TauRx Submits UK Marketing Authorisation Application for HMTM as a Treatment for Alzheimer's Disease

TauRx Pharmaceuticals Ltd, a global leader in tau-based research in Alzheimer's disease (AD), has announced the submission of a UK Marketing Authorisation Application (MAA) for hydromethylthionine mesylate (HMTM) for treatment of mild cognitive impairment (MCI-AD) and mild to moderate stages of dementia due to Alzheimer's disease.

HMTM has been designated by the UK's Medicines and Healthcare products Regulatory Agency (MHRA) for the Innovative Licensing and Access Pathway (ILAP). If successful, the UK could be the first country in which an accessible, safe, oral treatment, which targets the hallmark tau pathology of the disease, becomes available to patients.

The MAA is based on the totality of evidence available from the recently released 24-month Phase 3 LUCIDITY data and two earlier Phase 3 trials in mild to moderate AD. These studies have been consistent in showing benefit on measures of decline in cognition, ability to perform normal activities of daily living, and reduction in the rate of brain shrinkage.

Tau aggregation is strongly correlated with the rate and severity of cognitive decline, brain atrophy, and the damage to neurons responsible for the characteristic neurodegeneration of the disease. HMTM acts by selectively inhibiting the aggregation of tau-protein in brain nerve cells. It also has a second mode of action which results in enhancement of brain functioning.

Commenting on the submission, Professor Claude Wischik, Executive Chairman of TauRx, said: "This is a significant milestone for TauRx and is an important step in potentially bringing a new type of treatment and a new hope to patients and families who carry the burden of this terrible disease."

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ABOUT HMTM

Hydromethylthionine mesylate (HMTM) has been developed as a potential oral treatment for AD targeting tau aggregation. It also has a secondary tau-independent mode of action which increases acetylcholine levels in the hippocampus. The global Phase 3 clinical trial LUCIDITY has recently completed and the data were presented at the AD/PDtm 2024 Alzheimer's & Parkinson's Diseases Conference in Lisbon, Portugal, on 7 March. With over 3,000 subjects studied, HMTM has a strong safety profile and could be delivered with minimal patient and physician burden.

Neurofilament Light Chain (NfL) is an established biomarker for neurodegeneration. LUCIDITY showed a significant reduction in the change of NfL, in the randomised double-blind portion of the study at 12 months. Clinical measures of cognition showed improvement over baseline levels that was sustained over 18 months in MCI, as diagnosed in the LUCIDITY trial. Patients who were in the control group were unable to catch up in terms of cognitive function despite transitioning to 16mg per day HMTM (target dose) in the open-label phase after 12 months. This emphasises the importance of early intervention with effective therapies in AD.

ABOUT TAURx PHARMACEUTICALS LTD

TauRx was founded in 2002 in Singapore, with primary research facilities and operations based in Aberdeen, UK. In collaboration with the University of Aberdeen, the company has dedicated the past two decades to developing treatments and diagnostics for Alzheimer's and other neurodegenerative diseases due to pathological aggregation of tau and other proteins.

AD is a leading cause of disability and death throughout the world and is one of the most important global public health issues. TauRx will contribute to addressing this urgent unmet need with data from the totality of evidence available from LUCIDITY and the earlier AD trials in pursuit of regulatory approvals starting in UK. TauRx's hope is to make HMTM available for people living with Alzheimer's as soon as possible. Future research is planned for other related neurodegenerative diseases. https://taurx.com/