Positive proof-of-concept trial of Immunic's IMU-838 in PSC

US clinical-stage biopharma company Immunic (Nasdaq: IMUX) today announced positive top-line data from an investigator-sponsored Phase II proof-of-concept clinical trial of IMU-838 in primary sclerosing cholangitis (PSC), an uncommon live disease.

This single-arm, open-label, exploratory study was designed to investigate IMU-838's potential to improve various biochemical parameters in PSC patients and help determine whether any such activity warrants further investigation in randomized PSC trials. As previously announced, due to the COVID-19 pandemic, only 18 of the targeted 30 patients were enrolled in the study (intent-to-treat population, ITT), of whom only 11 patients completed the full IMU-838 treatment course and were evaluable over the 24-week treatment period (per-protocol population, PP).

The PP population experienced a statistically-significant decrease in serum alkaline phosphatase (ALP) levels (p=0.041) after 24 weeks of treatment using 30mg IMU-838 once daily, as compared to baseline. A consistent individual pattern of a stable decrease in ALP values was observed in the PP population between baseline and week 24, without any single patient showing an increase of more than 20% of ALP. As per the definition of the primary objective of the study, 27.3% of the patients in the PP population had a clinically relevant reduction of serum ALP higher than 25% at week 24, without an increase in liver biochemistry of more than 33%, as compared to baseline. Biochemical endpoints, such as changes in serum ALP, have been used in