TauRx Therapeutics Ltd has announced the initiation of two global Phase 3 clinical trials in mild to moderate Alzheimer’s disease.
These landmark studies could provide the first definitive data on a Tau-based approach to disease-modifying and preventative treatment of Alzheimer’s for at least the next 5-7 years, said Professor Claude Wischik, Chairman of TauRx and Professor of Old Age Psychiatry at the University of Aberdeen, in a press conference at the 5th Clinical Trials Conference on Alzheimer’s Disease (CTAD) in Monte Carlo, Monaco.
The studies culminate three decades of research by Professor Wischik and colleagues, including the original discovery of the Tau protein as the main constituent of the Tangle pathology of Alzheimer’s disease [‘Tau tangles’], the development of the first Tau Aggregation Inhibitor (TAI), and results from an earlier Phase 2 clinical trial involving more than 300 patients that showed a 90% reduction in the rate of disease progression over two years.
“These Phase 3 studies are bringing us closer to finding an effective treatment that can actually arrest the progression of the disease,” said Professor Wischik.
Professor Wischik continued, “We are building on over thirty years of research, and the encouraging results from our previous Phase 2 clinical trial in Alzheimer’s disease support an approach which targets the abnormal Tau aggregates in the brain.”
The studies, which have already starting enrolling in the U.S., aim to confirm the disease-modifying effects seen in the Phase 2 studies in mild to moderate patients over an 18-month timeframe.
The first study will involve 833 people with mild to moderate Alzheimer’s disease over 12 months. The second study will include 500 people with mild Alzheimer’s disease over 18 months.
The study drug, LMTXTM, is a second-generation TAI that targets the Tau tangles and their precursors, dissolving them in order to halt their harmful effects on memory.
LMTXTM also works on the early stage Tau aggregates (called ‘oligomers’) which are precursors to fully-formed tangles and are thought to be particularly toxic.
“Clinicians devoted to Alzheimer’s disease have been waiting for a promising agent with disease-modifying properties,” said Professor Serge Gauthier of the McGill Centre for Studies in Aging, Quebec, Canada.
“The basic science data for this agent, particularly in the tauopathies, looks sound and the interest among investigators and among families is high.” Professor Gauthier is a clinical investigator and scientific advisor for TauRx.
The tangles in the brain were first reported by Dr. Alois Alzheimer in 1907,8 starting the century-long journey to understand the pathology leading to their formation, their role in dementia, and, ultimately, how to stop their spread through the brain.
Professor Lon Schneider, MD, of the Keck School of Medicine at the University of Southern California, a scientific advisor for TauRx, said: “Successfully targeting Tau may be an important approach towards slowing and ideally halting the neuro-degeneration that is characteristic of Alzheimer’s disease or frontotemporal dementia. Clinicians need these Phase 3 studies to produce clear evidence that such an approach could lead to improved patient outcomes.”