AZTherapies, a company developing novel treatments for Alzheimer's disease based on technology licensed from Massachusetts General Hospital, has received notice from FDA that it may proceed with the phase III clinical study submitted in its IND application. This study is for its lead program, ALZT-OP1, a novel combination therapy for the prevention and treatment of early Alzheimer's disease (AD).

ALZT-OP1 is a multifunctional drug therapy consisting of the administration of two previously approved drugs that act on important mechanisms relevant to AD, with a new formulation and targeted delivery to ensure blood and brain concentrations necessary to achieve their actions. Drug A inhibits beta-amyloid peptide polymerization, and drug B inhibits the neuro-inflammatory response in patients with amnestic mild cognitive impairment (aMCI) due to suspected AD. These two drugs, which have excellent safety and tolerability profiles, are being repurposed using novel technology licensed from MGH and further developed in the company, to slow cognitive loss or prevent the onset of dementia due to AD.

"Confronting two triggering causes associated with AD progression simultaneously provides a new multifunctional treatment approach for modifying disease progression. Very early intervention, in subjects with the earliest clinical signs of dementia due to AD, will be key in preventing and/or delaying the onset of dementia," said company founder, Dr. David R. Elmaleh. "The in vitro and in vivo data support the potential for halting disease onset and progression."

The company reports that preparations currently are underway to initiate the study by the fourth quarter of this year. This phase III study is expected to be conducted in upwards of 10 countries around the world, including the U.S.