WINSTON-Targacept says it has successfully completed Phase I clinical trials with Dr. Falk Pharma to evaluate a new drug that targets chronic, debilitating diseases such as ulcerative colitis.

The drug is calledTC-2403-12, and it’s described as a neuronal nicotinic receptor agonist. It was derived from Targacept’s library of compounds.

TC-2403-12 is one of a new class of drugs that selectively modulates therapeutic targets in the human body known as neuronal nicotinic receptors. These receptors maintain and adjust nervous system activity and have been associated with many chronic, debilitating diseases, such as ulcerative colitis.

Targacept, which is based in Winston-Salem, and German company Dr. Falk formed an alliance in 2001 to develop a new class of drugs to treat ulcerative colitis, a relapsing and remitting disease that affects the innermost lining of the colon. The disease causes numerous debilitating effects on the gastrointestinal system. Symptoms include abdominal pain, diarrhea, fatigue, loss of appetite and loss of body fluids and nutrients.

No significant tolerability or safety concerns were suggested by either the single rising dose study or the 14-day multiple rising dose study of TC- 2403-12 dosed in an enema formulation to healthy volunteers, Targacept says. A Phase II placebo-controlled, dose-range-finding trial for proof of therapeutic concept is planned to start in the United States in early 2003, the company adds, with an oral, delayed release formulation of TC-2403-12 in active development.

“This compound has the potential to become the first new class of drug in the past 50 years targeted to treat ulcerative colitis,” Donald deBethizy, president and chief executive officer of Targacept, said in a statement. “There is tremendous need for a new treatment to help ulcerative colitis patients achieve a better quality of life.”


Dr. Falk Pharma: