AZTherapies Commences Phase III Pivotal Trial - for ALZT-OP1 in Early Alzheimer's Disease

BOSTON, Sept. 15, 2015 /PRNewswire/ -- AZTherapies, Inc. a company developing a novel combination therapy for Alzheimer's disease based on technology licensed from Massachusetts General Hospital, announced that it is commencing a Phase III clinical study for early Alzheimer's disease, after having received a SPA (Special Protocol Assessment) agreement notice from US Food and Drug Administration (FDA).

AZTherapies' experimental treatment is ALZT-OP1, a combination drug therapy consisting of the administration of two previously approved drugs for other indications that act on important mechanisms relevant to Alzheimer's disease (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 lowers cytokine production while drug B inhibits the neuroinflammatory response.  This combination therapy will be tested in patients with early signs of dementia due to suspected Alzheimer's disease. These two drugs, which have excellent safety and tolerability profiles, are being repurposed using novel technology licensed from Massachusetts General Hospital and further developed by the company, to slow cognitive loss and potentially prevent the onset of dementia due to AD.

In June 2015, the Company completed a pharmacokinetic study for both drugs in blood and cerebrospinal fluid (CSF) in 24 age matched normal control subjects following administration of a single and a two single  dose of each of the drugs.

On June 10, the company initiated the Phase III study for the US sites and on August 13, the company initiated the Phase III study in Australia and is planning to initiate the study in Europe.

"Confronting two triggering causes associated with Alzheimer's disease progression simultaneously provides a new multifunctional treatment approach for modifying disease progression," said Dr. David R. Elmaleh, an Associate Professor at Harvard Medical School and the Director of Contrast Media Chemistry at the Massachusetts General Hospital. Dr. Elmaleh is founder of AZTherapies, and a founder of several biotech companies including Molecular Insight, Mersana and co-founder of PureTech Health, which recently went public on The London Stock Exchange. "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, in-vivo, and the pharmacokinetic data support the potential for halting disease onset and progression."

"The use of these two safe, small molecules designed to modify disease behavior by intervening in the plaque formation process and by changing the course of the propagated neuron-inflammatory response is very promising venue for halting or continuously inhibiting disease progression in the early stages of AD onset," said Dr. Rudy Tanzi, the Joseph P. and Rose F. Kennedy Professor of Child Neurology and Mental Retardation at The Massachusetts General Hospital and world leader in AD genetic research and AD drug development.

"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, Effie Marie Cain Professor of Neurology, Baylor College of Medicine and a recognized expert in the field of Alzheimer's research and drug development.

"AZTherapies is poised to have a significant impact on the Alzheimer's population upon study enrollment, and we look forward launching this much anticipated trial," 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. The company's preclinical data and the recent clinical trial demonstrate the potential to change current AD drug regimens and support the importance of this Phase III Study."  Dr. 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 has received a SPA agreement with the FDA for their pivotal Phase 3 study, which provides clarity for the registration pathway.  In addition, the company has reached a broad agreement with FDA that they can use the 505(b)(2) pathway for eventual NDA submission. Both agreements are a great step forward for the development of this promising therapeutic," said Brenda Fielding, Vice President of Regulatory Affairs, Clinipace Worldwide. 

The company reports that preparations are currently underway to initiate the study, "A Phase III Safety and Efficacy Study of ALZT-OP1 in Subjects with Evidence of Early Dementia Due to Suspected Alzheimer's Disease," and expects to be actively recruiting patients in more than 10 countries, including the US, by the end of 2015.

About Alzheimer's Disease

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. AD currently ranks as the 6^th leading cause of death in the United States and remains an unmet medical need. Despite numerous recent advances, AD continues to pose a challenge for both diagnostic and treatment strategies.  An estimated 5.2 million Americans have the disease in 2014, including 200,000 individuals younger than age 65 who have younger-onset Alzheimer's.  It is estimated that by 2050, AD will triple and yearly cost will top one trillion dollars.

It is the Company's goal to become a leader in the world effort to modify disease treatment focusing on very earlier clinical signs of dementia, before neural damage causes progressive destruction, especially in those individuals at a higher risks to develop the disease.

About the Company 

AZTherapies (www.aztherapies.com) is a privately held drug development company founded in 2011 and headquartered in Boston, MA.  AZTherapies is innovating the discovery, development and commercialization of novel Alzheimer's disease therapeutics that will fundamentally improve patient treatment, quality of life and disease management. In addition to ALZT-OP1, the Company platform also includes ALZT-OP2, a disease modifying drug treatment in advanced preclinical stage development, AZHALER-D, a disposable novel inhaler in late stage development to be specifically used for the Company's proprietary inhaled drugs, and ALZT-QoL, a neurodegenerative drug treatment designed to improve the quality of life of subjects suffering from advanced stages of diagnosed AD using a compensatory ligand/receptor mechanism to improve brain network connectivity inhibition caused by AD.  AZTherapies' drugs are based on technologies developed at The Massachusetts General Hospital, a Harvard Medical School Teaching Hospital. The Company's 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.

About Clinipace Worldwide

Clinipace (www.clinipace.com) is a digital contract research organization (dCRO), pioneered an innovative, technology-amplified CRO service model to serve the unique needs of venture-backed, mid-tier and strategic pharmaceutical, biotechnology and medical device firms.

This press release contains forward-looking statements regarding AZTherapies' planned activities with respect to the clinical development of our candidates and other matters. Actual results may differ materially from those in these forward-looking statements. Risks include clinical trial conduct and results, FDA requirements, non-completion of our drugs in development, competing products, ability to secure financing, unseen market conditions and future hampering commercialization events, among others.

SOURCE AZTherapies, Inc.

Related Links

http://www.aztherapies.com