TauRx Therapeutics has begun a Phase III trial of LMTX in a type of frontotemporal dementia (FTD) known as behavioural-variant, or bvFTD, which can cause early changes in personality and loss of empathy.
The double-blind study is designed to evaluate the safety and efficacy of LMTX, a second-generation tau aggregation inhibitor (TAI) in sites located in Canada, US, UK, Germany, the Netherlands, Australia and Singapore.
Mayo Clinic spokesperson and study investigator, Professor Bradley Boeve, said clinicians have been refining the measures to use in an experimental trial in FTD spectrum disorders for years.
"The basic science data for this agent, particularly in the tauopathies, looks sound and the excitement among investigators and among families is high," Boeve said.
The placebo-controlled study expects to confirm results observed in pilot cases in a larger controlled 52-week trial in bvFTD patients.
TauRx received orphan designation for LMTX in 2010, which provides a basis for rapid approval for marketing if the trial is successful.
TauRx Therapeutics founder and CEO, the University of Aberdeen Professor Claude Wischik said; "We are building on over thirty years of research, and the encouraging results from our previous Phase II clinical trial in Alzheimer's Disease, which is also correlated with abnormal tau aggregates in the brain."
LMTX targets a brain process where a normal form of tau protein undergoes self aggregation because of binding with neuronal waste-products.
The aggregates use normal tau protein to propagate themselves indefinitely and converting it into the toxic aggregates thereby destroying the nerve cells where they are primarily formed.
The aggregates spread progressively and accelerate the destruction throughout the brain. LMTX stops the aggregation process in its tracks and releases the trapped tau protein in a form which can be easily cleared by nerve cells.