In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment. In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.
This trial phase represents an early or specialized stage of clinical development.
Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.
Mirum Pharmaceuticals (MIRM) is the sponsoring company for this trial. BiotechSign currently grades this company C (56/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.