The biopharmaceutical company Noscira* is conducting a clinical trial of a new drug, NP-12**, as a potential treatment for Progressive Supranuclear Palsy. NP-12 is also being tested in trials for Alzheimer's disease.
Previously, NP-12 has been tested on 160 healthy people, both young and old, in Phase I studies, and was found to be well tolerated. In addition, a study with 30 Alzheimer Disease patients dosed for 20 weeks has provided safety and tolerability data in patients.
In animal studies (using transgenic mice) NP-12 has been found to improve cognitive performance and to reduce amyloid deposits, hyperphosphorylation and tau aggregation, neuro-inflammation, and nerve cell loss - all of which are factors associated with Alzheimer's disease.
NP-12 is the only compound reported to date that is capable of acting on all of the histopathological lesions associated with Alzheimer's disease. That NP-12 has been shown to affect tau phosphorylation and aggregation, a feature of PSP as well as Alzheimer's disease, is the reason for thinking it may also have potential in the treatment of PSP.
The NP-12 PSP Study
Official title: A double-blind, placebo-controlled, randomized, parallel-group study evaluating the safety, tolerability, and efficacy of two different oral doses of NP031112, a GSK-3 inhibitor, versus placebo in the treatment of patients with mild to moderate Progressive Supranuclear Palsy.
Purpose: To determine if NP-12 is safe and effective in the treatment of mild to moderate PSP.
The study: Participants in the study will be randomized to receive 600mg NP-12, 800mg NP-12, or a placebo for 52 weeks.
Study completion date: April 2011
Participating centres: In the USA, Germany, Spain and UK. In the UK, four Centres, based in Cardiff, Liverpool, Newcastle and London are participating.
Around 6 patients are planned to be recruited to the study at each of the participating centres.
Three of the four UK Centres (Liverpool, London and Newcastle) are not looking to recruit additional patients at present. The centre at Cardiff may open to recruit patients in the near future.
How is NP-12 expected to work?
PSP is thought to be caused by a protein called tau sticking together and forming neurofibrillary tangles. These tangles build up in brain cells, damaging different areas of the brain.NP-12 is expected to work in PSP and Alzheimer's disease by blocking an enzyme called glycogen synthase kinase-3β (GSK-3).
GSK-3 is one of the main factors that makes the tau protein become ‘sticky' forming the neurofibrillary tangles. By blocking GSK-3, NP-12 is expected to reduce the amount of tau protein sticking together, thereby reducing the damage to brain cells.
It is hoped that NP-12 will not have the problematic toxicity of lithium. Both lithium and NP-12 act on the enzyme GSK-3 but NP-12 is a more specific inhibitor of GSK-3. The recent safety and toxicity trial with lithium as a potential treatment for PSP was terminated because of lithium's adverse effects on patients.
NP-12 is the only GSK-3 inhibitor under clinical development.
Dr Angela Wilson, The PSP Association's Director of Medical Awareness and Research said:
The Noscira NP-12 trial is a really exciting step forward in our quest to find a much needed effective treatment for PSP. NP-12 is a new drug and on the basis of the work undertaken so far looks promising. The drug must be safe to give to patients and that is one of the aspects that the current trial involving more than 100 PSP patients at centres across the United States and Europe is setting out to establish. The trial is also designed to evaluate efficacy of the compound. This aspect of the work will take around a year. We are still some way from having an effective treatment for PSP but Noscira's investment in PSP research is a much welcomed development and will help move us closer to our goal."
*Noscira, a subsidiary of the Zeltia Group (Spain's leading biotechnology and chemical company) is a biopharmaceutical company devoted to the research and development of innovative drugs for the treatment and prevention of diseases of the nervous system. Since its formation in 2000, Noscira has focused its activity on Alzheimer's disease and has two clinical compounds (NP-12 and NP-61) in the pipeline for treating Alzheimer's disease. For more information, visit www.noscira.com
**NP-12 is the commonly used name for NP031112. The drug is also known as Nypta®. The active ingredient is tideglusib.
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