In addition to IMAGO, two larger placebo-controlled phase 2 studies in ALGS are in progress, one of which has pruritus as the primary endpoint.
IMAGO is a phase 2 multicenter, randomized, placebo-controlled study of children with ALGS, 1-18 years of age, conducted in the United Kingdom.
Indication US prevalence EU prevalence 3 per 100,000 ALGS
9476 individuals 3 per 100,000 <1 per 100,000 PFIC 3159 individuals 1 per 100,000 40 per 100,000 PBC 126,979 individuals 30 per 100,000 14 per 100,000 PSC 44,222 individuals 3 per 100,000
Lumena's lead product candidate, LUM001, has been studied in more than 1,400 human subjects in 12 different clinical trials evaluating the product candidate as a treatment for elevated cholesterol levels, and is being evaluated in Phase 2 clinical trials in patients with rare cholestatic liver diseases, including ALGS
, PFIC, PBC and PSC.
Lumena is currently evaluating LUM001 in a Phase II study in children with ALGS and a Phase II study in adults with PBC.
About Alagille Syndrome ALGS is a rare genetic disorder that can affect the liver, heart, and other parts of the body.
Currently, there are no approved drugs to treat the cholestasis and pruritus associated with ALGS, and frequent options for children suffering with this disease are off-label treatments with limited efficacy and potential liver toxicity or surgical diversion of bile, which comes with risk of complications and quality of life issues.
based study which is expected to begin later in 2013 and will be seeking pediatric patients, two years of age and older, with ALGS.