ALBO Albireo Pharma Inc

Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, today announced late-breaker acceptances by the American Association for the Study of Liver Diseases (AASLD) for the Phase 3 PEDFIC 1 trial and PEDFIC 2 long-term extension trial. There will be an oral presentation of the results from PEDFIC 1, the global Phase 3 clinical trial evaluating the efficacy and safety of odevixibat in children with progressive familial intrahepatic cholestasis (PFIC) in the late-breaker session. PEDFIC 2 is an open-label Phase 3 extension study of the long-term efficacy and safety of odevixibat in children with PFIC, and interim results will be presented as a late-breaker poster. In addition, data on novel bile acid modulator approaches and new product candidate A3907 have also been accepted for poster presentations. The AASLD The Liver Meeting Digital Experience™ (TLMdX) is being held November 13-16.

“PEDFIC 1 achieved very high statistical significance on both the U.S. and EU primary endpoints and we are eager to share the full results from the PEDFIC program to date. Odevixibat sustained reductions in bile acids and pruritus, improved growth parameters, and was well tolerated with very low incidence of diarrhea across a wide range of PFIC patients,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Our scientific leadership and pipeline execution will also shine with exciting data introducing multiple new bile acid modulator approaches focusing on our novel compound A3907 in adult liver diseases.”

Abstracts are now available on the AASLD website. Details of the late-breaking abstracts will be presented in November and published in the December issue of HEPATOLOGY:

Presentation #L04: Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2: Results from PEDFIC 1, a Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial

Session: Late-breaking Oral Abstract Session 1 with presentation by lead author Richard J. Thompson, M.D., Ph.D., Professor of Molecular Hepatology at King's College London and principal investigator of the studyDate & Time: Sunday, Nov. 15, 5:30 p.m. EST

Poster #LP19: Long-term Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children with Progressive Familial Intrahepatic Cholestasis: Interim Results from PEDFIC 2, an Open-label Phase 3 Trial

Session: Late-breaking Abstract Posters

Abstracts accepted earlier in the month were published in the October issue of HEPATOLOGY and included one abstract selected by TLMdX as a Poster of Distinction for being in the top 10 percent of scored poster abstracts.

Poster #348: Discovery and Characterization of Novel Bile Acid Transporter Inhibitors with Potential for Treatment of Liver Diseases

Session: Biliary Transport and Metabolism

Poster of Distinction #509: A3907, a Novel Inhibitor of Bile Acid Transport in the Intestine and Kidney, Improves Markers of Metabolic and Hepatic Pathology, and Reduces Nonalcoholic Fatty Liver Disease Activity Score and Fibrosis Stage in a Diet-induced and Biopsy-confirmed Mouse Model of Nonalcoholic Steatohepatitis

Session: Experimental NAFLD and NASH

About OdevixibatOdevixibat is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. A highly potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), odevixibat acts locally in the small intestine. Odevixibat does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population. Albireo conducted the largest ever global Phase 3 trial in PFIC1 and PFIC2. The PEDFIC 1 trial evaluated odevixibat 40 µg/kg/day or 120 µg/kg/day or placebo in 62 patients, ages 6 months to 15.9 years. Positive results from the trial were announced on September 8, 2020: News Release – Albireo Phase 3 Trial Meets Both Primary Endpoints for Odevixibat in PFIC. Odevixibat is currently being evaluated in the ongoing PEDFIC 2 open-label trial (NCT03659916) and the BOLD Phase 3 trial in patients with biliary atresia (NCT04336722). Initiation of a pivotal Phase 3 trial of odevixibat for Alagille syndrome is also anticipated by the end of 2020.

Odevixibat has received fast track, rare pediatric disease and orphan drug designations in the United States. In addition, the FDA has granted orphan drug designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. The EMA has granted odevixibat orphan designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. Its Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plan for PFIC and biliary atresia. EMA has also granted orphan designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. Odevixibat has the potential to become the first approved drug treatment for patients with PFIC. The Company intends to complete regulatory filings in the EU and U.S. for odevixibat in PFIC no later than early 2021, in anticipation of potential regulatory approval, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.  

About AlbireoAlbireo Pharma is a clinical-stage biopharmaceutical company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases, and other adult liver 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 progressive familial intrahepatic cholestasis (PFIC) and biliary atresia, with a third Phase 3 trial being planned in Alagille syndrome. The Company expects to complete IND-enabling studies for new preclinical candidate A3907 this year. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. The Boston Business Journal named Albireo one of the 2020 Best Places to Work in Massachusetts for the second consecutive year. 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 or any other Albireo product candidate or program, including regarding expectations regarding the impact of COVID-19 on our business and our ability to adapt our approach as appropriate; the Phase 3 clinical program for odevixibat in patients with PFIC, the pivotal trial for odevixibat in biliary atresia (BOLD), and the planned pivotal trial for odevixibat in Alagille syndrome; the target indication(s) for development or approval, the size, design, population, location, conduct, cost, objective, enrollment, duration or endpoints of any clinical trial, or the timing for initiation or completion of or availability or reporting of results from any clinical trial, including the long-term open-label extension study for odevixibat in PFIC, the pivotal trial for odevixibat in biliary atresia, the planned pivotal trial for odevixibat in Alagille syndrome; the potential approval and commercialization of odevixibat; discussions with the FDA or EMA 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 effects of odevixibat of the treatment of PFIC patients and its potential to improve the current standard of care; the potential benefits of an orphan drug designation; the potential issuance of a rare pediatric disease priority review voucher; 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: negative impacts of the COVID-19 pandemic, including on manufacturing, supply, conduct or initiation of clinical trials, or other aspects of our business; whether favorable findings from clinical trials of odevixibat to date, including findings in indications other than PFIC, will be predictive of results from 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 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 pivotal program in biliary atresia or the planned pivotal program in Alagille syndrome, and the outcomes of such trials; 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, company’s clinical 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.            Media Contacts: Colleen Alabiso, 857-356-3905, colleen.alabiso@albireopharma.comHeather Anderson, 6 Degrees, 919-827-5539, handerson@6degreespr.com

Investor Contact: Hans Vitzthum, LifeSci Advisors, LLC., 857-272-6177