Tau-targeting Treatment Slows Cognitive, Functional Declines: Trial
Alzheimer’s patients given the experimental tau-targeting therapy HMTM in a Phase 3 clinical trial experienced a substantially slower decline in cognitive and functional measures than would be expected based on published research, according to new findings announced by HMTM’s developer TauRx Pharmaceuticals.
TauRx expects these results will support applications to gain regulatory approvals of HMTM for treating Alzheimer’s.
“The output indicates that participants receiving HMTM decline at a rate substantially less than is typical in Alzheimer’s based on published research. This was seen for both cognitive and functional endpoints across a broad range of severity from mild cognitive impairment (MCI) to moderate Alzheimer’s,” Claude Wischik, executive chairman and co-founder of TauRx, said in a press release. “We recognise and publicly thank the incredible commitment from participants and partners involved in LUCIDITY,” Wischik said.
Hydromethylthionine mesylate, or HMTM, is an oral medicine designed to reduce levels of tau tangles — irregular clumps of protein that are a hallmark of Alzheimer’s and are thought to help drive the disease’s progression. The investigational therapy also has been referred to as TRx0237, LMTM, and LMTX.
“Today with limited treatments for Alzheimer’s, the standard of care does not impact the underlying causes of symptom progression. HMTM aims to significantly slow disease progression, providing longer term benefits compared to medications brought to market almost twenty years ago,” Wischik said.
The Phase 3 clinical trial LUCIDITY (NCT03446001) enrolled 598 people with Alzheimer’s who were randomly assigned to one of two doses of HMTM (8 or 16 mg/day), or a placebo, for one year. The trial’s full design will be detailed in an upcoming paper in the Journal of Prevention of Alzheimer’s Disease.
In addition to positive effectiveness data, HMTM’s safety profile in the LUCIDITY study was generally positive and consistent with prior studies of the experimental therapy, according to Wischik.
“Our data analysis is ongoing and will be reported at a later date,” Wischik said, adding that an update will be provided June 9 at the 35th Global Conference of Alzheimer’s Disease International.
Based on these positive findings, TauRx is planning to begin seeking regulatory approvals of HMTM.
“Our expert advisors including Eversana [a life sciences commercial services company] are confident in our moving toward regulatory submission and gaining coverage for HMTM,” Wischik said.
Before the new data from LUCIDITY were announced, TauRx had received an innovation passport from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). This is the first step of the innovative licensing and access pathway (ILAP), intended to speed up new therapies’ development and approval times.
“The ILAP designation represents a clear signal of regulatory support for a prospective treatment breakthrough in Alzheimer’s, which remains one of the world’s greatest unmet medical needs. Dementia is a leading cause of death around the world, and the Innovation Passport, as the first stage of the ILAP scheme, enables access to the collaborative approach of regulators and associated health technology assessment bodies to both drug licensing and access throughout the UK,” Wischik said.