SAN CARLOS, Calif.– AcureX Therapeutics Corporation, a privately-held preclinical-stage biopharmaceutical company focused on developing potential disease altering therapeutics for neurodegenerative diseases, today announced that it was selected to receive a grant from The Michael J. Fox Foundation to accelerate the development of AcureX’s biomarker for use in Parkinson’s disease drug development and clinical trials.
Parkinson’s disease (PD) is a debilitating neurodegenerative disorder characterized by loss of dopaminergic (DA) neurons in the substantia nigra pars compacta. The causes of PD are largely unknown and the subject of intense investigation. However, a large body of evidence now supports that mitophagy, the normal process by which cells dispose of dysfunctional mitochondria, plays an important and early role in the pathogenesis of Parkinson’s disease. AcureX’s co-founder Prof. Xinnan Wang in Stanford University’s neurosurgery department discovered that the mitochondrial protein Miro1 functions as a privileged gatekeeper controlling the start of mitophagy. Further, Miro1 fails to be degraded resulting in mitophagy defects in PD patients of both genetic and sporadic forms. Recent studies have also shown that asymptomatic children of PD parents with known genetic risk factors display a defect in Miro1 degradation. Together, these data suggest that the presence of a Miro1 defect will serve as an important early diagnostic and prognostic biomarker of PD to accelerate drug discovery, drug development and clinical trials.
“We are grateful and proud to have the continued support of the Michael J. Fox Foundation to further develop AcureX’s biomarker for Parkinson’s disease,” said William D. Shrader, Ph.D., co-founder and CEO/CSO of AcureX Therapeutics. “MJFF has been a supporter of AcureX therapeutic programs, and their support of this biomarker will enable longitudinal studies to better understand disease progression and enable the identification and treatment of PD patients at the earliest possible time, significantly increasing the chances of therapies improving the quality of patients’ lives.”
Therapies that slow PD disease progression will benefit significantly from having a clinical biomarker to monitor target engagement and disease progression to guide drug development and de-risk clinical trials. “As we support therapeutic development — including from AcureX — toward new treatments and cures for Parkinson’s disease, we also pursue critical tools to select trial participants and to understand therapeutic impact quickly and objectively. Miro1 is a promising drug discovery tool and biomarker candidate, and the outcomes of this project to measure this pathway could speed new treatments and improve patient care,” said Brian Fiske, PhD, MJFF Chief Scientific Officer.
