EnVivo Pharmaceuticals Initiates COGNITIV AD, a Phase 3 Clinical Trial Program of Encenicline (EVP-6124) in Alzheimer's Disease

WATERTOWN, Mass. – January 22, 2014 – EnVivo Pharmaceuticals, a company dedicated to developing a broad range of novel therapies for central nervous system (CNS) diseases, today announced it has initiated COGNITIV AD, a Phase 3 clinical program evaluating its novel alpha-7 (α7) potentiator, encenicline-hydrochloride (EVP-6124), in patients with Alzheimer's disease (AD). The program is being conducted under a Special Protocol Assessment agreement with the FDA.

"We are pleased to advance encenicline into Phase 3 clinical development in Alzheimer's disease, a significant milestone for our company and promising step forward for patients who desperately need new therapies," said Deborah Dunsire, M.D., president and chief executive officer of EnVivo. "Prior clinical studies of encenicline have demonstrated clinically significant improvements in cognitive function in patients with Alzheimer's disease. For the millions of patients living with AD, we believe encenicline has the potential to make a meaningful difference."

Patient dosing has begun in the 26-week trial, which is designed to evaluate the safety and efficacy of two fixed doses of encenicline compared to placebo in patients with mild to moderate AD who are currently receiving stable treatment with or have been previously treated with an acetylcholinesterase inhibitor.

"There is a profound need for better therapies for the cognitive and functional impairments of Alzheimer's disease," said Lon S. Schneider, M.D., professor of psychiatry and the behavioral sciences, neurology, and gerontology at the University of Southern California (USC); director of the California Alzheimer's Disease Center at USC; clinical core of the NIH-funded USC Alzheimer's Disease Research Center and investigator in the COGNITIV AD Phase 3 clinical program. "Encenicline demonstrated clear, positive effects on cognition in the Phase 2 trial, and we should expect to confirm these outcomes in the large Phase 3 program."

About the COGNITIV AD Program

The COGNITIV AD Phase 3 clinical trial program consists of two randomized, double-blind, placebo-controlled trials, which will enroll approximately 1,600 patients at sites in the United States and other countries worldwide. Patients will be randomized to receive one of two doses of encenicline once daily or placebo. In addition to assessing the safety and tolerability of encenicline, efficacy will be evaluated by two co-primary endpoints, the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) as a measure of cognition and the Clinical Dementia Rating Sum of Boxes (CDR-SB) as a measure of clinical function. Secondary measurements include the Disability Assessment for Dementia (DAD), Neuropsychiatric Inventory (NPI), the Mini-Mental State Examination (MMSE) scale and the Controlled Oral Word Association Test (COWAT).

Prior to initiating this study, EnVivo successfully completed a Phase 2b clinical trial in which encenicline demonstrated statistically significant and clinically important improvement in cognition and clinical function compared to placebo in patients with mild to moderate Alzheimer's disease. In February 2013, the company initiated its Phase 3 clinical trial program evaluating encenicline in people with cognitive impairment associated with schizophrenia (COGNITIV CIAS) to assess the safety and efficacy of encenicline compared to placebo in patients with schizophrenia when added to chronic, stable, atypical antipsychotic therapy.

About Alzheimer's Disease

Alzheimer's disease is a complex neurodegenerative disease that affects the brain. One in eight Americans over the age of 65, or approximately five million people, currently have Alzheimer's disease, and with the aging of the baby boomer generation, the total number is expected to nearly triple by 2030. Alzheimer's disease is the sixth-leading cause of death in the United States. Over time, the disease leads to cellular loss and dysfunction in the brain, a gradual loss of memory, problems with reasoning or judgment, disorientation, difficulty in learning, loss of language skills, and decline in the ability to perform routine tasks and daily activities. People with Alzheimer's disease can also experience behavioral problems such as agitation, anxiety, delusions and hallucinations.

About Encenicline

EnVivo's lead compound, encenicline (EVP-6124), is a novel alpha-7 (α7) potentiator currently being evaluated in the ongoing Phase 3 COGNITIV clinical trials to improve cognition in patients with Alzheimer's disease (COGNITIV AD) and patients with cognitive impairment associated with schizophrenia (COGNITIV CIAS). Encenicline is an orally administered, brain penetrant, selective and potent agonist of the α7 receptor found on certain hippocampal and cortical neurons in the brain. Encenicline is believed to work in low concentrations as a co-agonist with naturally occurring acetylcholine to potentiate the response of α7 receptors – activated brain networks associated with sensory gating, attention and cognition – thus priming these networks for improved neural processing and improved cognitive performance in areas such as memory and executive function. 

EnVivo has a license agreement with Mitsubishi Tanabe Pharma Corporation (MTPC) for the development and commercialization of encenicline in Japan and several other Asian countries.

About EnVivo Pharmaceuticals

EnVivo Pharmaceuticals, Inc. and its subsidiaries ("EnVivo Pharmaceuticals" or "EnVivo") are dedicated to discovering and developing small molecule therapeutics for disorders of the central nervous system (CNS). EnVivo Pharmaceuticals, Inc., based in Watertown, Mass., is a privately held company working to develop its broad pipeline into a range of CNS therapies that explore novel mechanisms of action to potentially alter the progression of disease and provide improvement in cognitive and overall function. For more information about EnVivo, visit www.envivopharma.com.