EnVivo Pharmaceuticals Announces Initiation of Phase 3 Clinical Trial Program of EVP-6124 in Schizophrenia

Building on Positive Phase 2b Results, Studies to Evaluate EVP-6124 Effect on Cognition in Schizophrenia Patients

WATERTOWN, Mass.--(BUSINESS WIRE)--EnVivo Pharmaceuticals, a company dedicated to developing a broad range of novel central nervous system (CNS) therapies, today announced the initiation of its Phase 3 clinical trial program for EVP-6124, a novel alpha-7 agonist. Patient dosing has begun in two trials, which are designed to assess the safety and efficacy of EVP-6124 compared to placebo in patients with schizophrenia when added to chronic, stable, atypical antipsychotic therapy. These two 26-week multinational trials are designed to enroll approximately 700 patients each and both will evaluate two doses of EVP-6124 compared to placebo.

“While currently available atypical antipsychotic medications can be effective in improving the positive symptoms of schizophrenia, there are no currently approved treatments for the cognitive or negative symptoms, which greatly impact the daily lives of people living with this disease”

“While currently available atypical antipsychotic medications can be effective in improving the positive symptoms of schizophrenia, there are no currently approved treatments for the cognitive or negative symptoms, which greatly impact the daily lives of people living with this disease,” said Dana Hilt, M.D., senior vice president, clinical development and chief medical officer of EnVivo. “We have seen statistically significant and clinically meaningful results on global cognitive function, negative symptoms and clinical function with EVP-6124 in prior studies. We believe that EVP-6124 may address a significant need for treatment options that can enhance cognition and that can be administered along with atypical antipsychotics, the current standard of care for people living with schizophrenia.”

The Phase 3 clinical trial program includes two randomized, double-blind, placebo-controlled studies evaluating the safety and efficacy of two doses of once daily treatment with EVP-6124 as a pro-cognitive treatment compared to placebo when added to chronic, stable, atypical antipsychotic therapy in people with schizophrenia. The primary endpoints of the trials include effect on cognitive function as measured by the Measurement and Treatment Research to Improve Cognition in Schizophrenia (MATRICS™) Consensus Cognitive Battery (MCCB™) overall cognitive index and effect on clinical function as measured by the interview-based Schizophrenia Cognition Rating Scale (SCoRS). Secondary endpoints of the trial include the clinical efficacy of the two doses of EVP-6124 as measured by negative subscale of the Positive and Negative Syndrome Scale (PANSS), Clinical Global Impression – Severity (CGI-S), Clinical Global Impression – Change scale (CGI-C), and quality of life, using the EuroQoL-5D™ (EQ-5D). Safety will be determined by clinical and laboratory safety assessments.

“An estimated 2.4 million Americans have schizophrenia, almost all of whom have cognitive deficits which are the major cause of their functional impairment, even more so than psychotic symptoms. There is a critical need for safe and effective therapies that address these deficits in cognition because minimizing them is likely to have a positive impact on quality of life and independence,” said Herbert Y. Meltzer, M.D., Ph.D., professor of psychiatry and behavioral sciences, Northwestern University School of Medicine. “Studies like these Phase 3 clinical trials are essential for the medical community and regulatory authorities to evaluate new treatment options designed to address these unmet needs of patients with schizophrenia.”

About Schizophrenia

Schizophrenia is a psychiatric disorder that affects approximately 2.4 million Americans, or about one percent of the adult population, and is usually diagnosed between the ages of 15 and 35 years old. Symptoms of schizophrenia include positive and negative symptoms as well as cognitive impairment. “Positive” symptoms include hallucinations, delusions and paranoia. “Negative” symptoms include loss of motivation and interest in everyday activities, blunting of emotion, decrease in speech and social withdrawal. Increasingly, cognitive impairments such as difficulty paying attention, memory loss and problems processing information and making decisions are also recognized as core disabling symptoms of the disease. The overall annual cost of schizophrenia in the U.S. is more than $62 billion according to a study published in the Journal of Clinical Psychiatry.

About EVP-6124

EnVivo Pharmaceuticals’ lead compound, EVP-6124, is a selective, potent, brain penetrant, oral alpha-7 nicotinic agonist being developed as a long-term treatment to improve cognitive function with sustained effect in Alzheimer’s disease and schizophrenia. It is believed that EVP-6124 offers a novel mechanism of action by enhancing synaptic transmission in the brain and acting as a co-agonist in combination with Acetylcholine (ACh) to enhance cognition. By sensitizing the alpha-7 receptor, EVP-6124 makes it possible for smaller amounts of naturally occurring ACh to be effective in activating the A7 receptor. This mechanism of action could potentially alleviate the undesirable side effects caused by other systemic compounds (for example, acetylcholinesterase inhibitors, or AChEIs), which are dose-limited by side effects. EVP-6124 has also been shown to increase the levels of key neurotransmitters such as glutamate and dopamine in key brain regions in preclinical studies, which may be linked to enhanced cognition, not only in Alzheimer’s disease and schizophrenia, but also in other diseases.

EnVivo announced positive topline data from its double-blind, placebo-controlled Phase 2b clinical trial of EVP-6124 in schizophrenia in May 2011 and presented a comprehensive analysis of these findings in December 2011. The Phase 3 program of EVP-6124 in schizophrenia was announced in February 2013. The company also announced and presented positive findings from another double-blind, placebo-controlled Phase 2b clinical trial of EVP-6124 in Alzheimer’s disease in July and October 2012. A Phase 3 clinical trial of EVP-6124 in Alzheimer’s disease is expected to be initiated in 2013.

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. is a biopharmaceutical company located in Watertown, Mass. The company’s focus is on building an integrated company and it is working to convert its broad pipeline into a range of CNS therapies that leverage novel mechanisms of action by altering the progression of disease and providing improvement in cognitive and overall function. EnVivo’s lead product, EVP-6124, is an alpha-7 nicotinic acetylcholine receptor agonist. EVP-6124 is currently being evaluated in Phase 3 clinical trials in schizophrenia and will enter a Phase 3 clinical trial in Alzheimer’s disease in 2013. EnVivo’s other development programs include an epigenetics program based on Histone Deacetylase inhibition (HDACi), a Gamma Secretase Modulator program and a potent and selective PDE10 inhibitor program. For more information about EnVivo, visit www.envivopharma.com.