Phase 3 Clinical Trial Begins in Early Form of Dementia

AsiaNet 50575

MANCHESTER, England, Sept. 10 / PRN=KYODO JBN/ --

         - Investigational drug study follows earlier study with

         promising results in mild to moderate Alzheimer's patients

     TauRx Therapeutics today announced the initiation of a global Phase 3

clinical trial in a type of Frontotemporal Dementia (FTD) also known as Pick's

Disease. The announcement, which immediately follows The 8th International

Conference on Frontotemporal Dementias, held 5-7 September in Manchester,

England, underscores the need for new treatments for this form of dementia that

is similar to Alzheimer's Disease, except that it tends to damage different

areas of the brain and affects people as early as 40 years old.

     The study focuses on a type of FTD known as behavioural-variant, or bvFTD,

which can cause early changes in personality and loss of empathy. A large

percentage of these patients have a specific pathology that involves abnormal

collections of tau protein in the brain.

     The study drug, LMTX(R), targets a process in the brain whereby a normal

form of tau protein begins to self-aggregate due to binding neuronal

waste-products. Once the process has started, the aggregates are able to

propagate themselves indefinitely, using up normal tau protein and converting

it into the toxic aggregates. After destroying the nerve cells where they are

initially formed, the aggregates go on to infect nearby healthy neurons,

progressively spreading and accelerating the destruction throughout the brain.

LMTX(R) stops this aggregation process in its tracks and releases the trapped

tau protein in a form which can be easily cleared by nerve cells.

     In a pilot series of cases, LMTX(R) was found to arrest the progression of

the disease. LMTX(R) has been found to act in a similar way on the aggregation

of TDP-43 protein. Tau or TDP-43 aggregates each account for about 50% of

patients with this early form of dementia.

     Speaking to patients and caregivers at the FTD conference in Manchester,

Professor Bradley Boeve of the Mayo Clinic in the U.S., 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 3 double-blind placebo-controlled study is designed to evaluate

the safety and efficacy of LMTX(R) 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 timeframe. Participating study sites are located in Canada,

U.S., U.K., Germany, The Netherlands, Australia and Singapore. Because the

condition is relatively rare, TauRx was granted Orphan Designation for LMTX(R)

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 2

clinical trial in Alzheimer's Disease, which is also correlated with abnormal

tau aggregates in the brain."

     TauRx previously tested rember(R), the first-generation TAI on which LMTX(R)

is based, in a Phase 2 clinical trial involving 321 patients with mild and

moderate Alzheimer's Disease in the UK and Singapore. This study found a 90%

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.

     If successful, this will be the first investigational drug that is able to

arrest the progression of this disease. TauRx Therapeutics, a Singapore-based

company spun out of the University of Aberdeen, developed the novel treatment

based on an entirely new approach which targets aggregates of abnormal fibres

of tau protein that form inside nerve cells in the brain. The TauRx team have

since discovered that LMTX(R) could also have beneficial effects on other

proteins which aggregate abnormally, including TDP-43 in FTD and synuclein in

Parkinson's disease. In FTD, the frontal and temporal lobes are affected first,

which impacts behaviour and emotion. As the disease progresses, other parts of

the brain are affected, eventually producing a global dementia.

     Patients and caregivers are invited to sign up for future updates as more

news is available at http://www.PicksDementiaStudy.info.

     About Pick's Disease:

     According to the Association for Frontotemporal Degeneration (AFTD), bvFTD

- or Pick's disease as it was originally known - can cause early and

progressive changes in personality, emotional 'blunting' and loss of empathy. A

person with the disorder may have difficulty controlling their behaviour, which

can result in socially inappropriate responses or actions. Language may also be

impaired after behavioural changes take place, as well as neurological symptoms

such as movement and coordination difficulties. Over time, these symptoms

worsen. The bvFTD form of the disease is particularly aggressive and progresses

faster than Alzheimer's disease.

     About TauRx:

     TauRx Therapeutics was established in Singapore in 2002 with the aim of

developing new treatments and diagnostics for a range of neurodegenerative

diseases based on its technology platform. The TauRx team includes highly

skilled and internationally recognised pharmaceutical experts in drug discovery

and development. The company's Tau Aggregation Inhibitors (TAIs) include

rember(R) and LMTX(R), the second-generation drug that is being studied in

Phase 3 clinical trials in Alzheimer's and FTD. TauRx is headquartered in

Singapore with primary research facilities in Aberdeen, Scotland.

     SOURCE: TauRx Therapeutics