FORUM Pharmaceuticals Inc. Receives FDA Fast Track Designation for Lead Compound Encenicline to Treat Cognitive Impairment in Schizophrenia

- No Treatment Currently Available for Condition Affecting Nearly All Schizophrenia Patients -

June 22, 2015 08:30 AM Eastern Daylight Time
WALTHAM, Mass.--(BUSINESS WIRE)--FORUM Pharmaceuticals Inc., a biopharmaceutical company singularly focused on the development and delivery of innovative medicines to treat serious brain diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to encenicline, FORUM's lead compound in development to treat cognitive impairment in schizophrenia. The FDA's Fast Track program is designed to facilitate the development and expedite the review of drug candidates intended to treat serious or life-threatening conditions, and to demonstrate the potential to address unmet medical needs. Encenicline is a novel alpha 7 agonist in Phase 3 development by FORUM to treat cognitive impairment in schizophrenia and Alzheimer's disease. The Company recently completed patient enrollment in its pivotal Phase 3 COGNITIV SZ clinical trial program investigating the use of encenicline as a pro-cognitive therapy in patients with schizophrenia who are currently being treated with atypical antipsychotics.

"FORUM's ongoing Phase 3 COGNITIV SZ clinical trial program recently completed recruitment of more than 1,500 patients, and we expect to announce top-line results from those trials in the first half of 2016"

"We are pleased that the FDA has awarded Fast Track designation for our encenicline development program in schizophrenia, as we believe that it acknowledges the unmet need for a pro-cognitive therapy in patients with this disease," said Deborah Dunsire, M.D., President and Chief Executive Officer of FORUM Pharmaceuticals. "Cognitive impairment is a debilitating condition that can severely impact patients' ability to function effectively in activities of daily living, such as the ability to live independently, to work or attend school, to have relationships and to participate in social interactions and the community. Nearly all patients with schizophrenia are affected with cognitive impairment and no therapy is currently available."

"FORUM's ongoing Phase 3 COGNITIV SZ clinical trial program recently completed recruitment of more than 1,500 patients, and we expect to announce top-line results from those trials in the first half of 2016," continued Dr. Dunsire. "Encenicline is also being investigated to treat Alzheimer's disease, and patient enrollment is well underway in our ongoing multinational Phase 3 COGNITIV AD clinical trial program."

About the Encenicline Phase 3 Clinical Trial Program in Schizophrenia (COGNITIV SZ)

The COGNITIV SZ Phase 3 clinical trial program includes two randomized, double-blind, placebo-controlled studies evaluating the safety and efficacy of two oral doses of once daily treatment with encenicline as a pro-cognitive treatment compared to placebo when added to chronic, stable, atypical antipsychotic therapy in people with schizophrenia. 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™), 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 as measured by the 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. The two global 26-week trials enrolled a total of more than 1,500 patients at approximately 200 clinical sites.

About Encenicline

Encenicline is a novel, orally administered, highly brain-penetrant, selective, and potent agonist of the alpha 7 receptor found in hippocampal and cortical neurons involved in cognition. Encenicline is believed to work in low concentrations to prime the alpha 7 receptor to increase its response to acetylcholine, a naturally occurring neurotransmitter. This effect is thought to activate brain networks associated with sensory gating, attention, and cognition—thus enhancing these networks for improved neural processing and improved cognitive performance in areas such as memory and executive function. Encenicline is in development by FORUM Pharmaceuticals as a long-term treatment to improve cognitive function with sustained effect in schizophrenia and Alzheimer's disease. In preclinical studies, alpha 7 agonists increased neuronal synchronization and enhanced synaptic plasticity, measured by an increase in long term potentiation (LTP). These effects may be linked to enhanced cognition not only in Alzheimer's disease and schizophrenia, but suggest potential in other diseases with cognitive impairment.

Encenicline is the first alpha 7 agonist drug candidate to reach pivotal Phase 3 development. Two Phase 3 registration multi-center clinical trials (COGNITIV SZ) investigating the use of encenicline for cognitive impairment in schizophrenia have recently completed enrollment (www.clinicaltrials.gov registration numbers NCT01714661 and NCT01716975). Additionally, FORUM is investigating the use of encenicline for Alzheimer's disease through a Special Protocol Assessment with the FDA. Two Phase 3 registration multi-center trials (COGNITIV AD) investigating the use of encenicline for Alzheimer's disease are ongoing (www.clinicaltrials.gov registration numbers NCT01969123 and NCT01969136). FORUM has a license agreement with Mitsubishi Tanabe Pharma Corporation (MTPC) for the development and commercialization of encenicline in Alzheimer's disease in Japan and several other Asian countries.

About Schizophrenia

Schizophrenia is a psychiatric disorder that affects approximately 2.2 million Americans, or about one percent of the adult population worldwide, and is usually diagnosed between 15 and 35 years of age. Symptoms of schizophrenia typically include positive and negative symptoms and 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. Cognitive impairments such as difficulty in attention, memory loss and problems processing information and decision-making are recognized as core disabling symptoms of schizophrenia linked to the functional impairment in this condition. The overall annual cost of schizophrenia in the U.S. is estimated at more than $62 billion, according to a study published in the Journal of Clinical Psychiatry.

About FORUM Pharmaceuticals Inc.

FORUM Pharmaceuticals Inc. ("FORUM Pharmaceuticals" or "FORUM") is dedicated to developing groundbreaking medicines in support of all those who are affected by or caring for people with serious brain disease, to help them live fuller, more enriching lives. The Company's robust and diverse pipeline is focused on discovering and developing new treatments for important neurodegenerative diseases that explore novel mechanisms of action to potentially alter the progression of brain disease and provide improvement in cognitive and overall function. FORUM's lead compound, encenicline, is currently being evaluated in separate ongoing Phase 3 clinical trial programs: COGNITIV SZ, which looks to improve cognitive impairment in patients with schizophrenia, and COGNITIV AD, which aims to improve cognition in patients with Alzheimer's disease. The Company is also developing genetically targeted therapies, including FRM-0334, for the potential treatment of the ultra-orphan, rare disease frontotemporal dementia. Privately owned, FORUM Pharmaceuticals is based in Waltham, Mass. For more information about FORUM, visit www.forumpharma.com.

FORUM Pharmaceuticals and the FORUM octagon logo are trademarks of FORUM Pharmaceuticals Inc.

Contacts
Pure Communications, Inc.
Katie Engleman, 910-509-3977
[email protected]

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