Last 5th of October , Noscira announced that it has completed recruitment for
Phase II clinical trial with its drug tideglusib (Zentylor) in patients with
Progressive Supranuclear Palsy (PSP). This is an important milestone as it
indicates that the trial is progressing on schedule, particularly after the US
Food and Drug Administration (FDA) recently granted tideglusib Fast Track
designation for this disease (Fast track status is granted to facilitate
development and expedite the review of a drug for a serious or potentially
fatal illness and to meet an unmet medical need).
PSP is a deadly fast-progressing degenerative disease of the brain. Clinical
symptoms include frequent falls, paralysis of vertical eyes movement, akinetic
rigid syndrome, bulbar dysfunction and frontal subcortical dementia. Average
survival is 5-6 years from onset of symptoms. This rare disease affects 6 out
of every 100,000 people.
One of its causes is the overespression of GSK-3 (Glycogen Synthase Kinase-3)
that leads to hyperphosphorylation of the tau protein. Tau proteins are
proteins that stabilize microtubules. Its hyperphosphorylation (tau
inclusions, pTau) can result in the self-assembly of tangles of paired helical
filaments and straight filaments. This anomaly occurs in a number of
neurodegenerative diseases known collectively as taupathies, which include
Alzheimer's disease (AD), PSP and pick disease.
This clinical trail, with the name of TAUROS (TAU Restoration On PSP),
investigates the drug tideglusib , a GSK-3 inhibitor with oral bioavailability,
administered orally in daily doses for a year, as a treatment for PSP. The trial
involves 146 patients at 24 centres in Europe and the US.
Currently, Tideglusib is the only GSK-3 inhibitor in clinical development for
PSP. Prior to receiving Fast track designation from the FDA , tideglusib
obtained orphan designation for PSP from both the US and EU authorities in
2009.
Noscira is already working with tudeglusib (Nypta) in phase II clinical trail on
Alzheimer's disease after analysing the promising results from the preceding
Phase IIa trial, which were presented at the International Conference on
Alzheimer's disease in July 2010. Tieglusib is also the only GSK-3 inhibitor
under clinical development for Alzheimer's disease and it has proven to be
capable of acting on several of the histopathological lesions associated with
the disease in experimental models: it reduces phosphorylation of the tau
protein and hippocampal and entorhinal cortex neuron loss, improves spatial
memory deficits and significantly reduces the accumulation of amyloid plaques
in the brain. It also provides neuroprotection in vivo and has a potent
anti-inflammatory effect in a range of animal models.