Mirum Pharmaceuticals, Inc. presented data at the International Liver Congress™ of The European Association for the Study of the Liver (EASL) in London, United Kingdom. The first analysis featured a multinational survey of patient-reported outcomes showing the effects of pruritus in patients with primary sclerosing cholangitis (PSC). A second poster assessed gastrointestinal (GI) tolerability on time of dosing with ileal bile acid transporter inhibitors (IBATis) relative to meals.
“We are pleased to share new data at the 2022 EASL congress”
“We are pleased to share new data at the 2022 EASL congress,” said Dr. Pam Vig, head of research and development at Mirum. “The large multinational PSC survey shows that adults with PSC often experience significant and debilitating pruritus which impacts their day-to-day quality of life, despite use of off-label antipruritic medications. Thus, there remains an urgency for a treatment to address this most burdensome symptom, and we are excited to be evaluating the potential of volixibat, a minimally absorbed IBAT inhibitor, in patients with PSC.”
Summaries of data presented during the congress are enclosed below. View the presentations in full within our Publications & Presentations section on our website.
Poster 756: Impact of pruritus in primary sclerosing cholangitis (PSC): a multinational survey
By Dr. Kris Kowdley, et al.
The multinational survey, conducted by Hetz Israel, PSC Australia, PSC Partners, and PSC Support, assessed results from a collection of patient-reported outcomes from people living with PSC (n=482). The aim of the survey was to assess the presentation and severity of cholestatic pruritus and its broader burden on patients with this rare liver disease. The 39-question survey captured the age and gender of respondents as well as whether they also have inflammatory bowel disease. In addition to pruritus, inflammatory bowel disease and chronic liver disease associated with PSC can negatively impact the quality of life and can lead to depression and anxiety.
The survey results showed that:
Of the 91% of patients who reported experiencing itch following their diagnosis, 46.4% of respondents experienced >15 episodes (an episode is defined as a prolonged period of itching for at least 7 days).
Respondents were asked to rate the worst itch experienced in the last 24 hours, and the median worst itch was 6 out of 10 in those who responded (scale from 0-10). Their previous itching episode was reported as 8 out of 10.
Also Read: Datadog Launches Audit Trail to Help Businesses Achieve Their Compliance and Governance Objectives
31% of patients who reported having a current itching episode stated that they have been continuously itching for >12 months.
The survey assessed the overall impact of pruritus on respondents’ lives with responses indicating:
Itch was worst in the evening or nighttime (96%)
Mood changes including but not limited to anxiety, irritability, and feelings of hopelessness (58%)
Itch led to disruption of day-to-day activities (50%)
Itch lasted ≥1 month in duration (45%)
Disruption of daily responsibilities due to itch that lasted >30 days (32%), and reported it lasting >12 months (18%)
Missing school or work (22%)
Other findings showed that half of respondents (235/482) reported using ≥2 medications but 75% (177/235) described only partial or no relief with the interventions.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum’s approved medication is LIVMARLI® (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.