Hydromethylthionine Improved Cognition in Alzheimer Disease 

In the phase 3 LUCIDITY study (NCT03446001), people with early-stage Alzheimer disease (AD) treated with hydromethylthionine (HMTM; TauRx Therapeutics, Aberdeen, Scotland) had sustained improvement in cognition compared with their pretreatment baseline. 

The study compared participants treated with HMTM 16 mg/day with methylthioninium chloride (MTC) given at a dose of 4 mg twice weekly. Participants (n=598) had AD severity ranging from mild cognitive impairment (MCI) through to the moderate stage of the disease.

In the 105 participants with MCI who received 16 mg/day HMTM, there was statistically significant cognitive improvement of 2 units over the pretreatment baseline at 6 months (P=.0002). 

In the 147 participants with mild-to-moderate AD who received 16 mg/day HMTM, there was a 2.5-unit cognitive decline in the first 9 months and no further decline over the following 9 months. 

“This is the first time any treatment has produced evidence of sustained improvement over the individual’s own pretreatment baseline lasting 18 months at an early clinically detectable stage of AD, and stabilization of disease progression at more severe stages,” said Claude Wischik, professor, University of Aberdeen. “Tau pathology of the disease is now recognized as an important target for treatment, and it is encouraging that cognitive improvement is seen at such an early stage of the disease with a drug targeting tau.”

There were no treatment-related serious adverse events or evidence of amyloid-related imaging abnormalities. 

TauRx will present the study findings at the Clinical Trials on AD (CTAD) conference on November 30, 2022, in San Francisco, CA.