FDA Approval Sought to Begin Phase 2 Trial of KM-819
FAScinate Therapeutics has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 clinical trial evaluating KM-819 as a potential therapy for Parkinson’s disease, the company announced in a press release.
Parkinson’s disease is characterized by the death and dysfunction of neurons (nerve cells) that produce a chemical messenger called dopamine. KM-819 is an orally active molecule with the potential to be a disease-modifying treatment for Parkinson’s. It inhibits FAF1, a protein that promotes apoptosis, or regulated cell death.
Rather than working to alleviate the symptoms of dopaminergic neuronal death, KM-819 can prevent, by inhibiting FAF1, dopaminergic neurons from dying and potentially slow or stop Parkinson’s progression.
KM-819 also may have the potential to be used to treat other degenerative diseases, such as multiple system atrophy (MSA), another progressive neurodegenerative disorder.
In long-term preclinical studies KM-819 has shown superior effectiveness in neuroprotection in cell models and in dopaminergic neuron protection in the midbrain of animal models of Parkinson’s disease. The therapy also has been shown to improve behavioral tests.
In a previous Phase 1 clinical trial (NCT03022799) in healthy volunteers, the medication showed an excellent safety profile.
The trial was a placebo-controlled study divided into two parts, both of which used a dose-escalation protocol with KM-819 administered over the course of six weeks.
The proposed Phase 2 trial will assess KM-819’s safety, tolerability and pharmacokinetics — the way in which the medication is processed within the body — in healthy older adults and in Parkinson’s disease patients.
The study will be placebo-controlled and divided into multiple parts. Part 1 will focus on finding the appropriate dose in healthy individuals and individuals with Parkinson’s, while Part 2 will evaluate two ascending doses of KM-819 in patients with Parkinson’s disease.
FAScinate has licensed KM-819 from Kainos Medicine for clinical development and commercialization in the U.S. market as a therapy for Parkinson’s.