The company’s hydromethylthionine mesylate therapy reduced neurodegeneration during the pivotal trial.
TauRx Pharmaceuticals – a company focused on Alzheimer’s disease (AD) research – has announced results from its pivotal phase 3 LUCIDITY trial.
The study measured the effect of hydromethylthionine mesylate (HMTM) on neurofilament light change (NfL) – a vital brain neurodegeneration biomarker.
Blood concentration of NfL demonstrated a significant 93% reduction in change over the course of a year among individuals receiving HMTM at a dose of 16mg/day, in contrast to a control group, which linked notably with a tau biomarker.
Tau proteins and neurofilaments are important for neuronal structure and functionality within the brain. In AD, tau protein collects to produce toxic fibrils, which in turn damage neurons and sees NfL leaked into the bloodstream.
NfL concentration in blood is known to correlate with tau pathology and, therefore, cognitive decline and brain atrophy in AD. Consequently, this indicates NfL is a reliable method to measure the extent of neuronal damage caused by AD.
The new data has emerged following previously shared LUCIDITY topline results. This showed HMTM led to a sustained improvement in cognition.
Claude Wischik, executive chairman at TauRx, elaborated: “The NfL results demonstrate that a drug targeting tau pathology reduces the neurodegeneration which underlies the clinical decline in AD. They bring us a step closer to offering an effective new treatment option for people with AD.”
He added: “Because it is taken as a tablet and has a strong safety profile, HMTM would be readily accessible to people needing a disease-modifying treatment.”
Henrik Zetterberg, professor of neurochemistry at UCL Queen Square Institute of Neurology, concluded: “NfL is a well-studied biomarker with wide applicability to different neurological disorders, including AD.
“Clinical practice has been waiting decades to uncover meaningful advancements to address unmet needs of people with AD. These new results further support the importance of NfL as an AD biomarker both for diagnosis and measurement of treatment effect.”
TauRx now intends to submit the HMTM results from LUCIDITY and previous trials for regulatory approval in the US, UK and several other territories.