Synedgen to Present New Treatment Options for CF complications at North American Cystic Fibrosis Conference

Innovative Treatments to Improve GI & Lung Health and Antibiotic Effectiveness in CF Patients

Cystic fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and results in dysfunction of the lungs and the gastrointestinal (GI) tract. In CF patients, abnormally viscous and sticky mucus accumulates in the lungs. This mucus is very difficult to clear, blocks the airways and the glands, and creates an environment where bacteria can easily grow.

Synedgen researchers are developing targeted therapies that address CF complications at the pulmonary and GI surfaces that currently have limited treatment options and often lead to early mortality.

Two presentations detailing this research have been accepted for presentation at the 29^th annual North American Cystic Fibrosis Conference October 7-10, 2015 in Phoenix AZ.

The first presentation will highlight the work led by Synedgen’s collaborator, Dr. Steven Rowe and his team at the University of Alabama, Birmingham. Their studies focus on the powerful effect of Synedgen’s drug PAAG SYGN303 to prevent Distal Intestinal Obstructive Syndrome (DIOS) in mice that carry the CF gene mutation.

DIOS, formerly called meconium ileus (MI) equivalent, is associated with severe morbidity and prolonged hospitalizations in CF patients. Physiological changes in the CF gut lead to the accumulation of viscous mucus and fecal material in the intestines, which form obstructing impactions. DIOS is a condition unique to cystic fibrosis, occurring in about 10-22% of patients, and has a recurrence rate of approximately 50%. Current treatments for CF intestinal disease are non-specific and often ineffective.

Dr. Rowe’s team demonstrates that SYGN303 effectively reduces mucus impaction in the CF intestine, reduces inflammation and maintains healthy intestinal tissue while reducing the overall mortality by 90% when compared to the control. “This is a completely new approach to treating GI disease in cystic fibrosis,” commented Synedgen CEO Dr. William Wiesmann. “A targeted therapy for GI disease will meet a large unmet need for patients who suffer from DIOS and related complications.”

Synedgen VP of Research Dr. Stacy Townsend will highlight Synedgen’s work using PAAG SYGN113 to prevent Pseudomonas aeruginosa attachment to lung epithelial cells, and its ability to inhibit and remove biofilms from these cells. The bacteria tested are clinical isolates from CF patients that have multi-drug resistance and mucoid characteristics. P. aeruginosa infections are among the most common associated with CF.

Complications due to biofilm infections continue to rise primarily due to multi-drug resistance and the inability of current therapies to penetrate thick mucus and biofilms. Recurring infections exacerbate CF symptoms, cause a decline in pulmonary lung function, and increase morbidity and mortality. Dr. Townsend’s work shows that a single 4-hour treatment with SYGN113 significantly reduced multiple strains of P. aeruginosa biofilms from the lung epithelial cell surface from 87-100%. Pretreatment of lung cells with SYGN113 resulted in 100 times less bacteria attached to the surface of the lung cells after 1-hour. Extending treatment over 5-hours caused an 82-98% reduction in biofilm formation on the lung epithelial cells.

“The results of these two studies demonstrate new mechanisms of action for SYGN303 and SYGN113 relative to current standards of care,” stated Synedgen President Shenda Baker. “The ability to improve CF GI health and inhibit and remove biofilm growth provides a dramatic improvement over current drugs for treating mucus impaction, infection and exacerbations in CF patients.”

About Synedgen
Synedgen Inc. is an innovative biopharmaceutical company focused on targeting the mucosal interface to treat genetic and acquired disorders in the lungs and digestive tract. Synedgen’s Glycomics Technology Platform produces targeted drugs to meet unmet needs in treating inflammation, infection and mucosal damage. Synedgen’s Corporate Headquarters, Research Laboratories and Manufacturing Facility are in Claremont CA. Additional information can be found at Synedgen’s web site at www.synedgen.com.

About the North American Cystic Fibrosis Conference
The North American Cystic Fibrosis Conference (NACFC) provides a collaborative and educational forum for CF professionals to help advance CF research and care. This annual meeting brings together scientists, clinicians and caregivers from around the world to discuss and share ideas on the latest advances in CF research, care and drug development and to exchange ideas about ways to improve the health and quality of life for people with CF. More than 4,000 researchers and health care professionals will attend the Meeting.