EnVivo Pharmaceuticals announced positive topline results of its randomized, placebo-controlled phase II b clinical trial of EVP-6124, its potent, orally bio-available and selective alpha-7 agonist, in patients with schizophrenia. The data showed that EVP-6124 had a clinically meaningful and statistically significant impact on patients’ overall cognition - the trial’s pre-specified primary endpoint - when taken in combination with second-generation antipsychotics and as measured by the full CogState overall cognitive index, or “OCI” (p=0.05 for all patients treated with EVP-6124 versus placebo.) This positive effect on the OCI was supported by a strong positive trend for improved cognition on the MCCB Battery of cognition tests, which were conducted on all U.S. patients in the trial.

Additionally, results from this phase II b trial demonstrated that patients treated with EVP-6124 showed clinically meaningful and statistically significant effects in key secondary endpoints: improvement in clinical function (as assessed by the Schizophrenia Cognition Rating Scale (SCoRS)) and reduction of the negative symptoms of schizophrenia (as measured by the Negative Symptom Scale of the Positive and Negative Symptoms Scale (PANSS)). Importantly, EVP-6124 was generally safe and well-tolerated over the trial’s three-month dosing period.

“The often devastating effect of schizophrenia on the vocational and social function of patients, and the toll it takes on their families and society is undeniable. Much evidence points towards cognitive dysfunction as the principal cause of the functional decline in most patients. Hallucinations and delusions impact function to a lesser extent than does cognition. Present therapies address these symptoms but have a significantly weaker effect on the cognitive deficit. Thus, the effort to improve outcomes in schizophrenia has focused on developing safe and effective treatments for cognitive impairment, expecting them to lead to improved function and quality of life,” said Herbert Y Meltzer, MD, PhD, the Bixler/May/Johnson professor of psychiatry at Vanderbilt University. “EVP-6124 represents a novel, rational approach, supported by extensive basic research, to improve cognition in schizophrenia. This initial clinical trial is highly encouraging that this novel approach has the potential to address what many consider the greatest unmet pharmacologic need for 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 US is more than $62 billion according to a study published in the Journal of Clinical Psychiatry.

“We are extremely excited by these data, which show a compelling and positive impact on a wide range of schizophrenia patients’ cognitive functions as measured by the CogState overall cognition index. Additionally, the ability of EVP-6124 to reduce schizophrenia’s debilitating negative symptoms further illustrates the drug’s promise and broad potential,” said Kees Been, president and chief executive officer of EnVivo Pharmaceuticals. “There are no currently approved therapies to treat cognitive and negative symptoms of the disease, so the opportunity to potentially address both cognition and negative symptoms is exciting and something that we will carefully evaluate as we move forward into later stages of development.”

Dana Hilt, MD, senior vice president, clinical development and chief medical officer of EnVivo added, “We are committed to presenting more complete data in a peer reviewed forum and look forward to the opportunity to present detailed trial results at an upcoming medical conference. We are excited to continue to advance this program as well as obtain data from our ongoing phase II b trial of EVP-6124 in Alzheimer’s disease in the beginning of 2012.”

This phase II b trial evaluated the safety and efficacy of two doses of EVP-6124 (0.3 mg and 1.0 mg per day) versus placebo in chronic schizophrenia patients on stable second-generation anti-psychotic drugs (except clozapine). Each patient was treated for three months and a total of 319 patients were enrolled in the US, Russia, Ukraine and Serbia. The trial’s primary endpoint was overall cognition as measured by the CogState overall cognitive index and important secondary endpoints included cognition as assessed by the MCCB battery, clinical function (as measured by the SCoRS) and positive and negative symptoms (measured by PANSS). Safety and tolerability were also assessed.

The EnVivo Pharmaceuticals group, consisting of EnVivo Pharmaceuticals, Inc., and its subsidiaries, is a drug discovery and development company working to convert its world-class pipeline into a broad range of Central Nervous System (CNS) therapies and deliver novel treatments to patients. Its focus is on building an integrated company that leverages novel mechanisms of action to treat CNS diseases like schizophrenia and Alzheimer’s disease by altering the progression of disease and providing improvement in cognitive and overall function.