Primary Sclerosing Cholangitis (PSC) is chronic liver disease characterized by a progressive reduction or stoppage of bile flow with inflammation and fibrosis of the intrahepatic and extrahepatic bile ducts
Prevalence is 60 to 80 cases per 1 million population
Occurs mostly (~70%) in men
~75% of PSC patients also have IBD
Orphan indication with high unmet need
No approved treatment
Average life expectancy is 12-18 years without liver transplantation after diagnosis
An over $10B market opportunity (US and EU)
Beneficial effects of HTD1801 in PSC are expected to include:
● Hepatocyte protection
● Intestinal microbiota modification
Based on a rat bile duct ligation (BDL) model of cholestasis, HTD1801:
● Lowered the elevated serum ALP levels
● Ameliorated hepatocellular necrosis
● Reduced inflammation in the portal area
● Inhibited bile duct proliferation
● Increased antioxidative defense
● The “Market Spotlight: Primary Sclerosing Cholangitis” report (www.researchandmarkets.com) estimates that in 2016, there were 227,400 prevalent cases of primary
sclerosing cholangitis worldwide among adults aged 30 years and older, and forecasts that number to increase to 265,700 prevalent cases by 2025.
● In the US alone, the age-adjusted prevalance is estimated to be 4/100,000, resulting in approximately 13,000 cases.
● Globally, the prevalance appears to vary by population, ranging from 0-16.2/100,000.