Albireo Reports Fourth Quarter and Year-End 2019 Financial Results, and Provides Business Update

— Phase 3 odevixibat PFIC trial fully enrolled —
— Initiating pivotal trials in biliary atresia first half of 2020 and Alagille syndrome by year end —— Management to host conference call and webcast today at 10:00 a.m. ET —BOSTON, March 02, 2020 (GLOBE NEWSWIRE) — Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today provided a business update and reported financial results for the fourth quarter and year ended December 31, 2019.“In 2019, we took major steps forward in multiple development programs, advanced commercial preparations and evolved as an organization, laying the groundwork for Albireo to enter the next stage,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We have always believed in odevixibat’s potential across a range of cholestatic liver diseases, and 2020 will be a transformational year, as we expect results from our Phase 3 trial in PFIC, look forward to initiating a pivotal trial in biliary atresia and plan to begin a pivotal trial in Alagille syndrome following discussions with the FDA this quarter. Meanwhile, we eagerly anticipate the results from the elobixibat NASH/NAFLD study and are excited to have selected a lead candidate to advance from our robust library of preclinical compounds.”Recent HighlightsOdevixibatCompleted full randomization of the PEDFIC 1 Phase 3 study in progressive familial intrahepatic cholestasis (PFIC). The PEDFIC 1 trial is studying odevixibat in both PFIC type 1 and type 2 patients aged 6 months to 18 years. Patients randomized to odevixibat are being treated with high- (120µg/kg) or low- (40µg/kg) dose once-daily oral capsules or sprinkles. The trial uses Albireo’s planned commercial formulation of odevixibat, which does not require refrigeration. Albireo expects topline results mid-2020, and anticipates a potential first regulatory approval and launch in the second half of 2021.
 Continued enrolling patients in PEDFIC 2, the long-term, open-label extension study of PEDFIC 1. Cohort 1 of PEDFIC 2 is composed of patients who have rolled over from the PEDFIC 1 study. Albireo now has patients who have been treated with odevixibat for more than 1 year. Cohort 2 is expected to broaden the evidence base for odevixibat by including PFIC patients who were ineligible for PEDFIC 1. This includes patients with all types of PFIC, and patients younger than 6 months or older than 18 years of age.
 Received clearance from the U.S. Food and Drug Administration (FDA) for the company’s Investigational New Drug (IND) application to initiate a precedent-setting global pivotal trial in biliary atresia following agreement on a study design. Biliary atresia is a rare pediatric liver disease that is the leading cause of liver transplants among children, and for which there is no approved pharmacological therapy. The double-blind, placebo-controlled trial is designed to enroll approximately 200 patients at 70 sites. Patients will receive either placebo or high-dose (120µg/kg) odevixibat once daily. The primary endpoint is survival with native liver after 2 years of treatment. The FDA and European Commission have granted orphan designations for odevixibat in the treatment of biliary atresia. Albireo plans to initiate the trial in 1H 2020.
 Plans to commence an additional pivotal program in Alagille syndrome by the end of 2020, following scheduled FDA interactions in the first quarter. The FDA and the European Commission have granted orphan designations for odevixibat in the treatment of Alagille syndrome.
 Advanced preparations for commercialization across physician, patient and payer communities. The company initiated activities to develop a detailed understanding of treatment centers, continued to build the PFIC Voices initiative in collaboration with patients and families, and progressed the odevixibat value story and economic models for reimbursement.PipelineNearing completion of enrollment in the company’s Phase 2 multicenter, placebo-controlled clinical trial of elobixibat 5mg in non-alcoholic fatty liver disease (NAFLD)/nonalcoholic steatohepatitis (NASH). Albireo expects topline data by mid-2020. In addition, the company expects data from a second study being conducted in Japan with elobixibat 10mg and in combination with a bile acid sequestrant later this year or early 2021.
 Selected lead preclinical product candidate, filed for intellectual property protection and initiated IND-enabling studies. Fourth Quarter 2019 Financial ResultsRevenues were $6.4 million for the fourth quarter of 2019, compared to $0.6 million for the fourth quarter of 2018. The year-over-year increase was primarily the result of estimated milestone royalty revenue received from EA Pharma for elobixibat for the treatment of chronic constipation.
 R&D expense was $14.2 million for the fourth quarter of 2019, compared to $9.5 million for the fourth quarter of 2018. The year-over-year change was primarily the result of program expenses for odevixibat and elobixibat, as well as personnel costs, as we continue to increase our program activities and headcount.
 G&A expense was $6.2 million for the fourth quarter of 2019, compared to $5.8 million for the fourth quarter of 2018. The year-over-year change was primarily the result of personnel and related expenses, as we continue to increase our headcount.
 Net loss for the fourth quarter of 2019 was $7.5 million, or $(0.57) per share, compared to $15.9 million, or $(1.34) per share for the fourth quarter of 2018.Financial Results for the Year Ended December 31, 2019Revenues were $9.6 million for the year ended December 31, 2019, compared to $12.7 million for the year ended December 31, 2018. The year-over-year decline is primarily due to a milestone payment received in 2018 from EA Pharma due to the approval by the Japanese MHLW of elobixibat for the treatment of chronic constipation offset by royalty revenue from EA Pharma.
 R&D expense was $45.6 million for 2019 up from $31.7 million for the same period in 2018. The year-over-year increase was primarily the result of program expenses for odevixibat, elobixibat and preclinical work, as well as personnel costs, as we continue to increase our program activities and headcount.
 G&A expense was $23.0 million for 2019, compared to $18.1 million for the previous year. The year-over-year increase was primarily the result of personnel and stock-based compensation, as we continue to increase our headcount.
 Net loss for the year ended December 31, 2019, was $62.7 million, or $(5.04) per share, compared to $46.1 million, or $(3.94) per share for the year ended December 31, 2018.
 The company had cash and cash equivalents at December 31, 2019, of $131.8 million, which compares to $163.9 million at December 31, 2018.Financial Guidance
For the full year 2020, the company anticipates total expenses, including R&D and G&A expenses, to be around $100M. Following a February 2020 common stock offering of $43.2 million, Albireo now anticipates that its current cash balance will be sufficient to meet its operating needs into the second half 2021.Conference Call
As previously announced, Albireo will host a conference call and webcast today, March 2, 2020, at 10:00 a.m. ET. To access the live conference call by phone, dial 877-407-0792 (domestic) or 201-689-8263  (international), and provide the access code 13697430. A live audio webcast will be accessible from the Media & Investors page of Albireo’s website, http://ir.albireopharma.com/. To ensure a timely connection to the webcast, it is recommended that participants register at least 15 minutes prior to the scheduled start time. An archived version of the webcast will be available for replay on the Events & Presentations section of the Media & Investors page of Albireo’s website for 3 months following the event.