AZTherapies Inc., a company developing novel treatments for Alzheimer’s disease based on technology licensed from Massachusetts General Hospital, announced that it has received notice from FDA that it may proceed with the Phase 3 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.
ALZT-OP1 is a multifunctional drug therapy consisting of the administration of two previously approved drugs that act on important mechanisms relevant to Alzheimer’s disease, 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 Alzheimer’s disease (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 Alzheimer’s disease 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 Elmaleh, “The in-vitro and in-vivo data support the potential for halting disease onset and progression.”
“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’ multi-functional treatment approach which, if successful, could have a long-term beneficial effect on the course of AD progression,” commented Dr. Rachelle Doody, Cain Professor of Neurology, Baylor College of Medicine and a recognized key opinion leader (KOL) in the field of Alzheimer’s research. Doody was a principal investigator in studies that led to the approval of Aricept and Namenda, two currently FDA-approved drug treatments for improving Alzheimer’s disease symptoms.
“The company technology includes intellectual property protecting the drug combination, dosing, formulation and drug properties that will deliver the drug to both blood and brain through inhalation,” said Dr. Peter Conti, a professor of radiology, Biomedical Engineering and Pharmaceutical Sciences at the University of Southern California and a member of the Board of Directors at AZTherapies. Conti is an expert in the field of neuroimaging and positron emission tomography, and has been involved in the early use of Alzheimer’s diagnostics, such as F-18 Florbetapir, the amyloid-imaging tracer developed by Avid.
“AZTherapies intends to request a Special Protocol Assessment (SPA) from FDA and expects to reach an agreement on the design and size of a clinical trial in support of a 505(b)(2) NDA for the treatment of early Alzheimer’s disease (AD),” said Brenda Fielding, vice president of Regulatory Affairs, Clinipace Worldwide.
The company reported that preparations are currently underway to initiate the study, “A Phase 3 Safety and Efficacy Study of ALZT-OP1 in Subjects with Evidence of Amnestic Mild Cognitive Impairment due to Suspected Alzheimer’s Disease” by the fourth quarter of this year (Q4-2014). This Phase 3 study is expected to be conducted in upwards of 10 countries around the world, including the US.
Alzheimer’s Disease is an irreversible neurodegenerative disease, its onset and progression are associated with cognitive and functional decline causing a gradual daily life activity impairment, psychotic disturbances, and becomes fatal at its advanced stages. It currently ranks as the 6th leading cause of death in the United States and remains an unmet medical need. Despite numerous recent advances, Alzheimer’s disease continues to pose a challenge for both diagnostic and treatment strategies. An estimated 5.2 million Americans have Alzheimer’s disease in 2014, including 200,000 individuals younger than age 65 who have younger-onset Alzheimer’s. It is estimated that by 2050, Alzheimer’s disease will triple and yearly escalation cost to top one trillion dollars.
Date: May 23, 2014