FAScinate Therapeutics, Inc. Cleared for Phase 2 Clinical Trials of Company’s Parkinson’s Disease Treatment with Approval from U.S. Food and Drug Administration

FDA Approves FAScinate’s Application for Phase 2 Trials with “KM-819” Treatment



Clinical stage drug development company FAScinate Therapeutics, Inc.today announced that its Investigational New Drug (IND) application for Phase 2 trials of its Parkinson’s Disease treatment “KM-819” has been cleared by the United States Food and Drug Administration (FDA). The trial design, as approved by the FDA, is intended to evaluate the disease-modifying efficacy of KM-819 in slowing or stopping the progression of Parkinson’s disease. The goal is to evaluate the pharmacokinetics and safety and preliminary efficacy in early to mid-stage patients, including GBA mutation carriers, with two-year treatments.

The approved trial design has a dose escalation stage where the pharmacokinetics and safety will be assessed at several different doses beyond the dose tested in Phase 1 trials, and will be followed by a 24-month trial of the selected doses with both safety and efficacy endpoints of KM-819 on the progression of Parkinson’s Disease.

Parkinson’s Disease is a neurodegenerative disease, and the second most common central nervous system (CNS) disease following Alzheimer’s Disease. It disrupts normal movement behaviors and also causes several non-movement symptoms. It is known to be caused by the premature death of dopaminergic neuron cells in the midbrain. Currently, there are many symptomatic treatment drugs for the disease but effective disease-modifying therapies for stopping or slowing down the progression are completely absent despite significant need.

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. A higher level of FAF1 is found in Parkinson’s Disease patients and could contribute to the early cell death. Thus, FAF1 may be a significant target when it comes to the development of disease modifying treatments for Parkinson’s Disease

FAScinate Therapeutics said, "We are excited about the prospect of KM-819 for Parkinson’s disease with Phase 2 trials looking for evidence of disease modifying effects. KM-819 shows a promising neuroprotection efficacy in dopaminergic cells including GBA mutation, as well as clearing the 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 a superior neuroprotective efficacy in Parkinson’s Disease models, and entered into clinical trials with the aim of proving efficacy in human patients. KM-819 may be also explored in other indications for different 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, next to 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 % 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 one of the major unmedical needs 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 moving it to 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.

FAScinate Therapeutics, Inc. Cleared for Phase 2 Clinical Trials of Company’s Parkinson’s Disease Treatment with Approval from U.S. Food and Drug Administration