TauRx seeks UK regulatory approval for tau-targeting Alzheimer’s drug

If approved, HMTM would become the first oral Alzheimer’s treatment targeting the tau pathology to be made available to patients.

Alzheimer’s-focused biotech TauRx Pharmaceuticals has announced the submission of a UK Marketing Authorisation Application (MAA) for hydromethylthionine mesylate (HMTM) to treat Alzheimer’s patients. If successful, the company says the UK would become the first country to offer an oral treatment targeting the hallmark tau pathology of the disease to patients with mild cognitive impairment and mild to moderate dementia stages due to Alzheimer’s.

Alzheimer’s features two hallmark pathologies: tau and amyloid. While β-amyloid plaques have dominated research and drug development for the past 20 years, there are increasing questions around their clinical relevance in the disease.

Professor Claude Wischik, co-founder, chairman and CEO of TauRx

Rather than focus on amyloid plaques, TauRx is targeting misfolding tau protein, which leads to its aggregation and tangle formation, and which the company believes is more closely correlated with Alzheimer’s disease progression than β-amyloid. Tau aggregation is linked to cognitive decline, brain atrophy and neuronal damage, which are characteristic of neurodegeneration in Alzheimer’s disease.

In Alzheimer’s, tau tangles first destroy memory-critical nerve cells and then neurons in other brain parts as tau aggregation spreads. TauRx says that HMTM acts by selectively inhibiting tau-protein aggregation in brain nerve cells and enhancing brain functioning through a second mode of action that increases acetylcholine levels in the hippocampus. The drug has been designated by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the Innovative Licensing and Access Pathway (ILAP).

TauRx’s MAA submission is based on comprehensive evidence from the recent 24-month Phase 3 LUCIDITY study and two earlier Phase 3 trials in patients with mild to moderate Alzheimer’s. The studies showed benefits in measures of cognitive decline, the ability to perform daily activities, and reduction in the rate of brain shrinkage.

Along with his colleagues at the University of Aberdeen, Professor Claude Wischik, co-founder of TauRx, has spent nearly 30 years investigating the structure of tau tangles and their role in Alzheimer’s, frontotemporal dementia and other neurodegenerative diseases.

“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,” said Prof Wischik, Chairman and CEO of TauRx.

Photograph: Chalabala/Envato