FAScinate Therapeutics Completes Part 1a of Phase 2 Study of Company's Parkinson’s Disease Drug Candidate "KM-819"

Company-Sponsored Phase 2 Study is Evaluating the Efficacy of "KM-819" in Halting and/or Slowing the Progression of Parkinson’s Disease

FAScinate Therapeutics, Inc.

FAScinate Therapeutics, Inc.

We are pleased with the prospects of KM-819 as a disease-modifying treatment for Parkinson’s disease as we progress through Phase 2 trials. Part 1a showed favorable safety even at very high doses, and we are excited to now initiate Part 1b. KM-819 shows great promise.


Clinical stage drug development company FAScinate Therapeutics, Inc. today announced the successful completion of Part 1a, the initial stage of Phase 2 studies, for the Company's Parkinson’s Disease drug candidate "KM-819," with the results showing a favorable safety profile. Part 1a involves a dose-escalation approach with KM-819 in healthy elderly subjects, designed to assess the pharmacokinetics and safety of the drug candidate at several different doses beyond the dose tested in Phase 1 trials.

The subsequent stage, Part 1b, will be a dose-finding study of KM-819 in Parkinson’s Disease patients where the pharmacokinetics and safety profile will be evaluated at three doses, including a high dose tested in healthy test subjects. The results will then be used to select the doses used for the main Part 2 study, a 24-month trial of the selected doses evaluating both the safety and efficacy endpoints of KM-819 on the progression of Parkinson’s Disease.

KM-819 is a potent inhibitor of FAF1, a key regulatory protein in cell death pathways, apoptosis and necrosis, and leads to neuronal cell protection in pre-clinical studies. Furthermore, high levels of FAF1 lead to the suppression of autophagy, resulting in the accumulation of alpha-synuclein, which often appears in Parkinson’s patients as Lewy bodies in dopaminergic neuron cells in the midbrain. Thus, FAF1 may be a significant target when it comes to the development of disease-modifying treatments for Parkinson’s Disease. In various preclinical cell and animal studies, KM-819 demonstrated protective properties for dopaminergic cells and the inhibition of alpha-synuclein accumulation in such cells.

The Phase 2 trial of KM-819 in Parkinson’s disease was approved by the FDA in November of 2021, and Part 1a was initiated in July of 2022. Parkinson’s Disease is a neurodegenerative disease and the second most common central nervous system (CNS) disease following Alzheimer's Disease, affecting more than a million people in the US. It disrupts normal movement and also causes numerous non-movement symptoms. It is known to be caused by the premature death of dopaminergic neuron cells in the midbrain. Currently, there are many drugs that address the symptoms of the disease, but effective disease-modifying therapies for stopping or slowing down the progression of Parkinson's Disease in patients are nonexistent. KM-819 is designed to address this pressing need.

FAScinate Therapeutics said, "We are pleased with the prospects of KM-819 as a disease-modifying treatment for Parkinson’s disease as we progress through Phase 2 trials. Part 1a showed favorable safety even at very high doses, and we are excited to now initiate Part 1b. KM-819 shows great promise when it comes to the neuroprotection of dopaminergic cells, including GBA mutation, as well as in clearing alpha-synuclein.”

About KM-819

KM-819 is a small molecule developed as an inhibitor for FAF1, a proapoptotic protein, targeting various degenerative diseases. It has shown superior neuroprotective efficacy in Parkinson’s Disease models, and clinical trials are underway with the aim of proving its efficacy in human patients. KM-819 may be also explored as a potential treatment for other degenerative diseases, including multiple system atrophy (MSA). It shows an excellent safety profile in both long-term animal toxicology studies and the human clinical trial Phase 1 study that has been completed.

About Parkinson’s Disease

Parkinson’s Disease is the second most common chronic CNS disease after Alzheimer’s Disease, estimated to afflict 6 to 10 million patients worldwide and 1 million in the United States. It is more common in elderly people, with 2-3% of those aged 65 and over now affected, and there are expectations of an increase in the number of patients as the population continues to age. The current standard of care is symptomatic treatments by supplementing dopamine or dopamine agonists or analogous mechanisms, so a disease modifying treatment is an urgent need in order to address the impact of Parkinson’s Disease.

About FAScinate Therapeutics Inc.

FAScinate Therapeutics Inc., a subsidiary of the Korean Biotech company Kainos Medicine Inc., is a clinical stage biotechnology company based in San Diego, California focused on the development of novel therapeutics for central nervous system diseases. FAScinate has licensed KM-819 for clinical development and commercialization from Kainos Medicine, and is developing it for phase 2 study in the US for Parkinson’s Disease. For more information, please visit http://www.fascinatetherapeutics.com

Forward-Looking Statements

This press release contains forward-looking statements. These statements relate to, among other things, the potential of KM-819 as a disease-modifying therapeutic stopping or slowing the progression of Parkinson’s disease. These statements are identified by their use of terms and phrases such as “will,” “potentially,” “suggest,” “targeting,” “expect,” “aiming,” “prove,” “estimated,” and other similar terms and phrases. These statements are based on estimates, assumptions, and projections that may not be proven to be accurate, and actual results could differ materially from those anticipated due to known and unknown risks, uncertainties, and other factors. FAScinate Therapeutics undertakes no obligations to update publicly any forward-looking statements contained in this press release as a result of new information, future events or changes in FAScinate Therapeutics’s expectations.