Phase III MARCH study evaluating Livmarli shows benefits in progressive familial intrahepatic cholestasis

The primary endpoint of improvement in pruritus severity in PFIC2 was statistically significant (p=0.0098). The primary analysis was conducted in PFIC2 patients (n=31). The secondary analyses were evaluated in the All-PFIC cohort, which included PFIC2 as well as additional PFIC subtypes (n=64). The Full-Study population included All-PFIC as well as supplemental patients who had previously undergone surgery, had truncating mutations and other patients (n=93).

The results for the Primary endpoint, the change from baseline in ItchRO(Obs) severity, was -1.7 for Livmarli and -0.6 for placebo. The results for the secondary endpoint, the change from baseline in serum bile acid, was -176 for Livmarli and 11 for placebo. Livmarli’s safety and tolerability profile was comparable to previously published data and no new safety signals emerged in the MARCH study. The most common treatment emergent adverse event was diarrhea, which was predominantly mild, with no severe cases, and transient with a median duration of 5.5 days. One patient had a treatment emergent adverse event of mild diarrhea that led to discontinuation.