Professor Claude Wischik, CEO and Co-Founder; Dr Glenn Corr, COO and CBO; Dr Richard Stefanacci, CMO
Professor Claude Wischik, CEO and Co-Founder of TauRx, and Chairman of Mental Health (Clinical) at the venerable University of Aberdeen, made a pioneering discovery an impressive four decades ago—the neurofibrillary tangles witnessed in Alzheimer's disease are, in fact, constructed from subunits of the tau protein.
We are not just trying to create a medicine; we are trying to create a solution
“TauRx was established to bring this research to fruition in clinical practice, remaining steadfastly committed to tau-based Alzheimer’s research,” states Professor Wischik. “As the original discoverers of the tau protein pathology in Alzheimer’s disease, we have demonstrated that it is highly correlated with cognitive impairment across the range of the disease.”
This approach stands in contrast to the typical research strategy on the disease, which has historically centered on amyloid plaques—the other lesion commonly observed in the brains of Alzheimer's patients.
Why is this Work So Important?
The number of individuals living with dementia worldwide currently stands at a staggering 55 million, with Alzheimer's disease the cause for 60 to 70 percent of cases. Given the current absence of effective preventive measures to deter dementia, detecting the disease and developing accessible, efficacious treatment is an urgent necessity.
TauRx blazes a trail in the tau pathology of Alzheimer's disease, with an established link to disease progression. To that end, it is investigating a class of drugs known as Tau Aggregation Inhibitors (TAIs), which interfere with the abnormal aggregation of tau and subsequent formation of tau tangles.
Professor Wischik, Professor of Psychiatric Geratology at University of Aberdeen, has been deeply involved in all aspects of the organization's work, including toxicology studies, pharmacology, and basic science. The organization's lead molecule was developed through a Phase 2 trial study, followed by Phase 3 clinical trials with its second-generation tau aggregation inhibitor with enhanced bioavailability properties to allow for lower dosing. These early phase 3 clinical trials were conducted in mild and moderate Alzheimer’s, with a third study in people with a rarer form of dementia, behavioral variant frontotemporal dementia.
TauRx is currently completing a third global Phase 3 clinical trial in Alzheimer’s called LUCIDITY. Having progressed its deep science R&D, TauRx has its sights set on progressing regulatory submissions with a view to product registration on the back of these latest trial results and preparing for future commercialization.
In May 2022, TauRx was granted an Innovation Passport in the UK, which is the first step in the country’s Innovative Licensing and Access Pathway (ILAP) process. The UK regulator's passport programme is designed to facilitate the regulatory process, allowing innovative products to be introduced to the market quickly. With pressure also to educate stakeholders in tau science and the potential availability of new treatments and diagnostic approaches, TauRx is investing more on advocacy, data collection, and critical support initiatives. Yet R&D remains a driving force, and the organization continues to examine future molecules and evaluate the regulatory system to introduce novel treatments and patient pathways to the market.
Long-Term Partnerships Transforming the Treatment Pathway
GT Diagnostics is developing crucial digital tools for the timely detection and monitoring of disease progression. The creation of the company came from Professor Wischik’s experiences in clinical practice, particularly in the rural area of the northeast of Scotland, where assessments had to be done in people's homes. Collaboration between TauRx and Genting Berhad Group resulted in the formation of Genting TauRx Diagnostics Centre, trading as GT Diagnostics, and is aimed at transforming the neurodegenerative diagnostic landscape.
Better diagnostic and monitoring tools could significantly contribute to more patients being diagnosed early, more appropriate follow-up for patients and caregivers, and help transform the care pathway for those affected.
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TauRx was established to bring this research to fruition in clinical practice, remaining steadfastly committed to tau-based Alzheimer’s research
Journey Backed by Clinical and Basic Science
TauRx focuses on developing a valuable and easily accessible complete answer for supporting patients and their healthcare providers. It intends to continue its mission towards this goal by collaborating with a diverse range of businesses and pushing the boundaries of research in neurodegeneration.
“We are not just developing a medicine; we are creating a solutions,” says Dr. Richard Stefanacci, a practicing / geriatrician who focuses on care of people with Alzheimer’s and Chief Medical Officer of TauRx.
Moving forward, the company envisions forming new relationships with neurodegeneration stakeholders to expand its research beyond Alzheimer’s. Its goal is to successfully bring treatments to those with different forms of Tau pathologies as Tau is a driving force of other forms of dementia, including chronic traumatic encephalopathy, often associated with brain trauma.
The pipeline for TauRx and GT Diagnostics is ripe for exploitation with significant clinical and financial success.
TauRx’s Hydromethylthionine Mesylate (HMTM) Demonstrates Significant Reduction in Neurodegeneration in Alzheimer’s Disease (AD)
Monday, July 17, 2023
• Results from a prespecified analysis of the Phase 3 LUCIDITY trial show reduction in neurodegeneration biomarker (NfL) in AD for people receiving 16 mg/day of HMTM compared to controls
• HMTM is an oral drug with a strong safety profile
• Results announced in an oral presentation at the Alzheimer’s Association International Conference (AAIC) 2023
ABERDEEN, Scotland: TauRx Pharmaceuticals Ltd., a global leader in Tau-based research in Alzheimer’s disease (AD), today announced results from a prespecified analysis of the Phase 3 LUCIDITY trial that measured the impact of HMTM on neurofilament light chain (NfL), an established biomarker for brain neurodegeneration. Blood concentration of NfL showed a statistically significant 93% reduction in change over 12 months in participants receiving HMTM at a dose of 16 mg/day relative to the control group, which correlated significantly with a tau biomarker (p-tau 181) in blood.
Neurofilaments and tau proteins play crucial roles in maintaining the structure and proper function of neurons within the brain. In the context of Alzheimer's disease (AD), tau protein tends to aggregate, forming harmful fibrils that can inflict damage on neurons. The extent of this damage can be assessed by measuring the release of neurofilament protein into the bloodstream.
It is well-established that the concentration of neurofilament protein in the blood is linked to tau pathology, the severity of the disease, as well as cognitive decline and brain atrophy in AD. With the objective of mitigating tau pathology in AD, scientists developed HMTM, a tau aggregation inhibitor.
Observing the changes in NfL concentration brought about by HMTM has provided valuable insights, indicating a direct influence on the underlying disease pathology. This avenue of research holds promise for further understanding and potentially treating AD.
“NfL is a well-studied biomarker with wide applicability to different neurological disorders, including AD,” said Henrik Zetterberg, Professor of Neurochemistry, UCL Queen Square Institute of Neurology. “Clinical practice has been waiting decades to uncover meaningful advancements to address unmet needs of people with AD. These new results further support the importance of NfL as an AD biomarker both for diagnosis and measurement of treatment effect.”
“The NfL results demonstrate that a drug targeting tau pathology reduces the neurodegeneration that underlies clinical decline in AD,” said Claude Wischik, Executive Chairman, TauRx. “They bring us a step closer to offering an effective new treatment option for people with AD. Because it is taken as a tablet and has a strong safety profile, HMTM would be readily accessible to people needing a disease modifying treatment.”


