Primary sclerosing cholangitis (PSC) is a progressive, rare, and chronic cholestatic liver disorder that is characterized by thickening, inflammation, and fibrosis of the bile ducts. Both intra- and extra-hepatic bile ducts are affected. This generally leads to cholestasis, liver cirrhosis, and eventually liver failure.

Key Takeaways

• Datamonitor Healthcare estimates that in 2017, there were 232,900 prevalent cases of primary sclerosing cholangitis (PSC) worldwide among adults aged 30 years and over, and forecasts that number to increase to 270,300 prevalent cases by 2026.
• The majority of industry-sponsored drugs in active clinical development for PSC are in Phase II, with only one drug in Phase III. Therapies in the pipeline for PSC focus on a wide variety of targets. The majority of pipeline drugs in development are administered via the oral route, with the remainder being tested in intravenous and subcutaneous formulations.
• The only high-impact upcoming event for drugs in the PSC space is topline Phase II trial results for PLN-74809. The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 21.4%, and the average probability a drug advances from Phase III is 70%. Drugs, on average, take 10.0 years from Phase I to approval, compared to 8.8 years in the overall autoimmune/immunology space.
• The distribution of clinical trials across Phase I–III indicates that the majority of trials for PSC have been in the early and midphases of development, with 78% of trials in Phase I–II, and only 22% of trials in Phase III–IV.
• The US leads in terms of the number of PSC clinical trials globally, while the UK leads the major EU markets.
• In the PSC space, Gilead Sciences has two completed trials, and one ongoing trial.
• Gilead Sciences has conducted two Phase II trials for PSC, and one Phase III trial.