Conatus Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Conatus' drug candidate IDN-7314 for the treatment of primary sclerosing cholangitis (PSC), a disease affecting bile ducts in the liver which can lead to cirrhosis and liver failure. The FDA's Orphan Drug Designation program is intended to encourage the development of drugs and biologics that may provide benefit to patients suffering from rare diseases or conditions.
IDN-7314 is an orally active pan-caspase protease inhibitor designed to reduce the activity of enzymes that mediate inflammation and cell death (or apoptosis), which has demonstrated reduction of relevant biomarkers in two preclinical models of PSC. One nonclinical model, the Mdr2-/- (knockout) mouse model, is considered the current benchmark nonclinical model of PSC. A new preclinical model, second mitochondria-derived activator of caspases (SMAC)-mimetic induced PSC in mice, has recently been reported that reproduces much of the phenotype of human PSC. IDN-7314 significantly improved biochemical indices of hepatic and biliary damage in these murine models of PSC, and these results suggest the involvement of caspases in the progression of PSC.
"The FDA's Orphan Drug Designation of IDN-7314 for the treatment of PSC highlights the need for novel therapies for this patient population," said Conatus Co-Founder, President and Chief Executive Officer, Steven J. Mento, Ph.D. "There are no curative or disease-modifying treatments for PSC itself. Continued progression over time ultimately leads to liver transplant or liver failure. For Conatus, the Orphan Drug Designation provides a potential opportunity to address an important unmet medical need and expand the company's drug development pipeline beyond emricasan. We will continue to evaluate this opportunity along with others as we advance toward announcement of our initial pipeline plans later in 2017."