Albireo Pharma, Inc. (NASDAQ:ALBO) Q3 2019 Earnings Conference Call - Final Transcript
Nov 06, 2019 • 10:00 am ET
to provide patients with a well-tolerated treatment option that can reduce bile acid ease their pruritis and hopefully prevent or postpone a liver transplant. PEDFIC-1 is a randomized double-blind placebo-controlled clinical trial that is evaluating odevixibat in 60 patients ages six months to 18 years with PFIG type 1 or 2. Patients are receiving either 40 or 120 microgram per kilogram oral dose of the planned commercial formulation of odevixibat or placebo once-daily for 24 weeks.
The primary endpoint for the FDA evaluation is an assessment of the change in pruritis. The primary endpoint for the EMA is the serum bile acid responder rate. Enrollment in PEDFIC-1 has progressed well since our summer update. We continue to be encouraged by the number of patients who've been entering screening at the over 40 actively recruiting sites around the world. As of the end of October we'd randomized more than three-quarters of the patients needed to complete the trial. It's also important to note that we believe there are adequate additional patients in screening or prescreening today to complete randomization in this trial. Now we plan to announce when randomization is finished in the trial and we remain confident that we'll be in position to report top-line results from the trial in mid-2020. PFIG is an ultra-rare disease and the exact timing of the top line results will be determined by the last few patients. Assuming a positive outcome for this study that will put us firmly on track for potential end launch in 2021.
Meanwhile PEDFIC-1 patients have been rolling over to the voluntary extension portion of the study which we refer to as cohort 1 or the rollover cohort of our PEDFIC-2 trial. We now have patients who are approaching a year on odevixibat treatment. All patients in this 72-week extension trial receive the higher dose of odevixibat 120 micrograms per kilogram daily. In the third quarter we also screened our first patient in cohort 2 or the expanded cohort of PEDFIC-2. The expanded cohort is composed of PFIG patients who do not meet the eligibility criteria for PEDFIC-1 but who still have elevated serum bile acid levels and pruritis. This includes patients with all types of PFIG and patients younger than six months or older than 18 years of age. The second cohort reflects our commitment to providing a potential new treatment option for many different types of patients with PFIG if possible while maximizing the body of data and evidence for odevixibat. The new set of patients will be analyzed separately without affecting cohort one of PEDFIC-2.
Now as we continue to make great headway with our robust phase 3 program we also are deepening our engagement with key opinion leaders to provide critical feedback on our plans for odevixibat. For instance at the AASLD liver meeting which is taking place here in Boston this week we have multiple events that engage preeminent cholestatic liver disease clinicians from the U.S. Europe and around the world. We're advancing our