Frequently Asked Questions (FAQ)
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Resources - Frequently Asked Questions
TauRx reported rember™ phase 2 results in 2008. What has the company been doing since then?
TauRx has made significant R&D, infrastructural and commercial advancements since 2008.
On the R&D side, TauRx has discovered and is developing alternative Tau Aggregation Inhibitors (TAIs) as second-generation forms of the same active moiety present in rember™. These have been chemically optimized to have enhanced tolerability and bioavailability, enabling their use at higher doses than rember™. TauRx’s TAIs are backed by a robust patent estate.
TauRx’s lead second-generation TAI, LMTX, is now under investigation in clinical trials conducted under an open US IND. The results of these trials and those of a range of supportive preclinical studies are so promising that LMTX has been prioritized as a replacement for rember™ in our forthcoming phase 3 clinical program. Under a development plan agreed with US and EU regulatory authorities, we are now preparing to evaluate LMTX in pivotal international phase 3 trials in mild and moderate AD and related neurodegenerative diseases.
Structurally, TauRx has enhanced its R&D and commercial expertise with numerous appointments including that of Dr Hans Moebius as Chief Medical Officer. Dr Moebius led the clinical development and regulatory strategy supporting the launch of Memantine, Merz’s blockbuster therapy for the treatment of Alzheimer’s disease symptoms licensed to Lundbeck in the EU and Forest in the US, and he has led the development of numerous other CNS therapies. Also, we will shortly announce the appointment of several internationally recognized scientists in the field of neurodegenerative diseases to our Scientific Advisory Board.
Lastly, but importantly, we have also conducted a preliminary clinical study using rember™ in patients diagnosed with Frontotemporal dementia (FTD) with promising results. FTD is one of several lethal neurodegenerative diseases characterised by the formation of Tau aggregates for which no treatments are available. A pivotal clinical trial is planned in FTD and in a second Tau aggregate related disease. Due to the classification of FTD and the additional diseases we are considering as orphan indications, provisions agreed by the US and European regulatory authorities permit fast-tracked regulatory reviews and marketing approvals in orphan diseases where disease prevalence is low and there is a compelling medical need.
More details are available on our relaunched website.
How big a breakthrough are TauRx’s drugs?
TauRx’s drug rember™, a Tau Aggregation Inhibitors (TAI), is a breakthrough in that the phase 2 clinical trial with this drug was the first evidence of a drug actually modifying the Alzheimer’s disease process itself (slowing down and halting it) rather than just treating the symptoms. Currently available drugs treat symptoms only by offering a temporary boost to mental function, whereas rember™ continues to halt decline in mental function for as long as the drug is taken. Because of what we know about the correlation of the accumulation of Tau Aggregates with the onset of Alzheimer’s disease, there is a strong potential that TAIs could actually stop or even prevent disease if given at an early enough point in the disease process.
How big a breakthrough would a Tau Directed diagnostic be?
A Tau directed diagnostic, such as TauRx is developing in collaboration with Bayer Schering Pharma, could allow the identification of individuals with very early forms of the disease, increasing the chance for early intervention with disease modifying or preventative drugs such as LMTX. More information about preventative use is given below and on the company website. A Tau directed diagnostic would also allow treatment to be monitored and adjusted more accurately in patients at all stages of the disease to optimise dose levels according to disease severity and drug response.
When was the Bayer Schering Pharma deal signed? How much is the deal worth?
The collaboration was signed in May 2010. TauRx will provide its proprietary technology platform of ligands, specialised Tau-directed assays and its animal models to optimise the development of ligands. Diagnostic products arising from the collaboration will be developed by Bayer Schering Pharma, whereas compounds with therapeutic potential will be developed by TauRx. The financial terms of the collaboration are confidential.
Who else are you speaking with about collaborations?
The management and shareholders of TauRx are committed to the objective of delivering our therapies to the market. We see distinct financial advantages for our shareholders in retaining product rights until later stages of development and commercialisation. In support of the further clinical development of LMTX, we have entered into an agreement with QCTR, a CNS specialist clinical research organisation.
How are TauRx’s drugs new/different to what is already available for Alzheimer’s?
Current therapies for Alzheimer’s do not affect the progress of the disease itself, but only provide a temporary boost to mental function, with no long-term benefit. TauRx’s rember™ trial was the first successful trial of a new approach, in which a drug acts on the abnormal tangles of Tau protein that form in the brain cells of people with Alzheimer’s, and disrupts and ‘dissolves’ them. More importantly our TAIs act on the early stage pre-tangle aggregates that are thought to have particular toxicity for nerve cells. To our knowledge, no company other than TauRx has a TAI in preclinical or clinical development, although there is an increasing research momentum by discovery groups following TauRx’s lead, particularly given the failures of the amyloid-based approaches to date. However, the long time-frame required for clinical trials in AD means that these programs are unlikely to reach the market until after 2020.
Could TAIs be the magic bullet/miracle cure for Alzheimer’s? When will the phase 3 trials begin?
The rember™ results from our phase 2 trial are exciting and encouraging, and we are now preparing to do a further and bigger trial to confirm these results in people with mild and moderate AD. We will then seek to make this drug available as soon as possible for people in the mild to moderate stage of the disease.
At the same time we are conducting more research to investigate the future potential of this approach and type of drug - to find out if we can actually reverse the disease at the milder stages of the disease. Then we want to ultimately prevent it from occurring in the first place. That is the ultimate goal of TauRx and would have enormous benefit for millions of families worldwide, in addition to huge cost savings for national economies and health services.
Does it work on everyone with Alzheimer's?
This trial tested the drug in over 300 people with mild or moderate Alzheimer's and found these encouraging results. We do not have any evidence that the drug could dissolve the tangles in the brain of a person with advanced Alzheimer's in which many nerve cells will already have been destroyed. This will form part of our ongoing research.
How do TauRx’s TAIs work?
The drugs appear to be able to disrupt and ‘dissolve’ the tangles of Tau protein which form abnormal ‘aggregates’ in the brain cells of people with Alzheimer’s and ultimately destroy the cells. More importantly our TAIs act on the early stage pre-tangle Tau aggregates (“oligomers”) that are thought to be particularly toxic to nerve cells. We know that these tangles form earliest and most severely in the areas of the brain that are most critical for memory, and we have seen from the brain scans of people exposed to our TAIs during our phase 2 trial that these are the very areas of the brain where rember™ appeared to have the greatest effect in protecting against ongoing loss of nerve function.
At what stage could people with Alzheimer’s take TAIs?
It is known that disease related changes to the Tau protein can appear as much as 20 years before the onset of symptoms. Given these timings and the action of TAIs as demonstrated in our research studies and clinical trials, we have strong hopes that further breakthroughs in the management of AD are possible. The combination of a diagnostic such as we are developing in collaboration with BSP with a disease modifying or preventative drug has the potential to identify susceptible individuals and prevent the development of AD. Our phase 2 clinical trial in patients with mild and moderate severity AD demonstrated potential utility in these patient populations (to be confirmed in the upcoming phase 3 trials). It is unclear if TAIs would have utility in patients with advanced disease wherein significant nerve cell damage and cell death had already occurred. All these categories of drug use are being studied at TauRx.
How did the people on the rember™ trial improve?
Every person is unique, and Alzheimer’s affects people in varying ways. However people in the rember™ trial reported that they felt more confident and better able to cope with daily life, and did not experience the same degree of mental decline that they and their carers had expected. In terms of clinical readouts, rember™ showed evidence in the phase 2 trial of a significant reduction in the rate of clinical decline: 80% by weeks 50 and 102 of this trial, relative to controls, as measured using psychometric tools. Functional neuroimaging results from the trial supported the psychometric data: in patients exposed to rember™, the loss of function occurring in the areas of the brain known to be particularly effected by the Tau-tangle pathology of the disease was eliminated over 6 months.
What are phase 2 and phase 3 clinical trials?
A Phase 2 Clinical Trial – a trial that is conducted to evaluate clinical efficacy of a drug for a particular condition in hundreds of patients and to determine the common short-term side effects and risks
A Phase 3 Clinical Trial is an even bigger trial, which is needed to confirm the results, before the regulatory authorities approve the drug for wider use.
Are TAIs new drugs?
rember™, a TAI and the drug developed by scientists at the University of Aberdeen and tested in phase 2 trials, is a new version of an existing drug, but created especially for the treatment of AD. It is patented for use in Alzheimer’s by the company TauRx Therapeutics. As described above, TauRx has recently brought a number of proprietary (composition of matter) second-generation TAIs into development. One of these, LMTX, has been prioritised ahead of rember™ due to its superior tolerability and bioavailability profile as described in the above. The active ingredient in these compounds (methylthioninium, MT) is the same as an old drug existing as the chloride salt (MTC), which has been used in a variety of clinical indications for over a century. However, in the MTC form, the tolerability and bioavailability is limited. People should avoid sourcing MTC themselves for use for Alzheimer’s as it may not be pure and could prove harmful. In addition, the effective dosing range is limited by side effects and tolerability. This is why TauRx has developed LMTX which has been optimised for improved bioavailability and tolerability.
Can I buy MTC over the internet?
It can be dangerous to buy any drug over the internet or from other sources. This is because you will not know what you are buying, it may not be pure and could be toxic and cause harm. TauRx’s proprietary TAIs have been specially created to be pure with good absorption and tolerability profiles at therapeutic doses and their properties will be guaranteed and regulated by the authorities.
When will TAIs be available?
We need to carry out a phase 3 trial to confirm the therapeutic and tolerability profile of our TAI, LMTX, on a still larger scale, and to further refine this treatment. If these trials are successful, the company will apply to the appropriate regulatory bodies for authorisation to market LMTX as regulated and approved treatment for mild to moderate Alzheimer’s and one or more orphan indications. We expect to complete these trials as early as 2013 for AD.
Do TAIs and LMTX in particular have any side effects?
All drugs can cause side effects in some people, and people taking LMTX will be elderly and have a number of other health conditions in addition to Alzheimer’s.
However the evidence is that rember™ was generally well tolerated, although the dosing range was limited in terms of tolerability and side effects, leading to limitation in the maximum potential efficacy. The emerging data on LMTX indicates that TauRx’s second generation TAI has an especially good tolerability profile allowing dosing at higher levels and so possibly achieving better efficacy than the already striking effects seen with rember™ in the previously reported phase 2 trial. A feature of TauRx’s lead TAIs is that they discolour urine and this can cause staining which some people find distressing.
How can I/my relative get on a trial?
Plans are now being made for a phase 3 trial involving clinical teams in Europe and the US, and additional countries still to be confirmed. Doctors in the centres taking part will approach patients who would be suitable for the trial and explain to them what is involved.
People cannot offer themselves or their relatives for the trial. However we hope that if all goes to plan that we could complete the clinical trials needed for regulatory approval as early as 2013 for patients with AD.
Can I ask my doctor for this now?
Your doctor will be unable to prescribe this for you at the present time, as the drug cannot be approved by the regulatory bodies until we have confirmatory results from a phase 3 trial. However we hope that if all goes to plan we could complete these trials as early as 2013 for patients with AD.
In the meantime you are advised not to try to buy MTC over the internet or from other unapproved sources as there will be no guarantee of purity and it could cause harm.
As always you should discuss any concerns about your health with your GP.
Why can’t this be available now?
A phase 3 trial is necessary to confirm the promising benefits of the drug we saw in phase 2, and further refine the treatment. Only when the efficacy and safety of the drug have been confirmed will the regulatory bodies give approval for the drug to be made generally available to the wider population. But if we can proceed speedily with the phase 3 trial we are hoping that the necessary trials could be completed as early as 2013 for patients with AD.
How many people could benefit from this?
The World Health organisation estimates that by 2050 there will be more than one billion people across the world who are aged 65 and over. The research team has calculated that about half of them will have some Tau tangles in their brains which may eventually lead to Alzheimer’s. Should this research ultimately result in a drug that can be taken preventatively to avoid the disease developing – in addition to halting and reversing it once it has developed, many millions of families across the world will benefit. The economic savings to governments and to health services will also be huge.
Why did the University of Aberdeen need to form a company to carry out this research?
The role of the University – as a centre of international academic research expertise - is to initiate and incubate new ideas in treatment for a range of health conditions which are challenging our 21st century world. To develop these further however, a more commercial basis is needed which can attract the investment necessary to take these ideas to the market, and so a ‘spin-out’ company is formed. TauRx was formed as a spin-out company, registered in Singapore, in 2002. The company was further restructured in 2009 to form the TauRx Pharmaceuticals Group of companies composed of TauRx Therapeutics Ltd, Poredeen Pte. Ltd. and WisTa Laboratories Ltd. However TauRx has continued an extremely close relationship with the University, and company researchers are embedded within the University’s international team of experts in biosciences at the Institute of Medical Sciences, working in close partnership with clinical colleagues on Aberdeen’s unique health campus at Foresterhill and with chemists on the main University campus.
Will you be listing and when?
TauRx management and shareholders are constantly monitoring market conditions. We consider the assets of the company to be of a standard that would attract significant interest from financial markets in the context of a possible listing. If such a decision is made, it will be the subject of a separate announcement.
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