SP-304 - Guanylate Cyclase C (GC-C)

SP-304

SP-304 is a synthetic analog of uroguanylin, a natural gastro-intestinal hormone that is produced in the gastro-intestinal (GI) tract and is a key regulator of intestinal function. Uroguanylin works by activating a unique receptor on intestinal epithelial cells. The receptor, called the guanylate cyclase C (GC-C) receptor, promotes fluid and ion transport in the GI tract. Under normal conditions, the receptor is activated by the natural hormones uroguanylin and guanylin. Activation of the receptor leads to the transport of chloride and bicarbonate into the intestine. Water is carried with these ions into the lumen of the intestine, thereby producing a looser stool.

SP-304 acts in an identical manner as the natural hormone – as an agonist (i.e. activator) of the GC-C receptor and is administered orally. SP-304 remains in the gut, and is not systemically absorbed into the body, exerting its effect on GC-C receptors in the gut lining. As a result, SP-304 is anticipated to be very safe in clinical use.

Compound class

GC-C receptor agonist are a new class of drugs for gastrointestinal diseases. SP-304 is only one of two compounds in this class. There is strong pre-clinical and clinical evidence of safety, tolerability and efficacy for this class of compounds. SP-304 has demonstrated a good safety, tolerability and efficacy profile in the initial phase I trial in healthy volunteers.

Clinical development

Synergy is developing SP-304 for chronic constipation and constipation predominant irritable bowel syndrome (IBS-C). These are very common GI conditions with few treatments available. Synergy believes that SP-304 will prove to be the best compound in this drug class.

SP-304 is presently in early phase clinical development. The initial phase I clinical trial was completed in Augusut 2008. Results from this study will be presented at the 2009 Digestive Disease Week in Chicago, IL.

Intellectual property

Synergy has a strong intellectual property estate for SP-304. Our key patent, US7041786, issued on May 9, 2006 provides composition of matter protection on SP-304.

Market

The market for functional gastrointestinal disorders is one of the largest untapped pharmaceutical markets remaining. There are up to 35 million people in the Unites States that suffer from chronic constipation and IBS-C. Of those, about 25% have a severe condition. The prevalence is similar in other developed countries. In the US, there is at present, only one prescription product approved for these conditions.