ALBO Albireo Pharma Inc

Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced achievement of full patient enrollment in its Phase 2 clinical trial of elobixibat 5mg, a first-in-class, once-daily, orally available ileal bile acid transporter (IBAT) inhibitor for the treatment of nonalcoholic steatohepatitis (NASH) and nonalcoholic fatty liver disease (NAFLD).

Albireo is on track to report topline results in mid-2020. Albireo expects results from a second NASH/NAFLD trial with elobixibat, sponsored by its Japanese partner EA Pharma, late this year or early next year. These two proof-of-concept studies are designed to assess the combination of improvements in liver function and cardiovascular risk parameters with a favorable gastrointestinal tolerability profile. The combined data from both studies will inform next steps for Albireo. 

“This is the first clinical trial ever conducted in both NASH and NAFLD patients with an IBAT inhibitor,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We are pleased to have reached this critical milestone, which puts us on track to report topline data by the middle of this year. Our clinical and preclinical data indicate there is a strong rationale for bile acid modulation by IBAT inhibition as a therapeutic approach in NASH, given that this approach could have a positive effect on a combination of parameters like lipids, glucose, liver inflammation, liver fibrosis and elevated bile acids, a favorable tolerability profile, and once-a-day oral dosing for ease of use.”

The proof-of-concept Phase 2, multicenter, placebo-controlled trial enrolled 47 patients, and is designed to assess the safety and efficacy of a once-daily 5mg dose of elobixibat over 16 weeks in adult patients across 10 U.S. sites with biopsy-confirmed NASH, or a diagnosis of suspected NASH or NAFLD based on metabolic syndrome definitions. The primary endpoint is change from baseline in serum low-density lipoprotein cholesterol (LDL-C). Secondary endpoints include change in liver fat by imaging, and in alanine transaminase (ALT) and aspartate aminotransferase (AST). Exploratory endpoints include measures of glucose and insulin homeostasis, biomarkers for inflammation and fibrosis.

In addition, EA Pharma, the exclusive licensee of elobixibat for the treatment of GI disorders in Japan and select other countries in Asia, has sponsored an investigator-sponsored trial of elobixibat in Japan. The 16-week trial will enroll 100 NASH/NAFLD patients randomized to a once-daily 10mg dose of elobixibat, the bile acid sequestrant cholestyramine, the combination of cholestyramine and elobixibat 10mg, or placebo, with topline data anticipated by late 2020 or early 2021.

Albireo’s adult liver disease program also includes a lead product candidate that is a novel investigational preclinical bile acid modulator, for which the company expects to complete IND-enabling studies this year.

“We believe that, if successful, our approach to NASH/NAFLD could offer an effective and novel therapeutic approach as either monotherapy or combination therapy with a favorable safety profile in an area of massive unmet need,” Cooper said. “While we remain focused on advancing odevixibat in progressive familial intrahepatic cholestasis (PFIC) and other rare pediatric cholestatic liver diseases, our NASH/NAFLD program has potentially strong value for partnering opportunities.”

NAFLD affects about 25 percent of the world’s population and is a major cause of liver disease. NAFLD may progress to NASH, a common, serious and sometimes fatal chronic liver disease characterized by liver inflammation and damage caused by a buildup of fat in the liver. NASH patients have a 10-fold greater risk of liver-related mortality, compared with the general population, and NASH is projected to become the leading cause of liver transplants in the United States. Albireo estimates that NASH affects more than 9 million people in the United States and 10 million people in the European Union. There is currently no approved pharmacologic treatment for NASH.

About Elobixibat Elobixibat is a first-in-class, once-daily, orally-available ileal bile acid transporter (IBAT) inhibitor currently being evaluated in a Phase 2 clinical trial in nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH). In clinical studies, elobixibat demonstrated a decrease in LDL/H cholesterol, as well as decreased insulin resistance through an increase in GLP-1. The first IBAT inhibitor approved globally, elobixibat is approved in Japan for the treatment of patients with chronic constipation (excluding constipation caused by organic disease). It is marketed and sold in Japan under the trade name GOOFICE®.

About Albireo  Albireo Pharma is a clinical-stage biopharmaceutical company focused on the development of novel bile acid modulators to treat orphan pediatric liver diseases, and other liver and gastrointestinal diseases and disorders. Albireo’s lead product candidate, odevixibat, is being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in its initial target indication, progressive familial intrahepatic cholestasis (PFIC). Albireo’s clinical pipeline also includes two Phase 2 product candidates. Elobixibat is in Phase 2 development in NAFLD and NASH. Approved in Japan for the treatment of chronic constipation, elobixibat is the first ileal bile acid transporter (IBAT) inhibitor approved anywhere in the world.

Albireo was spun out from AstraZeneca in 2008. Albireo Pharma is located in Boston, Mass., and its key operating subsidiary is located in Gothenburg, Sweden. The Boston Business Journal named Albireo one of the 2019 Best Places to Work in Massachusetts. For more information on Albireo, please visit www.albireopharma.com.

Forward-Looking Statements This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding, among other things: the plans for, or progress, scope, cost, initiation, duration, enrollment,  results or timing for availability of results of, development of odevixibat, elobixibat or any other Albireo product candidate or program, including regarding the Phase 3 clinical program for odevixibat in patients with PFIC; the Phase 2 clinical trial for elobixibat in NAFLD/NASH, the target indication(s) for development, the size, design, population, location, conduct, objective, enrollment, duration or endpoints of any clinical trial, or the timing for initiation or completion of or reporting of results from any clinical trial, including the double-blind Phase 3 PFIC trial for odevixibat, and the long-term open-label extension study, or the Phase 2 trial for elobixibat in NAFLD/NASH; the potential approval and commercialization of odevixibat; discussions with the FDA regarding our programs; the potential benefits or competitive position of odevixibat, elobixibat, or any other Albireo product candidate or program or the commercial opportunity in any target indication; the potential benefits of an orphan drug designation; the pricing of odevixibat if approved; or Albireo’s plans, expectations or future operations, financial position, revenues, costs or expenses. Albireo often uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “planned,” “continue,” “guidance,” and similar expressions to identify forward-looking statements. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to: whether favorable findings from clinical trials of odevixibat to date, including findings in indications other than PFIC, will be predictive of results from the trials comprising the Phase 3 PFIC program or any other clinical trials of odevixibat; whether either or both of the FDA and EMA will determine that the primary endpoint for their respective evaluations and treatment duration of the double-blind Phase 3 trial in patients with PFIC are sufficient, even if the primary endpoint is met with statistical significance, to support approval of odevixibat in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or otherwise; the outcome and interpretation by regulatory authorities of the ongoing third-party study pooling and analyzing of long-term PFIC patient data; the timing for initiation or completion of, or for availability of data from, clinical trials of odevixibat, including the trials comprising the Phase 3 PFIC program; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or European Union; delays or other challenges in the recruitment of patients for, or the conduct of, the double-blind Phase 3 trial or other pivotal trials; and Albireo’s critical accounting policies. These and other risks and uncertainties that Albireo faces are described in greater detail under the heading “Risk Factors” in Albireo’s most recent Annual Report on Form 10-K or in subsequent filings that it makes with the Securities and Exchange Commission. As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur. Albireo cautions you not to place undue reliance on any forward-looking statement. In addition, any forward-looking statement in this press release represents Albireo’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Albireo disclaims any obligation to update any forward-looking statement, except as required by applicable law.

Investor Contact: Hans Vitzthum, LifeSci Advisors, LLC., 212-915-2568                                        Media Contact: Heather Anderson, 6 Degrees, 919-827-5539, handerson@6degreespr.com