed MD and MPH degrees, and neurology residency, at the University of Rochester. He was the initial Robert J Joynt Professor in the Department of Neurology, and is currently Professor of Neurology, at the University of Rochester. He was the founding Director of the Center of Health & Technology (CHET), and served as the Director of the Clinical and Translational Science Institute and Senior Associate Dean for Clinical Research at the University of Rochester. Dr Kieburtz was the past Chair of the Parkinson Study Group Executive Committee, and a past member of the Huntington Study Group Executive Committee. He has been global Principal Investigator for more than 50 multi-center and multi-national NIH, industry, and Foundation sponsored clinical trials, including the large NIH-sponsored multi-center NET-PD study. He was elected as a Fellow in the American Association for the Advancement of Sciences in 2014. He co-founded Clintrex Research Corporation in 2008, providing scientific and regulatory advisory services to companies developing CNS therapies. He serves on the scientific advisory boards of biotech and pharma companies, as well as for emerging therapeutic and data technology companies.

Associate Professor of neurology at Mayo Clinic Rochester and Director of the Autonomic Laboratories with interests in autonomic disorders and diseases caused by abnormal synuclein proteins.

Head of the Department of Neurology for Neurodegenerative Diseases at the University Hospital Bordeaux, Co-Chair of the French Reference Center for Multiple System Atrophy (MSA) and Deputy Director of the Institute for Neurodegenerative Diseases at the University Bordeaux.

News

Read our most recent press releases and company updates.

View All Press Releases

Contact Us

For more information regarding our company, partnerships, or drug candidate(s), please reach out to us info@ablitherapeutics.com.

Investor Relations

Mike Moyer, Life Science Advisors, mmoyer@lifesciadvisors.com, +1-617-308-4306