Updated on 10 September 2012
Speaking to patients and caregivers at the FTD conference, held between September 5 and 7, in Manchester, UK, Professor Bradley Boeve of the Mayo Clinic in the US, one of the investigators of the study, said: "Clinicians devoted to FTD clinical trial development have been refining the measures to use in an experimental trial in FTD spectrum disorders for years, and frankly have been waiting for a promising agent. The basic science data for this agent, particularly in the tauopathies, looks sound and the excitement among investigators and among families is high."
The phase III double-blind placebo-controlled study is designed to evaluate the safety and efficacy of LMTX, the second-generation Tau Aggregation Inhibitor (TAI) developed by TauRx. The study aims to confirm the results first seen in the pilot cases in a larger controlled clinical trial in bvFTD patients over a 52-week time-frame. Participating study sites are located in Canada, US, UK, Germany, The Netherlands, Australia and Singapore. Because the condition is relatively rare, TauRx was granted orphan designation for LMTX in 2010, which provides a basis for more rapid approval for marketing if the trial is successful.
"This is an important step forward in our quest to find an effective treatment, with a goal to actually arrest the progression of the disease," said Professor Claude Wischik, founder and CEO of TauRx Therapeutics and Professor of Old Age Psychiatry at the University of Aberdeen. "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."
TauRx previously tested rember, the first-generation TAI on which LMTX is based, in a phase II clinical trial involving 321 patients with mild and moderate Alzheimer's Disease in the UK and Singapore. This study found a 90 percent reduction in the rate of disease progression over two years in Alzheimer's Disease. Professor Wischik and his team have spent nearly 24 years investigating the structure and role of Tau tangles in the development of Alzheimer's disease, FTD and other neurodegenerative diseases. They were the original discoverers of the Tau protein pathology of Alzheimer's.
"It's very exciting news that a treatment is being tested for FTD in a clinical trial," said Penelope Roques of the Frontotemporal Dementia Support Group in the UK. "This is encouraging progress in a disease where there is currently no treatment available." The group has about 1,000 members across the UK, ranging from FTD patients, caregivers and family members.