Alzheimer's treatment enters phase III trial this year

AZTherapies, a Boston-based biomedical company investigating Massachusetts General Hospital-licensed treatments for Alzheimer's disease (AD), announced May 23 that the FDA has cleared the path for a phase III clinical trial for ALZT-OP1, a combination of two drugs with the aim of curbing the neurodegenerative disease in its earliest stages. 

ALZT-OP1 is comprised of two previously approved drugs that have been reformulated and targeted for essential blood and brain concentrations. The first drug inhibits the polymerization of beta-amyloid peptides and the second inhibits neuro-inflammatory response in cases of amnestic mild cognitive impairment (aMCI) in patients suspected of Alzheimer's disease in order to slow cognitive loss and provide a means of dementia prevention.

"Confronting two triggering causes associated with Alzheimer's disease progression simultaneously provides a new multifunctional treatment approach for modifying disease progression," said AZTherapies founder, David R. Elmaleh, MD, PhD, in a press release. "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. The in-vitro and in-vivo data support the potential for halting disease onset and progression."

The new international study, titled "A Phase III Safety and Efficacy Study of ALZT-OP1 in Subjects with Evidence of Amnestic Mild Cognitive Impairment due to Suspected Alzheimer's Disease," is expected to begin by the fourth quarter of this year and will be operating within the U.S. as well as up to 10 other countries.

"Alzheimer's disease modifying treatments should affect the underlying pathophysiology of the disease and add additional value to the benefits of the presently approved drugs. I am optimistic about AZTherapies' multifunctional treatment approach which, if successful, could have a long-term beneficial effect on the course of AD progression," said Rachelle Doody, MD, Cain Professor of Neurology at Baylor College of Medicine and a principal investigator in the studies involved in FDA-approved Alzheimer's drugs Aricept and Namenda, which are formulated to improve symptoms of disease.

The trial will be reliant on Alzheimer's associated neuroimaging in order to validate the drug treatment, which is delivered through inhalation.