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Aberdeen’s role in reshaping Alzheimer’s care as TauRx trial data strengthens case for oral treatment

By David Langstane Aberdeen’s life sciences sector has taken another important step onto the global stage with new clinical data ...

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By David Langstane

Aberdeen’s life sciences sector has taken another important step onto the global stage with new clinical data from TauRx that could change how Alzheimer’s disease is treated – and underline the north‑east’s growing role in Scotland’s brain‑health ambitions.

TauRx has announced that results from a confirmatory study evaluating its experimental Alzheimer’s drug hydromethylthionine mesylate (HMTM) in people with mild cognitive impairment and mild to moderate dementia due to Alzheimer’s disease have been published in The Journal of Prevention of Alzheimer’s Disease. The latest analysis bolsters earlier findings that the tablet‑based treatment may slow or halt underlying neurodegeneration when given early, and comes as Aberdeen’s wider brain‑health ecosystem gains momentum. (Anne Beau‑Lyon has also written a fuller analysis for Silicon Scotland on the nationwide drive to position Scotland at the forefront of brain‑health science and the fight against Alzheimer’s disease.)

Strengthening the clinical case for HMTM

The new paper revisits data from participants who received HMTM 16 mg/day in TauRx’s TRx‑237‑039 (LUCIDITY) Phase III trial. In the original study, a very low dose of HMTM was used as the control arm, rather than a true placebo, to maintain blinding in the face of “slight urinary colouration by HMTM”. The company later confirmed that this low dose “had some activity and so was not a true placebo”, making it harder to see the full treatment effect.

To address that, the confirmatory study “compared closely matched external placebo data to show statistically significant treatment effects.” TauRx’s analytics team matched HMTM‑treated participants against placebo controls drawn from the FDA‑sponsored Critical Path for AD (CPAD) database, meeting key inclusion and exclusion criteria (protocol TRx‑237‑080), “as well as with other control populations.”

The new publication, titled Assessment of clinical and neuroimaging efficacy of treatment targeting tau pathology in mild cognitive impairment and mild to moderate Alzheimer’s disease with hydromethylthionine mesylate using external control data, “reports statistically significant differences relative to three different external control populations. Key analyses suggest that unmeasured confounding is highly unlikely to explain the observed results.”

Crucially, the study reinforces earlier work indicating that HMTM may alter the course of disease when used at the stage of Mild Cognitive Impairment (MCI). “The study supports an earlier publication reporting that hydromethylthionine mesylate (HMTM) halted progression of neurodegeneration in Alzheimer’s disease (AD) and that participants with Mild Cognitive Impairment (MCI) who received HMTM 16 mg/day experienced statistically significant cognitive improvement over 18 months. Participants also displayed no evidence of statistically significant cognitive or functional decline over a period of two years.”

“Strongly support the effectiveness” of HMTM

Prof Bjoern Schelter, TauRx’s Chief Analytics Officer and lead author of the new paper, said the latest findings add important weight to the company’s case.

“The latest results strongly support the effectiveness of HMTM in people living with MCI and mild to moderate dementia due to AD.”

“By comparing participants receiving HMTM 16 mg/day with external control populations, we have been able to navigate the blinding problem and so demonstrate consistent and clinically meaningful differences in cognitive decline (ADAS‑Cog13) and rate of loss of brain volume at 78 and 104 weeks, as well as a global measure of progression of dementia (CDR‑SB) over 104 weeks.”

He added that the broader evidence package now points in the same direction:

“The latest results are consistent with our earlier report demonstrating that HMTM could have significant treatment effects on neurodegeneration, tau pathology and brain atrophy. The totality of the evidence now available reinforces our belief that HMTM has the potential to offer an accessible oral treatment option which could be delivered safely and with minimal patient/physician burden.”

The original LUCIDITY trial also underpins TauRx’s safety argument. “The original TRx‑237‑039 (LUCIDITY) trial confirmed HMTM has a benign safety profile, with headache (1.5%) and diarrhoea (1.2%) reported as the most frequent adverse effects at the 16 mg/day dose.”

HMTM remains an unlicensed medicine at this stage. “HMTM is an investigational drug and a Marketing Authorisation Application from TauRx Therapeutics Management Ltd is currently being evaluated by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).”

Aberdeen at the centre of a Scottish brain‑health strategy

Although the latest paper is global in scope, it shines a spotlight on work that has been driven for more than two decades from Aberdeen.

TauRx Therapeutics Management Ltd “is the UK subsidiary of TauRx Pharmaceuticals Ltd. TauRx was established in 2002 with a mission to discover, develop and commercialise innovative products for the diagnosis, treatment and cure of neurodegenerative diseases caused through protein aggregation.”

That mission has helped put tau – the protein HMTM is designed to target – at the heart of international conversations about disease‑modifying therapies for Alzheimer’s and related dementias. As Scotland rolls out a refreshed life sciences strategy with a strong emphasis on healthy ageing, neurodegeneration and data‑enabled medicine, TauRx’s maturing clinical programme provides a high‑profile example of what long‑term scientific investment in the north‑east can deliver.

For Aberdeen, the implications are both scientific and economic. A successful outcome to the ongoing MHRA review could see one of the first oral, tau‑targeting treatments for Alzheimer’s disease emerge from a company anchored in the city. That would not only put Aberdeen firmly on the map for global pharma and biotech investors, but also strengthen the case for continued investment in local clinical research capacity, skills and supporting infrastructure.

At the same time, the north‑east’s contribution goes beyond a single drug. By combining home‑grown therapeutics development, access to international trial networks and a growing base of clinicians and data scientists working on brain health, the region is becoming an integral part of a wider Scottish system focused on earlier detection, intervention and support for people at risk of dementia.

As TauRx awaits regulators’ verdict on HMTM, Aberdeen’s message to industry and policymakers is clear: the city is not just a backdrop to Scotland’s brain‑health ambitions – it is one of the places where those ambitions are being turned into tangible clinical, economic and societal impact.

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