AZTherapies announced that it has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with its Phase III clinical study investigating its lead program ALZT-OP1 for early Alzheimer’s’ Disease.
The company develops new treatments for Alzheimer’s based on technology licensed from Massachusetts General Hospital. ALZT-OP1 is a multifunctional drug therapy comprising two FDA-approved drugs that act on crucial mechanisms of AD for prevention and treatment. The treatment program is enhanced with a new formulation and targeted delivery to control proper blood and brain concentrations. The first drug in the combo treatment inhibits beta-amyloid peptide polymerization while the second suppresses neuro-inflammatory response in patients with amnestic mild cognitive impairment (aMCI) due to suspected presence of the disease.
The two FDA-approved drugs are being repurposed via MGH’s novel technology and are being developed by AZTherapies to slow cognitive loss or prevent the onset of AD-caused dementia. “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 AZTherapies founder, Dr. David Elmaleh. He said that data from both in vitro and in vivo studies suggest ALZT-OP1’s potential as Alzheimer’s disease treatment.
“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, VP of Regulatory Affairs, Clinipace Worldwide.
Alzheimer’s disease ranks as the sixth leading cause of death in the U.S., affecting about 5.2 million in the country, including patients younger than age 65. The disease is projected to triple this figure by 2050 and escalate yearly cost to over $1 trillion.
Preparations are ongoing to initiate a Phase III study investigating ALZT-OP1’s safety and efficacy in patients with amnestic mild cognitive impairment attributed to suspected AD. The study is expected to begin by the fourth quarter of 2014 across at least 10 countries around the world including the U.S.