Sucampo Pharmaceuticals Inc has begun a multi-center, Phase IIa safety and efficacy study for the treatment of Cystic Fibrosis (CF) with its proprietary agent SPI-8811.

The Sucampo study will randomize 24-30 patients with documented CF into three, open-label, dose-escalating cohorts at four sites throughout the United States. At least eight subjects will complete dosing in each cohort. The company is working closely with the Cystic Fibrosis Therapeutic Development Network on the study. The network was established by the Cystic Fibrosis Foundation to conduct early-phase clinical studies (Phase I and II) with novel therapies for CF.

CF is caused by a defective chloride channel called the cystic fibrosis transmembrane regulator, which prevents the transport of chloride ions between cells. The defect causes the body to develop thick, sticky mucous in the lung, pancreas and liver, and increases morbidity and mortality in CF patients. SPI-8811 is an ion transport modulator that facilitates transport of chloride ions across cell membranes by a process different from the defective one in CF patients. The agent's ability to activate chloride transport should reduce mucous viscosity and allow increased clearance of mucous in the lungs, pancreas and liver.

"Cystic Fibrosis is a devastating disease with many dire effects," said Sucampo CEO Myra L. Patchen. "This research holds a special place in our organization and we hope that SPI-8811 will prove beneficial in treating this terrible disease."

According to the foundation, CF is a genetic disease that affects approximately 30,000 Americans; 70,000 worldwide. People with CF have difficulty absorbing nutrients from food and develop recurrent lung infections. These lung infections and the subsequent pulmonary damage are often fatal. The median age of survival for a person with CF is in the early thirties.

SPI-8811 received orphan drug status for use in treating CF from the U.S. Food and Drug Administration (FDA) in December 2002. The FDA designation encourages research and development of new therapies for the more than 5,000 orphan (rare) diseases that affect fewer than 200,000 U.S. residents, diseases that affect 20 million Americans in total.