Phase 3 Clinical Trial Begins in Early Form of Dementia
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
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